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 range
18 Years
Sex
ALL
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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
. 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
. Type 1 or type 2 diabetes mellitus
. 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