Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)
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| ClinicalTrials.gov Identifier: NCT03579966 |
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Recruitment Status :
Recruiting
First Posted : July 9, 2018
Last Update Posted : July 9, 2018
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Sponsor:
Catalyst Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.
- Study Details
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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 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
Genetics Home Reference related topics:
Myasthenia gravis
MedlinePlus related topics:
Myasthenia Gravis
| Arm | Intervention/treatment |
|---|---|
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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 |
Primary Outcome Measures :
- 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:
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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 3,4-diaminopyridine 4-Aminopyridine Potassium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |