ALSPhase 2MD1003-ALSNCT03114215
Effect of MD1003 in Amyotrophic Lateral Sclerosis
A Phase 2 study testing MD1003 in people with ALS and Amyotrophic Lateral Sclerosis, run by MedDay Pharmaceuticals SA at 1 site in France.
Trial summary
This is a 6-month double blind randomized 2:1 placebo-controlled study with two arms (placebo, biotin 300 mg/day). The study will be followed by a 6-month extension phase during which all patients will receive biotin 300 mg/day. Read more about this study
- Who is it for?
- Ages 25 to 80 years
- Conditions the study is for
- ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- What is being tested?
- MD1003
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- 29 Jun 2016to Dec 2017
- Phase
- Phase 2
- 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 25 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age: 25 to 80 years, inclusive
- Male or female subjects with probable or confirmed ALS (revised international El Escorial criteria, Forbes et al., 2001).
- Patients presenting first motor deficits due to ALS for a maximum of three years at the first consultation in an ALS centre.
- Patients monitored for at least 6 months in an ALS centre or for whom the previous monitoring parameters are available (excepted for MIP and SNIP).
- Patients who have lost at least 5 points on the ALSFRS-R (ALS functional rating scale) during the last 12 months or at least 2 points during the preceding 6 months
- Patients who have been treated with riluzole for at least 3 months at a stable dose. In case of intolerance to this product or refusal for this treatment, patients who have not been treated with riluzole for at least 1 month before inclusion
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- For patients with spinal form (onset of the disease affecting limbs) or respiratory form, slow vital capacity > 60% of predicted value.
- For patients with a bulbar form, slow vital capacity > 60% of theoretical value or, if spirometry not assessable (severe bulbar disability), patient should not have significant abnormality in both nocturnal capnography and nocturnal oximetry (median pCO2 (carbon dioxide partial pressure ) < 52 mmHg, SaO2 (arterial oxygen saturation ) < 90% less than 5% of the time during night) less than 3 months prior inclusion.
- Patients who are willing to give written consent (or oral consent in the presence of a trusted person if the patient is no longer able to write)
- Patients likely to be able to participate in all scheduled evaluation and complete all required study procedures (except for spirometry in bulbar patients with severe disability).
Who cannot join
- Patients on non-invasive ventilation for respiratory insufficiency due to ALS for more than 10 hours a day
- Patients with an ALSFRS-R score at inclusion of < 20 (maximum score without disability = 48)
- Patients who have lost less than 5 points on the ALSFRS-R during the last year or less than 2 points during the preceding 6 months
- Patients with a gastrostomy
- Patients who have lost more than 15% of their reference weight (defined as weight before disease onset)
- Patient with dyspnoea at rest or with the least effort (score < 3 on the dyspnoea item of the ALSFRS-R)
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- Patients with dementia
- Patient with severe or rapidly progressive form of ALS for whom the investigator estimates the life expectancy less than 3 months
- Patients with another progressive disease that has not been stabilized at the time of inclusion
- Patients with cancer, except basal cell carcinoma, for less than 5 years, or who require continuous treatment for cancer even if it is older
- Pregnant women.
- Subject who are not covered by a social security scheme.
- Subject under temporary or permanent Judicial Protection.
- Contraception: Both male subjects, and female subjects who are not either surgically sterile (tubal ligation/obstruction or removal of ovaries or uterus) or post-menopausal (no spontaneous menstrual periods for at least one year confirmed by a negative hormone panel), must commit to using two highly effective method of birth control for the duration of the study and for two months after the treatment termination.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effect of MD1003 in Amyotrophic Lateral Sclerosis: a Randomized Double Blind Placebo Controlled Pilot Study
This is a 6-month double blind randomized 2:1 placebo-controlled study with two arms (placebo, biotin 300 mg/day). The study will be followed by a 6-month extension phase during which all patients will receive biotin 300 mg/day.
- Conditions
- ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- Being tested
- MD1003, Placebo oral capsule
- Keywords
- Biotin, MD1003, ALSFRS-R
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MD1003Drug
Also called BIOTIN
capsules 100mg 3 times per day
Placebo oral capsuleDrug
capsules 100mg lactose 3 times per day
| Study group | Role | What they receive |
|---|---|---|
| MD1003 | Active comparator | The investigational drug will consist in capsules of 100 mg biotin and excipients (lactose, magnesium stearate, croscarmellose sodium, Silica) tid during 12 months Receives: Drug: MD1003 |
| PLACEBO | Placebo comparator | This formulation consists in lactose powder and other excipients (magnesium stearate, croscarmellose sodium, Silica) as placebo, tid during 6 months and then switch to MD1003 tid during 6 additional months. Receives: Drug: MD1003; Drug: Placebo oral capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recording of adverse events
All adverse events in two groups will be recorded.
Measured: 6 months
Laboratory testing (haematology and biochemistry panel)
- RBC (red blood cell), WBC (white blood cell ), platelets
- Ferritin, CPK (creatine phosphokinase )
- Electrolytes, creatinine, glycaemia
- AST (aspartate aminotransferase ), ALT (alanine aminotransferase) , bilirubin, GGT (gamma-glutamyltransferase), alkaline phosphatase
- Triglyceride, cholesterol
- Haemostasis: APPT (activated partial thromboplastin time), PT (prothrombin time )
Measured: 6 months
Secondary outcomes (6)
Motor disability
this is evaluated using the ALSFRS-R scale (score of 48 points). Among the criteria used to evaluate the severity of ALS, the rate of the decline in the ALSFRS-R is the one that correlates most closely with the risk of death (Kimura et al., 2006).
Measured: 6 months
Severity
The severity of the disease defined as the ratio between the number of points lost on the ALSFRS-R score and the number of months that have elapsed (Kollewe et al., 2008).
Measured: 6 months
Slow vital capacity (SVC)
in liters
Measured: 6 months
Maximal inspiratory pressure (MIP)
in cm H2O
Measured: 6 months
Sniff nasal inspiratory pressure (SNIP)
in cm H20
Measured: 6 months
Weight
weight in kg
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2017
- Study start29 Jun 2016
- Primary completion12 Jun 2017
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ALSStudies 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
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The primary objective of the study is to evaluate the safety of biotin at 300 mg/day over placebo in patients with amyotrophic lateral sclerosis. This is a 6-month double blind randomized 2:1 placebo-controlled study with two arms (placebo, biotin 300 mg/day). The study will be followed by a 6-month extension phase during which all patients will receive biotin 300 mg/day.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- MedDay Pharmaceuticals SA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03114215
- Sponsor's protocol ID
- MD1003CT2015-02-ALS
- Acronym
- MD1003-ALS
- Status last verified
- Jun 2019
- First submitted
- 28 Mar 2017
- Last updated
- 4 Jun 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Juntas-Morales R, Pageot N, Bendarraz A, Alphandery S, Sedel F, Seigle S, Camu W. High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: A pilot study. EClinicalMedicine. 2020 Jan 27;19:100254. doi: 10.1016/j.eclinm.2019.100254. eCollection 2020 Feb.
PubMed 32140672
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of MD1003 in Amyotrophic Lateral Sclerosis: a Randomized Double Blind Placebo Controlled Pilot Study
- ClinicalTrials.gov ID
- NCT03114215
- Acronym / sponsor ID
- MD1003-ALS / MD1003CT2015-02-ALS
- Conditions
- ALS, Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- Keywords
- Biotin, MD1003, ALSFRS-R
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- MedDay Pharmaceuticals SAIndustry (pharma, biotech, devices)
- Study start
- 29 Jun 2016 (actual)
- Primary completion
- 12 Jun 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 14 Apr 2017 (actual)
- 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.
This is a 6-month double blind randomized 2:1 placebo-controlled study with two arms (placebo, biotin 300 mg/day). The study will be followed by a 6-month extension phase during which all patients will receive biotin 300 mg/day.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- The primary objective of the study is to evaluate the safety of biotin at 300 mg/day over placebo in patients with amyotrophic lateral sclerosis. This is a 6-month double blind randomized 2:1 placebo-controlled study with two arms (placebo, biotin 300 mg/day). The study will be followed by a 6-month extension phase during which all patients will receive biotin 300 mg/day.
| Arm | Type | Receives |
|---|---|---|
| MD1003 | Active comparator | Drug: MD1003The investigational drug will consist in capsules of 100 mg biotin and excipients (lactose, magnesium stearate, croscarmellose sodium, Silica) tid during 12 months |
| PLACEBO | Placebo comparator | Drug: MD1003, Drug: Placebo oral capsuleThis formulation consists in lactose powder and other excipients (magnesium stearate, croscarmellose sodium, Silica) as placebo, tid during 6 months and then switch to MD1003 tid during 6 additional months. |
Who may qualify
- Ages
- 25 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age: 25 to 80 years, inclusive
- Male or female subjects with probable or confirmed ALS (revised international El Escorial criteria, Forbes et al., 2001).
- Patients presenting first motor deficits due to ALS for a maximum of three years at the first consultation in an ALS centre.
- Patients monitored for at least 6 months in an ALS centre or for whom the previous monitoring parameters are available (excepted for MIP and SNIP).
- Patients who have lost at least 5 points on the ALSFRS-R (ALS functional rating scale) during the last 12 months or at least 2 points during the preceding 6 months
Key exclusion criteria (14 listed)
- Patients on non-invasive ventilation for respiratory insufficiency due to ALS for more than 10 hours a day
- Patients with an ALSFRS-R score at inclusion of < 20 (maximum score without disability = 48)
- Patients who have lost less than 5 points on the ALSFRS-R during the last year or less than 2 points during the preceding 6 months
- Patients with a gastrostomy
- Patients who have lost more than 15% of their reference weight (defined as weight before disease onset)
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 |
|---|---|---|
| MD1003Also: BIOTINcapsules 100mg 3 times per day | Drug | MD1003, PLACEBO |
| Placebo oral capsulecapsules 100mg lactose 3 times per day | Drug | PLACEBO |
Primary outcome
Recording of adverse events
Time frame: 6 months
All adverse events in two groups will be recorded.
Laboratory testing (haematology and biochemistry panel)
Time frame: 6 months
* RBC (red blood cell), WBC (white blood cell ), platelets * Ferritin, CPK (creatine phosphokinase ) * Electrolytes, creatinine, glycaemia * AST (aspartate aminotransferase ), ALT (alanine aminotransferase) , bilirubin, GGT (gamma-glutamyltransferase), alkaline phosphatase * Triglyceride, cholesterol * Haemostasis: APPT (activated partial thromboplastin time), PT (prothrombin time )
6 secondary outcomes
Motor disability
Time frame: 6 months
Severity
Time frame: 6 months
Slow vital capacity (SVC)
Time frame: 6 months
Maximal inspiratory pressure (MIP)
Time frame: 6 months
Sniff nasal inspiratory pressure (SNIP)
Time frame: 6 months
Weight
Time frame: 6 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 4 Jun 2019.
- Derived from this study: PubMed 32140672Juntas-Morales R, Pageot N, Bendarraz A, Alphandery S, Sedel F, Seigle S, Camu W. High-dose pharmaceutical grade biotin (MD1003) in amyotrophic lateral sclerosis: A pilot study. EClinicalMedicine. 2020 Jan 27;19:100254. doi: 10.1016/j.eclinm.2019.100254. eCollection 2020 Feb.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MD1003 in people with ALS and Amyotrophic Lateral Sclerosis, run by MedDay Pharmaceuticals SA at 1 site in France.
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 10 inclusion and 14 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 4 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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 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.
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?
Recording of adverse events (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03114215, which also lists 1 linked publication.
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 4 Jun 2019.