Trial Outcomes & Findings for PTG-300 in Subjects With Hereditary Hemochromatosis (NCT NCT04202965)
NCT ID: NCT04202965
Last Updated: 2025-08-20
Results Overview
Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
Week 24 (or End of Treatment if treated for less than 24 weeks)
Results posted on
2025-08-20
Participant Flow
Participant milestones
| Measure |
PTG-300
PTG-300 injected subcutaneously. PTG-300 doses individually titrated with doses ranging from 10 mg to 40 mg once weekly or twice a week.
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
PTG-300
PTG-300 injected subcutaneously. PTG-300 doses individually titrated with doses ranging from 10 mg to 40 mg once weekly or twice a week.
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Did not meet inclusion criteria
|
1
|
Baseline Characteristics
PTG-300 in Subjects With Hereditary Hemochromatosis
Baseline characteristics by cohort
| Measure |
PTG-300
n=16 Participants
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Age, Continuous
|
62.5 years
STANDARD_DEVIATION 12.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
BMI
|
28.0 kg/m^2
STANDARD_DEVIATION 3.87 • n=5 Participants
|
PRIMARY outcome
Timeframe: 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
Change from Baseline to Week 24 (or End of Treatment) in transferrin saturation (TSAT) as measured by blood laboratory tests.
Outcome measures
| Measure |
PTG-300
n=14 Participants
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Effect of PTG-300 on Transferrin Saturation
|
-6.07 percent saturation
Standard Deviation 19.4
|
PRIMARY outcome
Timeframe: 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
Change from Baseline to Week 24 (or End of Treatment) in serum iron as measured by blood laboratory tests.
Outcome measures
| Measure |
PTG-300
n=14 Participants
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Effect of PTG-300 on Serum Iron
|
-2.34 micromol/L
Standard Deviation 9.76
|
SECONDARY outcome
Timeframe: Over 24 weeksPopulation: Enrolled population
Change in number of phlebotomies in 24-weeks before treatment compared with the number of phlebotomies during 24-weeks of treatment with PTG-300.
Outcome measures
| Measure |
PTG-300
n=16 Participants
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Effect of PTG-300 on Phlebotomy Frequency
|
0.00 phlebotomies/week
Standard Deviation 0.01
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 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
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).
Outcome measures
| Measure |
PTG-300
n=15 Participants
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Mental Component Summary of SF-36
|
-0.251 units on a scale
Standard Deviation 9.379
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 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
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).
Outcome measures
| Measure |
PTG-300
n=15 Participants
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Physical Component Summary of SF-36
|
2.263 units on a scake
Standard Deviation 6.528
|
Adverse Events
PTG-300
Serious events: 1 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
PTG-300
n=16 participants at risk
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma pancreas
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
Other adverse events
| Measure |
PTG-300
n=16 participants at risk
PTG-300: Active treatment with PTG-300 subcutaneously
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
12.5%
2/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Gastrointestinal disorders
Dry mouth
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Gastrointestinal disorders
Nausea
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Gastrointestinal disorders
Vomiting
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Cardiac disorders
Supraventricular extrasystoles
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Fatigue
|
12.5%
2/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Inflammation
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site bruising
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site discomfort
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site erythema
|
12.5%
2/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site haematoma
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site haemorrhage
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site induration
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site pain
|
31.2%
5/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site pruritus
|
18.8%
3/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Injection site swelling
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
General disorders
Malaise
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Infections and infestations
Cellulitis
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Infections and infestations
Gastrointestinal bacterial overgrowth
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Injury, poisoning and procedural complications
Hand fracture
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Nervous system disorders
Dizziness
|
12.5%
2/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Nervous system disorders
Headache
|
12.5%
2/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Nervous system disorders
Paraesthesia
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Psychiatric disorders
Insomnia
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.2%
1/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
|
Vascular disorders
Hypertension
|
12.5%
2/16 • 6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
|
Additional Information
Clinical Study Director
Protagonist Therapeutics, Inc
Phone: 1-888-899-1543
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place