453 Studies Now Enrolling
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Charcot-Marie-Tooth Disease Types 1 and X
Study of Study Drug (ACE-083) in Patients with Carcot-Marie-Tooth Disease Types 1 and X
| Sponsor: | Acceleron Pharma Inc. |
| Enrolling: | Male and Female Patients |
| IRB Number: | AAAR8240 |
| U.S. Govt. ID: | NCT03124459 |
| Contact: | Thomas Brannagan, III, MD: 212-305-0405 / tb2325@cumc.columbia.edu |
Additional Study Information:
Patients with Charcot-Marie-Tooth (CMT) disease have a degenerative nerve disease that can cause muscle weakness and decreased muscle size in different muscles in their body. This study drug is designed to evaluate ACE-083 for its ability to increase the size of your muscles, which could improve your muscle strength. The purpose of this research study is to: Evaluate the safety and tolerability of the study drug in patients with CMT types 1 and X, measure the amount of study drug in the blood of CMT patients dosed with ACE-083, evaluate changes in muscle size, strength, and abilities (walking, sitting and standing up), and assess how you are feeling before, during, and after study drug treatment
This study is closed
Do You Qualify?
| Do you have independent ambulation for at least 10 meters, without a brace? | Yes | No |
| Are you genetically confirmed with CMT1 or CMTX or have a first degree relative that has genetic confirmation? | Yes | No |
| History of active malignancy (except basal cell carcinoma, cerivical carcinoma, or squamous cell carcinomas of skin)? | Yes | No |
| Previous exposure to any investigational agent potentially affecting muscle volume, muscle strength, or muscle or nerve function | Yes | No |
| Do you have severe ankle deformity that limits movement of your ankle? | Yes | No |