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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06302348 | A Study of Sepiapterin in Participants With Phenylketonuria (PKU) | PHASE3 | RECRUITING | 56 | — | — | Mar 4, 2024 | Feb 28, 2031 | May 6, 2026 | 8 | United States, Australia +4 |
Analysis for participants ≥30 months to \<6 years of age.
Analysis for participants ≥6 years to 16 years of age.
| Arm | Type | Description |
|---|---|---|
| Sepiapterin | EXPERIMENTAL | Participants will receive age- and weight-adjusted doses of sepiapterin orally once daily for up to 6 years. |
| Name | Type | Description |
|---|---|---|
| Sepiapterin | DRUG | Sepiapterin powder for oral use will be mixed in water or apple juice prior to administration. |
Key Inclusion Criteria: For all participants: * Women of childbearing potential must have a negative pregnancy test at Screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for at least 90 days after the last dose of ...
| 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 |