Warm Autoimmune Hemolytic Anemia (wAIHA)
Conditions
Keywords
Warm Autoimmune Hemolytic Anemia (wAIHA)
Brief summary
The purpose of this study is to evaluate the efficacy and safety of parsaclisib compared with placebo in participants with Primary Warm Autoimmune Hemolytic Anemia (wAIHA),
Detailed description
Prospective participants must have primary wAIHA as well as other protocol-defined criteria. After participants have been determined to be eligible for the study, they will be randomized to 2:1, with stratification factor of corticosteroid dose and hemoglobin (Hgb \<9 g/dL or ≥ 9 g/dL). Once a participant has completed the week 24 assessments in the double-blind period, the participant will have the opportunity to receive parsaclisib in the open-label treatment which will last up to another 24 weeks. Participants may then continue to receive parsaclisib in a long-term extension period.
Interventions
DRUGparsaclisinib
parsaclisib will be administered QD orally
DRUGplacebo
placebo will be administered QD orally follwed by Parsaclisinib in the open label period
Sponsors
Incyte Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Study will be a 24 week double-blind period followed by a 24 week open-label period, followed by a long term extension period.
Eligibility
Sex/Gender
ALL
Age
18 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of primary warm AIHA. * Participants who have at least 1 unsuccessful prior therapy for warm AIHA or unable to receive or tolerate other therapies. * Hemoglobin ≥ 6.5 to \< 10 g/dL with symptoms of anemia at screening. * FACIT-F score ≤ 43 at screening. * Willingness to avoid pregnancy or fathering children. * Willingness to receive PJP prophylaxis. * Further inclusion criteria apply.
Exclusion criteria
* Women who are pregnant, breastfeeding or who are planning a pregnancy. * Diagnosis of other types of AIHA (CAD, cold agglutinin syndrome, mixed-type AIHA or paroxysmal cold hemoglobinuria). * Secondary warm AIHA from any cause or diagnosis of Evans syndrome. * Splenectomy less than 3 months before randomization. * Participants with a history or ongoing significant illness as assessed by the investigator. * Participants with a current of medical history of a malignancy within the past 5 years except basal or squamous cell skin cancer that has been removed and considered cured, or superficial bladder cancer, prostate intraepithelial neoplasm, carcinoma in situ of the cervix, or other noninvasive or indolent malignancy. * Participants know to be infected with HIV, Hepatitis B, or hepatitis C. * Chronic or current active infectious disease requiring systemic antibiotics, antifungal, or antiviral treatment or exposure to a live vaccine. * Participants with laboratory values outside of the protocol defined ranges. * Further
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Attaining a Durable Hemoglobin Response | up to Week 24 | A durable hemoglobin response was defined as hemoglobin ≥10 grams per deciliter (g/dL) with an increase from Baseline of ≥2 g/dL not attributed to rescue therapy at ≥3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT) | Baseline; Week 24 | The 6MWT is used to evaluate submaximal exercise capacity. It is a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes. |
| Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56 | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (not at all) to 4 (very much), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit. |
| Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56 | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (not at all) to 4 (very much), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Percentage change from Baseline was calculated as (\[the post-Baseline value minus the Baseline value\]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit. |
| Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56 | Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit. |
| Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56 | Percentage change from Baseline was calculated as (\[the post-Baseline value minus the Baseline value\]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit. |
| Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24 | Baseline; Week 24 | The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (not at all) to 4 (very much), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. |
| Change From Baseline in Daily Corticosteroid Dose at Week 24 | Baseline; Week 24 | A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. |
| Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24 | Baseline; Week 24 | A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Percentage change from Baseline was calculated as (\[the post-Baseline value minus the Baseline value\]/Baseline value) x 100. |
| Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48 | Week 6 to Week 24; Week 24 to Week 48 | Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. |
| Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | up to 446 days | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. An AE could therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. |
| Number of Participants With Any ≥Grade 3 TEAE | up to 446 days | An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. The severity of AEs were assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal. |
| Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48 | Week 6 to Week 24; Week 24 to Week 48 | Transfusion was permitted as a rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of warm autoimmune hemolytic anemia (wAIHA). |
Countries
Austria, Belgium, Canada, France, Germany, Israel, Italy, Japan, Netherlands, Poland, Spain, United Kingdom, United States
Participant flow
Pre-assignment details
This study was designed to evaluate parsaclisib 2.5 mg QD compared with placebo over a 24-week double-blind treatment period followed by a 24-week open-label treatment period with parsaclisib. Participants could then continue to receive parsaclisib in a long-term extension period.
