Clinical trial · Interventional
Pyridostigmine and Amifampridine for Myasthenia Gravis
IMproving Symptomatic Treatment With Pyridostigmine and Amifampridine: a Randomized Double-blinded, Placebo Controlled Crossover Trial in Patients With Myasthenia Gravis (IMPACT-MG)
NCT05919407CI-TRIAL-00067531IMPACT-MGunknownPhase 3ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
A randomized, double-blind, placebo controlled, crossover intervention study evaluating the effect of pyridostigmine (part 1) and amifampridine (part 2) in Myasthenia Gravis (MG).
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myasthenia Gravis | — | UNRESOLVED | — |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Amifampridine (base) with modified release | Drug | — | UNRESOLVED |
| Placebo | Drug | — | UNRESOLVED |
| Pyridostigmine | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (4)
- type
- EXPERIMENTAL
- label
- Pyridostigmine
- description
- The dose of pyridostigmine will be based on the patient's prior experience with pyridostigmine under the assumption that the patient already gained sufficient experience during their disease course to know which dose is effective for them as patients are advised by their treating neurologist to continually adjust their dose based on their symptoms and side effects.
- interventionNames
- Drug: Pyridostigmine
- type
- PLACEBO_COMPARATOR
- label
- Placebo (pyridostigmine)
- description
- Same as "Experimental", however capsules contain placebo.
- interventionNames
- Drug: Placebo
- type
- EXPERIMENTAL
- label
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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: 1. A stable steroid regimen for 1 month 2. 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 start \> 6 months ago, complement inhibitors and Fc receptor inhibitors start \> 6 months ago and a stable regimen for 3 months. Additional inclusion criteria for part 2 To be eligible for participation in part 2 of the study patients must score \>10 points on the MGII questionnaire at inclusion. We will include a maximum of 5 patients with a MGFA class I (i.e. 20 percent of the total number of included patients) to ensure that this study accurately reflects the clinical population. Exclusion Criteria: 1. Use of intravenous immunoglobulin or plasma exchange \<4 weeks or planned during the trial. 2. Thymectomy \< 6 months, or thymectomy (expected) to take place during the trial 3. Use of other acetylcholinesterase 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 QT c-prolongation, any drug known to lower the epileptic threshold, 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.
References
Publications (2)
- DERIVEDRemijn-Nelissen L, Bakker WR, van den Hout WB, van Gelder T, Ruiter AM, Campman YJM, Badrising UA, Straathof CSM, Verschuuren JJGM, Tannemaat MR. Efficacy of Pyridostigmine in Myasthenia Gravis: A Randomized, Double-Blind, Placebo-Controlled Crossover Trial. Neurology. 2026 Apr 28;106(8):e214865. doi: 10.1212/WNL.0000000000214865. Epub 2026 Apr 7. PMID 41945881
- DERIVEDRemijn-Nelissen L, Bakker WR, van Gelder T, Moes DJAR, Ruiter AM, Campman YJM, Badrising UA, Straathof CSM, Verschuuren JJGM, Tannemaat MR. Efficacy and Safety of Amifampridine in Myasthenia Gravis: A Randomized, Double-Blind, Placebo-Controlled Crossover Trial. Neurology. 2026 Apr 28;106(8):e214715. doi: 10.1212/WNL.0000000000214715. Epub 2026 Apr 7. PMID 41945880