Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
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ClinicalTrials.gov Identifier: NCT01645787 |
Recruitment Status :
Completed
First Posted : July 20, 2012
Last Update Posted : May 18, 2016
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Condition or disease | Intervention/treatment | Phase |
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Spinal Muscular Atrophy | Drug: 4-aminopyridine Drug: Placebo | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 11 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action |
Study Start Date : | June 2012 |
Actual Primary Completion Date : | September 2015 |
Actual Study Completion Date : | September 2015 |

Arm | Intervention/treatment |
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Active Comparator: 4-aminopyridine (Ampyra)
10 mg tab/ 1 tab twice daily
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Drug: 4-aminopyridine
10 mg/twice daily
Other Names:
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Placebo Comparator: Sugar pill
Placebo 1 tab /twice daily
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Drug: Placebo
Crossover study involving one trial with sugar pill (placebo)
Other Name: Sugar pill |
- Six Minute Walk Test (6MWT) with Kinematic Evaluation of Gait [ Time Frame: Up to 21 Weeks ]The primary outcome measure will be distance walked in the 6MWT. This measure is an objective evaluation of functional capacity which measures the distance a person can walk quickly in six minutes and is most representative of a person's ability because the test intensity is self-selected. The 6MWT can be safely performed in ambulatory SMA patients and correlates with standard SMA outcome measures including timed walking tests. In SMA, the 6MWT may be more sensitive to clinically meaningful changes in patients with type 3 SMA as it is a direct measure of their functional mobility.
- Hammersmith Functional Motor Scale, Expanded (HFMSE) [ Time Frame: Up to 21 Weeks ]Assessments of motor function are clinically relevant and are a good adjunct to tests of walking ability. The HFMSE, a 33-item scale designed for SMA type 2 and 3 patients, and is associated with minimal patient burden requiring only standard equipment and is completed on average in less than 15 minutes. The HFMSE showed good test-retest reliability and is correlated with other clinical and physiological measures in SMA.
- Manual Muscle Testing (MMT)/Hand Held Dynamometer (HHD) [ Time Frame: Up to 21 Weeks ]MMT will involve pushing and pulling against the evaluators hand (MMT) and HHD will involve pushing or pulling as against a handheld measuring device. The purpose of these tests is to measure the strength in different muscles. The MMT involves testing fourteen muscle groups of the arm and leg on both sides of the body. The evaluator will alternate sides between tests. The measuring device will be used on 10 muscle groups on both sides of the body.
- Change in Motor Unit Number Estimation (MUNE)/Nerve Conduction Study (NCS) [ Time Frame: Baseline, Week 2 and Week 5 ]Motor Unit Number Estimation (MUNE) is a noninvasive test that identifies the number of surviving motor units (motor nerve cells and the territory of muscle fibers they control) using electrical muscle stimulation and recording the response. The nerve conduction study involves the administration of modest electrical stimulations (pulsations or throbbing sensations from low level electricity) to a total of 4 nerves in your right arm and leg while recording the response over a muscle innervated by each nerve.

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Ages Eligible for Study: | 18 Years to 50 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Aged 18 to 50 years at the time of enrollment
- Have genetically confirmed SMA 3 (homozygous absence of SMN1 exon 7)
- Ability to walk at least 25 meters without assistance
- Be free of major orthopedic deformities (i.e. scoliosis, contractures)
- Normal Cystatin C clearance (> 80 ml/min)
Exclusion Criteria:
- Patients with a history of seizures
- Patients with any renal impairment
- Inability to comply with the study procedures
- Unstable medical illness
- Any ventilatory assistance
- Taking experimental medication for SMA other than under this protocol
- Pregnancy or lactation
- Menstruating women, not sterilized or not using effective birth control
- Planning to undergo scoliosis surgery within the next 10 months
- Inability to give informed consent

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): NCT01645787
United States, New York | |
Columbia University Medical Center | |
New York, New York, United States, 10032 |
Principal Investigator: | Claudia A. Chiriboga, MD, MPH | Columbia University |
Responsible Party: | Claudia Chiriboga, Professor of Neurology and Pediatrics at CUMC, Columbia University |
ClinicalTrials.gov Identifier: | NCT01645787 |
Other Study ID Numbers: |
AAAI7400 |
First Posted: | July 20, 2012 Key Record Dates |
Last Update Posted: | May 18, 2016 |
Last Verified: | May 2016 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Spinal Muscular Atrophy 4-aminopyridine dalfampridine Ampyra |
Muscular Atrophy Muscular Atrophy, Spinal Atrophy Pathological Conditions, Anatomical Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Spinal Cord Diseases |
Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases Neuromuscular Diseases 4-Aminopyridine Potassium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |