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This open-label, single-arm multi-center study studying the safety and efficacy of TXA127 on non-ambulant patients with DMD Cardiomyopathy will comprise of two phases:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TXA127 SC 0.5mg/kg/day | Experimental | TXA127 (talfirastide) 0.5mg/kg/day given via subcutaneous injection for 6 months |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| talfirastide | Drug | TXA127 (talfirastide) is a pharmaceutically formulated angiotensin (1-7) heptapeptide, identical to the endogenously produced, non-hypertensive derivative of angiotensin II (Ang II). |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the safety of TXA127 in patients with DMD | Incidence of adverse events (AEs), their severity and relationship to treatment | 6 months plus 12 month extension |
| To evaluate the effects of treatment on ejection fraction (EF) | Percent change in EF, as measured by echocardiogram (ECHO); Absolute change in EF, as measured by echocardiogram | 6 months plus 12 month extension |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the effects of treatment on upper extremity muscle function | Percent change in upper extremity strength, as measured by grip strength with a dynamometer; Absolute change in upper extremity strength, as measured by grip strength with a dynamometer | 6 months plus 12 month extension |
| To evaluate the effects of treatment on fractional shortening (FS) |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the effects of treatment on exploratory DMD-related clinical signs, FVC | Forced Vital Capacity (FVC) % predicted Absolute and percent (where applicable) change in the following biomarkers in the blood:
| 6 months plus 12 month extension |
Inclusion Criteria:
Male subjects 16 years of age or older who provide informed consent and can follow up with protocol procedures. Parental or guardian consent is required for subjects at least 16 years of age but younger than 18 years of age.
Documented diagnosis of Duchenne muscular dystrophy by genetic mutation analysis.
Documented cardiomyopathy, as assessed by echocardiogram with:
Reproducible (+/- 10%) difference between screening and baseline of percent predicted FVC , using the best out of 3 efforts at each visit:
Subjects must be taking systemic glucocorticoids for at least six months prior to screening.
Subjects taking mineralocorticoid receptor antagonists, must be taking the drug for at least three months prior to screening
Non-ambulant and cared for by a trained caregiver
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Richard L Franklin, MD, PhD | Contact | 1-617-245-0289 | 101 | rfranklin@constanttherapeutics.com |
| Elizabeth Wagner, MS, MBA | Contact | 1-617-245-0289 | 102 | ewagner@constanttherapeutics.com |
| Name | Affiliation | Role |
|---|---|---|
| Richard L Franklin, MD, PhD | Constant Therapeutics LLC | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hadassah Medical Center | Recruiting | Jerusalem | Israel |
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| ID | Term |
|---|---|
| C580047 | Dmd-Associated Dilated Cardiomyopathy |
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| ID | Term |
|---|---|
| C118790 | angiotensin I (1-7) |
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|
Percent and absolute change in fractional shortening as measured by echocardiogram |
| 6 months plus 12 month extension |
| To evaluate the effects of treatment on exploratory DMD-related clinical signs, PEF |
Peak Expiratory Flow (PEF) % predicted (if available) Absolute and percent (where applicable) change in the following biomarkers in the blood:
|
| 6 months plus 12 month extension |
| To evaluate the effects of treatment on exploratory DMD-related biomarkers, Troponin | Absolute and percent (where applicable) change in Troponin | 6 months plus 12 month extension |
| To evaluate the effects of treatment on exploratory DMD-related biomarkers, BNP | Absolute and percent (where applicable) change in Brain Natriuretic Peptide (BNP) | 6 months plus 12 month extension |
| To evaluate the effects of treatment on exploratory DMD-related biomarkers, BDNF | Absolute and percent (where applicable) change in Brain-derived neurotrophic factor (BDNF) | 6 months plus 12 month extension |
| Sheba Medical Center | Recruiting | Ramat Gan | Israel |
|