Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03579966 |
Recruitment Status :
Active, not recruiting
First Posted : July 9, 2018
Last Update Posted : February 16, 2021
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Condition or disease | Intervention/treatment | Phase |
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Myasthenia Gravis, MuSK | Drug: Amifampridine Phosphate | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 70 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia Gravis |
Actual Study Start Date : | July 1, 2018 |
Estimated Primary Completion Date : | December 31, 2021 |
Estimated Study Completion Date : | June 30, 2022 |

Arm | Intervention/treatment |
---|---|
Experimental: amifampridine phosphate
tablets equivalent to 10mg amifampridine, 3 to 4 times per day
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Drug: Amifampridine Phosphate
tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day |
- Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) [ Time Frame: over 21 months ]Evaluate the long-term safety and tolerability amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. [ Time Frame: over 21 monts ] Descriptive statistics will be used to summarize study data.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Participated in the MSK-002 study
Exclusion Criteria:
- Epilepsy and currently on medication
- Clinically significant abnormalities in ECG, in the opinion of the Investigator

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03579966
United States, Ohio | |
Cleveland Clinic | |
Cleveland, Ohio, United States, 44195 | |
United States, Pennsylvania | |
Univerity of Pennsylvania | |
Philadelphia, Pennsylvania, United States, 19104 |
Principal Investigator: | Renato Mantegazza, MD | Carlo Besta Neurological Institute |
Responsible Party: | Catalyst Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT03579966 |
Other Study ID Numbers: |
MSK-003 |
First Posted: | July 9, 2018 Key Record Dates |
Last Update Posted: | February 16, 2021 |
Last Verified: | February 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Muscle Weakness Myasthenia Gravis Muscular Diseases Musculoskeletal Diseases Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Pathologic Processes Autoimmune Diseases of the Nervous System Neuromuscular Junction Diseases |
Neuromuscular Diseases Autoimmune Diseases Immune System Diseases Amifampridine Neuromuscular Agents Peripheral Nervous System Agents Physiological Effects of Drugs Potassium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |