Trial · NCT01645787

Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients

NCT01645787, Phase 2, Phase 3, Completed, Columbia University; results first posted 2024-09-03

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients. Trials, record NCT01645787. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01645787

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
NCT number: NCT01645787
Status: Completed
Phases: Phase 2, Phase 3
Enrollment: 11 participants, actual
Lead sponsor: Columbia University
Conditions: Spinal Muscular Atrophy
Interventions: 4-aminopyridine, Placebo
First posted: 2012-07-20
Last updated: 2024-09-03
Results first posted: 2024-09-03

Source ClinicalTrials.gov

Registered outcomes7

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Short Term)	Day 14 of each short-term intervention period	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.
Primary	Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Long Term)	Day 42 of each long-term intervention period	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.
Secondary	Hammersmith Functional Motor Scale, Expanded (HFMSE) (Short Term)	Day 14 of each short-term intervention period	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. The score range is 0 (all items failed) to 66 (all items achieved unaided), with higher score indicating higher level of motor function.
Secondary	Hammersmith Functional Motor Scale, Expanded (HFMSE) (Long Term)	Day 42 of each long-term intervention period	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. The score range is 0 (all items failed) to 66 (all items achieved unaided), with higher score indicating higher level of motor function.
Secondary	Manual Muscle Testing (MMT) Total Score (Short Term)	Day 14 of each short-term intervention period	MMT will involve pushing and pulling against the evaluators hand (MMT). The purpose of this test is to measure the strength in different muscles. MMT was performed on 28 muscle groups (8 muscle groups on each leg and 6 muscle groups on each arm), including proximal and distal musculature. The score range for each muscle group is 0 to 10, with a higher score indicating better muscle strength. The total score range is 0 to 280.
Secondary	Manual Muscle Testing (MMT) Total Score (Long Term)	Day 42 of each long-term intervention period	MMT will involve pushing and pulling against the evaluators hand (MMT). The purpose of this test is to measure the strength in different muscles. MMT was performed on 28 muscle groups (8 muscle groups on each leg and 6 muscle groups on each arm), including proximal and distal musculature. The score range for each muscle group is 0 to 10, with a higher score indicating better muscle strength. The total score range is 0 to 280.
Secondary	Motor Unit Number Estimation (MUNE)	Day 14 of each short-term intervention period	Motor Unit Number Estimation (MUNE) is a noninvasive test that identifies the number of 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 the right arm and leg while recording the response over a muscle innervated by each nerve. MUNE is calculated by determining the compound motor action potential (CMAP) amplitude or area under the curve of a distal muscle in response to supramaximal stimulation, and then dividing the result by the amplitude or area under the curve of a single motor unit action potential.

Source ClinicalTrials.gov

Outcomes7

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Short Term)	Day 14 of each short-term intervention period		meters	MEAN
PRIMARY	Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Long Term)	Day 42 of each long-term intervention period	Ten of 11 participants completed the long term study. One participant withdrew prior to the long term study due to worsening migraine.	meters	MEAN
SECONDARY	Hammersmith Functional Motor Scale, Expanded (HFMSE) (Short Term)	Day 14 of each short-term intervention period		score	MEAN
SECONDARY	Hammersmith Functional Motor Scale, Expanded (HFMSE) (Long Term)	Day 42 of each long-term intervention period	Ten of 11 participants completed the long term study. One participant withdrew prior to the long term study due to worsening migraine.	score	MEAN
SECONDARY	Manual Muscle Testing (MMT) Total Score (Short Term)	Day 14 of each short-term intervention period		score	MEAN
SECONDARY	Manual Muscle Testing (MMT) Total Score (Long Term)	Day 42 of each long-term intervention period	Ten of 11 participants completed the long term study. One participant withdrew prior to the long term study due to worsening migraine.	score	MEAN
SECONDARY	Motor Unit Number Estimation (MUNE)	Day 14 of each short-term intervention period	MUNE testing was conducted only during the short term study.	motor units	MEAN

