MedTrace Logo

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

NCT00925054 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2019-02-26

Study results available
· View outcomes & findings →

Summary

The purpose of this study is to evaluate whether weekly injections of phenylalanine ammonia lyase (rAvPAL-PEG) can reduce blood phenylalanine concentrations in PKU subjects and whether repeated administration is safe.

Conditions

Interventions

DRUG

rAvPAL-PEG 0.001 mg/kg

In Part 1, the planned starting dose levels is 0.001 mg/kg

DRUG

rAvPAL-PEG 0.003 mg/kg

In Part 1, the planned starting dose levels is 0.003 mg/kg

DRUG

rAvPAL-PEG 0.01 mg/kg

In Part 1, the planned starting dose levels is 0.01 mg/kg

DRUG

rAvPAL-PEG 0.03 mg/kg

In Part 1, the planned starting dose levels is 0.03 mg/kg

DRUG

rAvPAL-PEG 0.1 mg/kg

In Part 1, the planned starting dose levels is 0.1 mg/kg

Sponsors & Collaborators

Principal Investigators

  • Ari Gershman, MD · BioMarin Pharmaceutical

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
16 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-09-30
Primary Completion
2015-06-30
Completion
2015-06-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00925054 on ClinicalTrials.gov