Hereditary Hemochromatosis
Conditions
Brief summary
This study will be conducted at multiple sites and every patient will get treated with PTG-300. The objective of the study is to assess the effect of PTG-300 in treating adult hereditary hemochromatosis patients.
Detailed description
This study is a multicenter, open-label study. The objective of the study is to assess the effect of PTG-300 on transferrin saturation (TSAT) and serum iron in adult hereditary hemochromatosis patients.
Interventions
DRUGPTG-300
Active treatment with PTG-300 injected subcutaneously.
Sponsors
Protagonist Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single Arm Study
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Men and women aged 18 and older * Confirmed diagnosis of HFE-related hereditary hemochromatosis with prior genotype testing * Documented stable phlebotomy for ≥ 6 months * Screening hemoglobin \>11.5 g/dL * Documented evidence of prior serum ferritin ≥500 ng/mL * Serum ferritin \>50 ng/mL and \<300 ng/mL at screening
Exclusion criteria
* Clinically meaningful laboratory abnormality * Receiving iron chelation therapy * Receiving erythrocytapheresis * Pregnant or lactating females * Infection requiring hospitalization or intravenous antimicrobial therapy, or opportunistic infection within 3 months of dosing; any infections requiring antimicrobial therapy within 4 weeks of dosing. * Serious or unstable medical or psychiatric condition that would prevent subject providing informed consent * Organ damage from iron overload * Primary or secondary immunodeficiency * Positive hepatitis B or hepatitis C or known human immunodeficiency virus infection * Known history of autoimmune/inflammatory diseases * Any surgical procedures requiring general anesthesia within 1 month prior to screening or planned surgery during study * History of invasive malignancies within last 2 years, except non-malignant skin cancer and local cured prostate cancer, cervical cancer, or ductal carcinoma in situ * Receipt of an investigational agent within 30 days of screening
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Effect of PTG-300 on Transferrin Saturation | Week 24 (or End of Treatment if treated for less than 24 weeks) | Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests. |
| Effect of PTG-300 on Serum Iron | Week 24 (or End of Treatment if treated for less than 24 weeks) | Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Effect of PTG-300 on Phlebotomy Frequency | Over 24 weeks | Change in number of phlebotomies in 24-weeks before treatment compared with the number of phlebotomies during 24-weeks of treatment with PTG-300. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Mental Component Summary of SF-36 | Week 24 (or End of Treatment if treated for less than 24 weeks) | Change from Baseline to Week 24 (or End of Treatment) in Mental Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Mental Component Summary (MCS), for mental health can be calculated. The MCS ranges from 0 (low functioning) to 100 (high functioning). |
| Physical Component Summary of SF-36 | Week 24 (or End of Treatment if treated for less than 24 weeks) | Change from Baseline to Week 24 (or End of Treatment) in Physical Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Physical Component Summary (PCS), for mental health can be calculated. The PCS ranges from 0 (low functioning) to 100 (high functioning). |
Countries
Canada, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| PTG-300 PTG-300: Active treatment with PTG-300 subcutaneously | 16 |
| Total | 16 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 3 |
| Overall Study | Did not meet inclusion criteria | 1 |
Baseline characteristics
| Characteristic | PTG-300 |
|---|---|
| Age, Continuous | 62.5 years STANDARD_DEVIATION 12.3 |
| BMI | 28.0 kg/m^2 STANDARD_DEVIATION 3.87 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 16 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 15 Participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 16 |
| other Total, other adverse events | 12 / 16 |
| serious Total, serious adverse events | 1 / 16 |
Outcome results
Primary
Effect of PTG-300 on Serum Iron
Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests.
Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)
Population: Subjects who had paired values to allow determination of change from baseline
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| PTG-300 | Effect of PTG-300 on Serum Iron | -2.34 micromol/L | Standard Deviation 9.76 |
Primary
Effect of PTG-300 on Transferrin Saturation
Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.
Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)
Population: Subjects who had paired values to allow determination of change from baseline
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| PTG-300 | Effect of PTG-300 on Transferrin Saturation | -6.07 percent saturation | Standard Deviation 19.4 |
Secondary
Effect of PTG-300 on Phlebotomy Frequency
Change in number of phlebotomies in 24-weeks before treatment compared with the number of phlebotomies during 24-weeks of treatment with PTG-300.
Time frame: Over 24 weeks
Population: Enrolled population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| PTG-300 | Effect of PTG-300 on Phlebotomy Frequency | 0.00 phlebotomies/week | Standard Deviation 0.01 |
Other Pre-specified
Mental Component Summary of SF-36
Change from Baseline to Week 24 (or End of Treatment) in Mental Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Mental Component Summary (MCS), for mental health can be calculated. The MCS ranges from 0 (low functioning) to 100 (high functioning).
Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)
Population: Subjects who have a Score at Baseline and at Week 24 or End of Treatment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| PTG-300 | Mental Component Summary of SF-36 | -0.251 units on a scale | Standard Deviation 9.379 |
Other Pre-specified
Physical Component Summary of SF-36
Change from Baseline to Week 24 (or End of Treatment) in Physical Component Summary of the SF-36. The SF-36 is a questionnaire of health status based on 36 questions distributed into eight domains of daily life that are frequently evaluated as influenced by disease. Values in each domain range from 0 to 100, with higher scores indicating better health condition. A summary measure, Physical Component Summary (PCS), for mental health can be calculated. The PCS ranges from 0 (low functioning) to 100 (high functioning).
Time frame: Week 24 (or End of Treatment if treated for less than 24 weeks)
Population: Subjects who have a Score at Baseline and at Week 24 or End of Treatment
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| PTG-300 | Physical Component Summary of SF-36 | 2.263 units on a scake | Standard Deviation 6.528 |