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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 : July 9, 2018
Sponsor:
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

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 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


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




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.



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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

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
Contact: Adriana Manari 305-420-3200 ext 117 amanari@catalystpharma.com

Locations
United States, California
UC Irvine Not yet recruiting
Irvine, California, United States, 92868
Contact: Veena Mathew       vmathew@uci.edu   
United States, Illinois
Rush University Not yet recruiting
Chicago, Illinois, United States, 60612
Contact: Gilles Hoffman    312-563-6015    gilles_hoffman@rush.edu   
OSF Healthcare St. Francis Medical Center Not yet recruiting
Peoria, Illinois, United States, 61637
Contact: Christine Brown    309-655-3448    vhristine.s.brown@osfhealthcare.org   
United States, Kansas
University of Kansas Medical Center Recruiting
Fairway, Kansas, United States, 66205
Contact: Andrew Heim       aheim2@kumc.edu   
United States, Michigan
Wayne State University Not yet recruiting
Detroit, Michigan, United States, 48201
Contact: Kelly Jia    313-966-9047    xjia@med.wayne.edu   
Henry Ford Health System Not yet recruiting
Detroit, Michigan, United States, 48202
Contact: Ashkhen Movsisyan    313-916-4121    amovsis1@hfhs.org   
Michigan State University Not yet recruiting
East Lansing, Michigan, United States, 48824
Contact: Kimberly Patterson    517-884-2274    kimberly.patterson2@ht.msu.edu   
United States, Missouri
Unviersity of Missouri Not yet recruiting
Columbia, Missouri, United States, 65212
Contact: Li-Yan Yin       yinliya@health.missouri.edu   
United States, Ohio
University of Cincinnati Not yet recruiting
Cincinnati, Ohio, United States, 45219
Contact: Ashley Fisher    513-558-4874    fisheal@ucmail.uc.edu   
Cleveland Medical Center Not yet recruiting
Cleveland, Ohio, United States, 44106
Contact: Valerie Cwiklinski    216-983-5144    valerie.cwiklinski1@uhhospitals.org   
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Contact: Irys Caristo       caristi@ccf.org   
Ohio State University Recruiting
Columbus, Ohio, United States, 43221
Contact: Paige Matisak    614-685-5815    paige.matisak@osumc.edu   
United States, Pennsylvania
Univerity of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Thomas Rose    215-294-9132    thomas.rose@uphs.upenn.edu   
United States, Tennessee
Wesley Neurology Clinic Recruiting
Memphis, Tennessee, United States, 38018
Contact: Robert Henegar       rhenegar@wesleyneurology.com   
United States, Texas
Houston Methodist Recruiting
Houston, Texas, United States, 77030
Contact: Sharon Halton    713-441-5192    shalton@houstonmethodist.org   
Sponsors and Collaborators
Catalyst Pharmaceuticals, Inc.
Investigators
Principal Investigator: Renato Mantegazza, MD Carlo Besta Neurological Institute

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: July 9, 2018
Last Verified: July 2018
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

Additional relevant MeSH terms:
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
3,4-diaminopyridine
4-Aminopyridine
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action