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
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
67
Hôpital Bicêtre
Le Kremlin-Bicêtre, France
Groupe Hospitalier Pitié-Salpêtrière
Paris, France
MS-Ambulanz Fachkrankenhaus Hubertusburg
Wermsdorf, Germany
Hospital Duran i Reynals / Bellvitge
Barcelona, Spain
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Time frame: Baseline and 12 Months
Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%
at Months 9 and Months 12 compared to the best value among screening and baseline.
Time frame: Baseline, 9 months, 12 months
Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%
at Months 9 and Months 12 compared to the best value among screening and baseline
Time frame: Baseline, 9 months, 12 months
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
Quality of Life questionnaire
Time frame: 12 months
Qualiveen Questionnaire
Qualiveen to evaluate urinary function
Time frame: 12 Months
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