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Results for “Cystinosis”

46 trials

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Enrolling by Invitation

Showing 20 of 46 results

Not applicableEnrolling By InvitationNCT05146830
What this trial is testing

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

Who this might be right for
Cystinosis
Stephanie Cherqui 50
Testing effectiveness (Phase 2)Looking for participantsNCT05994534
What this trial is testing

PK and PD Study of NPI-001 and Cysteamine Bitartrate

Who this might be right for
Cystinosis
Nacuity Pharmaceuticals, Inc. 12
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 completedNCT01044186
What this trial is testing

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

Who this might be right for
Transfusional Iron Overload
Novartis Pharmaceuticals 30
Not applicableStudy completedNCT05545774
What this trial is testing

Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients

Who this might be right for
Cystinosis
Assistance Publique - Hôpitaux de Paris 20
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableStudy completedNCT04071548
What this trial is testing

The Effect of Exercise on Muscle Dysfunction in Cystinosis

Who this might be right for
Cystinosis
Stanford University 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
Not applicableLooking for participantsNCT05843851
What this trial is testing

Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria

Who this might be right for
CystinosisPrimary Hyperoxaluria
Cystinose Stiftung 200,000
Early research (Phase 1)Study completedNCT00001736
What this trial is testing

New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis

Who this might be right for
Cystinosis
National Eye Institute (NEI) 51
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
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 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 completedNCT00010426
What this trial is testing

Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis

Who this might be right for
Cystinosis
FDA Office of Orphan Products Development 30
Not applicableStudy completedNCT00100204
What this trial is testing

Salivary Proteins in Disease and Health

Who this might be right for
Diabetes MellitusSarcoidosisDental Caries+3 more
National Institute of Dental and Craniofacial Research (NIDCR) 264
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 applicableStudy completedNCT06027385
What this trial is testing

Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy

Who this might be right for
CystinosisCystinosis, NephropathicSpinal Muscular Atrophy
Cystinose Stiftung 300,000
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 completedNCT00004312
What this trial is testing

Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis

Who this might be right for
Cystinosis
National Center for Research Resources (NCRR) 400
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
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