Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01212744 | Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) | PHASE2 | COMPLETED | 16 | — | — | Mar 1, 2011 | Apr 1, 2015 | Feb 26, 2019 | 9 | United States |
| NCT00924703 | Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) | PHASE2 | COMPLETED | 68 | — | — | Jan 13, 2010 | Jan 31, 2019 | Oct 12, 2021 | 14 | United States |
| NCT00925054 | Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU | PHASE2 | COMPLETED | 40 | — | — | Sep 1, 2009 | Jun 1, 2015 | Feb 26, 2019 | 11 | United States |
| NCT00634660 | Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria | PHASE1 | COMPLETED | 25 | — | — | May 1, 2008 | Oct 1, 2009 | Feb 1, 2017 | 8 | United States |
Plasma Phe
Blood Phe Concentration (Change from Baseline) and Daily Dose in PAL-003 Subjects by Disposition (PAL-003 Population)
Specifically, safety was to be assessed by examining the incidence of all treatment-emergent adverse events reported during the study period and clinically significant changes in vital signs and clinical laboratory results, including development of rAvPAL-PEG antibodies.
| Arm | Type | Description |
|---|---|---|
| rAvPAL-PEG | EXPERIMENTAL | rAvPAL-PEG in varying doses |
| rAvPAL-PEG 0.001 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.001 mg/kg |
| rAvPAL-PEG 0.003 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.003 mg/kg |
| rAvPAL-PEG 0.01 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.01 mg/kg |
| rAvPAL-PEG 0.03 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.03 mg/kg |
| rAvPAL-PEG 0.1 mg/kg | EXPERIMENTAL | Subjects will start on rAvPAL-PEG 0.1 mg/kg |
| 0.001 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.001 mg/kg of rAvPAL-PEG. |
| 0.003 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.003 mg/kg of rAvPAL-PEG. |
| 0.01 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.01 mg/kg of rAvPAL-PEG. |
| 0.03 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.03 mg/kg of rAvPAL-PEG. |
| 0.1 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.1 mg/kg of rAvPAL-PEG. |
| 0.3 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 0.3 mg/kg of rAvPAL-PEG. |
| 1.0 mg/kg | ACTIVE_COMPARATOR | One subcutaneous injection of 1.0 mg/kg of rAvPAL-PEG. |
| Name | Type | Description |
|---|---|---|
| rAvPAL-PEG | DRUG | 0.06 mg/kg/day, 0.1 mg/kg/day, 0.2 mg/kg/day, 0.4 mg/kg/day, 0.6 mg/kg/day, 0.8 mg/kg/day |
| rAvPAL-PEG 0.001 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.001 mg/kg |
| rAvPAL-PEG 0.003 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.003 mg/kg |
| rAvPAL-PEG 0.01 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.01 mg/kg |
| rAvPAL-PEG 0.03 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.03 mg/kg |
| rAvPAL-PEG 0.1 mg/kg | DRUG | In Part 1, the planned starting dose levels is 0.1 mg/kg |
Inclusion Criteria: * Diagnosis of PKU with both of the following: current blood Phe concentration of ≥ 600 micromol/L at screening and average blood Phe concentration of ≥ 600 micromol/L over the past 3 years, using available data * Evidence that the subject is a non-responder to Kuvan® treatment ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| BioMarin Pharmaceutical Inc. | BMRN | 5 | PHASE3 | Pegvaliase, BMN 307 |
| PTC Therapeutics, Inc. | PTCT | 2 | PHASE3 | PTC923, Sepiapterin |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE1 | SAR444836 |
| Agios Pharmaceuticals, Inc. | AGIO | 1 | PHASE1 | AG-181 |
| Illumina, Inc. | ILMN | 1 | — | Undisclosed |
| Q32 Bio Inc | QTTB | 1 | — | HMI-102 |