NCT01744782

Who might be able to join this trial:

• You have been officially diagnosed with cystinosis.
• Your liver function test results have been stable (not significantly raised) in the 6 months before the screening appointment.
• Your kidney function levels have been stable in the 6 months before the screening appointment.
• Your kidneys are working at above a certain minimum level (a specific kidney filtration rate measurement must be above 20 mL/minute/1.73m²— confirm with trial site).
• If you are female, sexually active, and able to become pregnant, you must agree to use an approved form of birth control throughout the study (such as hormonal contraceptives, condoms with spermicide, an IUD, or a vasectomized partner).
• You or your parent or guardian must be willing and able to sign a consent form before joining the study.
• You have never previously taken any form of the medication cysteamine bitartrate.

Who may not be able to join:

• You have active inflammatory bowel disease, or have had part of your small intestine surgically removed.
• You have had a serious heart condition (such as a heart attack, heart failure, irregular heartbeat, or poorly controlled high blood pressure) within the past 90 days.
• You have had an active bleeding disorder within the past 90 days.
• You have had a cancer diagnosis within the past 2 years.
• Your blood count (haemoglobin level) is below a certain level, or is low enough that the doctor feels it would be unsafe for you to participate.
• You have a known allergy or sensitivity to a medication called penicillamine.
• You are female and are currently breastfeeding, pregnant, or planning to become pregnant.
• The trial doctor believes you are unwilling or unable to follow the study requirements.
• You have had a kidney transplant or are currently on dialysis.
• You are 6 years of age or older at the time of your screening appointment.

Important: Always verify eligibility with the trial site directly before applying.