Back to resultsMD1003-AMN MD1003 in Adrenomyeloneuropathy
Primary Purpose
Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
MD1003 100 mg capsule
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Adrenomyeloneuropathy focused on measuring AMN, MD1003, Adrenomyeloneuropathy, ALD, Adrenoleukodystrophy, Biotin, 2MWT, Two-minute walk test, TW25, Timed 25-Foot Walk
Eligibility 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
Sites / Locations
- Hôpital Bicêtre
- Groupe Hospitalier Pitié-Salpêtrière
- MS-Ambulanz Fachkrankenhaus Hubertusburg
- Hospital Duran i Reynals / Bellvitge
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
MD1003
Placebo
Arm Description
MD1003 100mg capsules, 1 capsule tid for 24 months
Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months
Outcomes
Primary Outcome Measures
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Secondary Outcome Measures
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.
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
Mean Change in TW25 (time to walk 25 feet)
Timed up and Go test (TUG)
Euroqol EQ-5D questionnaire
Quality of Life questionnaire
Qualiveen Questionnaire
Qualiveen to evaluate urinary function
Full Information
NCT ID
NCT02961803
First Posted
November 9, 2016
Last Updated
October 6, 2017
Sponsor
MedDay Pharmaceuticals SA
1. Study Identification
Unique Protocol Identification Number
NCT02961803
Brief Title
MD1003-AMN MD1003 in Adrenomyeloneuropathy
Official Title
MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
Study Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
October 2014 (undefined)
Primary Completion Date
June 2016 (Actual)
Study Completion Date
June 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MedDay Pharmaceuticals SA
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
Keywords
AMN, MD1003, Adrenomyeloneuropathy, ALD, Adrenoleukodystrophy, Biotin, 2MWT, Two-minute walk test, TW25, Timed 25-Foot Walk
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
67 (Actual)
8. Arms, Groups, and Interventions
Arm Title
MD1003
Arm Type
Experimental
Arm Description
MD1003 100mg capsules, 1 capsule tid for 24 months
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months
Intervention Type
Drug
Intervention Name(s)
MD1003 100 mg capsule
Other Intervention Name(s)
High Dose Biotin
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Time Frame
Baseline and 12 Months
Secondary Outcome Measure Information:
Title
Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%
Description
at Months 9 and Months 12 compared to the best value among screening and baseline.
Time Frame
Baseline, 9 months, 12 months
Title
Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%
Description
at Months 9 and Months 12 compared to the best value among screening and baseline
Time Frame
Baseline, 9 months, 12 months
Title
Mean Change in TW25 (time to walk 25 feet)
Time Frame
Baseline and 12 months
Title
Timed up and Go test (TUG)
Time Frame
12 Months
Title
Euroqol EQ-5D questionnaire
Description
Quality of Life questionnaire
Time Frame
12 months
Title
Qualiveen Questionnaire
Description
Qualiveen to evaluate urinary function
Time Frame
12 Months
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Patrick Aubourg, MD
Organizational Affiliation
Hopital Le Kremlin-Bicêtre
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Frederic Sedel, MD
Organizational Affiliation
Medday Pharmeuticals
Official's Role
Study Director
Facility Information:
Facility Name
Hôpital Bicêtre
City
Le Kremlin Bicêtre
ZIP/Postal Code
94275 Cedex
Country
France
Facility Name
Groupe Hospitalier Pitié-Salpêtrière
City
Paris
ZIP/Postal Code
75651
Country
France
Facility Name
MS-Ambulanz Fachkrankenhaus Hubertusburg
City
Wermsdorf
ZIP/Postal Code
04779
Country
Germany
Facility Name
Hospital Duran i Reynals / Bellvitge
City
Barcelona
ZIP/Postal Code
08908
Country
Spain
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
http://www.medday-pharma.com
Description
Sponsor
Learn more about this trial
MD1003-AMN MD1003 in Adrenomyeloneuropathy
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