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Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

NCT ID: NCT01197378

Last Updated: 2024-12-27

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-08-27

Study Completion Date

2017-06-26

Brief Summary

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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®.

Detailed Description

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This is a long-term, open-label, study to determine the safety and tolerability of twice a day treatment with cysteamine bitartrate delayed-release capsules (RP103). It will involve 6-9 monthly clinic visits followed by quarterly clinic visits for the duration of the study and home use of cysteamine bitartrate delayed-release capsules.

Initially, enrollment was open to those patients who had completed the previous Phase 3 Study (RP103-03, NCT01000961). Subsequently enrollment in Study RP103-04 was opened to additional participants, including children aged 1 to 6 years and renal transplant recipients, who had previously been on a stable dose of Cystagon® for at least 21 days.

Study with completed results acquired from Horizon in 2024.

Conditions

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Cystinosis

Keywords

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cysteamine Bitartrate

Cysteamine bitartrate delayed-release capsules were administered twice daily for up to 96 months.

Group Type EXPERIMENTAL

Cysteamine Bitartrate Delayed-release Capsules

Intervention Type DRUG

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.

Interventions

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Cysteamine Bitartrate Delayed-release Capsules

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 Type DRUG

Other Intervention Names

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RP103 PROCYSBI®

Eligibility Criteria

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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.
Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amgen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MD

Role: STUDY_DIRECTOR

Amgen

Locations

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California Pacific Medical Center (CPMC) Research Institute

San Francisco, California, United States

Site Status

Stanford University Medical School

Stanford, California, United States

Site Status

Emory Children's Center

Atlanta, Georgia, United States

Site Status

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Site Status

Texas Children's Hospital/Baylor University

Houston, Texas, United States

Site Status

Hospices Civils de Lyon

Lyon, , France

Site Status

Hôpital Arnaud Villeneuve - CHU Montpellier

Montpellier, , France

Site Status

Hopital Necker

Paris, , France

Site Status

Robert Debre Hospital

Paris, , France

Site Status

Radboud University Nijmegen Medical Center

Nijmegen, , Netherlands

Site Status

Countries

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United States France Netherlands

References

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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)

Langman CB, Greenbaum LA, Grimm P, Sarwal M, Niaudet P, Deschenes G, Cornelissen EA, Morin D, Cochat P, Elenberg E, Hanna C, Gaillard S, Bagger MJ, Rioux P. Quality of life is improved and kidney function preserved in patients with nephropathic cystinosis treated for 2 years with delayed-release cysteamine bitartrate. J Pediatr. 2014 Sep;165(3):528-33.e1. doi: 10.1016/j.jpeds.2014.05.013. Epub 2014 Jun 16.

Reference Type DERIVED
PMID: 24948347 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

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http://procysbi.com/

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

Other Identifiers

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2010-018365-34

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

RP103-04

Identifier Type: -

Identifier Source: org_study_id