Effect of MD1003 in Amyotrophic Lateral Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03114215
• Date of registration: 28/03/2017
• Prospective Registration: No
• Primary sponsor: MedDay Pharmaceuticals SA
• Public title: Effect of MD1003 in Amyotrophic Lateral Sclerosis MD1003-ALS
• Scientific title: Effect of MD1003 in Amyotrophic Lateral Sclerosis: a Randomized Double Blind Placebo Controlled Pilot Study
• Date of first enrolment: June 29, 2016
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03114215
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Other. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

France

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 (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

- 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).

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 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)

- 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: 25 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Motor Neuron Disease

ALS

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: MD1003

Drug: Placebo oral capsule

Primary Outcome(s)

Laboratory testing (haematology and biochemistry panel) [Time Frame: 6 months]

Recording of adverse events [Time Frame: 6 months]

Secondary Outcome(s)

Maximal inspiratory pressure (MIP) [Time Frame: 6 months]

Motor disability [Time Frame: 6 months]

Weight [Time Frame: 6 months]

Severity [Time Frame: 6 months]

Sniff nasal inspiratory pressure (SNIP) [Time Frame: 6 months]

Slow vital capacity (SVC) [Time Frame: 6 months]

Secondary ID(s)

MD1003CT2015-02-ALS

Source(s) of Monetary Support

Please refer to primary and secondary sponsors