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Also known as Recombinant collagen 7 (rC7), Recombinant collagen 7, rC7, Recombinant human collagen 7 (rC7)
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 by BridgeBio Pharma, Inc. for the treatment of recessive dystrophic epidermolysis bullosa, a rare genetic skin disorder. It is currently in Phase 2 clinical development and has been studied in three completed clinical trials.
PTR-01 is being developed to treat recessive dystrophic epidermolysis bullosa, a rare inherited condition that causes fragile skin and chronic blistering. All three completed clinical trials of PTR-01 enrolled patients with this condition, including adults and adolescents aged 12 years and older.
PTR-01 is being developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. BridgeBio is the sponsor of the clinical program evaluating PTR-01 in recessive dystrophic epidermolysis bullosa.
PTR-01 is in Phase 2 clinical development. It is an investigational therapy and has not been approved by the FDA. Three trials have been completed, including a Phase 1/2 trial and two Phase 2 studies, and no trials are currently active.
PTR-01 has been evaluated in three completed trials: NCT03752905, a Phase 1/2 trial in adults with recessive dystrophic epidermolysis bullosa; NCT04599881, a Phase 2 study; and NCT05143190, a Phase 2 extension study for patients previously treated with PTR-01. All were conducted in the United States.
Across the three completed PTR-01 trials, a total of 20 patients were enrolled. NCT03752905 enrolled 12 patients, NCT04599881 enrolled 6, and the extension study NCT05143190 enrolled 2. The trials were randomized, double-blind, and placebo-controlled, and enrolled patients aged 12 years and older.