AdrenomyeloneuropathyPhase 2/3NCT02961803
MD1003-AMN MD1003 in Adrenomyeloneuropathy
A Phase 2/3 study testing MD1003 100 mg capsule in people with Adrenomyeloneuropathy and Adrenoleukodystrophy, run by MedDay Pharmaceuticals SA at 4 sites in 3 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 60 years, men only
- Conditions the study is for
- Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
- What is being tested?
- MD1003 100 mg capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 67 participants
- Where
- 4 sitesin 3 countries
- Study dates
- Oct 2014to Jun 2017
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- Men only
- No healthy volunteers
- Adult
Who may be able to join
- 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
Show all 12Show fewer
- 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
- Not easily contactable by the investigator in case of emergency or not capable to call the investigator
Who cannot join
- 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.
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
France2 sites
Hôpital Bicêtre
Le Kremlin-Bicêtre, 94275 Cedex
Groupe Hospitalier Pitié-Salpêtrière
Paris, 75651
About this study
Official title: MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
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
Read the 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.
- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
- Being tested
- MD1003 100 mg capsule, Placebo
- Keywords
- AMN, MD1003, Adrenomyeloneuropathy, ALD, Adrenoleukodystrophy, Biotin, 2MWT, Two-minute walk test, TW25, Timed 25-Foot Walk
- Medical subject headings
- Adrenoleukodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MD1003 100 mg capsuleDrug
Also called High Dose Biotin
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| MD1003 | Experimental | MD1003 100mg capsules, 1 capsule tid for 24 months Receives: 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 Receives: Drug: MD1003 100 mg capsule; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Measured: Baseline and 12 Months
Secondary outcomes (6)
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.
Measured: Baseline, 9 months, 12 months
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
Measured: Baseline, 9 months, 12 months
Mean Change in TW25 (time to walk 25 feet)
Measured: Baseline and 12 months
Timed up and Go test (TUG)
Measured: 12 Months
Euroqol EQ-5D questionnaire
Quality of Life questionnaire
Measured: 12 months
Qualiveen Questionnaire
Qualiveen to evaluate urinary function
Measured: 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2016 (estimated)
- Study startOct 2014
- Primary completionJun 2016
- Study completionJun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AdrenomyeloneuropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 67 participants (actual)
Sponsor and investigators
- Lead sponsor
- MedDay Pharmaceuticals SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Patrick Aubourg, MD, Principal investigator, Hopital Le Kremlin-BicêtreFrederic Sedel, MD, Study director, Medday Pharmeuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02961803
- Sponsor's protocol ID
- MD1003CT2014-01AMN
- Status last verified
- Nov 2016
- First submitted
- 9 Nov 2016
- Last updated
- 9 Oct 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
- ClinicalTrials.gov ID
- NCT02961803
- Acronym / sponsor ID
- MD1003CT2014-01AMN
- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
- Keywords
- AMN, MD1003, Adrenomyeloneuropathy, ALD, Adrenoleukodystrophy, Biotin, 2MWT, Two-minute walk test
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 67 participants (actual)
- Lead sponsor
- MedDay Pharmaceuticals SAIndustry (pharma, biotech, devices)
- Study start
- Oct 2014
- Primary completion
- Jun 2016 (actual)
- Study completion
- Jun 2017 (actual)
- First posted
- 11 Nov 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MD1003 | Experimental | Drug: MD1003 100 mg capsuleMD1003 100mg capsules, 1 capsule tid for 24 months |
| Placebo | Placebo comparator | Drug: MD1003 100 mg capsule, Drug: PlaceboPlacebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (12 listed)
- 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 :
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MD1003 100 mg capsuleAlso: High Dose Biotin | Drug | MD1003, Placebo |
| Placebo | Drug | Placebo |
Primary outcome
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
Time frame: Baseline and 12 Months
6 secondary outcomes
Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%
Time frame: Baseline, 9 months, 12 months
Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%
Time frame: Baseline, 9 months, 12 months
Mean Change in TW25 (time to walk 25 feet)
Time frame: Baseline and 12 months
Timed up and Go test (TUG)
Time frame: 12 Months
Euroqol EQ-5D questionnaire
Time frame: 12 months
Qualiveen Questionnaire
Time frame: 12 Months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2017.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patrick Aubourg, MD principal investigator
Hopital Le Kremlin-Bicêtre
Frederic Sedel, MD study director
Medday Pharmeuticals
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing MD1003 100 mg capsule in people with Adrenomyeloneuropathy and Adrenoleukodystrophy, run by MedDay Pharmaceuticals SA at 4 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France, Germany, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline (time frame: Baseline and 12 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2017.