Treatment Use of 3,4-Diaminopyridine

Expanded access No longer available Vern C. Juel, M.D.

Summary

This protocol provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is now closed.

Timeline

Start: —
Primary completion: —
Completion: —

Outcome

Met primary endpoint

paper 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). PMID 29280483 ↗

Publications

Results Sanders DB, Juel VC, Harati Y, Smith AG, Peltier AC, Marburger T, Lou JS, Pascuzzi RM, Richman DP, Xie T, Demmel V, Jacobus LR, Ales KL, Jacobus DP; Dapper Study Team. 3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia. Muscle Nerve. 2018 Apr;57(4):561-568. doi: 10.1002/mus.26052. Epub 2018 Feb 2.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Amifampridine	Small molecule	—	—