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Also known as rivunatpagene miziparvovec
UX701 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Stage 1: UX701 Dose Level 1 | EXPERIMENTAL | Participants receive a single, peripheral intravenous (IV) infusion of UX701 at dose level 1. |
| Stage 1: UX701 Dose Level 2 | EXPERIMENTAL | Participants receive a single, peripheral IV infusion of UX701 at dose level 2. |
| Stage 1: UX701 Dose Level 3 | EXPERIMENTAL | Participants receive a single, peripheral IV infusion of UX701 at dose level 3. |
| Stage 1: UX701 Dose Level 4 | EXPERIMENTAL | Participants receive a single, peripheral IV infusion of UX701 at dose level 4. |
| Stage 2: UX701 at Selected Dose | EXPERIMENTAL | Participants randomized to UX701 receive a single, peripheral IV infusion of UX701 at the selected dose. |
| Stage 2: Standard of Care (SOC) to UX701 | EXPERIMENTAL | Participants randomized to SOC will continue their baseline SOC medications for 52 weeks, followed by a single, peripheral IV infusion of UX701 at the selected dose. Following UX701 administration, participants will be evaluated for modification of their SOC medications. |
| Name | Type | Description |
|---|---|---|
| UX701 | GENETIC | Nonreplicating, recombinant gene transfer vector |
| Standard of Care (SOC) | DRUG | SOC treatment (i.e., copper chelators and/or zinc) administered according to standard regimens. |
Key Inclusion Criteria: * Confirmed diagnosis of Wilson disease based on genetic confirmation of heterozygous or homozygous biallelic ATP7B mutation. * Stable Wilson disease as evidenced by ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at least 2 months at screening...
UX701 is an investigational gene therapy being developed for the treatment of Wilson Disease, a rare inherited disorder that causes copper accumulation in the body. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
UX701 targets the ATP7B gene, which is responsible for encoding a copper-transporting protein that is defective in Wilson Disease. By delivering a functional copy of this gene, the therapy aims to address the underlying genetic cause of the condition.
UX701 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol RARE. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for Wilson Disease.
UX701 is currently in Phase 1 clinical development. It is an investigational therapy, meaning it has not yet received FDA approval and is still being studied in clinical trials to assess its safety and effectiveness in patients with Wilson Disease.
UX701 is being studied in a Phase 1/2/3 clinical trial with the identifier NCT04884815. This trial is active but not recruiting participants, with an estimated enrollment of 82 adults with Wilson Disease across the United States, Portugal, Spain, and the United Kingdom.