NCT04982393 (BONAPH1DE) is a clinical trial in Primary Hyperoxaluria Type 1, sponsored by Alnylam Pharmaceuticals.

Who is sponsoring NCT04982393?

NCT04982393 is sponsored by Alnylam Pharmaceuticals.

What condition does NCT04982393 study?

NCT04982393 studies Primary Hyperoxaluria Type 1.

Is NCT04982393 still recruiting?

NCT04982393 is currently active, enrolled. Last updated 31 March 2026.

Last reviewed 2026-03-31 · How we verify

NCT04982393: BONAPH1DE

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)

Active, enrolled Last updated 31 March 2026

What this trial tests

trial in Primary Hyperoxaluria Type 1 in 207 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

13 December 2021

Primary endpoint
1 September 2028

1 September 2028

Quick facts

Lead sponsor	Alnylam Pharmaceuticals
Status	Active, enrolled
Study type	OBSERVATIONAL
Enrollment	207
Start date	13 December 2021
Primary completion	1 September 2028
Estimated completion	1 September 2028
Sites	30 locations across France, Italy, Netherlands, Belgium, United Kingdom, Germany, Israel, Canada

Conditions studied

Primary Hyperoxaluria Type 1 — all drugs for Primary Hyperoxaluria Type 1 →

Sponsor

Alnylam Pharmaceuticals — full company profile →

Who can join

Eligibility, any sex, with Primary Hyperoxaluria Type 1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is to describe the natural history and progression of patients diagnosed with PH1, and to characterize the long-term real-world safety and effectiveness of lumasiran.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

Therapeutic siRNA: State-of-the-Art and Future Perspectives.
Friedrich M, Aigner A. · · 2022 · cited 256× · PMID 35997897 · DOI 10.1007/s40259-022-00549-3
Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.
Liu M, Wang Y, Zhang Y, Hu D, et al · · 2025 · cited 62× · PMID 40059188 · DOI 10.1038/s41392-024-02112-8
Hepatocyte targeting <i>via</i> the asialoglycoprotein receptor.
Ramírez-Cortés F, Ménová P. · · 2025 · cited 22× · PMID 39628900 · DOI 10.1039/d4md00652f
Lumasiran in the Management of Patients with Primary Hyperoxaluria Type 1: From Bench to Bedside.
D'Ambrosio V, Ferraro PM. · · 2022 · cited 14× · PMID 35747094 · DOI 10.2147/ijnrd.s293682
Biotechnological Evolution of siRNA Molecules: From Bench Tool to the Refined Drug.
de Brito E Cunha D, Frederico ABT, Azamor T, Melgaço JG, et al · · 2022 · cited 14× · PMID 35631401 · DOI 10.3390/ph15050575
Lumasiran for primary hyperoxaluria type 1: What we have learned?
Gang X, Liu F, Mao J. · · 2022 · cited 13× · PMID 36704142 · DOI 10.3389/fped.2022.1052625
Current Approaches to Epigenetic Therapy.
Griazeva ED, Fedoseeva DM, Radion EI, Ershov PV, et al · · 2023 · cited 11× · PMID 37873808 · DOI 10.3390/epigenomes7040023

Verify or expand the search:

Other recruiting trials for Primary Hyperoxaluria Type 1

Currently open trials in the same condition.

NCT04580420 — Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD · Phase 2 · recruiting

Other Alnylam Pharmaceuticals trials

Trials by the same sponsor.

NCT07535606 — A Study to Evaluate ALN-4915 in Adult Healthy Volunteers · Phase 1 · recruiting
NCT07463846 — A Study to Evaluate ALN-2232 in Participants With Obesity · Phase 1, PHASE2 · recruiting
NCT07223203 — TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidos · Phase 3 · recruiting
NCT07465224 — A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus · Phase 2 · recruiting
NCT07358078 — DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04982393 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Alnylam Pharmaceuticals
Last refreshed: 31 March 2026

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04982393.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing