ClinicalTrials.gov
ClinicalTrials.gov Menu

Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03304054
Recruitment Status : Recruiting
First Posted : October 6, 2017
Last Update Posted : June 7, 2018
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.

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 : December 2018
Estimated Study Completion Date : February 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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
Contact: Gary Ingenito, MD, PhD 305-420-3200 gingenito@catalystpharma.com
Contact: Adriana Manari 305-420-3200 amanari@catalystpharma.com

Locations
United States, California
UC Irvine Not yet recruiting
Irvine, California, United States, 92868
Contact: Veena Mathew       vmathew@uci.edu   
University of Southern California Recruiting
Los Angeles, California, United States, 90033
Contact: Salma Akhter       salma.akhter@med.usc.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 Not yet 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 Not yet 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 Not yet 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 Neurologic Institute

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: June 7, 2018
Last Verified: June 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

Keywords provided by Catalyst Pharmaceuticals, Inc.:
MuSK antibody positive

Additional relevant MeSH terms:
Myasthenia Gravis
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Antibodies
3,4-diaminopyridine
4-Aminopyridine
Immunologic Factors
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action