Randomised controlled trial
Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid
- Randomised controlled trial
- phase 1
- double-blind
- placebo-controlled
- 2:1 randomisation
- ascending-dose cohorts
- open-label extension
Authors' conclusion
In this phase 1 trial of the recombinant human antibody NI006 for the treatment of patients with ATTR cardiomyopathy and heart failure, the use of NI006 was associated with no apparent drug-related serious adverse events.
Study context
- Population
- Patients with wild-type or variant ATTR cardiomyopathy and chronic HF
- Participants
- 40 patients
- Comparison
- NI006 vs placebo
- Primary endpoint
- Safety and pharmacokinetic profiles of NI006
Primary result
As reported in the abstract
Safety and pharmacokinetics
- No apparent drug-related serious adverse events with NI006
- Pharmacokinetic profile consistent with an IgG antibody
- No antidrug antibodies detected
Further findings
Cardiac imaging surrogates of amyloid load (scintigraphy tracer uptake, CMR extracellular volume), doses ≥10 mg/kg · 12 months
- Appeared to be reduced
Median NT-proBNP and troponin T
- Seemed to be reduced
Original publication
Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid. N Engl J Med. Published 20 May 2023. doi:10.1056/nejmoa2303765
Summarised from the published abstract; every number is checked against it