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Results for “Nephropathic Cystinosis”

31 trials

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Showing 20 of 31 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT04248439
What this trial is testing

Gene Therapy for Fanconi Anemia, Complementation Group A

Who this might be right for
Fanconi Anemia Complementation Group A
Rocket Pharmaceuticals Inc. 5
Large-scale testing (Phase 3)Study completedNCT01000961
What this trial is testing

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 43
Not applicableWithdrawnNCT02837523
What this trial is testing

Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis)

Who this might be right for
Renal Fanconi SyndromePhotophobiaHypophosphatemia
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Study completedNCT00872729
What this trial is testing

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 9
Not applicableStudy completedNCT02766855
What this trial is testing

Efficacy of Topical Cysteamine in Nephropathic Cyctinosis

Who this might be right for
Nephropathic CyctinosisCorneal Cystine Crystals
The Eye Center and The Eye Foundation for Research in Ophthalmology 26
Early research (Phase 1)UnknownNCT03814408
What this trial is testing

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

Who this might be right for
Fanconi Anemia Complementation Group A
Rocket Pharmaceuticals Inc. 2
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Not applicableLooking for participantsNCT03919981
What this trial is testing

CYSTEA-BONE Clinical Study

Who this might be right for
Nephropathic Cystinosis
Hospices Civils de Lyon 50
Testing effectiveness (Phase 2)Study completedNCT00061763
What this trial is testing

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

Who this might be right for
Beta-thalassemiaMyelodysplastic SyndromesFanconi Syndrome+2 more
Novartis Pharmaceuticals 175
Not applicableStudy completedNCT01432561
What this trial is testing

Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™

Who this might be right for
CystinosisNephropathic Cystinosis
University of California, San Diego 8
Not applicableStudy completedNCT00071903
What this trial is testing

The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study

Who this might be right for
Pseudotumor CerebriCystinosis
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 18
Not applicableStudy completedNCT00004350
What this trial is testing

Evaluation of Fanconi Syndrome and Cystinosis

Who this might be right for
CystinosisFanconi Syndrome
National Center for Research Resources (NCRR) 12
Testing effectiveness (Phase 2)Looking for participantsNCT06910813
What this trial is testing

DFT383 in Pediatric Participants With Nephropathic Cystinosis

Who this might be right for
Nephropathic Cystinosis
Novartis Pharmaceuticals 30
Not applicableEnrolling By InvitationNCT04246060
What this trial is testing

Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients

Who this might be right for
Nephropathic Cystinosis
Chiesi SA/NV 31
Not applicableStudy completedNCT00074516
What this trial is testing

Kidney Transplantation in Patients With Cystinosis

Who this might be right for
Cystinosis
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 11
Not applicableStudy completedNCT00499187
What this trial is testing

Fanconi Syndrome Due to ARVs in HIV-Infected Persons

Who this might be right for
HIV InfectionsFanconi SyndromeKidney Disease+1 more
Gilead Sciences 56
Testing effectiveness (Phase 2)Study completedNCT00001213
What this trial is testing

Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis

Who this might be right for
Cystinosis
National Eye Institute (NEI) 328
Testing effectiveness (Phase 2)Active Not RecruitingNCT04069533
What this trial is testing

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

Who this might be right for
Fanconi Anemia Complementation Group A
Rocket Pharmaceuticals Inc. 7
Testing effectiveness (Phase 2)Ended earlyNCT04069260
What this trial is testing

ELX-02 in Patients With Nephropathic Cystinosis

Who this might be right for
Genetic DiseaseNonsense MutationCystinosis
Eloxx Pharmaceuticals, Inc. 3
Large-scale testing (Phase 3)Study completedNCT01197378
What this trial is testing

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

Who this might be right for
Cystinosis
Amgen 60
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