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Results for “Single-gene Disorders”

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Showing 20 of 0 results

Not applicableStudy completedNCT02841501
What this trial is testing

Genetic Susceptibility Factors for Candidemia.

Who this might be right for
Invasive CandidiasisCandidemiaGenetic Predisposition to Disease
University Hospital, Lille 453
Testing effectiveness (Phase 2)Looking for participantsNCT07009002
What this trial is testing

BCOR and ZC3H12 Genes Knock-out CD19-targeting CAR-T Cell Therapy in r/r B Cell Lymphoma

Who this might be right for
B Cell Lymphoma
Chinese PLA General Hospital 30
Not applicableStudy completedNCT04223180
What this trial is testing

Rehabilomics Study in Stroke Patients After Robotic Rehabilitation

Who this might be right for
Stroke
Fondazione Don Carlo Gnocchi ETS 50
Testing effectiveness (Phase 2)UnknownNCT03872479
What this trial is testing

Single Ascending Dose Study in Participants With LCA10

Who this might be right for
Leber Congenital Amaurosis 10Inherited Retinal DystrophiesEye Diseases, Hereditary+4 more
Editas Medicine, Inc. 34
Testing effectiveness (Phase 2)Ended earlyNCT06515002
What this trial is testing

Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)

Who this might be right for
Cystic Fibrosis
Boehringer Ingelheim 5
Testing effectiveness (Phase 2)Study completedNCT03489291
What this trial is testing

Dose Confirmation Trial of AAV5-hFIXco-Padua

Who this might be right for
Hemophilia B
CSL Behring 3
Not applicableWithdrawnNCT00573092
What this trial is testing

Analyzing Gene Regions That May Interact With the Effectiveness of High Blood Pressure Drugs

Who this might be right for
Myocardial InfarctionCerebrovascular AccidentDeath, Sudden, Cardiac
University of Washington
Testing effectiveness (Phase 2)Looking for participantsNCT07282847
What this trial is testing

Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

Who this might be right for
Pompe Disease (Late-onset)Pompe Disease Late-OnsetLOPD
AskBio Inc 12
Testing effectiveness (Phase 2)Enrolling By InvitationNCT07113743
What this trial is testing

Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Who this might be right for
Chronic Granulomatous Disease (CGD)
National Institute of Allergy and Infectious Diseases (NIAID) 10
Testing effectiveness (Phase 2)Study completedNCT02269943
What this trial is testing

Safety and Efficacy of CC-486 in Previously Treated Patients With Locally Advanced or Metastatic Nasopharyngeal Carcinoma

Who this might be right for
Nasopharyngeal Neoplasms
Celgene 36
Large-scale testing (Phase 3)Study completedNCT03392974
What this trial is testing

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Who this might be right for
Hemophilia A
BioMarin Pharmaceutical 1
Very early researchLooking for participantsNCT06345898
What this trial is testing

Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)

Who this might be right for
X Linked Retinoschisis
West China Hospital 12
Early research (Phase 1)Study completedNCT02082860
What this trial is testing

Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria

Who this might be right for
Acute Intermittent Porphyria
Digna Biotech S.L. 8
Testing effectiveness (Phase 2)WithdrawnNCT01500824
What this trial is testing

A Clinical Study Testing The Safety And Efficacy Of Crizotinib In East Asian Patients With Anaplastic Lymphoma Kinase (ALK) Positive Advanced Non-Small Cell Lung Cancer

Who this might be right for
Non-Small Cell Lung Cancer
Pfizer
Very early researchActive Not RecruitingNCT06392724
What this trial is testing

Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
Peking Union Medical College Hospital 3
Early research (Phase 1)Study completedNCT02071862
What this trial is testing

Study of the Glutaminase Inhibitor CB-839 in Solid Tumors

Who this might be right for
Solid TumorsTriple-Negative Breast CancerNon Small Cell Lung Cancer+7 more
Calithera Biosciences, Inc 210
Testing effectiveness (Phase 2)Active Not RecruitingNCT05972629
What this trial is testing

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

Who this might be right for
Phenylketonuria
Sanofi 32
Testing effectiveness (Phase 2)Looking for participantsNCT06844214
What this trial is testing

Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

Who this might be right for
Myotonic Dystrophy
Sanofi 32
Not applicableUnknownNCT00549029
What this trial is testing

The Association of Genetic Polymorphisms With Statin-Induced Myopathy.

Who this might be right for
RhabdomyolysisMyopathy
National Taiwan University Hospital 150
Very early researchLooking for participantsNCT07371767
What this trial is testing

CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia

Who this might be right for
Hyperchylomicronemia
Shanghai Jiao Tong University School of Medicine 15
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