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Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

This study is not yet open for participant recruitment.
Verified October 2017 by Catalyst Pharmaceuticals, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03304054
First Posted: October 6, 2017
Last Update Posted: October 10, 2017
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.
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.
September 28, 2017
October 6, 2017
October 10, 2017
December 2017
December 2018   (Final data collection date for primary outcome measure)
MG-ADL [ Time Frame: Change from baseline in MG-ADL at Day 10 ]
myasthenia gravis activities of daily living scale
Same as current
Complete list of historical versions of study NCT03304054 on ClinicalTrials.gov Archive Site
QMG [ Time Frame: Change from baseline in QMG at Day 10 ]
quantitative myasthenia score
Same as current
Not Provided
Not Provided
 
Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG
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
Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.
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.
Interventional
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Myasthenia Gravis, Generalized
  • Drug: Amifampridine Phosphate
    tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day
  • Drug: Placebo Oral Tablet
    tablets matching amifampridine phosphate, 3 to 4 times a day
  • Experimental: amifamapridine phosphate tablets
    Intervention: Drug: Amifampridine Phosphate
  • Placebo Comparator: placebo tablets
    Intervention: Drug: Placebo Oral Tablet
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Not yet recruiting
60
February 2019
December 2018   (Final data collection date for primary outcome measure)

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).
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact: Gary Ingenito, MD, PhD 305-420-3200 gingenito@catalystpharma.com
Contact: Adriana Manari 305-420-3200 amanari@catalystpharma.com
United States
 
 
NCT03304054
MSK-002
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Catalyst Pharmaceuticals, Inc.
Catalyst Pharmaceuticals, Inc.
Not Provided
Principal Investigator: Renato Mantegazza, MD Carlo Besta Neurologic Institute
Catalyst Pharmaceuticals, Inc.
October 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP