Trial Outcomes & Findings for Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (NCT NCT03819660)

NCT ID: NCT03819660

Last Updated: 2023-11-30

Results Overview

Number of subjects with treatment emergent adverse events (TEAE).

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 13 participants
• Primary outcome timeframe: 18 months
• Results posted on: 2023-11-30

Participant Flow

Participant milestones

Measure	Amifampridine Phosphate The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily. Amifampridine Phosphate 10 MG Oral Tablet: Oral tablets
Overall Study STARTED	13
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

Baseline characteristics by cohort

Measure	Amifampridine Phosphate n=13 Participants The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily. Amifampridine Phosphate 10 MG Oral Tablet: Oral tablets
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	34.8 years STANDARD_DEVIATION 11.3 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Italy	6 Participants n=5 Participants
Region of Enrollment Serbia	7 Participants n=5 Participants
Height	171.9 CM n=5 Participants
Weight	67.1 Kg n=5 Participants
BMI	22.7 kg/m^2 n=5 Participants

PRIMARY outcome

Timeframe: 18 months

Population: TEAE = any AE started at or after the first dose of drug for the current Long-Term Study.

Number of subjects with treatment emergent adverse events (TEAE).

Outcome measures

Measure

Amifampridine Phosphate n=13 Participants The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily. Amifampridine Phosphate 10 MG Oral Tablet: Oral tablets

Long-term Safety and Tolerability of Amifampridine

5 Participants

SECONDARY outcome

Timeframe: Screening to end of study.

Population: 11 patients completed the screening Quality of Life Score and only 9 patients completed the EOS Quality of Life Score .

Quality of life (QoL): the Individualized Quality of Life for neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) was used for adult or pediatric patients, respectively. Since there were no pediatric patients, none of the patients completed the PEDSQL.

The INQol evaluated weakness, pain, fatigue, double vision, muscle locking, droopy eyelids, swallowing difficulties, activities, social relationships, emotions and body image. Each of these areas were measured in four categories as follows: 1- Incidence (0= No, 1 =Yes), 2-Severity (0= None to 7 = extreme), 3- Impact - (0= None to 6 = extreme) and 4-Importance (0= None to 6 = extreme). The numbers were summative and are input to the QOL calculation, which is a percentage of severity on a scale of 0-100. The mean value was taken across this population. The higher scores were a worse outcome.

Outcome measures

Measure	Amifampridine Phosphate n=11 Participants The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily. Amifampridine Phosphate 10 MG Oral Tablet: Oral tablets
To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL). screening	33.737 score on a scale Standard Deviation 17.677
To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL). End of Study	39.012 score on a scale Standard Deviation 14.337

Adverse Events

Amifampridine Phosphate

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Measure

Amifampridine Phosphate n=13 participants at risk The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily. Amifampridine Phosphate 10 MG Oral Tablet: Oral tablets

Nervous system disorders Intellectual Disability

7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.

Other adverse events

Measure	Amifampridine Phosphate n=13 participants at risk The dose of amifampridine was based on optimal neuromuscular benefit determined from the Run-in Period from SMA-001 or could be modified as the discretion of the Investigator. The maximum single dose was 20 mg. The dose range for patients 6 to 16 years of age was 15 to 60 mg, and for those 17 years and older the range was from 30 to 80 mg daily. Amifampridine Phosphate 10 MG Oral Tablet: Oral tablets
Gastrointestinal disorders Paraesthesia oral	23.1% 3/13 • Number of events 3 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
Gastrointestinal disorders ABDOMINAL PAIN UPPER	15.4% 2/13 • Number of events 2 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
General disorders HYPERPYREXIA	15.4% 2/13 • Number of events 2 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
General disorders FEELING HOT	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
General disorders INFLUENZA LIKE ILLNESS	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
General disorders MALAISE	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
Injury, poisoning and procedural complications FALL	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
General disorders POST LUMBAR PUNCTURE SYNDROME	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
Metabolism and nutrition disorders HYPOKALAEMIA	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
Nervous system disorders Paraesthesia	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
Nervous system disorders HEADACHE	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
Nervous system disorders TREMOR	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.
Skin and subcutaneous tissue disorders RASH	7.7% 1/13 • Number of events 1 • 27 months The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.

Additional Information

Gary Ingenito

Catalyst Pharmaceuticals, Inc.

Phone: 3054203200

Email: gingenito@catalystpharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place