Study design & results
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Trial intelligence / Profile preview
Phase 1 double-blind placebo-controlled ascending-dose study of CMP-CPS-001 in healthy volunteers and females with heterozygous OTC genotype
NL-OMON58964Phase 1Not yet recruiting
- Sponsor
- CAMP4 Therapeutics
- Start
- Aug 2025
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 1, double-blind trial of CMP-CPS-001 in urea cycle disorder (UCD) and ornithine transcarbamylase deficiency (2 arms), sponsored by CAMP4 Therapeutics. Primary endpoints: Incidence of adverse events, Vital signs and 3 more. Planned start Aug 2025; status: not yet recruiting.
02
Drugs
CMP-CPS-001
03
Conditions
urea cycle disorder (UCD)ornithine transcarbamylase deficiency
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