Clinical Studies Volunteers
The Cystinosis Research Foundation is dedicated to funding research that will lead to improved treatments and a cure for cystinosis. The following studies are being funded by the CRF to advance knowledge in key areas of cystinosis treatment. Click the following links to see the full information for each study.
CRF Announces Food & Drug Administration (FDA) Approval for Human Clinical Trials for Stem Cell and Gene Therapy for Cystinosis.
Individuals must be 18 years or older and meet specific eligibility requirements to participate. Click the link above if you are interested in participating. The subsequent phases of the clinical trials may include children, however, timing and approvals of this have yet to be determined. The trials are available to individuals worldwide, though priority will be given to U.S. patients.
Therapeutic Strategy for Cystinotic Nephropathy with iPS Cells – Study Conducted by Benjamin “Beno” Freedman, PhD at the University of Washington.
The goal of our CRF project is to use human mini-kidneys as surrogates for patients, to explore the potential of kidney regeneration, gene therapy, and drug discovery for cystinosis. We are seeking to recruit patients with cystinosis, and turn their urine into stem cells and kidney grafts, which we will test for transplantability. There is no obligation. Study participation is on a purely volunteer basis and is free of charge without compensation. If you are interested in joining the study or have questions about it, please contact Dr. Freedman at firstname.lastname@example.org with any inquiries.
ELOXX Phase 2 Clinical Trial in Montreal, Canada-Summer 2019
We are excited to let you know that there is another clinical trial open for volunteers. CRF has funded Dr. Paul Goodyer for many years and his work has resulted in this new clinical trial that will take place in Montreal, Canada. They are recruiting patients with nonsense mutations who are over 18 years of age with stable kidney function (eGRF>40 mL/min/1.73m2). ELOXX Recruitment letter.
Those interested and those who would like more information should contact Murielle Akpa, PhD, by phone: (514) 412-4400 ext. 22953 or by email: email@example.com . It should be emphasized that subjects will spend six weeks in Montreal and will have to discontinue cysteamine during the trial.
The Effect of Resistance Exercise on Muscle Dysfunction in Cystinosis
A research study being conducted by Richard Reimer, MD, Associate Professor of Neurology, Stanford University School of Medicine, Palo Alto, California, email firstname.lastname@example.org