Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

NCT ID: NCT00872729

Last Updated: 2024-12-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-05-31
• Study Completion Date: 2009-10-31

Brief Summary

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

Detailed Description

This is a single-dose, open-labeled, non-randomized, two-period study of Cysteamine Bitartrate Delayed-release Capsules (RP103) and Cystagon® in up to 10 patients (male or female) with nephropathic cystinosis under fasting conditions. It will involve a 4 night check-in to a clinical research center.

Study with completed results acquired from Horizon in 2024

Conditions

Cystinosis

Keywords

cystinosis; cysteamine; inheritable disease; orphan disease; CTNS protein, human; nephropathic cystinosis

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cystagon®

Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg

Group Type: ACTIVE_COMPARATOR

Cystagon®

Intervention Type: DRUG

Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.

Duration of Treatment and Dose: Reference Period up to four doses Q6H.

RP103

Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg

Group Type: EXPERIMENTAL

RP103

Intervention Type: DRUG

Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.

Duration of treatment and Dose: Single dose of Test Product at dose equivalent to Reference Product.

Interventions

Cystagon®

Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules, 150 mg/50 mg.

Duration of Treatment and Dose: Reference Period up to four doses Q6H.

Intervention Type: DRUG

RP103

Test Product: RP103 (Cysteamine Bitartrate) Delayed-release Capsules, 75 mg.

Duration of treatment and Dose: Single dose of Test Product at dose equivalent to Reference Product.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male and female subjects must have nephropathic cystinosis.
• Children less than 22.5 kg will only be included in the study if the investigator feels they can safely participate in the study including the required blood draw volume for the safety and PK/PD assessments.
• Subjects must be on a stable dose of Cystagon® at least 21 days prior to Screening.
• Subjects must be able to swallow a 150 mg Cystagon® capsule with the capsule intact.
• Within the last 2 months, no clinically significant change in liver function [i.e., ALT, AST, alkaline phosphatase, bilirubin (total and direct)] and renal function [i.e., serum creatinine, albumin, total protein] at Screening as determined by the Investigator.
• 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 or their authorized caregiver must provide written informed consent and assent (where applicable) prior to participation in the study.
• If in the opinion of the investigator, patients can safely provide the study required blood draw volume.
• Subjects must be willing and able to comply with the study restrictions and requirements.

Exclusion Criteria

• If, in the opinion of the investigator, the planned study dose would exceed the patient's tolerability of cysteamine based on their prior Cystagon® steady state drug requirements.
• Evidence of or verbal attestation of Helicobacter pylori infection, presently, or within the last 90 days prior to Screening.
• Subjects with a known history, currently or within the past 90 days prior to Screening, of the following conditions or other health issues that make it, in the opinion of the investigator, unsafe for them to participate, or whose concomitant medical problems preclude them from committing to the study schedule including the following: Crohn's disease, inflammatory bowel disease (if currently active) or have had prior resection of small intestine; • History of heart disease, e.g., myocardial infarction, heart failure, arrhythmias; Any bleeding disorder; Malignant disease; Severe liver disease as defined as ALT or AST > 2 times the upper limit of normal.
• Subjects who have had a kidney transplant.
• Subjects who are planning or are a registered candidate for a kidney transplant within 3 months of the Screening or have a serum creatinine > 2.4.
• Subjects with known hypersensitivity to cysteamine.
• If female (of child-bearing potential), are nursing, planning a pregnancy, known or suspected to be pregnant, or have a positive urine pregnancy screen.
• Patients with a hemoglobin level < 10.5.
• Subjects who have a made a blood donation within 60 days prior to study initiation.
• Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amgen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

MD

Role: STUDY_DIRECTOR

Amgen

Locations

University of California San Diego Medical Center

San Diego, California, United States

Countries

United States

References

Dohil R, Fidler M, Barshop BA, Gangoiti J, Deutsch R, Martin M, Schneider JA. Understanding intestinal cysteamine bitartrate absorption. J Pediatr. 2006 Jun;148(6):764-9. doi: 10.1016/j.jpeds.2006.01.050.

Reference Type: BACKGROUND

PMID: 16769383 (View on PubMed)

Fidler MC, Barshop BA, Gangoiti JA, Deutsch R, Martin M, Schneider JA, Dohil R. Pharmacokinetics of cysteamine bitartrate following gastrointestinal infusion. Br J Clin Pharmacol. 2007 Jan;63(1):36-40. doi: 10.1111/j.1365-2125.2006.02734.x.

Reference Type: BACKGROUND

PMID: 17229040 (View on PubMed)

Levtchenko EN, van Dael CM, de Graaf-Hess AC, Wilmer MJ, van den Heuvel LP, Monnens LA, Blom HJ. Strict cysteamine dose regimen is required to prevent nocturnal cystine accumulation in cystinosis. Pediatr Nephrol. 2006 Jan;21(1):110-3. doi: 10.1007/s00467-005-2052-0. Epub 2005 Oct 27.

Reference Type: BACKGROUND

PMID: 16252107 (View on PubMed)

Related Links

http://www.procysbi.com

RP103 (marketed as PROCYSBI) is now approved by the US FDA for management of nephropathic cystinosis in patients 6 years and older

http://biochemgen.ucsd.edu/Cystinosis/Index.htm

Click here for more information about UCSD's cystinosis program

Other Identifiers

RP103-01

Identifier Type: -

Identifier Source: org_study_id