Cystinosis Clinical Trial
OBJECTIVES:
I. Classify renal tubular defects using clinical and biochemical findings in patients with
Fanconi syndrome and cystinosis.
PROTOCOL OUTLINE:
Patients receive a clinical and biochemical evaluation, including a psychometric assessment
and molecular, renal, and thyroid studies.
;
Primary Purpose: Screening
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
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Completed |
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N/A | |
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Completed |
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Phase 3 | |
Completed |
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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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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 |
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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 |
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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
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Phase 1 | |
Recruiting |
NCT05959668 -
Development of Health-related Quality of Life Instrument for Patients With Cystinosis
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Withdrawn |
NCT02124070 -
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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Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria
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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
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Phase 3 | |
Active, not recruiting |
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Stem Cell Gene Therapy for Cystinosis
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Phase 1/Phase 2 | |
Recruiting |
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