Clinical Trials

Current Studies

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Study 165-508

Recruiting

A Multicentre, Observational Study to Evaluate the Real-World Outcomes of PALYNZIQ^® (Pegvaliase) in Subjects with Phenylketonuria in Germany

• Indication

  Phenylketonuria (PKU)

• Investigational therapeutic

  PALYNZIQ^® (pegvaliase)

• Study Type

  Phase 4

• Goal

  In addition to the efficacy and safety profile of pegvaliase that has been established in clinical trials; there remains an important need to provide data to prescribers and the phenylketonuria (PKU) community on the real-world usage of pegvaliase, and associated outcomes, to support clinical practice optimization and provide greater understanding of important clinical issues in PKU

• Status

  This study is currently recruiting participants

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Completed Studies

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Study 165-304

An Open-label Extension Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase (> 40mg/day Dose) in Adults with Phenylketonuria.

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  PALYNZIQ^® (pegvaliase, rAvPAL-PEG or BMN 165)

• Study Type

  Phase 3

• Goal

  Evaluate long-term safety and efficacy of Pegvaliase in (> 40mg/day dose) in adults with PKU

• Status

  This study has been completed

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Study 165-302

A Four-Part, Phase 3, Randomized, Double-Blind, Placebo-Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self Administered by Adults with Phenylketonuria

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  PALYNZIQ^® (pegvaliase, rAvPAL-PEG or BMN 165)

• Study Type

  Phase 3

• Goal

  The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in adults with PKU

• Status

  This study has been completed

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Study PAL-003

Long-term Extension of a Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous Doses of rAvPAL-PEG in Subjects with PKU

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  PALYNZIQ^® (pegvaliase, rAvPAL-PEG or BMN 165)

• Study Type

  Phase 2

• Goal

  Evaluate the long-term safety, tolerability and effectiveness of injections of an investigational drug for people with PKU who have trouble controlling and maintaining their Phe levels

• Status

  This study has been completed

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Study 165-205

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients with Phenylketonuria for 24 Weeks

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  PALYNZIQ^® (pegvaliase, rAvPAL-PEG or BMN 165)

• Study Type

  Phase 2

• Goal

  Evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of hypersensitivity reactions in patients with PKU

• Status

  This study has been completed

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Study PAL-004

A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of Ravpal-PEG Administered Daily in Subjects with Phenylketonuria

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  PALYNZIQ^® (pegvaliase, rAvPAL-PEG or BMN 165)

• Study Type

  Phase 2

• Goal

  Evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU

• Status

  This study has been completed

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Study 165-301

A Phase 3, Open-Label, Randomized, Multi-Center Study to Assess the Safety and Tolerability of an Induction, Titration, and Maintenance Dose Regimen of Pegvaliase Self-Administered by Adults with Phenylketonuria Not Previously Treated with Pegvaliase

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  PALYNZIQ^® (pegvaliase, rAvPAL-PEG or BMN 165)

• Study Type

  Phase 3

• Goal

  Evaluate the safety and tolerability of injections of an investigational drug (also called PEG PAL) for people with PKU who have not received Pegvaliase in the past

• Status

  This study has been completed

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