Fibromyalgia
Conditions
Keywords
Phase 2, Fibromyalgia, rozanolixizumab
Brief summary
The purpose of the study is to evaluate efficacy and safety of rozanolixizumab to treat adult study participants with severe fibromyalgia syndrome (FMS).
Interventions
DRUGrozanolixizumab
Study participants will receive rozanolixizumab during the dosing periods as pre-defined.
OTHERPlacebo
Study participants will receive Placebo during the dosing periods as pre-defined.
Sponsors
UCB Biopharma SRL
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Study participant must be ≥18 years and ≤70 years of age at the time of signing the informed consent form (ICF) * Study participant with a diagnosis of fibromyalgia as defined by the 2016 Revisions to the 2010/2011 fibromyalgia diagnostic criteria (American College of Rheumatology Preliminary Diagnostic Criteria) plus the following characteristics during the Screening Period: 1. Brief Pain Inventory-short form (BPI-SF) interference score ≥6. 2. Study participant has been diagnosed with fibromyalgia syndrome (FMS) for at least 6 months. 3. Study participant has been having FMS symptomatology for at least 2 years before enrollment - Capable of giving signed informed consent as described in the Protocol which includes compliance with the requirements and restrictions listed in the ICF and in the Study Protocol
Exclusion criteria
* Study participant has been diagnosed with fibromyalgia syndrome (FMS) for \>15 years * Study participant has any systemic autoimmune inflammatory disease * Study participant has any medical or psychiatric or separate chronic pain condition that, in the opinion of the investigator, could jeopardize or would compromise the study participant's ability to participate in this study or the ability to assess FMS-related pain * Study participant has severe renal impairment, defined as estimated glomerular filtration rate \<30 mL/min/1.73 m\^2, (calculated using Modification of Diet in Renal Disease \[MDRD\] study equation), at Screening visit * Study participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator * Study participant has chronic inflammatory demyelinating polyneuropathy * Study participant has a current or medical history of primary immunodeficiency * Study participant is pregnant or lactating * Study participant * Has suicide attempt in the past 2 years (including an active attempt, interrupted attempt, or aborted attempt), * OR had suicidal ideation with at least some intent to act in the past 6 months as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening or Baseline (Visit 3); * OR is otherwise judged clinically to be at a serious suicidal risk based on the investigator's judgment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 12 Weeks of Treatment | At 12 weeks of treatment | The BPI-SF was a self-administered questionnaire used to evaluate the severity of a study participant's pain and the impact of this pain on the study participant's daily functioning. The BPI-SF assesses for the location of pain, pain intensity and functional interference from pain. The 7 BPI-SF interference items included: general activity, mood, walking ability, normal work (including housework), relations with other people, sleep, and enjoyment of life. Each item was rated on a 0 (did not interfere) to 10 (completely interfere) scale with a recall period of 24 hours. The BPI-SF interference score ranges 0-70. Higher scores indicated greater interference. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With TEAEs Leading to Withdrawal of IMP | From Baseline till end of Safety Follow-up (up to Week 33) | An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE was defined as any AE with an onset date on or after the first dose of IMP in Run-In and on or before the earliest of the following: the last date of infusion (including Run-Out) +56 days, final contact date, or death. |
| Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 24 Weeks of Treatment | At 24 weeks of treatment | The BPI-SF was a self-administered questionnaire used to evaluate the severity of a study participant's pain and the impact of this pain on the study participant's daily functioning. The BPI-SF assesses for the location of pain, pain intensity and functional interference from pain. The 7 BPI-SF interference items included: general activity, mood, walking ability, normal work (including housework), relations with other people, sleep, and enjoyment of life. Each item was rated on a 0 (did not interfere) to 10 (completely interfere) scale with a recall period of 24 hours. The BPI-SF interference score ranges 0-70. Higher scores indicated greater interference. |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | From Baseline till end of Safety Follow-up (up to Week 33) | An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE was defined as any AE with an onset date on or after the first dose of investigational medicinal product (IMP) in Run-In and on or before the earliest of the following: the last date of infusion (including Run-Out) +56 days, final contact date, or death. A TEAE is also defined as any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment. |
