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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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30 May 2016 |
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Main ID: |
NCT01645787 |
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Date of registration:
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05/07/2012 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
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Scientific title:
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Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action |
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Date of first enrolment:
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June 2012 |
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Target sample size:
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11 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01645787 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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United States
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Contacts
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Name:
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Claudia A. Chiriboga, MD, MPH |
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Address:
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Telephone:
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Email:
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Affiliation:
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Columbia University |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Aged 18 to 50 years at the time of enrollment
2. Have genetically confirmed SMA 3 (homozygous absence of SMN1 exon 7)
3. Ability to walk at least 25 meters without assistance
4. Be free of major orthopedic deformities (i.e. scoliosis, contractures)
5. Normal Cystatin C clearance (> 80 ml/min)
Exclusion Criteria:
1. Patients with a history of seizures
2. Patients with any renal impairment
3. Inability to comply with the study procedures
4. Unstable medical illness
5. Any ventilatory assistance
6. Taking experimental medication for SMA other than under this protocol
7. Pregnancy or lactation
8. Menstruating women, not sterilized or not using effective birth control
9. Planning to undergo scoliosis surgery within the next 10 months
10. Inability to give informed consent
Age minimum:
18 Years
Age maximum:
50 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Spinal Muscular Atrophy
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Intervention(s)
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Drug: 4-aminopyridine
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Drug: Placebo
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Primary Outcome(s)
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Six Minute Walk Test (6MWT) with Kinematic Evaluation of Gait
[Time Frame: Up to 21 Weeks]
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Secondary Outcome(s)
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Change in Motor Unit Number Estimation (MUNE)/Nerve Conduction Study (NCS)
[Time Frame: Baseline, Week 2 and Week 5]
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Hammersmith Functional Motor Scale, Expanded (HFMSE)
[Time Frame: Up to 21 Weeks]
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Manual Muscle Testing (MMT)/Hand Held Dynamometer (HHD)
[Time Frame: Up to 21 Weeks]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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