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Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients

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ClinicalTrials.gov Identifier: NCT03781479
Recruitment Status : Completed
First Posted : December 20, 2018
Last Update Posted : July 28, 2020
Sponsor:
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE December 18, 2018
First Posted Date  ICMJE December 20, 2018
Last Update Posted Date July 28, 2020
Actual Study Start Date  ICMJE January 21, 2019
Actual Primary Completion Date July 23, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 18, 2018)
Change from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) [ Time Frame: Change from baseline in HFMSE score at the end of week 6 (end of Period 1) and change from baseline in HFMSE scare at the end of week 8 (end of Period 2) ]
standardized measurement of muscle function
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients
Official Title  ICMJE A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
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 ambulatory patients diagnosed with SMA Type 3. The study is planned to include approximately 12 male and female SMA Type 3 patients. The planned duration of participation for each patient is approximately 2 months, based upon length of dose titration and excluding the screening period, which can last up to 14 days. Patients should only be taking the assigned investigational product (amifampridine phosphate 10 mg tablets or matching placebo tablets), no new therapies are permitted during the study.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Muscular Atrophy, Spinal
Intervention  ICMJE
  • Drug: Amifampridine Phosphate
    Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.
    Other Name: 3,4 diaminopyridine phosphate
  • Drug: Placebo Oral Tablet
    Placebo Oral Tablet
Study Arms  ICMJE
  • Experimental: amifampridine phophate - placebo
    Oral tablets, 30 to 80 mg per day in divided doses 3 to 4 times a day for 4 weeks
    Interventions:
    • Drug: Amifampridine Phosphate
    • Drug: Placebo Oral Tablet
  • Experimental: placebo - amifampridine phophate
    Oral tablets, 30 to 80 mg per day in divided doses 3 to 4 times a day for 4 weeks
    Interventions:
    • Drug: Amifampridine Phosphate
    • Drug: Placebo Oral Tablet
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 18, 2018)
12
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 23, 2020
Actual Primary Completion Date July 23, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. 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.
  2. Male or female between the ages of 6 and 50 years.
  3. Genetically confirmed diagnosis of SMA Type 3.
  4. Able to walk independently for at least 30 meters.
  5. 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.
  6. Able to swallow oral medication.
  7. 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.
  8. 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 Criteria:

  1. Epilepsy and currently on medication for epilepsy.
  2. Concomitant use of medicinal products with a known potential to cause QTc prolongation.
  3. Patients with long QT syndromes.
  4. An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator.
  5. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
  6. Treatment with an investigational drug (other than amifampridine), device, or biological agent within 6 months prior to Screening or while participating in this study.
  7. Surgery for scoliosis or joint contractures within the previous 6 months.
  8. 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.
  9. History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).
  10. Less than a 3-point improvement in HFSME from start of the Open label Run -in period to end of Run-in (Day 0).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 50 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03781479
Other Study ID Numbers  ICMJE SMA-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Catalyst Pharmaceuticals, Inc.
Study Sponsor  ICMJE Catalyst Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Lorenzo Maggi, MD Carlo Besta Institute, Milan, Italy
PRS Account Catalyst Pharmaceuticals, Inc.
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP