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Results for “Genetic Therapy”

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Enrolling by Invitation

Showing 20 of 0 results

Early research (Phase 1)Study completedNCT04095273
What this trial is testing

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

Who this might be right for
Advanced Solid Tumors
Bayer 56
Testing effectiveness (Phase 2)WithdrawnNCT06833931
What this trial is testing

PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Who this might be right for
Duchenne Muscular Dystrophy (DMD)
PepGen Inc
Not applicableWithdrawnNCT01182857
What this trial is testing

Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents

Who this might be right for
ADA-SCID
National Human Genome Research Institute (NHGRI)
Not applicableLooking for participantsNCT07429851
What this trial is testing

CompArative Analysis Between, Thymic, pulmonaRy and Pancreatic Well Differentiated High Grade Neuroendocrine Tumors

Who this might be right for
Neuroendocrine Carcinoma of LungNeuroendocrine Carcinoma of Thymus
European Institute of Oncology 34
Not applicableUnknownNCT06448702
What this trial is testing

Evaluation of Efficacy and Safety of Enhanced External Counter-pulsation on Patients With Atopic Dermatitis and Inflammatory Skin Disease Requiring Wet Wrap Therapy

Who this might be right for
Atopic DermatitisInflammatory Skin Disease
The University of Hong Kong 40
Large-scale testing (Phase 3)UnknownNCT04553419
What this trial is testing

Antibiotic Treatment Of Staphylococcus Aureus In Stable People With CF

Who this might be right for
Cystic Fibrosis
University of British Columbia 86
Testing effectiveness (Phase 2)Ended earlyNCT05429372
What this trial is testing

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Who this might be right for
Muscular Dystrophy, Duchenne
Pfizer 10
Not applicableStudy completedNCT03797677
What this trial is testing

MN4000 for Treatment of CF and MND Patients in the Home Setting

Who this might be right for
Cystic FibrosisMotor Neuron DiseaseAirway Clearance Impairment
Hill-Rom 10
Not applicableStudy completedNCT03741881
What this trial is testing

A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6)

Who this might be right for
Haemophilia AHaemophilia A With InhibitorsHaemophilia B+1 more
Novo Nordisk A/S 231
Testing effectiveness (Phase 2)Study completedNCT04978428
What this trial is testing

Epidiolex in Obsessive Compulsive Disorder and Related Disorders

Who this might be right for
Obsessive-Compulsive DisorderTrichotillomania (Hair-Pulling Disorder)Tourette Syndrome+1 more
University of Chicago 4
Testing effectiveness (Phase 2)Active Not RecruitingNCT03770572
What this trial is testing

Gene Therapy for Children With CLN3 Batten Disease

Who this might be right for
CLN3Batten Disease
Neela Therapeutics 7
Large-scale testing (Phase 3)Active Not RecruitingNCT05444257
What this trial is testing

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

Who this might be right for
Cystic Fibrosis
Vertex Pharmaceuticals Incorporated 822
Not applicableLooking for participantsNCT05818319
What this trial is testing

Cystic Fibrosis in the Kidney: Monitoring the Effectiveness of Elexacaftor/tezacaftor/ivacaftor in Urine After a Short Pause of Therapy

Who this might be right for
Cystic Fibrosis (CF)CFTR Gene Mutation
University of Aarhus 30
Testing effectiveness (Phase 2)Study completedNCT00229619
What this trial is testing

Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

Who this might be right for
Anemia, AplasticRed-Cell Aplasia, PureAnemia, Diamond-Blackfan
National Heart, Lung, and Blood Institute (NHLBI) 11
Not applicableNot Yet RecruitingNCT07653139
What this trial is testing

Patient Preferences in Treatment of Haemophilia A With and Without Inhibitors: a Discrete Choice Experiment in Germany

Who this might be right for
Haemophilia A
Novo Nordisk A/S 150
Not applicableEnrolling By InvitationNCT06879665
What this trial is testing

TANDem-2: Closing the Gap to Interventions for TAND

Who this might be right for
Adult Caregivers of Individuals With TSCTuberous Sclerosis Complex (TSC)
Vrije Universiteit Brussel 30
Testing effectiveness (Phase 2)Active Not RecruitingNCT05228145
What this trial is testing

Gene Therapy Study for Children With CLN5 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis CLN5
Neurogene Inc. 6
Not applicableStudy completedNCT02740153
What this trial is testing

PCORI Urea Cycle Disorder Study

Who this might be right for
Urea Cycle Disorders
Children's National Research Institute 187
Testing effectiveness (Phase 2)WithdrawnNCT02904772
What this trial is testing

Alipogene Tiparvovec for the Treatment of LPLD Patients

Who this might be right for
LPL Deficiency
UniQure Biopharma B.V.
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
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