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Results for “Nephropathic Cystinosis”

32 trials

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Showing 20 of 32 results

Not applicableNot Yet RecruitingNCT05903365
What this trial is testing

Observational Follow-up Study of Haplo-identical Transplants in Fanconi Disease

Who this might be right for
Fanconi Syndrome
Assistance Publique - Hôpitaux de Paris 18
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
Testing effectiveness (Phase 2)UnknownNCT04069533
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
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
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
Not applicableLooking for participantsNCT07036731
What this trial is testing

A Study Comparing the Necessity of a Second Transurethral Resection in High-Risk Non-Muscle-Invasive Bladder Cancer Patients With Negative Results From Post-Initial Resection Urine Genome-Wide Low-Depth Sequencing

Who this might be right for
Non Muscle Invasive Bladder Cancer
The First Affiliated Hospital with Nanjing Medical University 428
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
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
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
Large-scale testing (Phase 3)Study completedNCT01733316
What this trial is testing

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

Who this might be right for
Cystinosis
Amgen 41
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
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 applicableLooking for participantsNCT06065852
What this trial is testing

National Registry of Rare Kidney Diseases

Who this might be right for
Adenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis+81 more
UK Kidney Association 35,000
Not applicableUnknownNCT01327807
What this trial is testing

Cure Cystinosis International Registry

Who this might be right for
CystinosisNephropathic CystinosisRenal Fanconi Syndrome
Cystinosis Research Foundation 750
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
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
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
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
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
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