Adrenomyeloneuropathy MedDRA version: 17.0 Level: LLT Classification code 10069075 Term: Adrenomyeloneuropathy without cerebral involvement System Organ Class: 100000004850
Conditions
Interventions
Product Name: biotin
Product Code: MD1003
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: D-BIOTIN
Current Sponsor code: MD1003
Concentration unit: mg milligram(s)
Concentration type: equal
Co
Sponsors
MEDDAY SAS
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - Men of 18 to 60 years old, inclusive - 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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 60 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Brain MRI abnormalities with a Loes score > 12 or with gadolinium enhacement - 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 evolutive malignancy - Any new medication for AMN including Fampridine initiated less than 1 month prior to inclusion - Contraindications 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 - Patient not easily reachable by the investigator in case of emergency or not capable to call the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the superiority of MD1003 (biotin) at 300 mg/day over placebo in the clinical improvement of patients with Adrenomyeloneuropathy;Secondary Objective: To evaluate safety of MD1003 in patients with Adrenomyeloneuropathy;Primary end point(s): Mean change of 2 minutes walking test (2MWT) between M12 and baseline;Timepoint(s) of evaluation of this end point: Baseline, 12 months, 24 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20% at M9 and M12 compared to the best value among screening and baseline? - Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20% at M9 and M12 compared to the best value among screening and baseline - Timed up and Go test (TUG) - Euroqol EQ-5D questionnaire - Qualiveen to evaluate urinary function - To evaluate the safety of high dose of biotin assessed by: adverse events, clinical examination (vital signs, ECG), standard laboratory tests (hematology, biochemistry) Exploratory studies (optional): 1) Brain MRI with conventional and non conventional sequences: a) diffusion tensor imaging (DTI) parameters in the upper cervical spinal cord and brain; b) magnetic resonance spectroscopy (MRS) in the cerebral white matter. 2) Evaluation of muscle strength by the use of a dynamometer 3) Nerve conduction velocities in lower limbs (NCV);Timepoint(s) of evaluation of this end point: Baseline, 9 months, 12 months, 18 months, 24 months | — |
Countries
Spain
Contacts
Public ContactProfesor de Investigación ICREA
Dra Aurora Pujol
Outcome results
None listed