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Pegvaliase (BMN 165) and Kuvan® for PKU

Current Clinical Trials: PKU

Study: 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)

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 PKUtrial

Status: This study is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

Study: 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: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 2

Goal: This study is evaluating 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 is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

Study: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Indication: Phenylketonuria (PKU), Hyperphenylalaninaemia

Investigational Therapeutic: Kuvan

Study Type: Observational

Goal: The objective of this study is to evaluate the safety of long-term treatment with Kuvan

Status: This study is currently recruiting participants

Additional information: View on Clinicaltrials.gov

Study: Kuvan®‘s Effect on the Cognition of Children With Phenylketonuria (KOGNITO)

Indication: Phenylketonuria (PKU)

Investigational Therapeutic: The Investigational Medicinal Product (IMP) for this study is Kuvan® (sapropterin dihydrochloride).

Study Type: Phase 4

Goal: A Phase IV Open-Label, Single-Cohort Study of the Long-
Term Neurocognitive Outcomes in 4 to 5 Year-Old Children with Phenylketonuria Treated with Sapropterin Dihydrochloride (Kuvan®) for 7 Years

Status: This study is ongoing, but not recruiting participants

Additional information: View on Clinicaltrials.gov

Study: Kuvan® in Phenylketonuria Patients Less Than 4 Years Old (SPARK)

Indication: Phenylketonuria (PKU)

Investigational Therapeutic: The Investigational Medicinal Product (IMP) for this study is Kuvan® (sapropterin dihydrochloride).

Study Type: Phase 3b

Goal: This study has been designed to evaluate the efficacy after 26 weeks of Kuvan® treatment + Phe-restricted diet therapy in increasing dietary Phe tolerance, as compared to dietary therapy alone in <4 year-old infants and children with phenylketonuria (PKU). Phe tolerance will be defined as the amount of dietary Phe (mg/kg/day) ingested while maintaining blood Phe levels within the range of 120-360 μmol/L (defined as ≥120 to < 360 μmol/L).

Status: This study is completed (awaiting final results)

Additional information: View on Clinicaltrials.gov

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Completed Clinical Studies: PKU

Study: Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

Indication: Phenylketonuria (PKU)

Investigational Therapeutic: Kuvan (formerly Phenoptin™)

Study Type: Phase 3

Goal: The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria

Status: This study has been completed

Additional information: View on Clinicaltrials.gov

Study: A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects with Phenylketonuria

Indication: Phenylketonuria (PKU)

Investigational Therapeutic: Kuvan (formerly Phenoptin™)
 
Study Type: Phase 3

Goal: This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU

Status: This study has been completed

Additional information: View on Clinicaltrials.gov

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

Indication: Phenylketonuria (PKU)

Investigational Therapeutic: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 2

Goal: The primary objective of the study is to 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

Additional information: View on Clinicaltrials.gov

Study: 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: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 2

Goal: The purpose of this study is to 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

Additional information: View on Clinicaltrials.gov

Study: 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: Pegvaliase (rAvPAL-PEG or BMN 165)

Study Type: Phase 3

Goal: This study is to 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

Additional information: View on Clinicaltrials.gov

Clinical Trial

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.
Read more »

Clinical Trial

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
Read more »

Clinical Trial

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
Read more »

Clinical Trial

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Read more »

Clinical Trial

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Read more »

Clinical Trial

A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects with Phenylketonuria
Read more »

Clinical Trial

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

Clinical Trial

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
Read more »

Clinical Trial

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
Read more »

Clinical Trial

Kuvan®‘s Effect on the Cognition of Children With Phenylketonuria (KOGNITO)
Read more »

Clinical Trial

Kuvan® in Phenylketonuria Patients Less Than 4 Years Old (SPARK)
Read more »

BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

Brineura
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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