drugset / Trial / NCT04360434
First-in-Human Study of NI006 in Patients With Amyloid Transthyretin Cardiomyopathy
RandomizedParallel-groupQuadruple-blindTreatment
Summary
A phase 1, randomized, placebo-controlled, double-blind, dose escalation trial combining single-ascending dose and multiple-ascending dose phases of NI006 or placebo, followed by an open-label extension phase in subjects with Amyloid Transthyretin Cardiomyopathy (ATTR-CM).
Timeline
- Start
- 2020-02-10
- Primary completion
- 2023-07-26
- Completion
- 2023-07-26
Publications
- Kahr PC, Aus dem Siepen F, Lairez O, Donal E, Damy T, Kristen AV, Quarta CC, Mercuri MF, Keppner-Witter S, Herrmann-Keiner E, Tichy M, Michalon A, Grimm J, Nitsch RM, Hock C, Garcia-Pavia P, van der Meer P. Cliramitug for depletion of cardiac amyloid transthyretin: long-term follow-up of the NI006-101 trial. Nat Med. 2026 Jun 26. doi: 10.1038/s41591-026-04487-3. Online ahead of print.
- Garcia-Pavia P, Aus dem Siepen F, Donal E, Lairez O, van der Meer P, Kristen AV, Mercuri MF, Michalon A, Frost RJA, Grimm J, Nitsch RM, Hock C, Kahr PC, Damy T. Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid. N Engl J Med. 2023 Jul 20;389(3):239-250. doi: 10.1056/NEJMoa2303765. Epub 2023 May 20.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | ALXN2220 | Unknown | 0.3 mg/kg | Intravenous |
| Subject | ALXN2220 | Unknown | 30 mg/kg | Intravenous |
| Subject | ALXN2220 | Unknown | 60 mg/kg | Intravenous |
Indications
No indication recorded.