Participants by arm
| Arm | Count |
|---|---|
| Parsaclisib Participants received parsaclisib 2.5 milligrams (mg) once daily (QD) for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for an additional 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. | 7 |
| Placebo Followed by Parsaclisib Participants received matching placebo QD for 24 weeks during the double-blind treatment period. Participants who completed the double-blind treatment period, tolerated study treatment, and, in the investigator's opinion, benefited from treatment had the option of continuing into an open-label treatment period for 24 weeks of parsaclisib 2.5 mg QD, and then the long-term extension period to receive parsaclisib 2.5 mg QD for up to 2 years. Participants may have received parsaclisib before reaching Week 24. | 6 |
| Total | 13 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| 24-week Double-blind Period | Adverse Event | 1 | 0 |
| 24-week Double-blind Period | Physician Decision | 1 | 0 |
| 24-week Double-blind Period | Study Terminated by Sponsor | 1 | 1 |
| 24-week Double-blind Period | Withdrawal by Subject | 0 | 1 |
| 24-week Open-label Period | Adverse Event | 1 | 2 |
| 24-week Open-label Period | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | Placebo Followed by Parsaclisib | Total | Parsaclisib |
|---|---|---|---|
| Age, Continuous | 57.8 years STANDARD_DEVIATION 15.54 | 60.23 years STANDARD_DEVIATION 14.42 | 62.3 years STANDARD_DEVIATION 14.28 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 1 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 4 Participants | 11 Participants | 7 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Asian | 1 Participants | 2 Participants | 1 Participants |
| Race/Ethnicity, Customized Missing | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized White/Caucasian | 4 Participants | 10 Participants | 6 Participants |
| Sex: Female, Male Female | 6 Participants | 10 Participants | 4 Participants |
| Sex: Female, Male Male | 0 Participants | 3 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 6 | 0 / 11 |
| other Total, other adverse events | 4 / 6 | 10 / 11 |
| serious Total, serious adverse events | 0 / 6 | 6 / 11 |
Outcome results
Primary
Percentage of Participants Attaining a Durable Hemoglobin Response
A durable hemoglobin response was defined as hemoglobin ≥10 grams per deciliter (g/dL) with an increase from Baseline of ≥2 g/dL not attributed to rescue therapy at ≥3 of the 4 available visits at Week 12 and/or later during the 24-week double-blind treatment period.
Time frame: up to Week 24
Population: Safety Analysis Set: all randomized participants who received at least 1 dose of study drug. Treatment groups were determined according to the actual treatment the participant received on Day 1 regardless of assigned study treatment. Only participants with available data were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Parsaclisib | Percentage of Participants Attaining a Durable Hemoglobin Response | 33.3 percentage of participants |
| Placebo | Percentage of Participants Attaining a Durable Hemoglobin Response | 25.0 percentage of participants |
Secondary
Change From Baseline in Daily Corticosteroid Dose at Week 24
A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; Week 24
Population: Safety Analysis Set. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Parsaclisib | Change From Baseline in Daily Corticosteroid Dose at Week 24 | Baseline | 13.3 milligrams | Standard Deviation 5.77 |
| Parsaclisib | Change From Baseline in Daily Corticosteroid Dose at Week 24 | Change from Baseline at Week 24 | 8.1 milligrams | Standard Deviation 14.02 |
| Placebo | Change From Baseline in Daily Corticosteroid Dose at Week 24 | Baseline | 20.8 milligrams | Standard Deviation 10.21 |
| Placebo | Change From Baseline in Daily Corticosteroid Dose at Week 24 | Change from Baseline at Week 24 | -4.8 milligrams | Standard Deviation 10.41 |
Secondary
Change From Baseline in Hemoglobin at Each Post-Baseline Visit
Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit.
Time frame: Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56
Population: Safety Analysis Set. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 12 | 13.8 grams per liter | Standard Deviation 19.69 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 6 | 2.7 grams per liter | Standard Deviation 6.38 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 16 | 19.5 grams per liter | Standard Deviation 15.76 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 20 | 15.3 grams per liter | Standard Deviation 6.08 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 8 | 12.7 grams per liter | Standard Deviation 14.07 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 24 | 19.5 grams per liter | Standard Deviation 14.8 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 4 | -4.4 grams per liter | Standard Deviation 14.29 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Double-blind Period | -62.4 grams per liter | — |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 10 | 16.5 grams per liter | Standard Deviation 17.41 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Baseline | 86.5 grams per liter | Standard Deviation 15.31 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 2 | -9.3 grams per liter | Standard Deviation 29.09 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 26 | 14.0 grams per liter | Standard Deviation 9.6 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Open-label Period | 127.7 grams per liter | — |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 3 | 23.0 grams per liter | — |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 56 | 17.5 grams per liter | Standard Deviation 12.02 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 28 | 18.9 grams per liter | Standard Deviation 19.58 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 64 | 8.0 grams per liter | — |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Long-term Extension Period | 86.0 grams per liter | — |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 32 | 19.0 grams per liter | Standard Deviation 31.32 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 1 | 40.0 grams per liter | — |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 36 | 22.8 grams per liter | Standard Deviation 18.01 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Baseline | 85.3 grams per liter | Standard Deviation 12.97 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 40 | 21.5 grams per liter | Standard Deviation 12.02 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 30 | 8.1 grams per liter | Standard Deviation 32.67 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 44 | 25.0 grams per liter | Standard Deviation 21.21 |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 2 | 32.0 grams per liter | — |
| Placebo | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 48 | 22.5 grams per liter | Standard Deviation 21.92 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 3 | 20.3 grams per liter | Standard Deviation 9.95 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Baseline | 98.4 grams per liter | Standard Deviation 21.53 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 2 | 8.7 grams per liter | Standard Deviation 11.76 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 4 | 14.2 grams per liter | Standard Deviation 13.14 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 6 | 14.3 grams per liter | Standard Deviation 16.83 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 8 | 15.0 grams per liter | Standard Deviation 18.95 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 10 | 16.5 grams per liter | Standard Deviation 16.32 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 12 | 20.5 grams per liter | Standard Deviation 11.01 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 16 | 22.4 grams per liter | Standard Deviation 16.41 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 20 | 7.7 grams per liter | Standard Deviation 1.53 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 24 | 19.3 grams per liter | Standard Deviation 22.28 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Double-blind Period | 11.0 grams per liter | — |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 26 | 3.0 grams per liter | Standard Deviation 24.33 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 28 | 8.3 grams per liter | Standard Deviation 26.48 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 30 | 27.0 grams per liter | — |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 32 | 26.0 grams per liter | Standard Deviation 11.31 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 36 | 18.5 grams per liter | Standard Deviation 13.44 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 40 | 26.5 grams per liter | Standard Deviation 7.78 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 44 | 27.5 grams per liter | Standard Deviation 14.85 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 48 | 32.0 grams per liter | — |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Open-label Period | 124.0 grams per liter | — |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Week 56 | 36.0 grams per liter | — |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 1 | 11.0 grams per liter | Standard Deviation 7.62 |
| Parsaclisib | Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 2 | 12.5 grams per liter | Standard Deviation 17.52 |
Secondary
Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit
The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (not at all) to 4 (very much), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit.
