MD1003-AMN MD1003 in Adrenomyeloneuropathy
- Conditions
- AdrenoleukodystrophyAMNAdrenomyeloneuropathy
- Interventions
- Drug: MD1003 100 mg capsuleDrug: Placebo
- Registration Number
- NCT02961803
- Lead Sponsor
- MedDay Pharmaceuticals SA
- 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 Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 67
- ABCD1 gene mutation identified
- Elevated plasma VLCFA
- Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk
- EDSS score ≥ 3.5 and ≤ 6.5
- Normal brain MRI or brain MRI showing :
- abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4
- and/or stable (≥6 months) cerebral demyelination without gadolinium enhancement with a Loes score ≤12.
- Appropriate steroid replacement if adrenal insufficiency is present
- Likely to be able to participate in all scheduled evaluation visits and complete all required study procedures
- Signed and dated written informed consent to participate in the study in accordance with local regulations
- Affiliated to a Health Insurance
- Brain MRI abnormalities with a Loes score > 12 or with gadolinium enhancement
- Any progressive neurological disease other than AMN
- Impossibility to perform the walk tests and the TUG test
- Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or any progressive malignancy
- Any new medication for AMN including Fampridine initiated less than 1 month prior to inclusion
- Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants.
- Inclusion in another therapeutic clinical trial for ALD
- Not easily contactable by the investigator in case of emergency or not capable to call the investigator
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description MD1003 MD1003 100 mg capsule MD1003 100mg capsules, 1 capsule tid for 24 months Placebo Placebo Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months Placebo MD1003 100 mg capsule Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months
- Primary Outcome Measures
Name Time Method Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline Baseline and 12 Months
- Secondary Outcome Measures
Name Time Method Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20% Baseline, 9 months, 12 months at Months 9 and Months 12 compared to the best value among screening and baseline
Timed up and Go test (TUG) 12 Months Euroqol EQ-5D questionnaire 12 months Quality of Life questionnaire
Mean Change in TW25 (time to walk 25 feet) Baseline and 12 months Qualiveen Questionnaire 12 Months Qualiveen to evaluate urinary function
Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20% Baseline, 9 months, 12 months at Months 9 and Months 12 compared to the best value among screening and baseline.
Trial Locations
- Locations (4)
Hospital Duran i Reynals / Bellvitge
🇪🇸Barcelona, Spain
Groupe Hospitalier Pitié-Salpêtrière
🇫🇷Paris, France
Hôpital Bicêtre
🇫🇷Le Kremlin Bicêtre, France
MS-Ambulanz Fachkrankenhaus Hubertusburg
🇩🇪Wermsdorf, Germany