Clinical trials: Severe Combined Immunodeficiency
Severe Combined Immunodeficiency, X-Linked
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity.
- Trial name
- NCT03601286
- Testing
- Lentiviral vector transduced CD34+ cells
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Lymphoma, Non-Hodgkin, HIV Infections
Phase II Trial of Sequential Chemotherapy and Radiotherapy for AIDS-Related Primary Central Nervous System Lymphoma
To estimate the response rate, overall and disease-free survival, toxicities, factors associated with outcome, and effect on quality of life in patients with AIDS-related primary CNS lymphoma treated with CHOD (cyclophosphamide…
- Trial name
- NCT00000801
- Testing
- Filgrastim, Vincristine sulfate, Doxorubicin hydrochloride
- Where
- 7 sites in United States
- Volunteers
- 33 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Severe Combined Immunodeficiencies (SCID)
Follow-up of the Cohort of Newborns Screened at Birth Using TREC Analysis
Since September 2025, neonatal screening for severe combined immunodeficiency (SCID) has been generalized in France. These genetic disorders, which are asymptomatic at birth, cause severe immunodeficiency, exposing infants to…
- Trial name
- DépisTrec (NCT07704281)
- Testing
- Data collection
- Where
- France
- Volunteers
- 450 people
- Sponsor
- Nantes University Hospital
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)
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
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.
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
Severe Combined Immunodeficiency
Autologous Gene Therapy for Artemis-Deficient SCID
This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene.
- Trial name
- NCT03538899
- Testing
- AProArt-CD34, CliniMACS® CD34 Reagent System cell sorter device, Busulfan
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- University of California, San Francisco
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
x Linked Combined Immunodeficiency
cliniMACs HUD for T Cell Depletion
This protocol is designed to enable access to related or unrelated CD34 cells manufactured using the CliniMACS (Miltenyi) under the HUD designation for patients needing T cell depleted allogeneic grafts for hematopoietic stem…
- Trial name
- NCT02915406
- Testing
- CliniMACs
- Where
- United States
- Sponsor
- University of Miami
Gene Therapy
Gene Therapy for X Linked Severe Combined Immunodeficiency
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be…
- Trial name
- NCT04286815
- Testing
- Lentiviral Vector Gene Therapy
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Children's Hospital of Chongqing Medical University
Bone Marrow Failure, Osteopetrosis
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in…
- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Trial name
- NCT01019876
- Testing
- Fludarabine, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Columbia University
Severe Combined Immunodeficiency, Atypical
Generalized Neonatal Screening of Severe Combined Immunodeficiencies
Severe combined Immunodeficiencies ( SCID ) are a group of inherited diseases of the immune system by characterised profound abnormalities of T cell development .
- Trial name
- DEPISTREC (NCT02244450)
- Testing
- SCID screening
- Where
- 51 sites in France
- Volunteers
- 190,539 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Pneumonia, Pneumocystis Carinii, HIV Infections
Trimetrexate Plus Leucovorin Calcium Rescue Versus Sulfamethoxazole-Trimethoprim in the Treatment of Pneumocystis Carinii Pneumonia (PCP) in Patients With AIDS
To compare the safety and effectiveness of an investigational drug therapy (trimetrexate plus leucovorin calcium) with that of conventional therapy (sulfamethoxazole-trimethoprim) in the treatment of moderately severe…
- Trial name
- NCT00001014
- Testing
- Trimetrexate glucuronate, Sulfamethoxazole-Trimethoprim, Leucovorin calcium
- Where
- 6 sites in United States
- Volunteers
- 302 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies
OBJECTIVES: I. Provide curative immunoreconstituting allogeneic bone marrow transplantation for patients with primary immunodeficiencies. II.
- Conditions
- Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease
- Trial name
- NCT00006054
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
Severe Combined Immunodeficiency
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
This is a study of stem cell transplantation with TBX-1400 in pediatric subjects with severe combined immunodeficiency (SCID). The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability…
- Trial name
- NCT02860559
- Testing
- TBX-1400
- Where
- 2 sites in Israel
- Volunteers
- 8 people
- Sponsor
- Taiga Biotechnologies, Inc.
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
Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders
This study is to discover whether children with severe combined immunodeficiency disease (SCID) or other primary immunodeficiency disorder (PID) for which no satisfactory treatment other than stem cell transplantation (SCT)…
- Conditions
- Severe Combined Immunodeficiency Disease, Severe Primary Immunodeficiency Disorder, Undefined T Cell Deficiency Disorder, Wiskott-Aldrick Syndrome
- Trial name
- MASCI (NCT00579137)
- Testing
- Campath -1H, Fludarabine, Anti-CD45
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Haematological Malignancy, Acute Leukemia
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts
To date, allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes, haemoglobinopathies…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT04331483)
- Testing
- Physical activity program
- Where
- France
- Sponsor
- Centre Leon Berard