|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
18 March 2024 |
|
Main ID: |
NCT03304054 |
|
Date of registration:
|
28/09/2017 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG
|
|
Scientific 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 |
|
Date of first enrolment:
|
April 18, 2018 |
|
Target sample size:
|
93 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/ct2/show/NCT03304054 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
|
|
Phase:
|
Phase 3
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Renato Mantegazza, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Carlo Besta Neurologic Institute |
| | |
|
Key inclusion & exclusion 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).
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Myasthenia Gravis, Generalized
|
|
Intervention(s)
|
|
Drug: Placebo Oral Tablet
|
|
Drug: Amifampridine Phosphate
|
|
Primary Outcome(s)
|
|
Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results
[Time Frame: Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early).]
|
|
Secondary Outcome(s)
|
|
Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results
[Time Frame: Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early).]
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|