Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Condition: Cystinosis
MeSH terms: Cystinosis
Intervention: Cysteamine Bitartrate Delayed-release Capsules
Intervention description: Participants who entered the trial from the RP103-03 study continued treatment with cysteamine bitartrate every 12 hours at the last dose level prescribed during their participation in that study.
Participants not entering the trial from Study RP103-03 were started on twice a day administration of cysteamine bitartrate at a total daily RP103 dose of 70% of their pre-study total daily stable Cystagon® dose.
Intervention MeSH terms: Cysteamine
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 60.0
Sex: All
Minimum age: 1 Year
Healthy volunteers: No
Keywords: cystinosis; cysteamine; inheritable disease; orphan disease; CTNS protein, human; metabolic disease; nephropathic cystinosis

Summary:

      Cystinosis is an inherited disease that if untreated, results in kidney failure as early as
      the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate
      immediate release) which must be taken every six hours for the rest of the patient's life to
      prevent complications of cystinosis. Cysteamine bitartrate delayed-release capsules (RP103)
      is a formulation of cysteamine bitartrate that is being studied to see if it can be given
      less frequently, once every 12 hours, and have similar results to four times a day Cystagon®.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male and female subjects must have completed the last visit of Study RP103-03 and be
             willing to continue with RP103 treatment.

        OR for patients who did not complete the RP103-03 study:

          -  Male and female subjects must have cystinosis.

          -  Subjects must be on a stable dose of Cystagon® at least 21 days prior to Screening.

          -  Within the last 6 months, no clinically significant change from normal in liver
             function tests (i.e., alanine aminotransferase [ALT], aspartate aminotransferase
             [AST], total bilirubin) and renal function (i.e., estimated glomerular filtration rate
             [eGFR]) at Screening as determined by the Investigator.

          -  Subjects with an eGFR corrected for body surface area > 30 mL/min/1.73m².

          -  Sexually active female subjects of childbearing potential (i.e., not surgically
             sterile [tubal ligation, hysterectomy, or bilateral oophorectomy] or at least 2 years
             naturally postmenopausal) must agree to utilize the same acceptable form of
             contraception from Screening through completion of the study.

          -  Subjects must be willing and able to comply with the study restrictions and
             requirements.

          -  Subjects or their parent or guardian must provide written informed consent and assent
             (where applicable) prior to participation in the study.

        Exclusion Criteria:

          -  Patients enrolled in the previous Study RP103-03 who did not complete their last
             scheduled Study visit or who do not wish to continue on treatment with RP103.

        AND for patients who did not complete the RP103-03 study:

          -  Subjects less than 1 year old

          -  Subjects with a known history, currently of the following conditions or other health
             issues that make it, in the opinion of the investigator, unsafe for them to
             participate: inflammatory bowel disease (if currently active) or have had prior
             resection of small intestine; Heart disease (e.g., myocardial infarction, heart
             failure, unstable arrhythmias or poorly controlled hypertension) 90 days prior to
             Screening; Active bleeding disorder 90 days prior to Screening; Malignant disease
             within the last 2 years.

          -  Patients with a hemoglobin level < 10 g/dL at Screening or a level that, in the
             opinion of the investigator, makes it unsafe for the subject to participate.

          -  Subjects with known hypersensitivity to cysteamine or penicillamine.

          -  Female subjects who are nursing, planning a pregnancy, known or suspected to be
             pregnant, or have a positive serum pregnancy screen.

          -  Subjects who, in the opinion of the Investigator, are not able or willing to comply
             with the protocol.