| Mean 7-day Average Daily Pain Score Assessed With Pain Numeric Rating Scale (NRS) at 12 Weeks of Treatment | At 12 Weeks of treatment | The Pain Numeric Rating Scale (NRS) was a scale in which a respondent selected a whole number that best described How much pain have you experienced on average over the past 24 hours? The 11-point Pain NRS ranged 0 (no pain) to 10 (pain as bad as you can imagine). Mean pain scores were derived the average of the daily assessment over the past 7 days. The higher score represented worst possible pain. |
| Mean 7-day Fatigue Score Assessed With Fatigue Numeric Rating Scale at 12 Weeks of Treatment | At 12 weeks of treatment | The Fatigue Numeric Rating Scale (NRS) was a scale in which a respondent selected a whole number that best described How much fatigue have you experienced on average over the past 24 hours? The 11-point Fatigue NRS ranged 0 (no fatigue) to 10 (fatigue as bad as you can imagine). Mean fatigue scores were derived the average of the daily assessment over the past 7 days. Higher score represented worst possible fatigue. |
| Revised Fibromyalgia Impact Questionnaire (FIQR) Score at 12 Weeks of Treatment | At 12 weeks of treatment | The Revised Fibromyalgia Impact Questionnaire (FIQR) was a 21-item questionnaire with a recall period of 7 days. The FIQR included 3 domains: activities, overall impact, and symptoms. Each item was based on an 11-point numeric rating scale. The FIQR total score was calculated by taking the sum of the following: Activities domain subtotal divided by 3, overall impact domain subtotal and symptoms domain subtotal divided by 2. The total score ranged from 0 to 100, with 0 denoting the best possible condition and 100 denoting the worst possible condition. Higher scores indicated more severe impact. |
Countries
United Kingdom
Participant flow
Recruitment details
The study started to enroll participants in December 2022 and concluded in July 2024.
Pre-assignment details
The Participant Flow refers to the Randomized Set.
Participants by arm
| Arm | Count |
|---|---|
| Sequence 1: RLZ 12w + RLZ 12w Participants received placebo, subcutaneously (SC), once weekly (QW) for 2 weeks (Run-in Period), then rozanolixizumab (RLZ) 560 milligrams (mg), SC, QW, weekly (w) for 24 weeks (Treatment Period 1 + Treatment Period 2), followed by placebo SC, QW for 2 weeks (Runout Period). The safety follow-up (SFU) included all the participants who received the investigational medicinal product (IMP), regardless of discontinuation. | 22 |
| Sequence 2: PBO + RLZ 12w Participants received placebo SC, QW for 2 weeks (Run-in Period), then placebo SC, QW for 12 weeks (Treatment Period 1), followed by rozanolixizumab 560 mg, SC, QW for 12 weeks (Treatment Period 2), followed by placebo SC, QW for 2 weeks (Runout Period). The SFU included all the participants who received the IMP, regardless of discontinuation. | 20 |
| Sequence 3: PBO + PBO Participants received placebo SC, QW for 2 weeks (Run-in Period), then placebo SC, QW for 24 weeks (Treatment Period 1 + Treatment Period 2), followed by placebo SC, QW for 2 weeks (Run-out Period). The SFU included all the participants who received the IMP, regardless of discontinuation. | 21 |
| Total | 63 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Treatment Period 1 (12 Weeks) | Adverse Event | 2 | 0 | 1 |
| Treatment Period 1 (12 Weeks) | Consent Withdrawn by Study Participant (Not Due to Adverse Event) | 0 | 1 | 0 |
| Treatment Period 1 (12 Weeks) | Lost to Follow-up | 1 | 0 | 0 |
| Treatment Period 2 (12 Weeks) | Consent Withdrawn by Study Participant (Not Due to Adverse Event) | 1 | 0 | 0 |
| Treatment Period 2 (12 Weeks) | Lack of Efficacy | 0 | 1 | 0 |
| Treatment Period 2 (12 Weeks) | New Work Commitments | 1 | 0 | 0 |
Baseline characteristics
| Characteristic | Sequence 1: RLZ 12w + RLZ 12w | Sequence 2: PBO + RLZ 12w | Sequence 3: PBO + PBO | Total |
|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 1 Participants | 0 Participants | 1 Participants | 2 Participants |
| Age, Categorical Between 18 and 65 years | 21 Participants | 20 Participants | 20 Participants | 61 Participants |
| Age, Continuous | 46.3 years STANDARD_DEVIATION 9.9 | 47.8 years STANDARD_DEVIATION 10.7 | 47.3 years STANDARD_DEVIATION 9.5 | 47.1 years STANDARD_DEVIATION 9.9 |
| Race/Ethnicity, Customized Asian | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Black or African American | 0 Participants | 1 Participants | 1 Participants | 2 Participants |
| Race/Ethnicity, Customized Hispanic or Latino | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Not Hispanic or Latino | 22 Participants | 20 Participants | 20 Participants | 62 Participants |
| Race/Ethnicity, Customized White | 22 Participants | 19 Participants | 19 Participants | 60 Participants |
| Sex: Female, Male Female | 21 Participants | 17 Participants | 17 Participants | 55 Participants |
