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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PTG-300 | Experimental | PTG-300 Subcutaneous |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PTG-300 | Drug | Active treatment with PTG-300 injected subcutaneously. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Week 24 (or End of Treatment if treated for less than 24 weeks) |
| 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. | Week 24 (or End of Treatment if treated for less than 24 weeks) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Over 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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). |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Protagonist Investigational Site | Fayetteville | Georgia | 30214 | United States | ||
| Protagonist Investigational Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37863080 | Derived | Kowdley KV, Modi NB, Peltekian K, Vierling JM, Ferris C, Valone FH, Gupta S. Rusfertide for the treatment of iron overload in HFE-related haemochromatosis: an open-label, multicentre, proof-of-concept phase 2 trial. Lancet Gastroenterol Hepatol. 2023 Dec;8(12):1118-1128. doi: 10.1016/S2468-1253(23)00250-9. Epub 2023 Oct 17. |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | PTG-300 | PTG-300: Active treatment with PTG-300 subcutaneously |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Subjects who had paired values to allow determination of change from baseline | Posted | Mean | Standard Deviation | percent saturation | Week 24 (or End of Treatment if treated for less than 24 weeks) |
|
|
6 months
Safety was assessed using the National Institutes of Health (NIH) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 criteria.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | PTG-300 | PTG-300: Active treatment with PTG-300 subcutaneously | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Adenocarcinoma pancreas | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (Unspecified) | Systematic Assessment | Pre-existing condition diagnosed during study. Not related to study drug |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Study Director | Protagonist Therapeutics, Inc | 1-888-899-1543 | ptgxclintrials@ptgx-inc.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Sep 14, 2020 | Apr 18, 2023 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D006432 | Hemochromatosis |
| ID | Term |
|---|---|
| D008664 | Metal Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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Single Arm Study
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| Week 24 (or End of Treatment if treated for less than 24 weeks) |
| 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). | Week 24 (or End of Treatment if treated for less than 24 weeks) |
| Bethesda |
| Maryland |
| 20817 |
| United States |
| Protagonist Investigational Site | Charlotte | North Carolina | 28210 | United States |
| Protagonist Investigational Site | Conroe | Texas | 77384 | United States |
| Protagonist Investigational Site | Dallas | Texas | 75246 | United States |
| Protagonist Investigational Site | Houston | Texas | 77030 | United States |
| Protagonist Investigational Site | Houston | Texas | 77058 | United States |
| Protagonist Investigational Site | Richmond | Virginia | 23226 | United States |
| Protagonist Investigational Site | Seattle | Washington | 98105 | United States |
| Protagonist Investigational Site | Halifax | Nova Scotia | Canada |
| Protagonist Investigational Site | Toronto | Ontario | M6H 3M1 | Canada |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| BMI | Mean | Standard Deviation | kg/m^2 |
|
|
| 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. | Subjects who had paired values to allow determination of change from baseline | Posted | Mean | Standard Deviation | micromol/L | Week 24 (or End of Treatment if treated for less than 24 weeks) |
|
|
|
| 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. | Enrolled population | Posted | Mean | Standard Deviation | phlebotomies/week | Over 24 weeks |
|
|
|
| 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). | Subjects who have a Score at Baseline and at Week 24 or End of Treatment | Posted | Mean | Standard Deviation | units on a scale | Week 24 (or End of Treatment if treated for less than 24 weeks) |
|
|
|
| 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). | Subjects who have a Score at Baseline and at Week 24 or End of Treatment | Posted | Mean | Standard Deviation | units on a scake | Week 24 (or End of Treatment if treated for less than 24 weeks) |
|
|
|
| 16 |
| 1 |
| 16 |
| 12 |
| 16 |
|
| Dry mouth | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Supraventricular extrasystoles | Cardiac disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Inflammation | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site bruising | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site discomfort | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site erythema | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site haematoma | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site haemorrhage | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site induration | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site pain | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site pruritus | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Injection site swelling | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Malaise | General disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Cellulitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Gastrointestinal bacterial overgrowth | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Hand fracture | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (Unspecified) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Bronchospasm | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA (Unspecified) | Systematic Assessment |
|
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| D019190 | Iron Overload |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |