Cystinosis
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CLINICAL_TRIALS trial_result · 2026-08-06Clinical Trial Results: Stem Cell Gene Therapy for Cystinosis [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating a stem cell gene therapy for cystinosis completed, with results posted.
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: PK and PD Study of NPI-001 and Cysteamine Bitartrate [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/2 PK and PD study of NPI-001 in combination with cysteamine bitartrate for cystinosis, and th…
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CLINICAL_TRIALS trial_result · 2026-03-31Clinical Trial Update: Clinical and Biological Study of Sub-pigmentation During Infantile Cystinosis [NA]
The document summarizes the completion of a clinical and biological study on sub-pigmentation in infants with cystinosis.
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CLINICAL_TRIALS trial_result · 2026-01-14Clinical Trial Update: Stem Cell Gene Therapy for Cystinosis [Phase 1, Phase 2]
The clinical trial update reports completion of the Phase 1/2 study evaluating stem cell gene therapy for cystinosis.
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CLINICAL_TRIALS trial_result · 2025-12-19Clinical Trial Update: A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications [NA]
A completed cohort study in cystinosis reports on patient compliance with cysteamine therapy and associated neurological complications.
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CLINICAL_TRIALS trial_result · 2025-11-24Clinical Trial Update: Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
The clinical trial evaluating neuromuscular characteristics in late adolescent and adult patients with cystinosis has completed.
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CLINICAL_TRIALS trial_result · 2024-07-03Clinical Trial Update: A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
A long-term follow-up study is investigating outcomes in cystinosis patients who previously received CTNS-RD-04, with enrollment by invitati…
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CLINICAL_TRIALS trial_result · 2024-02-05Clinical Trial Update: Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) [Phase 3]
Phase 3 clinical trial update for Cystadrops in pediatric cystinosis patients aged six months to less than two years completed.
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CLINICAL_TRIALS trial_result · 2018-07-24Clinical Trial Update: Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) [Phase 3]
Phase 3 long-term safety follow-up of cysteamine bitartrate delayed-release capsules (RP103) in cystinosis has been completed, with results …
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CLINICAL_TRIALS trial_result · 2018-01-16Clinical Trial Update: Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis [Phase 3]
Phase 3 safety and effectiveness study of RP103 (cysteamine bitartrate delayed-release capsules) in cystinosis patients has completed and re…
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CLINICAL_TRIALS trial_result · 2017-10-06Clinical Trial Update: Kidney Transplantation in Patients With Cystinosis
The National Institute of Diabetes and Digestive and Kidney Diseases-sponsored trial evaluating kidney transplantation in cystinosis patient…
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study
This completed case-control study examined whether susceptibility to thrombosis contributes to pseudotumor cerebri in patients with nephropa…
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Salivary Proteins in Disease and Health
An NIDCR-sponsored clinical trial update discusses salivary proteins in health and disease and lists studied conditions, including diabetes …
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CLINICAL_TRIALS trial_result · 2017-05-24Clinical Trial Update: Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis [Phase 3]
Phase 3 open-label trial evaluating safety and superior effectiveness of cysteamine bitartrate delayed-release capsules (RP103) in Cystinosi…
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CLINICAL_TRIALS trial_result · 2015-08-26Clinical Trial Update: The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients [EARLY_Phase 1]
An early-phase clinical trial investigated how CTNS deletion affects TRPV1 receptor function in patients with cystinosis; the study has comp…
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CLINICAL_TRIALS trial_result · 2015-03-25Clinical Trial Update: Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis [NA]
It reports completion of a randomized trial evaluating a new formulation of ophthalmic cysteamine hydrochloride for corneal cystine accumula…
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CLINICAL_TRIALS trial_result · 2014-11-19Clinical Trial Update: Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis [Phase 3]
Phase 3 trial comparing cysteamine bitartrate delayed-release (RP103) to Cystagon in patients with cystinosis completed; results were posted…
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CLINICAL_TRIALS trial_result · 2014-10-28Clinical Trial Update: Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis [Phase 1, Phase 2]
A Phase 1/2 pilot trial compared RP103 with Cystagon for cystinosis to assess safety, tolerability, and pharmacokinetics/pharmacodynamics, w…
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CLINICAL_TRIALS trial_result · 2014-07-22Clinical Trial Update: Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis [Phase 2]
A Phase 2 clinical trial update reports results for cysteamine ophthalmic drops aimed at reducing corneal crystals in patients with cystinos…
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CLINICAL_TRIALS trial_result · 2013-10-09Clinical Trial Update: Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ [NA]
A healthy volunteer study evaluated how food affects the absorption of Cystagon (cysteamine bitartrate); results were posted after completio…
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CLINICAL_TRIALS trial_result · 2012-06-20Clinical Trial Update: N Acetyl Cysteine for Cystinosis Patients [Phase 4]
A Phase 4 trial of N-acetylcysteine in cystinosis patients has completed.
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CLINICAL_TRIALS trial_result · 2008-03-04Clinical Trial Update: New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis [Phase 1]
Phase 1 trial evaluating a new cysteamine eye drop formulation to treat corneal crystals in cystinosis completed.