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Cystinosis clinical trials at UCSD

2 research studies open to eligible people

Showing trials for
  • Stem Cell Gene Therapy for Cystinosis

    open to eligible people ages 18 years and up

    This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)

    La Jolla, California

  • Cure Cystinosis International Registry

    open to all eligible people

    Cure Cystinosis International Registry (CCIR) is an online, patient self-identifying registry developed by medical and scientific experts specifically for the cystinosis community. CCIR's sole purpose is to identify people with cystinosis worldwide in an effort to accelerate novel treatments and a cure for cystinosis. CCIR provides a safe and secure platform for: - sharing anonymous medical information about cystinosis with researchers, clinicians and patients - disseminating information about research opportunities - connecting researchers/investigators and prospective participants * Interested cystinosis patients may register themselves with CCIR online at * No personal information is shared outside of CCIR. Individual identities are known only to appropriate CCIR staff. If a participant is matched to a clinical trial, the participant receives a notice from CCIR, after which they can decide whether they wish to contact the study sponsor.

    San Diego, California

Our lead scientists for Cystinosis research studies include .

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