| Sex: Female, Male Male | 1 Participants | 3 Participants | 4 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 63 | 0 / 22 | 0 / 41 | 0 / 18 | 0 / 19 | 0 / 20 | 0 / 22 | 0 / 20 | 0 / 21 |
| other Total, other adverse events | 24 / 63 | 21 / 22 | 37 / 41 | 18 / 18 | 19 / 19 | 16 / 20 | 3 / 22 | 8 / 20 | 3 / 21 |
| serious Total, serious adverse events | 0 / 63 | 0 / 22 | 3 / 41 | 0 / 18 | 0 / 19 | 0 / 20 | 0 / 22 | 1 / 20 | 2 / 21 |
Outcome results
Primary
Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 12 Weeks of Treatment
The BPI-SF was a self-administered questionnaire used to evaluate the severity of a study participant's pain and the impact of this pain on the study participant's daily functioning. The BPI-SF assesses for the location of pain, pain intensity and functional interference from pain. The 7 BPI-SF interference items included: general activity, mood, walking ability, normal work (including housework), relations with other people, sleep, and enjoyment of life. Each item was rated on a 0 (did not interfere) to 10 (completely interfere) scale with a recall period of 24 hours. The BPI-SF interference score ranges 0-70. Higher scores indicated greater interference.
Time frame: At 12 weeks of treatment
Population: Full Analysis Set (FAS) included all study participants who received any study treatment, including during the run-in period, had a baseline value and at least one post-baseline efficacy endpoint assessment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Placebo (PBO) | Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 12 Weeks of Treatment | 6.30 score on a scale |
| RLZ 12 Weeks | Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 12 Weeks of Treatment | 5.76 score on a scale |
p-value: 0.12995% CI: [-1.24, 0.16]Longitudinal linear mixed effect model
Secondary
Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 24 Weeks of Treatment
The BPI-SF was a self-administered questionnaire used to evaluate the severity of a study participant's pain and the impact of this pain on the study participant's daily functioning. The BPI-SF assesses for the location of pain, pain intensity and functional interference from pain. The 7 BPI-SF interference items included: general activity, mood, walking ability, normal work (including housework), relations with other people, sleep, and enjoyment of life. Each item was rated on a 0 (did not interfere) to 10 (completely interfere) scale with a recall period of 24 hours. The BPI-SF interference score ranges 0-70. Higher scores indicated greater interference.
Time frame: At 24 weeks of treatment
Population: FAS included all study participants who received any study treatment, including during the run-in period, had a baseline value and at least one post-baseline efficacy endpoint assessment. Here 'overall number of participants analyzed' signifies participants evaluable for this Outcome measure.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Placebo (PBO) | Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 24 Weeks of Treatment | 6.30 score on a scale |
| RLZ 12 Weeks | Brief Pain Inventory Short Form (BPI-SF) Average Interference Score at 24 Weeks of Treatment | 5.79 score on a scale |
p-value: 0.35895% CI: [-1.6, 0.58]Longitudinal mixed effects model
Secondary
Mean 7-day Average Daily Pain Score Assessed With Pain Numeric Rating Scale (NRS) at 12 Weeks of Treatment
The Pain Numeric Rating Scale (NRS) was a scale in which a respondent selected a whole number that best described How much pain have you experienced on average over the past 24 hours? The 11-point Pain NRS ranged 0 (no pain) to 10 (pain as bad as you can imagine). Mean pain scores were derived the average of the daily assessment over the past 7 days. The higher score represented worst possible pain.
Time frame: At 12 Weeks of treatment
Population: FAS included all study participants who received any study treatment, including during the run-in period, had a baseline value and at least one post-baseline efficacy endpoint assessment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Placebo (PBO) | Mean 7-day Average Daily Pain Score Assessed With Pain Numeric Rating Scale (NRS) at 12 Weeks of Treatment | 6.59 score on a scale |
| RLZ 12 Weeks | Mean 7-day Average Daily Pain Score Assessed With Pain Numeric Rating Scale (NRS) at 12 Weeks of Treatment | 6.63 score on a scale |
p-value: 0.87895% CI: [-0.55, 0.64]Longitudinal mixed effects model
Secondary
Mean 7-day Fatigue Score Assessed With Fatigue Numeric Rating Scale at 12 Weeks of Treatment
The Fatigue Numeric Rating Scale (NRS) was a scale in which a respondent selected a whole number that best described How much fatigue have you experienced on average over the past 24 hours? The 11-point Fatigue NRS ranged 0 (no fatigue) to 10 (fatigue as bad as you can imagine). Mean fatigue scores were derived the average of the daily assessment over the past 7 days. Higher score represented worst possible fatigue.
