MD1003-AMN MD1003 in Adrenomyeloneuropathy

NCT02961803 | Completed Phase 2 DMC

• 1 other identifier: MD1003CT2014-01AMN
• Study Type: interventional
• Target: 67
• Locations: 3 countries
• Sites: 4

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

Conditions

Adrenomyeloneuropathy; Adrenoleukodystrophy; AMN

Keywords

AMN; MD1003; Adrenomyeloneuropathy; ALD; Adrenoleukodystrophy; Biotin; 2MWT; Two-minute walk test; TW25; Timed 25-Foot Walk

Outcome Measures

Primary Outcomes (1)

• Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline

  Baseline and 12 Months

Secondary Outcomes (6)

• Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%

  Baseline, 9 months, 12 months

• Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%

  Baseline, 9 months, 12 months

• Mean Change in TW25 (time to walk 25 feet)

  Baseline and 12 months

• Timed up and Go test (TUG)

  12 Months

• Euroqol EQ-5D questionnaire

  12 months

Study Arms (2)

MD1003

EXPERIMENTAL

MD1003 100mg capsules, 1 capsule tid for 24 months

Drug: MD1003 100 mg capsule

Placebo

PLACEBO COMPARATOR

Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months

Drug: MD1003 100 mg capsule; Drug: Placebo

Interventions

MD1003 100 mg capsule DRUG

Also known as: High Dose Biotin

MD1003; Placebo

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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
• Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants.
• Not easily contactable by the investigator in case of emergency or not capable to call the investigator

Sponsors & Collaborators

• MedDay Pharmaceuticals SA: lead

Study Sites (4)

Hôpital Bicêtre

Le Kremlin-Bicêtre, 94275 Cedex, France

Location

Groupe Hospitalier Pitié-Salpêtrière

Paris, 75651, France

Location

MS-Ambulanz Fachkrankenhaus Hubertusburg

Wermsdorf, 04779, Germany

Location

Hospital Duran i Reynals / Bellvitge

Barcelona, 08908, Spain

Location

MeSH Terms

Conditions

Adrenoleukodystrophy

Interventions

Biotin

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Coenzymes; Enzymes and Coenzymes

Study Officials

• Patrick Aubourg, MD

  Hopital Le Kremlin-Bicêtre

  PRINCIPAL INVESTIGATOR

• Frederic Sedel, MD

  Medday Pharmeuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 9, 2016
• First Posted: November 11, 2016
• Study Start: October 1, 2014
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2017
• Last Updated: October 9, 2017

Record last verified: 2016-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2); DE (1); ES (1)