Trial reportMuscle & nerve2018
3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia.
Trial report in Muscle & nerve, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01765140 (Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome), which is not on this map. Cited by 17 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome
Who cites it
17 citing papers in PubMed.
- Amifampridine Phosphate (Firdapse) Is Effective in a Confirmatory Phase 3 Clinical Trial in LEMS.Journal of clinical neuromuscular disease · 2019Trial
- Longitudinal overview of symptomatic and immunosuppressive drugs in LEMS.Journal of neuromuscular diseases · 2026Article
- Paraneoplastic Lambert-Eaton myasthenic syndrome associated with non-small cell lung cancer: data from the European LEMS registry and systematic review.Neurological research and practice · 2025Article
- Amifampridines are the Most Effective Drugs for Treating Lambert-Eaton Myasthenic Syndrome With a Focus on Pediatric Lambert-Eaton Myasthenic Syndrome.Journal of clinical neurology (Seoul, Korea) · 2024Review
- Simulations of active zone structure and function at mammalian NMJs predict that loss of calcium channels alone is not sufficient to replicate LEMS effects.Journal of neurophysiology · 2023Article
- Amifampridine for Lambert-Eaton myasthenic syndrome.Australian prescriber · 2022Review
- The European Lambert-Eaton Myasthenic Syndrome Registry: Long-Term Outcomes Following Symptomatic Treatment.Neurology and therapy · 2022Article
- Activation of Voltage-Gated NaBiomedicines · 2022Article
- Effects of 3,4-diaminopyridine on myasthenia gravis: Preliminary results of an open-label study.Frontiers in pharmacology · 2022Article
- Myasthenia Gravis and Lambert-Eaton Myasthenic Syndrome: New Developments in Diagnosis and Treatment.Neuropsychiatric disease and treatment · 2022Review
- Presynaptic Paraneoplastic Disorders of the Neuromuscular Junction: An Update.Brain sciences · 2021Review
- Primary mitochondrial myopathy: Clinical features and outcome measures in 118 cases from Italy.Neurology. Genetics · 2020Article
- Validation of the triple timed up-and-go test in Lambert-Eaton myasthenia.Muscle & nerve · 2019Article
- Review
- Lambert-Eaton Myasthenic syndrome: early diagnosis is key.Degenerative neurological and neuromuscular disease · 2019Review
- Low specificity of voltage-gated calcium channel antibodies in Lambert-Eaton myasthenic syndrome: a call for caution.Journal of neurology · 2018Article
- A high-affinity, partial antagonist effect of 3,4-diaminopyridine mediates action potential broadening and enhancement of transmitter release at NMJs.The Journal of biological chemistryArticle
Corrections and comments
- Commented on by
- Commented on byReply.2018
Authors and funding
15 authors.
Funding
Abstract
introduction3,4-diaminopyridine has been used to treat Lambert-Eaton myasthenia (LEM) for 30 years despite the lack of conclusive evidence of efficacy.
methodsWe conducted a randomized double-blind placebo-controlled withdrawal study in patients with LEM who had been on stable regimens of 3,4-diaminopyridine base (3,4-DAP) for ≥ 3 months. The primary efficacy endpoint was >30% deterioration in triple timed up-and-go (3TUG) times during tapered drug withdrawal. The secondary endpoint was self-assessment of LEM-related weakness (W-SAS).
resultsThirty-two participants were randomized to continuous 3,4-DAP or placebo groups. None of the 14 participants who received continuous 3,4-DAP had > 30% deterioration in 3TUG time versus 72% of the 18 who tapered to placebo (P < 0.0001). W-SAS similarly demonstrated an advantage for continuous treatment over placebo (P < 0.0001). Requirement for rescue and adverse events were more common in the placebo group. DISCUSSION: This trial provides significant evidence of efficacy of 3,4-DAP in the maintenance of strength in LEM. Muscle Nerve 57: 561-568, 2018.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.