Time frame: Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56
Population: Safety Analysis Set. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Baseline | 28.5 scores on a scale | Standard Deviation 5.96 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 8 | 8.2 scores on a scale | Standard Deviation 5.63 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 12 | 7.6 scores on a scale | Standard Deviation 6.58 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 16 | 3.8 scores on a scale | Standard Deviation 5.91 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 20 | 2.0 scores on a scale | Standard Deviation 9.09 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 24 | 3.3 scores on a scale | Standard Deviation 2.63 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Double-blind Period | 20.0 scores on a scale | — |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 56 | 1.0 scores on a scale | — |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Long-term Extension Period | 1.0 scores on a scale | — |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 3 | 0.0 scores on a scale | — |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 28 | 6.5 scores on a scale | Standard Deviation 6.86 |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 32 | -9.0 scores on a scale | Standard Deviation 13.89 |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 40 | 1.0 scores on a scale | — |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 48 | 5.0 scores on a scale | — |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Baseline | 31.3 scores on a scale | Standard Deviation 5.12 |
| Placebo | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Open-label Period | -2.0 scores on a scale | — |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 28 | 6.0 scores on a scale | — |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Baseline | 31.1 scores on a scale | Standard Deviation 7.78 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 32 | -1.0 scores on a scale | — |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 16 | 6.2 scores on a scale | Standard Deviation 7.36 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 12 | 3.6 scores on a scale | Standard Deviation 8.56 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 20 | 4.5 scores on a scale | Standard Deviation 7.78 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 8 | 2.4 scores on a scale | Standard Deviation 7.16 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Week 24 | 5.0 scores on a scale | Standard Deviation 5.2 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at Follow-up Visit 3 | -1.0 scores on a scale | Standard Deviation 2.83 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Double-blind Period | -8.0 scores on a scale | Standard Deviation 9.9 |
| Parsaclisib | Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Open-label Period | 8.0 scores on a scale | — |
Secondary
Number of Participants With Any ≥Grade 3 TEAE
An AE was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug. The severity of AEs were assessed using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.
Time frame: up to 446 days
Population: Safety Analysis Set
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Parsaclisib | Number of Participants With Any ≥Grade 3 TEAE | 1 Participants |
| Placebo | Number of Participants With Any ≥Grade 3 TEAE | 3 Participants |
| Parsaclisib | Number of Participants With Any ≥Grade 3 TEAE | 6 Participants |
Secondary
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it was considered drug-related. An AE could therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. TEAEs were defined as AEs reported for the first time or the worsening of pre-existing events after the first dose of study drug.
Time frame: up to 446 days
Population: Safety Analysis Set
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Parsaclisib | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | 4 Participants |
| Placebo | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | 4 Participants |
| Parsaclisib | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | 7 Participants |
Secondary
Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24
A new or increased dose of corticosteroids (prednisone or equivalent) from the Day 1 dose was permitted as rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA. Percentage change from Baseline was calculated as (\[the post-Baseline value minus the Baseline value\]/Baseline value) x 100.
Time frame: Baseline; Week 24
Population: Safety Analysis Set. Only participants with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Parsaclisib | Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24 | 81.0 percentage change | Standard Deviation 140.21 |
| Placebo | Percentage Change From Baseline in Daily Corticosteroid Dose at Week 24 | -22.0 percentage change | Standard Deviation 60.07 |
Secondary
Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit
Percentage change from Baseline was calculated as (\[the post-Baseline value minus the Baseline value\]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visits 1, 2, and 3 occurred 4, 8, and 12 weeks, respectively, after the end of treatment visit.
