Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

NCT03819660 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: SMA-002
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.

Conditions

Spinal Muscular Atrophy Type 3

Keywords

Type 3

Outcome Measures

Primary Outcomes (1)

• Long-term Safety and Tolerability of Amifampridine

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

  18 months

Secondary Outcomes (1)

• 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 to end of study.

Study Arms (1)

amifampridine phosphate

EXPERIMENTAL

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.

Drug: Amifampridine Phosphate 10 MG Oral Tablet

Interventions

Amifampridine Phosphate 10 MG Oral Tablet DRUG

Oral tablets

amifampridine phosphate

Eligibility Criteria

• Age: 6 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participated in the SMA-001 study
• Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.
• Female patients of childbearing potential must have a negative pregnancy test (urine human chorionic gonadotropin \[HCG\] at the end of SMA-001 study); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment.
• Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires.

You may not qualify if:

• Epilepsy and currently on medication.
• Uncontrolled asthma.
• Concomitant use with sultopride.
• Concomitant use with medicinal products with a narrow therapeutic window.
• Concomitant use with medicinal products with a known to cause QTc prolongation.
• Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
• Subjects with congenital QT syndromes.
• Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
• Intolerable amifampridine-related side effects
• Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
• Any medical condition that, in the opinion of the Investigator, might interfere with the patient's participation in the study, poses an added risk for the patient, or confound the assessment of the patient.
• History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).

Sponsors & Collaborators

• Catalyst Pharmaceuticals, Inc.: lead

Study Sites (1)

Neurological Institute Carlo Besta

Milan, Lombardy, 20133, Italy

Location

MeSH Terms

Conditions

Spinal Muscular Atrophies of Childhood

Interventions

Amifampridine; Tablets

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Dosage Forms; Pharmaceutical Preparations

Limitations and Caveats

The results of efficacy are limited to the assessment of quality of life using the INQoL. The premature interruption of the study determined that few patients had available data of INQoL from Month 6 onwards.

Results Point of Contact

• Title: Gary Ingenito
• Organization: Catalyst Pharmaceuticals, Inc.

gingenito@catalystpharma.com

3054203200

Study Officials

• Lorenzo Maggi, MD

  Carlo Besta Institute, Milan, Italy

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label, extension study , long term safety and tolerability

Study Record Dates

• First Submitted: January 23, 2019
• First Posted: January 28, 2019
• Study Start: March 7, 2019
• Primary Completion: September 13, 2021
• Study Completion: September 13, 2021
• Last Updated: November 30, 2023
• Results First Posted: November 30, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)