Cystinosis Clinical Trial
Official title:
Cure Cystinosis International Registry
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.
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.
;
Time Perspective: Cross-Sectional
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT05545774 -
Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
|
||
Completed |
NCT01432561 -
Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagonâ„¢
|
N/A | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Completed |
NCT04125927 -
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
|
Phase 3 | |
Completed |
NCT00071903 -
The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study
|
N/A | |
Recruiting |
NCT05994534 -
PK and PD Study of NPI-001 and Cysteamine Bitartrate
|
Phase 1/Phase 2 | |
Completed |
NCT00004312 -
Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis
|
N/A | |
Completed |
NCT00004350 -
Evaluation of Fanconi Syndrome and Cystinosis
|
N/A | |
Enrolling by invitation |
NCT03655223 -
Early Check: Expanded Screening in Newborns
|
||
Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
|
||
Completed |
NCT02533076 -
The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients
|
Phase 0 | |
Enrolling by invitation |
NCT05146830 -
A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
|
||
Completed |
NCT00872729 -
Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
|
Phase 1/Phase 2 | |
Completed |
NCT00001736 -
New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis
|
Phase 1 | |
Recruiting |
NCT05959668 -
Development of Health-related Quality of Life Instrument for Patients With Cystinosis
|
||
Withdrawn |
NCT02124070 -
Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
|
Phase 1/Phase 2 | |
Completed |
NCT01614431 -
N Acetyl Cysteine for Cystinosis Patients
|
Phase 4 | |
Recruiting |
NCT05843851 -
Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria
|
N/A | |
Completed |
NCT01000961 -
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
|
Phase 3 | |
Active, not recruiting |
NCT03897361 -
Stem Cell Gene Therapy for Cystinosis
|
Phase 1/Phase 2 |