A Long-Term Open-Label, Safety and Superior Effectiveness Study ofCysteamine Bitartrate Delayed-release Capsules (RP103) in Patients withCystinosis

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 60

Inclusion Criteria

Male or female with a documented diagnosis of cystinosis.
2. On a stable dose of Cystagon® for at least 21 days prior to
Screening.
3. WBC cystine level > 1 nmol ½ cystine/mg of protein on
average over at least 2 measurements collected during the 2
years prior to Screening.
4. No clinically significant change in liver function tests, i.e. 1.5
times ULN for ALT and AST, and/or 1.5 times ULN for total
bilirubin, within 6 months prior to Screening.
5. No clinically significant change in renal function, i.e.
estimated GFR within 6 months prior to Screening.
6. Must have an estimated GFR > 20 mL/minute/1.73 m2 (using
the equation from Schwartz 2009 J Am Soc Nephrol 20:629-
637) 1.
7. Female subjects who are sexually active and of childbearing
potential, i.e. not surgically sterile (tubal ligation, bilateral
oopherectomy, or hysterectomy) or at least 2 years naturally
postmenopausal must agree to use an acceptable form of
contraception from Screening through completion of the
study. Acceptable forms of contraception for this study
include hormonal contraceptives (oral, implant, transdermal
patch, or injection) at a stable dose for at least 3 months prior
to Screening, barrier (spermicidal condom or diaphragm with
spermicide), IUD, or a partner who has been vasectomized
for at least 6 months.
8. Subject or their parent or guardian must provide written
informed consent and assent (where applicable) prior to
participation in the study.

Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Exclusion Criteria:
1. Younger than 12 years of age.
2. Current history of the following conditions or any other
health issues that make it, in the opinion of the Investigator,
unsafe for study participation:
- Inflammatory bowel disease if currently active, or
prior resection of small intestine;
- Heart disease (e.g. myocardial infarction, heart
failure, unstable arrhythmias, or poorly controlled
hypertension) within 90 days prior to Screening;
-Active bleeding disorder within 90 days prior to
Screening;
- History of malignant disease within 2 years prior to
Screening.
3. Hemoglobin level of < 9 g/dL at Screening or, in the opinion
of the Investigator, a hemoglobin level that would make it
unsafe for study participation.
4. Known hypersensitivity to cysteamine and penicillamine.
5. Female subjects who are nursing, planning a pregnancy, or
are known or suspected to be pregnant.
6. Subjects who, in the opinion of the Investigator, are not able
or willing to comply with study requirements.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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