Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PTR-01 · 3 trials · 1 indication
Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)
Safety and tolerability, as assessed by treatment-emergent adverse events
Change in a majority of target lesions of at least 2 levels using a 7-point (1-7) Global Impression of Change instrument (7 being the worst)
Safety and tolerability, as assessed by infusion-associated reactions (IAR)
Safety and tolerability, as assessed by immunogenicity through anti-drug antibody (ADA) testing
| Arm | Type | Description |
|---|---|---|
| PTR-01 | EXPERIMENTAL | All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg once monthly for a total of 5 doses. |
| PTR-01 3 mg/kg | EXPERIMENTAL | All patients will receive a PTR-01 dose of 3.0 mg/kg once weekly every week for a total of 4 doses, followed by a dose of 3.0 mg/kg every other week for a total of 7 doses. |
| PTR-01 0.1 mg/kg | EXPERIMENTAL | Three intravenous infusions of PTR-01 at 0.1 mg/kg with doses 2 weeks apart. |
| PTR-01 0.3 mg/kg | EXPERIMENTAL | Three intravenous infusions of PTR-01 at 0.3 mg/kg with doses 2 weeks apart. |
| PTR-01 1.0 mg/kg | EXPERIMENTAL | Three intravenous infusions of PTR-01 at 1.0 mg/kg with doses 2 weeks apart. |
| Normal Saline | PLACEBO_COMPARATOR | Saline control to mimic PTR-01. |
| PTR-01 3.0 mg/kg | EXPERIMENTAL | Three intravenous infusions of PTR-01 at 3.0 mg/kg with doses 2 weeks apart. |
| Name | Type | Description |
|---|---|---|
| PTR-01 | DRUG | Intravenous recombinant collagen 7 |
| Normal saline | DRUG | Saline control |
Inclusion Criteria: 1. Willing to provide informed consent form, or if 12 to \<18 years of age, legal guardian has provided informed consent form and the minor has signed an assent form acknowledging that they understand and agree to study procedures. 2. Has satisfactorily completed participation i...
PTR-01 is an investigational small molecule being developed for recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder. It is currently in Phase 2 clinical development and has not been approved by the FDA. The drug is being studied in patients aged 12 years and older.
PTR-01 is being developed by BridgeBio Pharma, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol BBIO. The company is conducting clinical trials of PTR-01 for the treatment of recessive dystrophic epidermolysis bullosa.
PTR-01 is currently in Phase 2 clinical development for recessive dystrophic epidermolysis bullosa. It is an investigational drug and has not received FDA approval. The drug has completed three clinical trials, including a Phase 1/2 study and two Phase 2 studies.
PTR-01 has completed three clinical trials: NCT03752905, a Phase 1/2 study in adult patients with recessive dystrophic epidermolysis bullosa (RDEB); NCT04599881, a Phase 2 study; and NCT05143190, a Phase 2 extension study for patients previously treated with PTR-01. All trials were conducted in the United States.
PTR-01 is the primary name used in clinical trials for this investigational treatment. No alternative names have been reported in the clinical trial data for this drug. It is being studied specifically for recessive dystrophic epidermolysis bullosa.