A Randomized, Crossover, Pharmacokinetic and PharmacodynamicStudy to Determine the Safety and Efficacy of Cysteamine BitartrateDelayed-release Capsules (RP103), Compared to Cystagon® in Patientswith Nephropathic Cystinosis

• Conditions: Cystinosis; MedDRA version: 12.1Level: LLTClassification code 10011777Term: Cystinosis

• Registration Number: EUCTR2009-017882-42-NL
• Lead Sponsor: Raptor Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06-25
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1.Male or female subject with a documented diagnosis of
nephropathic cystinosis.
2. Subject must be on a stable dose of Cystagon® sufficient to
maintain their white blood cell (WBC) cystine level at = 1.0
nmol/half-cystine/mg protein.
3. Subject must be able to swallow their typically administered
Cystagon® capsule with the capsule intact.
4. Within the last 6 months, no clinically significant change from
normal in liver function tests [i.e., 1.5 times ULN for ALT and
AST, and/or 1.5 times ULN for total bilirubin] and renal function
[i.e., estimated GFR (corrected for body surface area)] at
Screening as determined by the Investigator.
5. Subject must have an estimated GFR (corrected for body surface
area) > 30 mL/minute/1.73 m2.
6. 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. The 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, diaphragm with spermicide), IUD, or a
partner who has been vasectomized for at least 6 months. For
pre-pubescent children, a documented attestation of abstinence
from their parent or guardian will be acceptable.
7. Subject must be willing and able to comply with the study
restrictions and requirements.
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:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Subject’s age < 6 years old or subjects weight < 21 kg.
2. Subjects with current history of the following conditions or any
other health issues that make it, in the opinion of the Investigator,
unsafe for them to participate:
- 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)
90 days prior to Screening;
- Active bleeding disorder 90 days prior to Screening;
- History of malignant disease within the last 2 years.
3. Subject with a hemoglobin level of < 10 g/dL at Screening or, in
the opinion of the Investigator, a hemoglobin level that would
make it unsafe for the subject to participate.
4. Subjects receiving any form of cysteamine medication through a
gastric tube.
5. Subjects who are receiving maintenance dialysis or who have
had a kidney transplant.
6. Subjects who are on an active kidney transplant list or who are
planning to receive a kidney transplant within 3 months of
Screening.
7. Subjects with known hypersensitivity to cysteamine and
penicillamine.
8. Female subjects who are nursing, planning a pregnancy, known
or suspected to be pregnant, or with a positive serum pregnancy
screen.
9. Subjects who have a made a blood donation within 30 days of
Screening.
10. Subjects who, in the opinion of the Investigator, are not able or
willing to comply with the protocol.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To demonstrate that at steady-state, patients receiving every 6 hour<br>(Q6H) treatment of Cystagon® can maintain a comparable depletion<br>of white blood cell (WBC) cystine levels after receiving every 12<br>hour (Q12H) treatment regimen of RP103;Secondary Objective: To assess safety and tolerability of RP103.<br>To assess the steady-state pharmacokinetics (PK) and<br>pharmacodynamics (PD) of RP103 compared to Cystagon®.;Primary end point(s): Comparable depletion of steady-state cysteamine-trough WBC<br>cystine levels (Days 5, 6 and 7 of Week 6 (Period 1) and Week 9<br>(Period 2) between Cystagon® and RP103. If the one-sided test<br>of non-inferiority, conducted at the nominal level of 0.02104, is<br>rejected at a non-inferiority margin of 0.3, we will conclude that<br>RP103 is non-inferior to Cystagon® with an overall significance<br>level of 0.025.