Clinical Trials

Current Studies

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Study 165-306 (PEGASUS)

Recruiting

A Phase 3 Multi Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12 17) with Phenylketonuria Featuring an Open Label Randomized Two Arm (Active vs Diet Only Control) Design

• Indication

  Phenylketonuria

• Investigational therapeutic

  BMN165

• Study Type

  Phase 3

• Goal

  Evaluate safety and efficacy of BMN 165 in adolescents with PKU when administered as an Induction/Titration and Maintenance dose regimen.

• Status

  Recruiting

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Study 165-305

A Phase 3, Open-Label, Multi-Center Study to Assess the Safety and Efficacy of BMN 165 in Japanese Subjects 18 Years of Age and Older With Phenylketonuria

• Indication

  Phenylketonuria

• Investigational therapeutic

  BMN165

• Study Type

  Phase 3

• Goal

  The primary objective of the study is to evaluate the efficacy and safety of BMN 165 in Japanese subjects ≥ 18 years of age with PKU when administered as an Induction/Titration and Maintenance dose regimen.

• Status

  This study is ongoing, but not recruiting participants.

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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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Study 165-501

Recruiting

A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (pegvaliase) in Subjects with Phenylketonuria

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  N/A

• Study Type

  Phase 4

• Goal

  – To quantify and characterize the risk of protocol-defined safety events in incident-users receiving pegvaliase for the treatment of PKU in a real-world setting.

  – To quantify and characterize the risk of protocol-defined safety events in subjects receiving pegvaliase for the treatment of PKU in a real-world setting.

• Status

  This study is currently recruiting participants

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Study 165-504

Recruiting

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

• Indication

  Phenylketonuria (PKU)

• Investigational Therapeutic

  Pegvaliase

• Study Type

  Phase 4

• Goal

  This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and breastfeeding

• 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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