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Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

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ClinicalTrials.gov Identifier: NCT03304054
Recruitment Status : Recruiting
First Posted : October 6, 2017
Last Update Posted : August 8, 2019
Sponsor:
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
Myasthenia Gravis, Generalized Drug: Amifampridine Phosphate Drug: Placebo Oral Tablet Phase 3

Detailed Description:
Randomized, double-blind, placebo-controlled, parallel group study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in patients with MuSK-MG. In addition, a sample of AChR-MG patients will be assess for efficacy and safety of amifampridine phosphate. Planned duration of participation for each patient is at least 38 days, excluding the screening period. Eligible patients will be titrated to an efficacious dose of amifampridine phosphate and those who demonstrate improvement will be randomized to either placebo or amifampridine, in a double-blind fashion, for 10 days.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Placebo-control, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients With MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis Patients
Actual Study Start Date : March 7, 2018
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine


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

Placebo Comparator: placebo tablets Drug: Placebo Oral Tablet
tablets matching amifampridine phosphate, 3 to 4 times a day




Primary Outcome Measures :
  1. MG-ADL [ Time Frame: Change from baseline in MG-ADL at Day 10 ]
    myasthenia gravis activities of daily living scale


Secondary Outcome Measures :
  1. QMG [ Time Frame: Change from baseline in QMG at Day 10 ]
    quantitative myasthenia score



Information from the National Library of Medicine

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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:

  1. 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.
  2. Male or female ≥18 years of age.
  3. Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed at Screening or by previous antibody test, with report available.
  4. Confirmatory EMG or EMG report.
  5. Myasthenia Gravis Foundation of America (MGFA) Class II to IV at Screening.
  6. MG-ADL score of ≥6 at Screening, with more than 50% of this score attributed to non-ocular items.
  7. Patients receiving steroids or pyridostigmine should not have any modification of drug regimen during the month before Screening.
  8. 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.
  9. 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.

Exclusion Criteria:

  1. Epilepsy and currently on medication.
  2. Concomitant use of medicinal products with a known potential to cause QTc prolongation.
  3. Patients with long QT syndromes.
  4. History of thymectomy within 12 months before Screening.
  5. An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator.
  6. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
  7. 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.
  8. Use of rituximab or other similar biologic medications for immunomodulation within 6 months prior to Screening.
  9. Treatment with an investigational drug (other than amifampridine) or device within 30 days before Screening or while participating in this study.
  10. 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.
  11. History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).

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): NCT03304054


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: Aron Schwartz       aron.schwartz@uphs.upenn.edu   
Sponsors and Collaborators
Catalyst Pharmaceuticals, Inc.
Investigators
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Principal Investigator: Renato Mantegazza, MD Carlo Besta Neurologic Institute

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Responsible Party: Catalyst Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03304054     History of Changes
Other Study ID Numbers: MSK-002
First Posted: October 6, 2017    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
Keywords provided by Catalyst Pharmaceuticals, Inc.:
MuSK antibody positive
Additional relevant MeSH terms:
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Myasthenia Gravis
Nervous System Diseases
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Amifampridine
Antibodies
Immunologic Factors
Physiological Effects of Drugs
Neuromuscular Agents
Peripheral Nervous System Agents
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action