CystinosisCCIRNCT01327807
Cure Cystinosis International Registry
An observational study of Cystinosis and Nephropathic Cystinosis, run by Cystinosis Research Foundation at 1 site in United States.
Trial summary
Cure Cystinosis International Registry (CCIR) is an online, patient self-identifying registry developed by medical and scientific experts specifically for the cystinosis community. CCIR's sole purpose is to identify people with cystinosis worldwide in an effort to accelerate novel treatments and a cure for cystinosis. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
- How many people
- 750 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2010to Dec 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: International cystinosis community
Who may be able to join
- Diagnosis of cystinosis
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
University of California, San Diego
San Diego, California, 92103-8450
Betty L Cabrera, M.P.H., 619-471-9554, curator@cystinosisregistry.org
Jerry A Schneider, M.D.
About this study
Cure Cystinosis International Registry (CCIR) is an online, patient self-identifying registry developed by medical and scientific experts specifically for the cystinosis community.
CCIR's sole purpose is to identify people with cystinosis worldwide in an effort to accelerate novel treatments and a cure for cystinosis.
Read more
CCIR provides a safe and secure platform for:
- sharing anonymous medical information about cystinosis with researchers, clinicians and patients
- disseminating information about research opportunities
- connecting researchers/investigators and prospective participants *
Interested cystinosis patients may register themselves with CCIR online at http://www.cystinosisregistry.org.
- No personal information is shared outside of CCIR. Individual identities are known only to appropriate CCIR staff. If a participant is matched to a clinical trial, the participant receives a notice from CCIR, after which they can decide whether they wish to contact the study sponsor.
Read the detailed description
Significance and Purpose:
Many different resources and tools are necessary to make significant advances in medical research. Progress in rare diseases such as cystinosis can often be impeded by the lack of information available about the disease and limited access to volunteers eligible for clinical trials. Therefore, patients who are willing to provide information about how the disease has affected them and also make themselves available to participate in trials are among the most valuable resources the investigators have to fight a disease. However, the research community desperately needs the right tool that will permit access to these resources.
A tool widely used to conveniently collect both data about a disease and information about potential clinical trial participants is a patient registry. A patient registry is any system that allows for the organized collection of data about disease outcomes in affected populations for a scientific, clinical, or policy purpose. The Cystinosis Research Foundation (CRF) has aligned itself with cystinosis medical experts and organizations worldwide to create the first ever international, online patient registry for cystinosis, Cure Cystinosis International Registry (CCIR). The express purpose of CCIR is to make anonymous information available to the research community and thus promote accelerated research in advanced treatments and ultimately a cure for cystinosis.
Objectives:
The objectives of CCIR are:
- Evaluate epidemiology and clinical characteristics of cystinosis around the world.
- Evaluate and compare the diagnosis, treatment, and kidney transplant rates among cystinosis communities from different geographical areas.
- Enhance the understanding of how cystinosis affects quality of life.
CCIR Registration:
Interested cystinosis patients may register themselves with CCIR online at http://www.cystinosisregistry.org. Registration is easy and secure. Simply go to the website and create a CCIR account and complete a survey. The CCIR website is currently available in English and Spanish, and will soon be available in French, Portuguese, and possibly other languages.
Benefits to CCIR participants include instant access to the registry's accumulated survey results (reported as anonymous group data), and opportunities to submit questions to cystinosis experts and to learn of clinical trial opportunities. No personal information is shared outside of CCIR. Individual identities are known only to appropriate CCIR staff.
- Conditions
- Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
- Keywords
- Registries, Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome, Cystine, Kidney, Rare Diseases
- Medical subject headings
- Cystinosis, Fanconi Syndrome, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cystinsosis patients | Those with a diagnosis of cystinosis. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Age at treatment initiation and the effect on disease outcomes
Measured: Lifetime
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2011 (estimated)
- Study startAug 2010
- Primary completionDec 2020 (estimated)
- Study completionDec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for CystinosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Time perspective
- Cross-sectional
- Enrolment
- 750 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cystinosis Research Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jerry A. Schneider, Cystinosis Research Foundation
- Investigators
- Jerry A Schneider, M.D., Principal investigator, University of California, San Diego
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01327807
- Sponsor's protocol ID
- CCIR100913
- Acronym
- CCIR
- Status last verified
- Mar 2014
- First submitted
- 29 Mar 2011
- Last updated
- 12 Mar 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Cherqui S. Cysteamine therapy: a treatment for cystinosis, not a cure. Kidney Int. 2012 Jan;81(2):127-9. doi: 10.1038/ki.2011.301.
PubMed 22205430Gangoiti JA, Fidler M, Cabrera BL, Schneider JA, Barshop BA, Dohil R. Pharmacokinetics of enteric-coated cysteamine bitartrate in healthy adults: a pilot study. Br J Clin Pharmacol. 2010 Sep;70(3):376-82. doi: 10.1111/j.1365-2125.2010.03721.x.
PubMed 20716238
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cure Cystinosis International Registry
- ClinicalTrials.gov ID
- NCT01327807
- Acronym / sponsor ID
- CCIR / CCIR100913
- Conditions
- Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome
- Keywords
- Registries, Cystinosis, Nephropathic Cystinosis, Renal Fanconi Syndrome, Cystine, Kidney, Rare Diseases
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 750 participants (estimated)
- Lead sponsor
- Cystinosis Research FoundationUniversity, hospital or other
- Study start
- Aug 2010
- Primary completion
- Dec 2020 (estimated)
- Study completion
- Dec 2022 (estimated)
- First posted
- 4 Apr 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Cure Cystinosis International Registry (CCIR) is an online, patient self-identifying registry developed by medical and scientific experts specifically for the cystinosis community.
CCIR's sole purpose is to identify people with cystinosis worldwide in an effort to accelerate novel treatments and a cure for cystinosis.
CCIR provides a safe and secure platform for:
- sharing anonymous medical information about cystinosis with researchers, clinicians and patients
- disseminating information about research opportunities
- connecting researchers/investigators and prospective participants *
Interested cystinosis patients may register themselves with CCIR online at http://www.cystinosisregistry.org.
- No personal information is shared outside of CCIR. Individual identities are known only to appropriate CCIR staff. If a participant is matched to a clinical trial, the participant receives a notice from CCIR, after which they can decide whether they wish to contact the study sponsor.
Detailed description
Significance and Purpose:
Many different resources and tools are necessary to make significant advances in medical research. Progress in rare diseases such as cystinosis can often be impeded by the lack of information available about the disease and limited access to volunteers eligible for clinical trials. Therefore, patients who are willing to provide information about how the disease has affected them and also make themselves available to participate in trials are among the most valuable resources the investigators have to fight a disease. However, the research community desperately needs the right tool that will permit access to these resources.
A tool widely used to conveniently collect both data about a disease and information about potential clinical trial participants is a patient registry. A patient registry is any system that allows for the organized collection of data about disease outcomes in affected populations for a scientific, clinical, or policy purpose. The Cystinosis Research Foundation (CRF) has aligned itself with cystinosis medical experts and organizations worldwide to create the first ever international, online patient registry for cystinosis, Cure Cystinosis International Registry (CCIR). The express purpose of CCIR is to make anonymous information available to the research community and thus promote accelerated research in advanced treatments and ultimately a cure for cystinosis.
Objectives:
The objectives of CCIR are:
- Evaluate epidemiology and clinical characteristics of cystinosis around the world.
- Evaluate and compare the diagnosis, treatment, and kidney transplant rates among cystinosis communities from different geographical areas.
- Enhance the understanding of how cystinosis affects quality of life.
CCIR Registration:
Interested cystinosis patients may register themselves with CCIR online at http://www.cystinosisregistry.org. Registration is easy and secure. Simply go to the website and create a CCIR account and complete a survey. The CCIR website is currently available in English and Spanish, and will soon be available in French, Portuguese, and possibly other languages.
Benefits to CCIR participants include instant access to the registry's accumulated survey results (reported as anonymous group data), and opportunities to submit questions to cystinosis experts and to learn of clinical trial opportunities. No personal information is shared outside of CCIR. Individual identities are known only to appropriate CCIR staff.
Study design
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cystinsosis patients | – | –Those with a diagnosis of cystinosis. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- International cystinosis community
Key inclusion criteria (1 listed)
- Diagnosis of cystinosis
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Age at treatment initiation and the effect on disease outcomes
Time frame: Lifetime
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2014.
- Results publication: PubMed 22205430Cherqui S. Cysteamine therapy: a treatment for cystinosis, not a cure. Kidney Int. 2012 Jan;81(2):127-9. doi: 10.1038/ki.2011.301.
- Results publication: PubMed 20716238Gangoiti JA, Fidler M, Cabrera BL, Schneider JA, Barshop BA, Dohil R. Pharmacokinetics of enteric-coated cysteamine bitartrate in healthy adults: a pilot study. Br J Clin Pharmacol. 2010 Sep;70(3):376-82. doi: 10.1111/j.1365-2125.2010.03721.x.
- CCIR Patient PortalLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jerry A Schneider, M.D. principal investigator
University of California, San Diego
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystinosis and Nephropathic Cystinosis, run by Cystinosis Research Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Mar 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Betty L Cabrera, M.P.H (858-822-3747, curator@cystinosisregistry.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Age at treatment initiation and the effect on disease outcomes (time frame: Lifetime). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01327807, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2014; AzTrials last refreshed its copy on 6 Nov 2026.