Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Expanded Access Givosiran in Acute Hepatic Porphyria (Japan)
jRCT2071200074Completed
- Sponsor
- Alnylam Pharmaceuticals
- Start
- Feb 2021
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Open-label, single-arm trial of givosiran in acute porphyria, acute intermittent porphyria (AIP), hereditary coproporphyria (HCP) and 2 more, sponsored by Alnylam Pharmaceuticals. Primary endpoints: Urinary aminolevulinic acid (ALA) levels, Urinary porphobilinogen (PBG) levels and 4 more. Started Feb 2021; status: completed.
02
Drugs
givosiran
03
Conditions
acute porphyriaacute intermittent porphyria (AIP)hereditary coproporphyria (HCP)variegate porphyria (VP)ALA dehydratase deficiency porphyria (ADP)
Beyond the preview
Go deeper on jRCT2071200074.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on jRCT2071200074.