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Results for “Haploinsufficiency”

12 trials

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Showing 12 of 12 results

Early research (Phase 1)UnknownNCT05235438
What this trial is testing

Safety and Toxicity Study of IMM27M in Patients With Advanced Solid Tumor

Who this might be right for
OncologyCTLA4 Haploinsufficiency
ImmuneOnco Biopharmaceuticals (Shanghai) Inc. 16
Not applicableNot Yet RecruitingNCT05292768
What this trial is testing

Are Mast Cells Involved in Autoinflammatory Diseases

Who this might be right for
Autoinflammatory DiseaseFMFTRAPS+3 more
Assistance Publique - Hôpitaux de Paris 590
Testing effectiveness (Phase 2)Looking for participantsNCT06662188
What this trial is testing

JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency

Who this might be right for
SHANK3 HaploinsufficiencyPhelan-McDermid Syndrome
Jaguar Gene Therapy, LLC 6
Testing effectiveness (Phase 2)WithdrawnNCT03733067
What this trial is testing

Safety and Efficacy of Abatacept for Treating Chronic Cytopenia in Cytotoxic T-Lymphocyte Antigen 4 (CTLA4) Haploinsufficiency

Who this might be right for
CTLA4 HaploinsufficencyChronic Cytopenia
National Institute of Allergy and Infectious Diseases (NIAID)
Not applicableUnknownNCT04701229
What this trial is testing

Haploinsufficiency of the RBM22 and SLU7 Genes in Del(5q) Myelodysplastic Syndromes

Who this might be right for
Myelodysplastic SyndromesMyelodysplastic Syndrome With Isolated Del(5Q)Myelodysplastic Syndrome With Del(5Q)
University Hospital, Brest 100
Large-scale testing (Phase 3)Study completedNCT04623398
What this trial is testing

Effect of Lithium in Patients With Autism Spectrum Disorder and Phelan-McDermid Syndrome (SHANK3 Haploinsufficiency)

Who this might be right for
Autism Spectrum Disorder
Assistance Publique - Hôpitaux de Paris 22
Not applicableUnknownNCT04377867
What this trial is testing

New Biomarkers for Diagnosis and Follow-up of Patients With LRBA or CTLA4 Deficiencies

Who this might be right for
LRBA DeficiencyCTLA4 Haploinsufficiency
Marmara University 30
Not applicableStudy completedNCT00597987
What this trial is testing

BRCA1 Haploinsufficiency and Gene Expression

Who this might be right for
BRCA1 Mutations
Memorial Sloan Kettering Cancer Center 120
Not applicableLooking for participantsNCT07413211
What this trial is testing

Genetic Developmental and Epileptic Encephalopathy Natural History Study for Clinical Trial Readiness

Who this might be right for
MEF2CDEE
Weill Medical College of Cornell University 22,068
Not applicableStudy completedNCT00001639
What this trial is testing

Evaluation of Patients With Unresolved Chromosome Abnormalities

Who this might be right for
AbnormalitiesFailure to ThriveMental Retardation+1 more
National Human Genome Research Institute (NHGRI) 263
Not applicableLooking for participantsNCT07014020
What this trial is testing

RB001 Gene Therapy Study in Children With SHANK3-related Phelan McDermid Syndrome (PMS)

Who this might be right for
Phelan-McDermid SyndromeSHANK3 Haploinsufficiency
Peking University First Hospital 8
Not applicableStudy completedNCT05040256
What this trial is testing

Neurologic and Immunologic Characteristics of CTLA-4 and LRBA Hereditary Deficiency

Who this might be right for
CTLA4 Haploinsufficiency
University Hospital, Montpellier 20