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Effect of MD1003 in Amyotrophic Lateral Sclerosis (MD1003-ALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03114215
Recruitment Status : Unknown
Verified April 2017 by MedDay Pharmaceuticals SA.
Recruitment status was:  Active, not recruiting
First Posted : April 14, 2017
Last Update Posted : August 2, 2017
Sponsor:
Information provided by (Responsible Party):
MedDay Pharmaceuticals SA

Brief 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.

Condition or disease Intervention/treatment Phase
ALS Amyotrophic Lateral Sclerosis Motor Neuron Disease Drug: MD1003 Drug: Placebo oral capsule Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention 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 (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Other
Official Title: Effect of MD1003 in Amyotrophic Lateral Sclerosis: a Randomized Double Blind Placebo Controlled Pilot Study
Actual Study Start Date : June 29, 2016
Actual Primary Completion Date : June 12, 2017
Estimated Study Completion Date : December 2017


Arm Intervention/treatment
Active Comparator: MD1003
The investigational drug will consist in capsules of 100 mg biotin and excipients (lactose, magnesium stearate, croscarmellose sodium, Silica) tid during 12 months
Drug: MD1003
capsules 100mg 3 times per day
Other Name: BIOTIN

Placebo Comparator: PLACEBO
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.
Drug: MD1003
capsules 100mg 3 times per day
Other Name: BIOTIN

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




Primary Outcome Measures :
  1. Recording of adverse events [ Time Frame: 6 months ]
    All adverse events in two groups will be recorded.

  2. 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 )


Secondary Outcome Measures :
  1. Motor disability [ Time Frame: 6 months ]
    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).

  2. Severity [ Time Frame: 6 months ]
    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).

  3. Slow vital capacity (SVC) [ Time Frame: 6 months ]
    in liters

  4. Maximal inspiratory pressure (MIP) [ Time Frame: 6 months ]
    in cm H2O

  5. Sniff nasal inspiratory pressure (SNIP) [ Time Frame: 6 months ]
    in cm H20

  6. Weight [ Time Frame: 6 months ]
    weight in kg



Information from the National Library of Medicine

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Ages Eligible for Study:   25 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03114215


Locations
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France
Hopital Gui De Chauliac
Montpellier, France, 34000
Sponsors and Collaborators
MedDay Pharmaceuticals SA

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Responsible Party: MedDay Pharmaceuticals SA
ClinicalTrials.gov Identifier: NCT03114215     History of Changes
Other Study ID Numbers: MD1003CT2015-02-ALS
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: August 2, 2017
Last Verified: April 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by MedDay Pharmaceuticals SA:
Biotin
MD1003
ALSFRS-R

Additional relevant MeSH terms:
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Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Biotin
Carboxymethylcellulose Sodium
Vitamin B Complex
Vitamins
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Laxatives
Gastrointestinal Agents