# Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

> **NCT01016392** · — · COMPLETED · sponsor: **BioMarin Pharmaceutical** · enrollment: 627 (actual)

## Conditions studied

- Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

## Interventions

_None listed._

## Key facts

- **NCT ID:** NCT01016392
- **Lead sponsor:** BioMarin Pharmaceutical
- **Sponsor class:** INDUSTRY
- **Phase:** —
- **Study type:** OBSERVATIONAL
- **Status:** COMPLETED
- **Start date:** 2009-12
- **Primary completion:** 2021-05-31
- **Final completion:** 2021-05-31
- **Target enrollment:** 627 (ACTUAL)
- **Last updated:** 2022-04-15


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01016392

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01016392, "Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency". Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/clinical/NCT01016392. Licensed CC0.

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