Effect of MD1003 in amyotrophic lateral sclerosis: a randomized, double blind placebo controlled study

• Register: EUCTR
• Last refreshed on: 10 December 2019
• Main ID: EUCTR2015-005810-31-FR
• Date of registration: 24/10/2016
• Prospective Registration: No
• Primary sponsor: MEDDAY PHARMACEUTICALS
• Public title: Effect of MD1003 in amyotrophic lateral sclerosis: a randomized, double blind placebo controlled study
• Scientific title: Effect of MD1003 in amyotrophic lateral sclerosis: a randomized, double blind placebo controlled study - MD1003-ALS
• Date of first enrolment: 03/06/2016
• Target sample size: 30
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-005810-31
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: yes; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

France

Contacts

• Name: Clinical Trial Information
• Affiliation: MEDDAY PHARMACEUTICALS

• Name: Clinical Trial Information
• Affiliation: MEDDAY PHARMACEUTICALS

Key inclusion & exclusion criteria

Inclusion criteria:
- 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 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.
- 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 < 52 mmHg, SaO2 < 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).

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion criteria:
- 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 ideal weight
- Patient with dyspnoea at rest or with the least effort (score < 3 on the dyspnoea item of the ALSFRS-R)
- 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.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Amyotrophic lateral sclerosis
MedDRA version: 19.0 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Code: MD1003
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: BIOTIN
CAS Number: 58-85-5
Current Sponsor code: MD1003
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: Investigation of the effect of biotin on the parameters of clinical progression and on the prognosis of ALS

Timepoint(s) of evaluation of this end point: every 3-month

Primary end point(s): Safety
- Incidence of adverse events
- Laboratory testing abnormalities
- RBC, WBC, platelets
- Ferritin, CPK
- Electrolytes, creatinine, glycaemia
- AST, ALT, bilirubin, GGT, alkaline phosphatase
- Triglyceride, cholesterol
- Haemostasis: APPT, PT

Main Objective: Investigation of the safety of biotin in ALS

Secondary Outcome(s)

Secondary end point(s): Efficacy
- Motor disability (ALSFRS-R score)
- Disease severity
- Respiratory parameters
- Slow vital capacity (SVC)
- Maximal inspiratory pressure (MIP)
- Sniff nasal inspiratory pressure (SNIP).
- Weight

Timepoint(s) of evaluation of this end point: every 3-month

Secondary ID(s)

MD1003CT2015-02-ALS

Source(s) of Monetary Support

MEDDAY PHARMACEUTICALS

Ethics review

Status: Approved
Approval date:
Contact: