Last reviewed · How we verify
NCT03505125: 165-901
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
trial in Phenylketonuria (PKU) in 63 participants. Completed in 31 July 2019.
31 July 2019
Quick facts
| Lead sponsor | BioMarin Pharmaceutical |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 63 |
| Start date | 31 March 2018 |
| Primary completion | 31 July 2019 |
| Estimated completion | 31 July 2019 |
| Sites | 14 locations across France, Italy, United Kingdom, Germany, Canada, United States, Turkey (Türkiye) |
Conditions studied
- Phenylketonuria (PKU) — all drugs for Phenylketonuria (PKU) →
Sponsor
BioMarin Pharmaceutical — full company profile →
Who can join
Adults 18 to 70, any sex, with Phenylketonuria (PKU). Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.
Publications & conference data
1 peer-reviewed publication reference this trial (live from Europe PMC):
-
A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trials.
Burton BK, Skalicky A, Baerwald C, Bilder DA, et al · · 2021 · cited 4× · PMID 34815941 · DOI 10.1016/j.ymgmr.2021.100810
Verify or expand the search:
- PubMed search for NCT03505125
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Phenylketonuria (PKU)
Currently open trials in the same condition.
- NCT07255599 — Evaluation of BH4 Responsiveness in Our PKU Patients · Phase 1 · active not recruiting
- NCT06969209 — Brain Aging in Phenylketonuria · recruiting
- NCT06305234 — A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) · recruiting
- NCT05813678 — A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) · recruiting
Other BioMarin Pharmaceutical trials
Trials by the same sponsor.
- NCT07477691 — Immune Modulation During Palynziq® Treatment in Adults (IMPALA) · Phase 4 · not yet recruiting
- NCT07126262 — A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months · Phase 2 · recruiting
- NCT06309979 — A Study to Assess Growth in Children With Idiopathic Short Stature · recruiting
- NCT06455059 — Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia · Phase 3 · active not recruiting
- NCT06305234 — A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) · recruiting
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 NCT03505125 (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 BioMarin Pharmaceutical
- Last refreshed: 14 January 2020
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/NCT03505125.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing