Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes (NCT02562066) | Clinical Trial Compass
CompletedPhase 3
Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
United States20 participantsStarted 2016-01
Plain-language summary
This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 and above) diagnosed with certain genetic subtypes of CMS and demonstrated open label (amifampridine phosphate) or history of sustained amifampridine benefit from treatment.
Who can participate
Age range2 Years – 70 Years
SexALL
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Inclusion criteria
✓. Patient's or parent 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, or the patient's legal guardian or caregiver with durable power of attorney can provide written informed consent. An assent form must also be signed if in the judgement of the IRB the children are capable of providing assent.
✓. Male or female age 2 and above.
✓. Body weight ≥10 kg.
✓. Genetically-confirmed CMS involving acetylcholine receptor defect, Rapsyn deficiency, MuSK deficiency, Dok-7 deficiency, SYT2 deficiency,SNAP25B deficiency, and fast channel syndrome.
✓. MFM 20 or 32 score equal or less than 48 or 76, respectively, at Screening.
✓. In patients naïve to 3,4-DAP or amifampridine phosphate, improvement of \>20% in MFM20 or MFM32 scores after open label period of up titration of dose
✓. In patients previously stabilized on 3,4-DAP or amifampridine phosphate, history of meaningful improvement in motor function (in opinion of investigator)
✓. Willingness of patients receiving pyridostigmine, prednisone, albuterol, ephedrine, or fluoxetine to remain on a stable dose of these medications throughout the study interval.
Exclusion criteria
✕. CMS subtype diagnosis of acetylcholinesterase deficiency, slow-channel syndrome, LRP4 deficiency, and plectin deficiency.
✕. Cardiac conduction defects on Screening ECG.
What they're measuring
1
Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis
Timeframe: Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29
✕. Pregnancy or breastfeeding at Screening or planning to become pregnant at any time during the study.
✕. Any systemic bacterial or other infection, which is clinically significant in the opinion of the investigator and has not been treated with appropriate antibiotics.