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Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)

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ClinicalTrials.gov Identifier: NCT03579966
Recruitment Status : Recruiting
First Posted : July 9, 2018
Last Update Posted : August 8, 2019
Sponsor:
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

Study Description
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Brief Summary:
Evaluate the long-term safety of amifampridine phosphate in the symptomatic relief of antibody positive MuSK-MG.

Condition or disease Intervention/treatment Phase
Myasthenia Gravis, MuSK Drug: Amifampridine Phosphate Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
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 : June 2020
Estimated Study Completion Date : September 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: amifampridine phosphate
tablets equivalent to 10mg amifampridine, 3 to 4 times per day
 Drug: Amifampridine Phosphate
tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Stanley Iyadurai, MD, PhD 305-420-3200 siyadurai@catalystpharma.com

Locations
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United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Contact: Irys Caristo       caristi@ccf.org   
United States, Pennsylvania
Univerity of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Thomas Rose    215-294-9132    thomas.rose@uphs.upenn.edu   
Sponsors and Collaborators
Catalyst Pharmaceuticals, Inc.
Investigators
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Principal Investigator: Renato Mantegazza, MD Carlo Besta Neurological Institute
More Information
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Responsible Party: Catalyst Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03579966     History of Changes
Other Study ID Numbers: MSK-003
First Posted: July 9, 2018    Key Record Dates
Last Update Posted: August 8, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
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