Clinical trials: Severe Combined Immunodeficiency
Severe Combined Immunodeficiency Due to ADA Deficiency
Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)
STRIMVELIS is a medicinal product that restores adenosine deaminase (ADA) function in hematopoietic cell lineages, thereby preventing impaired immune function.
- Trial name
- NCT03232203
- Testing
- STRIMVELIS
- Where
- Italy
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Fondazione Telethon
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
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
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
Severe Combined Immunodeficiency
The Experience of Screening for SCID
This project will evaluate the impact of including Severe Combined Immunodeficiency into the newborn bloodspot screening panel. It will recruit parents and health professionals primarily from the sites where this new form of…
- Trial name
- NCT05651113
- Testing
- SCID Screening
- Where
- United Kingdom
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- King's College London
Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Human Placental-Derived Stem Cell Transplantation
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease
- Trial name
- HPDSC (NCT01586455)
- Testing
- Human Placental Derived Stem Cell
- Where
- 3 sites in United States
- Volunteers
- 43 people
- Sponsor
- New York Medical College
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
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
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
Adenosine Deaminase Deficiency
Evaluation of Adenosine Deaminase (ADA) Enzyme Deficiency in Patients With Lymphopenia and/or Elevated Immunoglobulin E
This study was designed as an observational, prospective, epidemiological screening study. Patients who have been admitted to the center and whose lymphopenia and/or Immunoglobulin E elevation has been detected in at least one…
- Trial name
- NCT05300373
- Testing
- ADENOSINE DEAMINASE TEST
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- TRPHARM
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
Severe Combined Immunodeficiency, Neonatal Screening
Neonatal Screening of Severe Combined Immunodeficiencies
The goal of the proposed research is to observe the prevalence and establish the validity of a newborn screening method for severe combined immunodeficiency (SCID).
- Trial name
- NCT02590328
- Where
- China
- Volunteers
- 180,000 people
- Sponsor
- Children's Hospital of Fudan University
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
Peanut Allergy
ARTEMIS Peanut Allergy In Children
The purpose of this study is to demonstrate the efficacy and safety of AR101 through oral immunotherapy (OIT) in peanut-allergic children.
- Trial name
- ARTEMIS (NCT03201003)
- Testing
- AR101 powder provided in capsules & sachets
- Where
- 18 sites in France, Germany and 5 more
- Volunteers
- 175 people
- Sponsor
- Aimmune Therapeutics, Inc.
Severe Combined Immunodeficiency
Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow Allograft
To investigate the presence of seconday of lymphoid organs in patients who underwent allogenic hematopoietic stem cell transplantation 15 to 45 years ago.
- Trial name
- LymphImaging (NCT04246840)
- Where
- France
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Association Pour l'Aide a la Recherche en Immunologie Pediatrique
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)
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.
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)