Clinical Trial Update: Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety [Phase 2, Phase 3]

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Summary

A terminated Phase 2/3 trial evaluated long-term tetrahydrobiopterin treatment in children with phenylketonuria to assess phenylalanine tolerance and safety.

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Diseases / indications

• Phenylketonuria

Drugs mentioned

• tetrahydrobiopterin (tetrahydrobiopterin)

Snippet

Sponsor: Orphanetics Pharma Entwicklungs GmbH | Conditions: Phenylalanine Hydroxylase Deficiencies | Phase 2, Phase 3 | Status: TERMINATED