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Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

NCT ID: NCT03819660

Last Updated: 2023-11-30

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-07

Study Completion Date

2021-09-13

Brief Summary

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

Detailed Description

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This open-label outpatient extension study was designed to evaluate the long-term safety and tolerability of amifampridine in patients diagnosed with SMA Type 3. In addition, the evaluation of the effects of amifampridine on the QoL was performed. The study was planned to enroll those patients who had completed the SMA-001 study and after all final evaluations for that study had been completed, or those who demonstrated benefit after completing the dose titration period but failed to meet the randomization criteria on Day 0 of SMA-001.

The duration of participation for each patient was expected to be at least 12 months as patients could continue in the study until amifampridine was approved by Regulatory Agencies or the clinical development of amifampridine was terminated for this indication. In addition to amifampridine, patients continued to receive previous concomitant medications, as needed.

Conditions

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Spinal Muscular Atrophy Type 3

Keywords

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Type 3

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open-label, extension study , long term safety and tolerability
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Amifampridine Phosphate 10 MG Oral Tablet

Intervention Type DRUG

Oral tablets

Interventions

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Amifampridine Phosphate 10 MG Oral Tablet

Oral tablets

Intervention Type DRUG

Eligibility Criteria

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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

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).
Minimum Eligible Age

6 Years

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Catalyst Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Lorenzo Maggi, MD

Role: PRINCIPAL_INVESTIGATOR

Carlo Besta Institute, Milan, Italy

Locations

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Neurological Institute Carlo Besta

Milan, Lombardy, Italy

Site Status

Countries

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Italy

Provided Documents

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Document Type: Study Protocol: original protocol

View Document

Document Type: Study Protocol: Amendment 1

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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SMA-002

Identifier Type: -

Identifier Source: org_study_id