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: 1. 6-month open-label treatment phase: Male DMD patients with documented cardiomyopathy, will receive a daily subcutaneous injection of TXA127 0.5 mg/kg. Treatment will be provided for 6 months. Treatment safety will be assessed by collection and review of AEs, vital signs, ECGs, physical examinations, PFTs, and laboratory parameters on Day 1, Month 1, and Month 6. Ejection Fraction, upper extremity strength and biomarker levels will be assessed at these study visits as well. In addition, an abbreviated safety visit will be conducted at Month 3. 2. 12-month optional extension phase: Patients will continue the same study drug regime for an additional 12 months. The primary objective of this phase is to obtain long-term safety data. Efficacy data will also be collected. Safety, efficacy, and exploratory biomarkers will be assessed at Month 12 and Month 18, using the same methods as in the treatment phase. In addition, abbreviated safety visits will be conducted at Month 9 and Month 15.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
TXA127 (talfirastide) is a pharmaceutically formulated angiotensin (1-7) heptapeptide, identical to the endogenously produced, non-hypertensive derivative of angiotensin II (Ang II).
Hadassah Medical Center
Jerusalem, Israel
RECRUITINGSheba Medical Center
Ramat Gan, Israel
RECRUITINGTo evaluate the safety of TXA127 in patients with DMD
Incidence of adverse events (AEs), their severity and relationship to treatment
Time frame: 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
Time frame: 6 months plus 12 month extension
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
Time frame: 6 months plus 12 month extension
To evaluate the effects of treatment on fractional shortening (FS)
Percent and absolute change in fractional shortening as measured by echocardiogram
Time frame: 6 months plus 12 month extension
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