Studies
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.
Pridopidine EAP2
This EAP will provide access to pridopidine for up to 200 patients with ALS who are ineligible for clinical trials. Pridopidine will be given at a dose of 45 mg twice daily p.o. (or via feeding tube).
Each patient will be followed for 2 years with regularly scheduled visits. The screening and baseline visits will be performed in person; subsequent visits may occur in person or remotely. Recommended in-person visits will occur at Weeks 12, 52, 78, and end of treatment (Week 104 or early termination). If the patient is unable to complete the visits in person, these visits may also be completed remotely.
Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis
RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).
Intermediate Expanded Access Protocol CNMAu8.EAP04
An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic Lateral Sclerosis for NIH Grant RFA-NS-23-012
HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.