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MD1003-AMN MD1003 in Adrenomyeloneuropathy

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ClinicalTrials.gov Identifier: NCT02961803
Recruitment Status : Completed
First Posted : November 11, 2016
Last Update Posted : October 9, 2017
Sponsor:
Information provided by (Responsible Party):
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

Condition or disease Intervention/treatment Phase
Adrenomyeloneuropathy Adrenoleukodystrophy AMN Drug: MD1003 100 mg capsule Drug: Placebo Phase 2 Phase 3

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 67 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
Study Start Date : October 2014
Actual Primary Completion Date : June 2016
Actual Study Completion Date : June 2017


Arm Intervention/treatment
Experimental: MD1003
MD1003 100mg capsules, 1 capsule tid for 24 months
Drug: MD1003 100 mg capsule
Other Name: High Dose Biotin

Placebo Comparator: Placebo
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
Other Name: High Dose Biotin

Drug: Placebo



Primary Outcome Measures :
  1. Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline [ Time Frame: Baseline and 12 Months ]

Secondary Outcome Measures :
  1. 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.

  2. 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

  3. Mean Change in TW25 (time to walk 25 feet) [ Time Frame: Baseline and 12 months ]
  4. Timed up and Go test (TUG) [ Time Frame: 12 Months ]
  5. Euroqol EQ-5D questionnaire [ Time Frame: 12 months ]
    Quality of Life questionnaire

  6. Qualiveen Questionnaire [ Time Frame: 12 Months ]
    Qualiveen to evaluate urinary function



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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02961803


Locations
France
Hôpital Bicêtre
Le Kremlin Bicêtre, France, 94275 Cedex
Groupe Hospitalier Pitié-Salpêtrière
Paris, France, 75651
Germany
MS-Ambulanz Fachkrankenhaus Hubertusburg
Wermsdorf, Germany, 04779
Spain
Hospital Duran i Reynals / Bellvitge
Barcelona, Spain, 08908
Sponsors and Collaborators
MedDay Pharmaceuticals SA
Investigators
Principal Investigator: Patrick Aubourg, MD Hopital Le Kremlin-Bicêtre
Study Director: Frederic Sedel, MD Medday Pharmeuticals

Additional Information:
Responsible Party: MedDay Pharmaceuticals SA
ClinicalTrials.gov Identifier: NCT02961803     History of Changes
Other Study ID Numbers: MD1003CT2014-01AMN
First Posted: November 11, 2016    Key Record Dates
Last Update Posted: October 9, 2017
Last Verified: November 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by MedDay Pharmaceuticals SA:
AMN
MD1003
Adrenomyeloneuropathy
ALD
Adrenoleukodystrophy
Biotin
2MWT
Two-minute walk test
TW25
Timed 25-Foot Walk

Additional relevant MeSH terms:
Adrenoleukodystrophy
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
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases
Biotin
Vitamin B Complex
Vitamins
Micronutrients
Growth Substances
Physiological Effects of Drugs