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Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (SMA3)

Primary Purpose

Muscle Atrophy

Status
Terminated
Phase
Phase 2
Locations
Italy
Study Type
Interventional
Intervention
Amifampridine Phosphate 10 MG Oral Tablet
Sponsored by
Catalyst Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscle Atrophy focused on measuring Type 3

Eligibility Criteria

6 Years - 50 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Individuals eligible to participate in this study must meet all the following inclusion criteria:

  1. Participated in the SMA-001 study
  2. 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.
  3. 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.
  4. 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:

CRITERIA FOR INCLUSION AND EXCLUSION:

Individuals eligible to participate in this study must meet all the following inclusion criteria:

  1. Participated in the SMA-001 study
  2. 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.
  3. 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.
  4. 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:

Individuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study:

  1. Epilepsy and currently on medication.
  2. Uncontrolled asthma.
  3. Concomitant use with sultopride.
  4. Concomitant use with medicinal products with a narrow therapeutic window.
  5. Concomitant use with medicinal products with a known to cause QTc prolongation.
  6. Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
  7. Subjects with congenital QT syndromes.
  8. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
  9. Intolerable amifampridine-related side effects
  10. Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
  11. 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.
  12. History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).

Sites / Locations

  • Neurological Institute Carlo Besta

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

amifampridine phosphate

Arm Description

Oral tablets, 15 to 80 mg per day in divided doses 3 to 4 times a day for up to 18 months.

Outcomes

Primary Outcome Measures

The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0
Safety analyses will be conducted on the safety population (i.e. all patients who receive at least 1 dose of amifampridine. The safety analysis will be descriptive and will be presented on observed data only. All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of TEAEs will be summarized by system organ class, preferred term, relationship to treatment, and severity.

Secondary Outcome Measures

Full Information

First Posted
January 23, 2019
Last Updated
March 14, 2022
Sponsor
Catalyst Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03819660
Brief Title
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
Acronym
SMA3
Official Title
Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
Study Type
Interventional

2. Study Status

Record Verification Date
March 2022
Overall Recruitment Status
Terminated
Why Stopped
Development of indication not being pursued
Study Start Date
March 7, 2019 (Actual)
Primary Completion Date
September 13, 2021 (Actual)
Study Completion Date
September 13, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Catalyst Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
Detailed Description
This open-label, long term safety study is designed to evaluate the safety of amifampridine phosphate in ambulatory patients diagnosed SMA Type 3 over an extended period. The study is planned to include approximately 12 male and female SMA Type 3 patients. The planned duration of participation for each patient may be up to 1 year. Patients must have demonstrated benefit during the initial SMA-001 trial.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscle Atrophy
Keywords
Type 3

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Open-label, long term safety treatment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
amifampridine phosphate
Arm Type
Experimental
Arm Description
Oral tablets, 15 to 80 mg per day in divided doses 3 to 4 times a day for up to 18 months.
Intervention Type
Drug
Intervention Name(s)
Amifampridine Phosphate 10 MG Oral Tablet
Intervention Description
Oral tablets
Primary Outcome Measure Information:
Title
The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0
Description
Safety analyses will be conducted on the safety population (i.e. all patients who receive at least 1 dose of amifampridine. The safety analysis will be descriptive and will be presented on observed data only. All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of TEAEs will be summarized by system organ class, preferred term, relationship to treatment, and severity.
Time Frame
18 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Individuals eligible to participate in this study must meet all the following inclusion criteria: 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. Exclusion Criteria: CRITERIA FOR INCLUSION AND EXCLUSION: Individuals eligible to participate in this study must meet all the following inclusion criteria: 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. Exclusion Criteria: Individuals who met any of the exclusion criteria in the original protocol or those listed below are not eligible to participate in the study: 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).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Lorenzo Maggi, MD
Organizational Affiliation
Carlo Besta Institute, Milan, Italy
Official's Role
Principal Investigator
Facility Information:
Facility Name
Neurological Institute Carlo Besta
City
Milano
State/Province
Lombardy
ZIP/Postal Code
20133
Country
Italy

12. IPD Sharing Statement

Learn more about this trial

Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

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