Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease (NCT03124459) | Clinical Trial Compass
TerminatedPhase 2
Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
Stopped: (Investigation of ACE-083 for use in patients with CMT is being discontinued as it did not achieve functional secondary endpoints in the A083-03 trial.
United States63 participantsStarted 2017-07-31
Plain-language summary
This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with Charcot-Marie-Tooth Disease Type 1 and Type X (CMT1 and CMTX), to be conducted in two parts. Part 1 is non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.
Who can participate
Age range18 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Age ≥ 18 years
✓. Diagnosis of CMT1 or CMTX confirmed by:
✓. Clinical presentation and electrodiagnostics
✓. Genetically-confirmed CMT1 or CMTX for the patient or first-degree relative
✓. Part 1:
✓. Six-minute walk distance (6MWD) of at least 150 meters (without a brace or walker)
✓. Independent ambulation for at least 10 meters, without a brace
✓. Left and right ankle plantar flexion MRC grade 4+ to 5, inclusive
Exclusion criteria
✕. History of active malignancy, with the exception of fully excised or treated basal cell carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
✕. Symptomatic cardiopulmonary disease, significant functional impairment, significant orthopedic or neuropathic pain, or other co morbidities that in the opinion of the investigator would limit a patient's ability to complete strength and/or functional assessments on study
What they're measuring
1
Part 1: Frequency of Adverse Events
Timeframe: From initiation of treatment (Study Day 1) to end of follow-up period for Part 1 (Study Day 141).
2
Part 2: Percent Change in Muscle Volume to the End of the Double-blind Placebo-controlled Portion of the Study.
Timeframe: From initiation of treatment (Study Day 1) to end of follow-up period of the double-blind placebo-controlled portion of the study (Study Day 190).
Trial details
NCT IDNCT03124459
SponsorAcceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
✕. Thyroid disorder unless condition is stable with no change in treatment for at least 4 weeks before the first dose and no expected change for duration of study
✕. Renal impairment (serum creatinine ≥ 2 times the upper limit of normal (ULN\])
✕. Increased risk of bleeding (i.e., due to hemophilia, platelet disorders, or use of any anticoagulation/platelet modifying therapies up to 2 weeks prior to Study Day 1 and for duration of study; low dose aspirin \[≤ 100 mg daily\] is permitted)
✕. Severe deformity or ankle fixation that would sufficiently limit passive range of motion to affect assessment of dorsiflexion strength