Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

NCT03304054 | Completed Phase 3 Results FDA Drug

• 1 other identifier: MSK-002
• Study Type: interventional
• Target: 93
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Myasthenia Gravis, Generalized

Keywords

MuSK antibody positive

Outcome Measures

Primary Outcomes (1)

• Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results

  Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos

  Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early).

Secondary Outcomes (1)

• Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results

  Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early).

Study Arms (2)

amifamapridine phosphate tablets

EXPERIMENTAL

Drug: Amifampridine Phosphate

placebo tablets

PLACEBO COMPARATOR

Drug: Placebo Oral Tablet

Interventions

Amifampridine Phosphate DRUG

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

amifamapridine phosphate tablets

Placebo Oral Tablet DRUG

tablets matching amifampridine phosphate, 3 to 4 times a day

placebo tablets

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 ≥18 years of age.
• Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed at Screening or by previous antibody test, with report available.
• Confirmatory EMG or EMG report.
• Myasthenia Gravis Foundation of America (MGFA) Class II to IV at Screening.
• MG-ADL score of ≥6 at Screening, with more than 50% of this score attributed to non-ocular items.
• Patients receiving steroids or pyridostigmine should not have any modification of drug regimen during the month before Screening.
• 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.

You may not qualify if:

• Epilepsy and currently on medication.
• Concomitant use of medicinal products with a known potential to cause QTc prolongation.
• Patients with long QT syndromes.
• History of thymectomy within 12 months before Screening.
• 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.
• 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.
• Use of rituximab or other similar biologic medications for immunomodulation within 6 months prior to Screening.
• Treatment with an investigational drug (other than amifampridine) or device within 30 days before Screening or 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).

Sponsors & Collaborators

• Catalyst Pharmaceuticals, Inc.: lead

Study Sites (2)

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Univerity of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Gary Ingenito
• Organization: Catalyst Pharmaceuticals, Inc.

gingenito@catalystpharma.com

305-420-3200

Study Officials

• Renato Mantegazza, MD

  Carlo Besta Neurologic Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 28, 2017
• First Posted: October 6, 2017
• Study Start: April 18, 2018
• Primary Completion: January 31, 2020
• Study Completion: March 15, 2020
• Last Updated: March 12, 2024
• Results First Posted: August 13, 2021

Record last verified: 2021-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)