Cystinosis Clinical Trial
Official title:
Clinical and Biological Study of Sub-pigmentation During Infantile Cystinosis
The purpose of this study is to determine the molecular mechanism of this disease and to research the relationship between cystinosis and skin phenotype.
n/a
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Basic Science
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
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Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
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| Completed |
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Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™
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N/A | |
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Baby Detect : Genomic Newborn Screening
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| Completed |
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Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
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Phase 3 | |
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The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study
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N/A | |
| Recruiting |
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PK and PD Study of NPI-001 and Cysteamine Bitartrate
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Phase 1/Phase 2 | |
| Completed |
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Evaluation of Fanconi Syndrome and Cystinosis
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N/A | |
| Completed |
NCT00004312 -
Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis
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N/A | |
| Enrolling by invitation |
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Early Check: Expanded Screening in Newborns
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| Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
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||
| Completed |
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The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients
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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 |
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Development of Health-related Quality of Life Instrument for Patients With Cystinosis
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| Withdrawn |
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Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
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Phase 1/Phase 2 | |
| Completed |
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N Acetyl Cysteine for Cystinosis Patients
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Phase 4 | |
| Recruiting |
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N/A | |
| Completed |
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Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
|
Phase 3 | |
| Active, not recruiting |
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Stem Cell Gene Therapy for Cystinosis
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Phase 1/Phase 2 |