Time frame: At 12 weeks of treatment
Population: FAS included all study participants who received any study treatment, including during the run-in period, had a baseline value and at least one post-baseline efficacy endpoint assessment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Placebo (PBO) | Mean 7-day Fatigue Score Assessed With Fatigue Numeric Rating Scale at 12 Weeks of Treatment | 7.26 score on a scale |
| RLZ 12 Weeks | Mean 7-day Fatigue Score Assessed With Fatigue Numeric Rating Scale at 12 Weeks of Treatment | 6.98 score on a scale |
p-value: 0.35295% CI: [-0.86, 0.31]Longitudinal mixed effects model
Secondary
Number of Participants With TEAEs Leading to Withdrawal of IMP
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE was defined as any AE with an onset date on or after the first dose of IMP in Run-In and on or before the earliest of the following: the last date of infusion (including Run-Out) +56 days, final contact date, or death.
Time frame: From Baseline till end of Safety Follow-up (up to Week 33)
Population: The SS-t included all study participants who received any study treatment, including during Run-In period.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo (PBO) | Number of Participants With TEAEs Leading to Withdrawal of IMP | 0 Participants |
| RLZ 12 Weeks | Number of Participants With TEAEs Leading to Withdrawal of IMP | 3 Participants |
| TP1-PBO | Number of Participants With TEAEs Leading to Withdrawal of IMP | 1 Participants |
| TP2-RLZ/RLZ | Number of Participants With TEAEs Leading to Withdrawal of IMP | 0 Participants |
| TP2-PBO/RLZ | Number of Participants With TEAEs Leading to Withdrawal of IMP | 0 Participants |
| TP2-PBO/PBO | Number of Participants With TEAEs Leading to Withdrawal of IMP | 1 Participants |
| Run-Out and SFU-RLZ/RLZ | Number of Participants With TEAEs Leading to Withdrawal of IMP | 0 Participants |
| Run-Out and SFU-PBO/RLZ | Number of Participants With TEAEs Leading to Withdrawal of IMP | 0 Participants |
| Run-Out and SFU-PBO/PBO | Number of Participants With TEAEs Leading to Withdrawal of IMP | 2 Participants |
Secondary
Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study
An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE was defined as any AE with an onset date on or after the first dose of investigational medicinal product (IMP) in Run-In and on or before the earliest of the following: the last date of infusion (including Run-Out) +56 days, final contact date, or death. A TEAE is also defined as any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment.
Time frame: From Baseline till end of Safety Follow-up (up to Week 33)
Population: The Safety Set as treated (SS-t) included all study participants who received any study treatment, including during Run-In period.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo (PBO) | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 44 Participants |
| RLZ 12 Weeks | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 22 Participants |
| TP1-PBO | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 41 Participants |
| TP2-RLZ/RLZ | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 18 Participants |
| TP2-PBO/RLZ | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 19 Participants |
| TP2-PBO/PBO | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 19 Participants |
| Run-Out and SFU-RLZ/RLZ | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 10 Participants |
| Run-Out and SFU-PBO/RLZ | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 12 Participants |
| Run-Out and SFU-PBO/PBO | Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the Study | 11 Participants |
Secondary
Revised Fibromyalgia Impact Questionnaire (FIQR) Score at 12 Weeks of Treatment
The Revised Fibromyalgia Impact Questionnaire (FIQR) was a 21-item questionnaire with a recall period of 7 days. The FIQR included 3 domains: activities, overall impact, and symptoms. Each item was based on an 11-point numeric rating scale. The FIQR total score was calculated by taking the sum of the following: Activities domain subtotal divided by 3, overall impact domain subtotal and symptoms domain subtotal divided by 2. The total score ranged from 0 to 100, with 0 denoting the best possible condition and 100 denoting the worst possible condition. Higher scores indicated more severe impact.
Time frame: At 12 weeks of treatment
Population: FAS included all study participants who received any study treatment, including during the run-in period, had a baseline value and at least one post-baseline efficacy endpoint assessment.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| Placebo (PBO) | Revised Fibromyalgia Impact Questionnaire (FIQR) Score at 12 Weeks of Treatment | 70.70 score on a scale |
| RLZ 12 Weeks | Revised Fibromyalgia Impact Questionnaire (FIQR) Score at 12 Weeks of Treatment | 62.30 score on a scale |
p-value: 0.00395% CI: [-13.84, -2.96]Longitudinal mixed effects model