Time frame: Baseline; Day 1; Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, and every 8 weeks post-Week 56
Population: Safety Analysis Set. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 4 | -3.5 percentage change | Standard Deviation 16.87 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 16 | 21.7 percentage change | Standard Deviation 16.02 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 8 | 14.0 percentage change | Standard Deviation 14.59 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 20 | 18.5 percentage change | Standard Deviation 9.32 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 2 | -12.3 percentage change | Standard Deviation 41.18 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 24 | 22.7 percentage change | Standard Deviation 15.52 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 10 | 17.8 percentage change | Standard Deviation 17.44 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Double-blind Period | -87.9 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 6 | 3.4 percentage change | Standard Deviation 7.42 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 12 | 13.6 percentage change | Standard Deviation 21.15 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Follow-up Visit 3 | 23.7 percentage change | — |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Open-label Period | 31.7 percentage change | — |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 40 | 29.7 percentage change | Standard Deviation 18.67 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 56 | 24.3 percentage change | Standard Deviation 18.21 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 28 | 23.4 percentage change | Standard Deviation 25.16 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 64 | 10.1 percentage change | — |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Long-term Extension Period | 8.9 percentage change | — |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 32 | 23.6 percentage change | Standard Deviation 40.57 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Follow-up Visit 1 | 41.2 percentage change | — |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 36 | 29.3 percentage change | Standard Deviation 26.69 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 26 | 15.2 percentage change | Standard Deviation 9.34 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Follow-up Visit 2 | 33.0 percentage change | — |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 44 | 34.9 percentage change | Standard Deviation 31.46 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 30 | 6.6 percentage change | Standard Deviation 36.17 |
| Placebo | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 48 | 31.6 percentage change | Standard Deviation 32.12 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Follow-up Visit 3 | 23.4 percentage change | Standard Deviation 13.52 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 2 | 8.2 percentage change | Standard Deviation 13.82 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 4 | 14.2 percentage change | Standard Deviation 13.68 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 6 | 14.2 percentage change | Standard Deviation 18.21 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 8 | 14.3 percentage change | Standard Deviation 19.65 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 10 | 16.5 percentage change | Standard Deviation 17.59 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 12 | 20.7 percentage change | Standard Deviation 12.36 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 16 | 23.2 percentage change | Standard Deviation 17.81 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 20 | 7.6 percentage change | Standard Deviation 2.11 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 24 | 19.4 percentage change | Standard Deviation 22.7 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Double-blind Period | 12.0 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 26 | 3.4 percentage change | Standard Deviation 24.52 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 28 | 9.7 percentage change | Standard Deviation 27.56 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 30 | 30.0 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 32 | 29.0 percentage change | Standard Deviation 12.41 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 36 | 20.6 percentage change | Standard Deviation 14.85 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 40 | 29.6 percentage change | Standard Deviation 8.46 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 44 | 30.7 percentage change | Standard Deviation 16.35 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 48 | 35.6 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Change from Baseline at End of Treatment, Open-label Period | 25.3 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Week 56 | 40.0 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Follow-up Visit 1 | 11.5 percentage change | Standard Deviation 8.19 |
| Parsaclisib | Percentage Change From Baseline in Hemoglobin at Each Post-Baseline Visit | Follow-up Visit 2 | 14.5 percentage change | Standard Deviation 19.47 |
Secondary
Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit
The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (not at all) to 4 (very much), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Percentage change from Baseline was calculated as (\[the post-Baseline value minus the Baseline value\]/Baseline value) x 100. End of Treatment, Double-blind Period: assessment performed for participants who discontinued treatment before Week 24. End of Treatment, Open-label Period: assessment performed for participants who discontinued treatment after Week 24 and before Week 56. Follow-up Visit 3 occurred 12 weeks after the end of treatment visit.
Time frame: Baseline; Day 1; Weeks 8, 12, 16, 20, 24, 28, 32, 40, 48, 56, and every 16 weeks post-Week 56
Population: Safety Analysis Set. Only participants with available data were analyzed,
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 20 | 9.1 percentage change | Standard Deviation 31.07 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 8 | 29.6 percentage change | Standard Deviation 21.4 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 12 | 28.1 percentage change | Standard Deviation 26.1 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | End of Treatment, Double-blind Period | 95.2 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 16 | 12.4 percentage change | Standard Deviation 20.21 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 24 | 10.1 percentage change | Standard Deviation 6.71 |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 28 | 22.6 percentage change | Standard Deviation 26.8 |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 32 | -26.9 percentage change | Standard Deviation 40.55 |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 40 | 2.9 percentage change | — |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 48 | 14.7 percentage change | — |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | End of Treatment, Open-label Period | -5.4 percentage change | — |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 56 | 2.9 percentage change | — |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | End of Treatment, Long-term Extension Period | 2.9 percentage change | — |
| Placebo | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Follow-up Visit 3 | 0.0 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | End of Treatment, Open-label Period | 25.0 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 32 | -4.8 percentage change | — |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Follow-up Visit 3 | -5.1 percentage change | Standard Deviation 10.41 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 20 | 12.8 percentage change | Standard Deviation 24.79 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 16 | 21.9 percentage change | Standard Deviation 27.65 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 24 | 14.8 percentage change | Standard Deviation 16.97 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 12 | 15.30252 percentage change | Standard Deviation 32.05 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | End of Treatment, Double-blind Period | -32.4 percentage change | Standard Deviation 42.62 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 8 | 8.2 percentage change | Standard Deviation 21.25 |
| Parsaclisib | Percentage Change From Baseline in the FACIT-F Score at Each Post-Baseline Visit | Week 28 | 18.8 percentage change | — |
Secondary
Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48
Transfusion was permitted as a rescue treatment. Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of warm autoimmune hemolytic anemia (wAIHA).
Time frame: Week 6 to Week 24; Week 24 to Week 48
Population: Safety Analysis Set. Only participants with available data were analyzed. The number of participants analyzed at Weeks 6 to 24 reflects the number of participants who received study drug for at least 43 days. The number of participants analyzed at Weeks 24 to 48 reflects the number of participants who received study drug during the open-label period.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Parsaclisib | Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48 | Week 6 to Week 24 | 0.0 percentage of participants |
| Placebo | Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48 | Week 24 to Week 48 | 25.0 percentage of participants |
| Parsaclisib | Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48 | Week 6 to Week 24 | 42.9 percentage of participants |
| Parsaclisib | Percentage of Participants Who Received Transfusions From Week 6 to Week 24 and From Week 24 to Week 48 | Week 24 to Week 48 | 25.0 percentage of participants |
Secondary
Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48
Rescue medication was to be considered if the absolute hemoglobin level continued to decline, there was a \> 1 g/dL decrease in hemoglobin from the prior assessment, or the participant developed new or worsening symptoms of wAIHA.
Time frame: Week 6 to Week 24; Week 24 to Week 48
Population: Safety Analysis Set. Only participants with available data were analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Parsaclisib | Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48 | Week 6 to Week 24 | 0.0 percentage of participants |
| Placebo | Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48 | Week 24 to Week 48 | 0.0 percentage of participants |
| Parsaclisib | Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48 | Week 6 to Week 24 | 0.0 percentage of participants |
| Parsaclisib | Percentage of Participants Who Required Rescue Therapy at Any Visit From Week 6 Through Week 24, and From Week 24 to Week 48 | Week 24 to Week 48 | 20.0 percentage of participants |
Secondary
Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24
The FACIT-F scale was developed to assess anemia-related fatigue. The FACIT-F is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function over the past 7 days. A clinically meaningful change in the FACIT-F score was defined as ≥3-point increase from Baseline. Item scores range from 0 (not at all) to 4 (very much), and the total score ranges from 0 to 52; lower scores indicate greater fatigue. Change from Baseline was calculated as the post-Baseline value minus the Baseline value.
Time frame: Baseline; Week 24
Population: Safety Analysis Set. Only participants with available data were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Parsaclisib | Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24 | 66.7 percentage of participants |
| Placebo | Percentage of Participants With a ≥3-point Increase From Baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F) Score at Week 24 | 50.0 percentage of participants |
Secondary
Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT)
The 6MWT is used to evaluate submaximal exercise capacity. It is a self-paced measurement of the distance that a participant can quickly walk on a flat, hard surface in a period of 6 minutes.
Time frame: Baseline; Week 24
Population: Safety Analysis Set. Only participants with available data were analyzed,
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Parsaclisib | Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT) | 0.0 percentage of participants |
| Placebo | Percentage of Participants With a 50 Meter Increase From Baseline to Week 24 in a 6-minute Walk Test (6MWT) | 100.0 percentage of participants |