Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

NCT ID: NCT03304054

Last Updated: 2024-03-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 93 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-04-18
• Study Completion Date: 2020-03-15

Brief Summary

Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.

Detailed Description

Randomized, double-blind, placebo-controlled, parallel group study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in patients with MuSK-MG. In addition, a sample of AChR-MG patients will be assess for efficacy and safety of amifampridine phosphate. Planned duration of participation for each patient is at least 38 days, excluding the screening period. Eligible patients will be titrated to an efficacious dose of amifampridine phosphate and those who demonstrate improvement will be randomized to either placebo or amifampridine, in a double-blind fashion, for 10 days.

Conditions

Myasthenia Gravis, Generalized

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

amifamapridine phosphate tablets

Group Type: EXPERIMENTAL

Amifampridine Phosphate

Intervention Type: DRUG

tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

placebo tablets

Group Type: PLACEBO_COMPARATOR

Placebo Oral Tablet

Intervention Type: DRUG

tablets matching amifampridine phosphate, 3 to 4 times a day

Interventions

Amifampridine Phosphate

tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

Intervention Type: DRUG

Placebo Oral Tablet

tablets matching amifampridine phosphate, 3 to 4 times a day

Intervention Type: DRUG

Eligibility Criteria

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 ≥18 years of age.

3. Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed at Screening or by previous antibody test, with report available.

4. Confirmatory EMG or EMG report.

5. Myasthenia Gravis Foundation of America (MGFA) Class II to IV at Screening.

6. MG-ADL score of ≥6 at Screening, with more than 50% of this score attributed to non-ocular items.

7. Patients receiving steroids or pyridostigmine should not have any modification of drug regimen during the month before Screening.

8. 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.

9. 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. Concomitant use of medicinal products with a known potential to cause QTc prolongation.

3. Patients with long QT syndromes.

4. History of thymectomy within 12 months before Screening.

5. An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator.

6. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.

7. Patients receiving immunomodulatory treatment (e.g. plasma exchange [PE], therapeutic plasma exchange [TPE], intravenous immunoglobulin G [IVIG]) should not have any treatment in the previous 4 weeks prior to Randomization or at any time during the study.

8. Use of rituximab or other similar biologic medications for immunomodulation within 6 months prior to Screening.

9. Treatment with an investigational drug (other than amifampridine) or device within 30 days before Screening or while participating in this study.

10. 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.

11. History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Catalyst Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Renato Mantegazza, MD

Role: PRINCIPAL_INVESTIGATOR

Carlo Besta Neurologic Institute

Locations

Cleveland Clinic

Cleveland, Ohio, United States

Univerity of Pennsylvania

Philadelphia, Pennsylvania, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

MSK-002

Identifier Type: -

Identifier Source: org_study_id