GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)

Plain-language summary

This study examined the drug GM604 compared with placebo in patients with amyotrophic lateral sclerosis (ALS). It included patients over 18 with symptom onset within the past 24 months who met the El Escorial criteria for a definite ALS diagnosis. The study was conducted in Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

Genervon Biopharmaceuticals, LLC

Intervention

GM604, Placebo comparator

Start date

8/1/2013

Estimated completion

7/1/2014

Countries

United States

Related genes

Locations

• Boston, United States
• New York, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. Patients with ALS: Familial and Sporadic ALS, with symptom onset \< or equal to 24 months.
2. At least 18 years of age
3. Subjects meet the El Escorial criteria of definite criteria for a diagnosis of ALS.
4. Subjects can be on a stable dose of riluzole for at least a month or not taking or initiating riluzole for the duration of the trial.
5. Not on any experimental medication for the last 1 month or five times the half-life of experimental medication.
6. At screening, must have a Forced Vital Capacity (FVC) ≥ 65% of predicted capacity for age, height and gender.
7. Have fully completed informed consent form
8. Ability to comply with study procedures
9. Women of child-bearing age must be on birth control. Pregnancy test should be done in women in child bearing age.
10. Medically safe to have lumbar puncture to collect CSF

Exclusion Criteria:

1. History of liver disease, severe renal failure, diabetes, coronary heart disease, cancer
2. Clinically significant EKG abnormality at screening
3. Any comorbid condition which would make completion of the trial unlikely
4. FVC \< 65%
5. Presence of a bleeding disorder
6. Allergy to local anesthetics
7. Problem with CSF pressure
8. Topical or other skin infection at the lumbar puncture site
9. BMI \> 32 kg/m2
10. Medical or surgical conditions in which a lumbar puncture is contraindicated
11. Use of any anti-platelet or anticoagulant drugs, such as plavix, aggrenox, ticlid, warfarin or coumadin

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