Source ClinicalTrials.gov

Results by group14

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Short Term)	Short Term 4-aminopyridine (Ampyra)	293.05	90% Confidence Interval 233.60 to 352.50	meters
Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Short Term)	Short Term Placebo	299.50	90% Confidence Interval 240.05 to 358.95	meters
Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Long Term)	Long Term 4-aminopyridine (Ampyra)	299.10	90% Confidence Interval 235.93 to 362.27	meters
Six Minute Walk Test (6MWT) With Kinematic Evaluation of Gait (Long Term)	Long Term Placebo	295.20	90% Confidence Interval 232.03 to 358.37	meters
Hammersmith Functional Motor Scale, Expanded (HFMSE) (Short Term)	Short Term 4-aminopyridine (Ampyra)	50.84	90% Confidence Interval 48.53 to 53.16	score
Hammersmith Functional Motor Scale, Expanded (HFMSE) (Short Term)	Short Term Placebo	50.43	90% Confidence Interval 48.11 to 52.74	score
Hammersmith Functional Motor Scale, Expanded (HFMSE) (Long Term)	Long Term 4-aminopyridine (Ampyra)	50.90	90% Confidence Interval 48.49 to 53.31	score
Hammersmith Functional Motor Scale, Expanded (HFMSE) (Long Term)	Long Term Placebo	50.30	90% Confidence Interval 47.89 to 52.71	score
Manual Muscle Testing (MMT) Total Score (Short Term)	Short Term 4-aminopyridine (Ampyra)	181.86	90% Confidence Interval 170.50 to 193.23	score
Manual Muscle Testing (MMT) Total Score (Short Term)	Short Term Placebo	182.23	90% Confidence Interval 170.86 to 193.59	score

1 to 10 of 14Source ClinicalTrials.gov

See all 14 results by group

Participant flow42

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
First Intervention Short Term (2 Weeks)	STARTED	4-aminopyridine, Then Placebo	6
First Intervention Short Term (2 Weeks)	STARTED	Placebo, Then 4-aminopyridine	5
First Intervention Short Term (2 Weeks)	COMPLETED	4-aminopyridine, Then Placebo	6
First Intervention Short Term (2 Weeks)	COMPLETED	Placebo, Then 4-aminopyridine	5
First Intervention Short Term (2 Weeks)	NOT COMPLETED	4-aminopyridine, Then Placebo	0
First Intervention Short Term (2 Weeks)	NOT COMPLETED	Placebo, Then 4-aminopyridine	0
Washout (1 Week)	STARTED	4-aminopyridine, Then Placebo	6
Washout (1 Week)	STARTED	Placebo, Then 4-aminopyridine	5
Washout (1 Week)	COMPLETED	4-aminopyridine, Then Placebo	6
Washout (1 Week)	COMPLETED	Placebo, Then 4-aminopyridine	5

1 to 10 of 42Source ClinicalTrials.gov

See all 42 participant flow

Adverse events32

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Upset Stomach	Gastrointestinal disorders	4-aminopyridine (Short Term)	1	11
other	Upset Stomach	Gastrointestinal disorders	Placebo (Short Term)	0	11
other	Upset Stomach	Gastrointestinal disorders	4-aminopyridine (Long Term)	0	11
other	Upset Stomach	Gastrointestinal disorders	Placebo (Long Term)	0	10
other	Nausea	General disorders	4-aminopyridine (Short Term)	0	11
other	Nausea	General disorders	Placebo (Short Term)	1	11
other	Nausea	General disorders	4-aminopyridine (Long Term)	0	11
other	Nausea	General disorders	Placebo (Long Term)	1	10
other	Fall	General disorders	4-aminopyridine (Short Term)	2	11
other	Fall	General disorders	Placebo (Short Term)	2	11

1 to 10 of 32Source ClinicalTrials.gov

See all 32 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	4-aminopyridine (Short Term)	0	11
Total, all-cause mortality	Placebo (Short Term)	0	11
Total, all-cause mortality	4-aminopyridine (Long Term)	0	11
Total, all-cause mortality	Placebo (Long Term)	0	10
Total, other adverse events	4-aminopyridine (Short Term)	9	11
Total, other adverse events	Placebo (Short Term)	4	11
Total, other adverse events	4-aminopyridine (Long Term)	11	11
Total, other adverse events	Placebo (Long Term)	3	10
Total, serious adverse events	4-aminopyridine (Short Term)	0	11
Total, serious adverse events	Placebo (Short Term)	0	11

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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