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

• Register: ClinicalTrials.gov
• Last refreshed on: 3 May 2021
• Main ID: NCT01854294
• Date of registration: 08/05/2013
• Prospective Registration: Yes
• Primary sponsor: Genervon Biopharmaceuticals, LLC
• Public title: GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS) GALS
• Scientific title: GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)
• Date of first enrolment: August 2013
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01854294
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Hiroshi Mitsumoto, MD
• Affiliation: Columbia Medical Center NY

• Name: Merit Cudkowicz, MD
• Affiliation: Massachusetts General Hospital

Key inclusion & exclusion criteria

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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Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: GM604

Drug: Placebo comparator

Primary Outcome(s)

Tolerability by measuring the ability to complete the first 2 weeks of active treatment in the study [Time Frame: Baseline, week 2, week 12]

Efficacy by percent change in biomarker in th CSF at week 12 from baseline [Time Frame: baseline, week 2, week 12]

Safety by measuring 1. adverse event frequency and severity, changes in vital signs, clinical laboratory values. 2. Serious adverse event frequency [Time Frame: baseline, week 2, week 12]

Secondary Outcome(s)

Mortality rate [Time Frame: baseline, week 2, week 6, week 12]

ALSFRS-R (Amyotrophic Laeral Sclerosis Functional Rating Scale - Revised) [Time Frame: Symptom onset, screening, baseline, week 2, week 6, week 12]

Biomarker in blood [Time Frame: baseline, week 2, week 6, week 12]

Forced Vital Capacity (FVC) [Time Frame: Symptom onset, baseline, week 2, week 6, week 12]

Time Up and Go (TUG) [Time Frame: Symptom onset, baseline, week 2, week 6, week 12]

muscle strength [Time Frame: Symptom onset, baseline, week 2, week 6, week 12]

Secondary ID(s)

GALS 001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Columbia University

Massachusetts General Hospital