Clinical trials: ADA-SCID
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 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.
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
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
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
- NCT02999984
- Testing
- Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, Los Angeles
ADA-SCID, Adenosine Deaminase Deficiency
EZN-2279 in Patients With ADA-SCID
The purpose of this study is to evaluate the safety, efficacy, and pharmacokinetics of EZN-2279 in patients with ADA-deficient combined immunodeficiency currently being treated with Adagen.
- Conditions
- ADA-SCID, Adenosine Deaminase Deficiency, Severe Combined Immunodeficiency
- Trial name
- NCT01420627
- Testing
- EZN-2279, Adagen
- Where
- 6 sites in United States
- Volunteers
- 7 people
- Sponsor
- Leadiant Biosciences, Inc.