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NCT03359551: NIGHT
Natural History of the Progression of Choroideremia Study
trial in Choroideremia in 319 participants. Completed in 1 October 2020.
1 October 2020
Quick facts
| Lead sponsor | NightstaRx Ltd, a Biogen Company |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 319 |
| Start date | 30 June 2015 |
| Primary completion | 1 October 2020 |
| Estimated completion | 1 October 2020 |
| Sites | 20 locations across Denmark, France, Finland, Netherlands, United Kingdom, Germany, Canada, United States |
Conditions studied
- Choroideremia — all drugs for Choroideremia →
Sponsor
NightstaRx Ltd, a Biogen Company
Who can join
18 and older, male only, with Choroideremia. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The objective of this natural history study is to gain a better understanding of the progression of choroideremia (CHM) and add to the knowledge base for this rare disease.
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
-
Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease.
Ong T, Pennesi ME, Birch DG, Lam BL, et al · · 2019 · cited 46× · PMID 30617669 · DOI 10.1007/s11095-018-2564-5 -
Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial.
MacLaren RE, Fischer MD, Gow JA, Lam BL, et al · · 2023 · cited 36× · PMID 37814062 · DOI 10.1038/s41591-023-02520-3 -
CHOROIDEREMIA: Retinal Degeneration With an Unmet Need.
Pennesi ME, Birch DG, Duncan JL, Bennett J, et al · · 2019 · cited 30× · PMID 31021898 · DOI 10.1097/iae.0000000000002553 -
Progress in the development of novel therapies for choroideremia.
Cehajic Kapetanovic J, Patrício MI, MacLaren RE. · · 2019 · cited 20× · PMID 32002021 · DOI 10.1080/17469899.2019.1699406 -
Choroideremia Gene Therapy.
Lam BL, Davis JL, Gregori NZ. · · 2021 · cited 15× · PMID 34584056 · DOI 10.1097/iio.0000000000000385 -
Bilateral visual acuity decline in males with choroideremia: a pooled, cross-sectional meta-analysis.
Bozkaya D, Zou H, Lu C, Tsao NW, et al · · 2022 · cited 5× · PMID 35034620 · DOI 10.1186/s12886-022-02250-z
Verify or expand the search:
- PubMed search for NCT03359551
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Choroideremia
Currently open trials in the same condition.
- NCT06375239 — Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degener · recruiting
- NCT04483440 — Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia · Phase 1 · active not recruiting
Other NightstaRx Ltd, a Biogen Company trials
Trials by the same sponsor.
- NCT03584165 — Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X · Phase 3 · enrolling by invitation
- NCT04926129 — Natural History of the Progression of X-Linked Retinitis Pigmentosa · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT03359551 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 9 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 NightstaRx Ltd, a Biogen Company
- Last refreshed: 10 February 2023
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/NCT03359551.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing