Effect of MD1003 in Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis Clinical Trial

— MD1003-ALS  

Official title:

Effect of MD1003 in Amyotrophic Lateral Sclerosis: a Randomized Double Blind Placebo Controlled Pilot Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03114215
• Other study ID #: MD1003CT2015-02-ALS
• Status: Completed
• Phase: Phase 2
• Start date: June 29, 2016
• Est. completion date: December 2017

Study information

• Verified date: June 2019
• Source: MedDay Pharmaceuticals SA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: December 2017
• Est. primary completion date: June 12, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 25 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• ALS
• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• MD1003
capsules 100mg 3 times per day
• Placebo oral capsule
capsules 100mg lactose 3 times per day

Locations

Country	Name	City	State
France	Hopital Gui De Chauliac	Montpellier

Sponsors (1)

Lead Sponsor	Collaborator
MedDay Pharmaceuticals SA

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Recording of adverse events	All adverse events in two groups will be recorded.	6 months
Primary	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 )	6 months
Secondary	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).	6 months
Secondary	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).	6 months
Secondary	Slow vital capacity (SVC)	in liters	6 months
Secondary	Maximal inspiratory pressure (MIP)	in cm H2O	6 months
Secondary	Sniff nasal inspiratory pressure (SNIP)	in cm H20	6 months
Secondary	Weight	weight in kg	6 months