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Results for “Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency”

28 trials

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Showing 20 of 28 results

Not applicableEnrolling By InvitationNCT03478670
What this trial is testing

Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Who this might be right for
Immunologic Deficiency Syndromes
Fondazione Telethon 50
Not applicableStudy completedNCT00001255
What this trial is testing

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study

Who this might be right for
Severe Combined Immunodeficiency
National Human Genome Research Institute (NHGRI) 10
Not applicableLooking for participantsNCT03645460
What this trial is testing

Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)

Who this might be right for
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Shenzhen Geno-Immune Medical Institute 10
Not applicableStudy completedNCT00043225
What this trial is testing

The Role of Bacteria and Genetic Variations in Cystic Fibrosis

Who this might be right for
Psuedomas InfectionCystic Fibrosis
National Heart, Lung, and Blood Institute (NHLBI) 76
Early research (Phase 1)Study completedNCT00008450
What this trial is testing

Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant

Who this might be right for
Adenosine Deaminase DeficiencyAutosomal Recessive DisorderImmune System Disorder+4 more
Fred Hutchinson Cancer Center 6
Testing effectiveness (Phase 2)Study completedNCT01852071
What this trial is testing

Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene

Who this might be right for
ADA-SCID
University of California, Los Angeles 46
Testing effectiveness (Phase 2)Study completedNCT03765632
What this trial is testing

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Who this might be right for
Severe Combined Immunodeficiency Due to ADA Deficiency
Great Ormond Street Hospital for Children NHS Foundation Trust 13
Testing effectiveness (Phase 2)Study completedNCT02999984
What this trial is testing

Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID

Who this might be right for
Severe Combined Immunodeficiency Due to ADA Deficiency
University of California, Los Angeles 10
Post-approval studies (Phase 4)WithdrawnNCT03311074
What this trial is testing

Retroviral Insertion Site Methodology Study

Who this might be right for
Immune System Diseases
Fondazione Telethon
Early research (Phase 1)Study completedNCT00018018
What this trial is testing

Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency

Who this might be right for
Severe Combined Immunodeficiency Syndrome
National Human Genome Research Institute (NHGRI) 8
Testing effectiveness (Phase 2)Study completedNCT01279720
What this trial is testing

Gene Therapy ADA Deficiency

Who this might be right for
Adenosine Deaminase Deficiency
Great Ormond Street Hospital for Children NHS Foundation Trust 8
Not applicableEnrolling By InvitationNCT04049084
What this trial is testing

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

Who this might be right for
Adenosine Deaminase DeficiencySevere Combined Immunodeficiency (SCID)
University of California, Los Angeles 70
Large-scale testing (Phase 3)WithdrawnNCT04140539
What this trial is testing

A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

Who this might be right for
Severe Combined Immunodeficiency Due to ADA Deficiency
University of California, Los Angeles
Not applicableUnknownNCT05300373
What this trial is testing

Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E

Who this might be right for
Adenosine Deaminase Deficiency
TRPHARM 100
Not applicableUnknownNCT01346150
What this trial is testing

Patients Treated for SCID (1968-Present)

Who this might be right for
SCIDADA-SCIDXSCID+3 more
National Institute of Allergy and Infectious Diseases (NIAID) 1,007
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)
Testing effectiveness (Phase 2)Study completedNCT01380990
What this trial is testing

Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency

Who this might be right for
Adenosine Deaminase DeficiencySevere Combined Immunodeficiencies (SCID)
Great Ormond Street Hospital for Children NHS Foundation Trust 36
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 applicableUnknownNCT04959890
What this trial is testing

Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Who this might be right for
Severe Combined Immunodeficiency Due to ADA Deficiency
Fondazione Telethon 15
Not applicableStudy completedNCT03232203
What this trial is testing

Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Who this might be right for
Severe Combined Immunodeficiency Due to ADA Deficiency
Fondazione Telethon 16
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