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 Treatment 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 approach Dr. Freedman or Nancy Stack email@example.com for more information. Dr. Freedman can also be contacted at firstname.lastname@example.org with any inquiries.
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). Use this link for the recruitment letter in French.
Those interested and 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 6 weeks in Montreal and will have to discontinue cysteamine during the trial.
Cystinosis Recruitment Oct 25, 2017
Dr. Doris Trauner and her research group at the UCSD School of Medicine are conducting a research study to evaluate the relationship between sleep, memory, and thinking in adults with cystinosis. We are looking for adults with cystinosis to participate in an overnight sleep study and cognitive testing focusing on memory.
Volunteers Needed for a Research Study of Bone Health and Muscle Strength
Children and adults with cystinosis are at risk for musculoskeletal disease. This research study, performed by Dr. Mary Leonard and Dr. Paul Grimm, will be the first comprehensive evaluation of bone structure and muscle strength in children and adults with cystinosis.
Research Study of Bone Marrow Transplant for Cystinosis Patients from Matched Related Donor – The clinical research study is being conducted at the Ronald Reagan UCLA Medical Center.