Clinical trials: Severe Combined Immunodeficiency Syndrome
Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID)
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1
This study will evaluate the safety and effectiveness of insulin-like growth factor-1 (IGF-1) to treat patients with X-linked severe combined immunodeficiency (XSCID).
- Conditions
- Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence
- Trial name
- NCT00490100
- Testing
- Increlex
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Immune System Diseases
Retroviral Insertion Site Methodology Study
Adenosine deaminase (ADA) is an enzyme involved in the development and functioning of the immune system. Deficiency of ADA results in severe combined immunodeficiency (SCID), a fatal inherited immune disorder.
- Trial name
- NCT03311074
- Testing
- Strimvelis
- Sponsor
- Fondazione Telethon
Severe Combined Immunodeficiency
Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID
The purpose of this study is to learn what factors influence adolescent girls' decisions regarding testing for carrier status of X-Linked Severe Combined Immunodeficiency (XSCID).
- Trial name
- NCT00006335
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
ADA-SCID
Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents
Background: Severe combined immunodeficiency (SCID) is a rare inherited disorder in which certain white blood cells have impaired function and are unable to properly fight infections.
- Trial name
- NCT01182857
- Sponsor
- National Human Genome Research Institute (NHGRI)
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)
SCID
Conditioning SCID Infants Diagnosed Early
The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation.
- Trial name
- CSIDE (NCT03619551)
- Testing
- Busulfan, Cell processing for TCRαβ+/CD19+ depletion
- Where
- 38 sites in United States, Canada
- Volunteers
- 56 people
- Sponsor
- Center for International Blood and Marrow Transplant Research
Sarcoma, Kaposi, HIV Infections
A Comparison of DOX-SL Versus Adriamycin Plus Bleomycin Plus Vincristine in the Treatment of Severe AIDS-Related Kaposi's Sarcoma
To determine the efficacy of Stealth liposomal doxorubicin hydrochloride (DOX-SL) in the treatment of severe AIDS-related Kaposi's sarcoma (KS) by comparison with the established therapy ABV: Adriamycin…
- Trial name
- NCT00002318
- Testing
- Doxorubicin hydrochloride (liposomal), Bleomycin sulfate, Vincristine sulfate
- Where
- 28 sites in United States
- Volunteers
- 225 people
- Sponsor
- Sequus Pharmaceuticals
Immunologic Deficiency Syndrome, Agammaglobulinemia
Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients
The objective of this study is to determine if the safety and tolerability of Immune Globulin Intravenous (Human), 10% caprylate/chromatography (IGIV-C)purified is similar when infused at two different infusion rates.
- Conditions
- Immunologic Deficiency Syndrome, Agammaglobulinemia, Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome
- Trial name
- NCT00220766
- Testing
- Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified, Dextrose, 5% in Water
- Where
- 13 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Grifols Therapeutics LLC
Bronchiectasis, Adenosine Deaminase Deficiency
Observational Study Evaluating the Prevalence of Enzyme Deficiency in Pulmonology Clinics (ADA)
This observational study was designed as a prospective epidemiological screening study. Patients who applied to the centers participating in the study, bronchiectasis was detected on at least one computed tomography of the lungs…
- Conditions
- Bronchiectasis, Adenosine Deaminase Deficiency, Pulmonary Disease
- Trial name
- NCT05300347
- Testing
- adenosine deaminase enzyme deficiency test
- Where
- Turkey (Türkiye)
- Volunteers
- 500 people
- Sponsor
- TRPHARM
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Haematological Malignancy, Acute Leukemia
Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation
Allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes haemoglobinopathies, bone…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT05298930)
- Testing
- Physical activity program
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Centre Leon Berard
Immunologic Deficiency Syndromes
Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Adenosine deaminase (ADA) enzyme deficiency results in severe combined immunodeficiency (SCID), a fatal autosomal recessive inherited immune disorder.
- Trial name
- NCT03478670
- Testing
- Strimvelis
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Fondazione Telethon
Severe Combined Immunodeficiency
Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim
The goal of this study is to develop a novel approach to hematopoietic stem cell transplantation for children with Severe Combined Immunodeficiency Disease (SCID) that eliminates the use of toxic chemotherapy conditioning and…
- Trial name
- NCT01182675
- Testing
- Transplant Conditioning with Mobilization Only, Transplant Conditioning with Mobilization and Alemtuzumab
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of California, San Francisco
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)
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
Severe Combined Immunodeficiency
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
This study will monitor the long-term effects of gene therapy in patients with severe combined immunodeficiency disease (SCID) due to a deficiency in an enzyme called adenosine deaminase (ADA).
- Trial name
- NCT00001255
- Testing
- ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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