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Results for “Adenosine Deaminase Deficiency”

32 trials

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Showing 20 of 32 results

Early research (Phase 1)Study completedNCT02022696
What this trial is testing

Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector

Who this might be right for
Adenosine Deaminase DeficiencyADA-SCID
National Human Genome Research Institute (NHGRI) 1
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
Testing effectiveness (Phase 2)Study completedNCT00794508
What this trial is testing

MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID

Who this might be right for
Severe Combined Immunodeficiency
Donald B. Kohn, M.D. 10
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
Large-scale testing (Phase 3)Study completedNCT01420627
What this trial is testing

EZN-2279 in Patients With ADA-SCID

Who this might be right for
ADA-SCIDAdenosine Deaminase DeficiencySevere Combined Immunodeficiency
Leadiant Biosciences, Inc. 7
Testing effectiveness (Phase 2)Study completedNCT00599781
What this trial is testing

Gene Therapy for ADA-SCID

Who this might be right for
Severe Combined Immunodeficiency Syndrome
IRCCS San Raffaele 8
Testing effectiveness (Phase 2)Looking for participantsNCT05432310
What this trial is testing

Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector

Who this might be right for
Adenosine Deaminase Severe Combined Immune Deficiency
University of California, Los Angeles 20
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
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 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 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
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
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
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
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
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 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 applicableUnknownNCT05300360
What this trial is testing

Prevalence of Adenosine Deaminase (ADA) Enzyme Deficiency Disease in Adult Patients With Pulmonary Alveolar Proteinosis

Who this might be right for
Pulmonary Alveolar Proteinosis
TRPHARM 15
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 applicableEnrolling By InvitationNCT01186913
What this trial is testing

Natural History Study of SCID Disorders

Who this might be right for
Severe Combined Immunodeficiency (SCID)Leaky SCIDOmenn Syndrome+3 more
National Institute of Allergy and Infectious Diseases (NIAID) 690
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