Status:
COMPLETED
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients
Lead Sponsor:
Catalyst Pharmaceuticals, Inc.
Conditions:
Muscular Atrophy, Spinal
Eligibility:
All Genders
6-50 years
Phase:
PHASE2
Brief Summary
A two-period, two-treatment, crossover study to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ambulatory patients diagnosed with spinal muscular atrophy (SMA) Type 3.
Detailed Description
This randomized (1:1), double-blind, placebo-controlled, 2-period, 2-treatment, crossover, outpatient study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in ...
Eligibility Criteria
Inclusion
- 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.
- Male or female between the ages of 6 and 50 years.
- Genetically confirmed diagnosis of SMA Type 3.
- Able to walk independently for at least 30 meters.
- Not taking Nusinersen for the treatment of SMA (Nusinersen should be stopped at least 6 months before screening). Salbutamol is permitted only if the dose has been stable during the 6 months before screening.
- Able to swallow oral medication.
- Female patients of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin \[HCG\] at Screening); 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.
Exclusion
- Epilepsy and currently on medication for epilepsy.
- Concomitant use of medicinal products with a known potential to cause QTc prolongation.
- Patients with long QT syndromes.
- An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator.
- Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
- Treatment with an investigational drug (other than amifampridine), device, or biological agent within 6 months prior to Screening or while participating in this study.
- Surgery for scoliosis or joint contractures within the previous 6 months.
- 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).
- Less than a 3-point improvement in HFSME from start of the Open label Run -in period to end of Run-in (Day 0).
Key Trial Info
Start Date :
January 21 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 23 2020
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT03781479
Start Date
January 21 2019
End Date
July 23 2020
Last Update
June 1 2021
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Neurological Institute Carlo Besta
Milan, Lombardy, Italy, 20133