Clinical Trial: MD1003-AMN MD1003 in Adrenomyeloneuropathy
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
Brief Summary: The primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy
Detailed Summary: AMN and progressive multiple sclerosis share some similarities including progressive spastic paraparesis and secondary energy failure leading to progressive axonal degeneration. Therefore, it was hypothesized that high doses of biotin might be efficient in patients with AMN.
Sponsor: MedDay Pharmaceuticals SA
Current Primary Outcome: Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline [ Time Frame: Baseline and 12 Months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20% [ Time Frame: Baseline, 9 months, 12 months ]at Months 9 and Months 12 compared to the best value among screening and baseline.
- Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20% [ Time Frame: Baseline, 9 months, 12 months ]at Months 9 and Months 12 compared to the best value among screening and baseline
- Mean Change in TW25 (time to walk 25 feet) [ Time Frame: Baseline and 12 months ]
- Timed up and Go test (TUG) [ Time Frame: 12 Months ]
- Euroqol EQ-5D questionnaire [ Time Frame: 12 months ]Quality of Life questionnaire
- Qualiveen Questionnaire [ Time Frame: 12 Months ]Qualiveen to evaluate urinary function
Original Secondary Outcome: Same as current
Information By: MedDay Pharmaceuticals SA
Dates:
Date Received: November 9, 2016
Date Started: October 2014
Date Completion: August 2017
Last Updated: November 15, 2016
Last Verified: November 2016
