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YOLT-203 · 1 trial · 2 indications
To evaluate safety and tolerability by assessing the incidence and severity of adverse events (AEs) and serious adverse events (SAEs) after single intravenous infusion of YOLT-203 in Chinese patients with primary hyperoxaluria type 1 (PH1).
| Arm | Type | Description |
|---|---|---|
| YOLT-203 Treatment Group | EXPERIMENTAL | Single intravenous infusion of YOLT-203 is administered to enrolled subjects, with a total planned enrollment of 3 to 9 patients. For pediatric subjects aged ≥6 years and \<18 years old, two weight-based dose cohorts are designed: 0.45 mg/kg (maximum capped dose of 35 mg) and 0.6 mg/kg (maximum capped dose of 45 mg). For adult subjects aged ≥18 years old, two fixed-dose cohorts are adopted at 35 mg and 45 mg respectively. |
| Name | Type | Description |
|---|---|---|
| YOLT-203 | GENETIC | YOLT-203 is an investigational gene editing therapy being evaluated for the treatment of Primary Hyperoxaluria Type 1 (PH1). It is administered via intravenous infusion over a period of 1 hour. |
Inclusion Criteria: Subjects must meet ALL of the following criteria to be eligible for participation in this study: Age ≥ 6 years at the time of signing the informed consent form. Documented confirmation of Primary Hyperoxaluria Type 1 (PH1) diagnosis via genetic analysis prior to enrollment. Me...
YOLT-203 is an investigational gene therapy being developed for Primary Hyperoxaluria Type 1 (PH1), a rare inherited metabolic disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The therapy is designed to address the underlying genetic cause of PH1.
YOLT-203 is being developed by cbdMD, Inc., a company publicly traded under the ticker symbol YCBD. The company is conducting a Phase 1 exploratory study of YOLT-203 in patients with Primary Hyperoxaluria Type 1.
YOLT-203 is in Phase 1 clinical development. It is an investigational gene therapy and has not yet received regulatory approval. The ongoing Phase 1 study is currently recruiting participants with Primary Hyperoxaluria Type 1.
YOLT-203 is being evaluated in a Phase 1 exploratory study with the identifier NCT07776626. This open-label, uncontrolled trial is recruiting approximately 9 patients with Primary Hyperoxaluria Type 1 in China. The study is currently active and recruiting participants aged 6 years and older.
Yes, YOLT-203 is a gene therapy being developed for Primary Hyperoxaluria Type 1. It is currently in Phase 1 clinical trials and is considered investigational. The therapy is being studied in a small, uncontrolled trial to assess its safety and potential efficacy.