Improving symptomatic treatment with pyridostigmine and amifampridine in patients with myasthenia gravis

Phase 1

• Conditions: Myasthenia Gravis; MedDRA version: 24.0Level: LLTClassification code 10085562Term: AChR myasthenia gravisSystem Organ Class: 100000004852; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2021-004110-20-NL
• Lead Sponsor: eiden University Medical Center

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-07-12
• Last Update Posted: 4 March 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

1. Age >18 years
2. AChR positive myasthenia gravis (ocular or generalized)
3. Current use of pyridostigmine
4. MGFA Clinical Classification I-IV
5. Receiving a stable dose of MG treatment (other than pyridostigmine). If applicable:
a. A stable steroid regimen for 1 month
b. Nonsteroidal immunosuppressants:
i. Azathioprine, mycophenolate mofetil, cyclosporine or other nonsteroid immunosuppressive agents start > 3 months ago and a stable regimen for 1 month.
ii. Rituximab, complement inhibitors and Fc receptor inhibitors start > 6 months ago and a stable regimen for 3 months.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 17
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 7

Exclusion Criteria

1. Use of intravenous immunoglobulin or plasma exchange <4 weeks
2. Thymectomy < 6 months, or thymectomy (expected) to take place during the trial
3. Use of other AChE inhibitors than pyridostigmine
4. Pregnancy, lactation or intention to become pregnant during the study
5. Treatment with amifampridine is contraindicated. Contraindications include a history of epilepsy, uncontrolled asthma, inherited QT syndrome / a prolonged QT interval (as indicated by ECG), any drug known to cause QTc-prolongation, concomitant use of sultopride, a known hypersensitivity reaction to the active substance or to any of the excipients.
6. The patient is unable to fill out the study questionnaires or be interviewed in Dutch, or is unable to undergo the tests needed for the study, or is unable to give informed consent for participation in the study.
7. The investigator can exclude patients for this trial which are deemed not suitable for any reason.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified