Clinical trials: ADA SCID
Severe Combined Immunodeficiency Syndrome
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency
This study will evaluate a new method for delivering gene transfer therapy to patients with severe combined immunodeficiency disease (SCID) due to a defective adenosine deaminase (ADA) gene.
- Trial name
- NCT00018018
- Testing
- CD34+ cells transduced with ADA retrovir
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
Immunologic Deficiency Syndromes
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
This is a phase I/II protocol to evaluate the safety and efficacy of ADA gene transfer into hematopoietic stem/progenitor cells for the treatment of adenosine deaminase (ADA)-deficiency.
- Trial name
- Gene-ADA (NCT00598481)
- Testing
- Gene Therapy, Busulfan
- Where
- 2 sites in Israel, Italy
- Volunteers
- 12 people
- Sponsor
- Fondazione Telethon
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency (SCID)
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human…
- Trial name
- NCT04049084
- Testing
- autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV)
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 70 people
- Sponsor
- University of California, Los Angeles
Adenosine Deaminase Deficiency, ADA-SCID
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
This is a clinical gene transfer study that aims to verify the safety and efficacy of the use of the EFS-ADA lentiviral vector to introduce the human adenosine deaminase (ADA) gene into the hematopoietic progenitors of patients…
- Trial name
- NCT02022696
- Testing
- Lentiviral Gene Transfer
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Adenosine Deaminase Severe Combined Immunodeficiency
Registry Study of Revcovi Treatment in Patients With ADA-SCID
This registry study is being conducted in patients with adenosine deaminase severe combined immune deficiency (ADA-SCID) who require enzyme replacement therapy (ERT) treatment with Revcovi.
- Trial name
- NCT03878069
- Testing
- elapegademase-lvlr
- Where
- 14 sites in United States
- Volunteers
- 32 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Severe Combined Immunodeficiency Syndrome
Gene Therapy for ADA-SCID
This study investigated the safety and efficacy of different gene therapy approaches for Severe Combined Immunodeficiency (SCID) caused by the deficiency of adenosine deaminase (ADA) enzyme.
- Trial name
- NCT00599781
- Testing
- gene transduced PBL and/or gene transduced HSC
- Volunteers
- 8 people
- Sponsor
- IRCCS San Raffaele
Severe Combined Immunodeficiency (SCID), Leaky SCID
Natural History Study of SCID Disorders
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions.
- Conditions
- Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
- Trial name
- NCT01186913
- Where
- 44 sites in United States, Canada
- Volunteers
- 690 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Severe Combined Immunodeficiency Due to ADA Deficiency
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic…
- Trial name
- NCT03765632
- Testing
- Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), Busulfan, Peg-Ada
- Where
- United Kingdom
- Volunteers
- 13 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Adenosine Deaminase Deficiency, Severe Combined Immunodeficiencies (SCID)
Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency
This is a historically controlled, non-randomized Phase I/II clinical trial to assess the safety and efficacy of autologous transplantation of CD34+ hematopoietic stem/progenitor cells (HSPCs), obtained from infants affected by…
- Trial name
- NCT01380990
- Testing
- Infusion of autologous EFS-ADA LV CD34+ cells, Haematopoietic Stem Cell Transplantation (HSCT), Busulfan
- Where
- United Kingdom
- Volunteers
- 36 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
SCID, ADA-SCID
Patients Treated for SCID (1968-Present)
Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant, gene therapy, or enzyme…
- Conditions
- SCID, ADA-SCID, XSCID, Leaky SCID
- Trial name
- NCT01346150
- Where
- 36 sites in United States, Canada
- Volunteers
- 1,007 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Adenosine Deaminase Severe Combined Immune Deficiency
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human…
- Trial name
- NCT05432310
- Testing
- A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, Los Angeles
Severe Combined Immunodeficiency Due to ADA Deficiency
A Clinical Study to Enable Process Validation of Commercial Grade OTL-101
The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process.
- Trial name
- NCT04140539
- Testing
- OTL-101
- Where
- United States
- Sponsor
- University of California, Los Angeles
Adenosine Deaminase Deficiency
Investigation of ADA Enzyme Deficiency
This observational study was designed as a prospective epidemiological screening study. Patients who applied to the centers participating in the study and were found to be lymphopenic in at least one examination will be included…
- Trial name
- NCT05300334
- Testing
- adenosine deaminase test
- Where
- Turkey (Türkiye)
- Volunteers
- 800 people
- Sponsor
- TRPHARM
ADA-SCID
Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from the bone marrow (BM) of ADA-deficient SCID infants and children following human ADA cDNA…
- Trial name
- NCT01852071
- Testing
- Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- University of California, Los Angeles
Psuedomas Infection, Cystic Fibrosis
The Role of Bacteria and Genetic Variations in Cystic Fibrosis
This study will examine 1) the role of hereditary factors in cystic fibrosis; i.e., the relationship of the disease to specific gene variations, and 2) the role of bacterial products involved in lung infections substances…
- Trial name
- NCT00043225
- Where
- 4 sites in United States
- Volunteers
- 76 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Adenosine Deaminase Deficiency, Autosomal Recessive Disorder
Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant
This pilot clinical trial studies total-body irradiation followed by cyclosporine and mycophenolate mofetil in treating patients with severe combined immunodeficiency (SCID) undergoing donor bone marrow transplant.
- Conditions
- Adenosine Deaminase Deficiency, Autosomal Recessive Disorder, Immune System Disorder, Purine-Nucleoside Phosphorylase Deficiency
- Trial name
- NCT00008450
- Testing
- Allogeneic Bone Marrow Transplantation, Cyclosporine, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Fred Hutchinson Cancer Center
Wiskott- Aldrich Syndrome, ADA Deficient SCID
Molecular and Clinical Studies of Primary Immunodeficiency Diseases
This study will try to identify mutations in the genes responsible for primary immunodeficiency disorders (inherited diseases of the immune system) and evaluate the course of these diseases in patients over time to learn more…
- Trial name
- NCT00006319
- Where
- United States
- Volunteers
- 266 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
This is a Phase I/II trial of in vivo lentiviral gene therapy for treating adenosine deaminase severe combined immunodeficiency (ADA-SCID) using a self-inactivating lentiviral vector (LV) ivlv-ADA to functionally correct the…
- Trial name
- NCT03645460
- Testing
- Direct intravenous injection of ivlv-ADA lentiviral vector
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute