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Results for “ADA Deficient SCID”

31 trials

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Showing 20 of 31 results

Not applicableUnknownNCT00006319
What this trial is testing

Molecular and Clinical Studies of Primary Immunodeficiency Diseases

Who this might be right for
Wiskott- Aldrich SyndromeADA Deficient SCID
National Human Genome Research Institute (NHGRI) 266
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
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 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
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
Not applicableStudy completedNCT03878069
What this trial is testing

Registry Study of Revcovi Treatment in Patients With ADA-SCID

Who this might be right for
Adenosine Deaminase Severe Combined Immunodeficiency
Chiesi Farmaceutici S.p.A. 32
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
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
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
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
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 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 applicableUnknownNCT05300334
What this trial is testing

Investigation of ADA Enzyme Deficiency

Who this might be right for
Adenosine Deaminase Deficiency
TRPHARM 800
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 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 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 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
Not applicableUnknownNCT05300347
What this trial is testing

Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA)

Who this might be right for
BronchiectasisAdenosine Deaminase DeficiencyPulmonary Disease
TRPHARM 500
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
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