A Study of TAK-994 in Adults With Type 1 and Type 2 Narcolepsy

Phase 2 Terminated 97 enrolled Takeda

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The main aims of the study are: * To check for side effects from TAK-994 and check what dose of TAK-994 participants can tolerate. * To check what dose range provides adequate relief of narcolepsy symptoms. * To check how much TAK-994 stays in the blood of participants, over time. The study will have 4 parts. Participants can only join 1 of the parts. A. Participants with type 1 narcolepsy will take either TAK-994 or placebo tablets for 28 days. A placebo looks just like TAK-994 but will not have any medicine in it. B. Participants with type 1 narcolepsy will take 1 of 3 doses of TAK-994 or placebo tablets for 56 days. C. Participants with type 1 narcolepsy in China only will take TAK-994 or placebo tablets for 56 days. D. Participants with type 2 narcolepsy will take either TAK-994 or placebo tablets for 28 days.

Timeline

Start: 2020-05-27
Primary completion: 2021-11-05
Completion: 2021-11-05

Outcome

Met primary endpoint

Stopped: “A safety signal has emerged in Phase 2 studies of TAK-994. As an immediate precautionary measure, Takeda has suspended dosing of patients and has decided to stop Phase 2 studies early.”

registry analysis (superiority test); Part B: Placebo vs Part B: TAK-994 30 mg; p <0.001; LS Mean Difference 26.4 (95% CI 20.07 to 32.73); MMRM; NCT04096560 ↗

registry analysis (superiority test); Part B: Placebo vs Part B: TAK-994 90 mg; p <0.001; LS Mean Difference 29.9 (95% CI 23.68 to 36.07); MMRM; NCT04096560 ↗

registry analysis (superiority test); Part B: Placebo vs Part B: TAK-994 180 mg; p <0.001; LS Mean Difference 35.0 (95% CI 28.73 to 41.34); MMRM; NCT04096560 ↗

Publications

Dauvilliers Y, Mignot E, Del Rio Villegas R, Du Y, Hanson E, Inoue Y, Kadali H, Koundourakis E, Meyer S, Rogers R, Scammell TE, Sheikh SI, Swick T, Szakacs Z, von Rosenstiel P, Wu J, Zeitz H, Murthy NV, Plazzi G, von Hehn C. Oral Orexin Receptor 2 Agonist in Narcolepsy Type 1. N Engl J Med. 2023 Jul 27;389(4):309-321. doi: 10.1056/NEJMoa2301940.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	TAK-994	Unknown	30 mg	Oral
Subject	TAK-994	Unknown	90 mg	Oral
Subject	TAK-994	Unknown	120 mg	Oral
Subject	TAK-994	Unknown	180 mg	Oral