Clinical trials: SCID
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
X-linked Severe Combined Immunodeficiency, X-SCID
Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
Background: X-linked severe combined immunodeficiency (XSCID) is a rare inherited disorder that affects the immune system. It is caused by a change in the IL2RG gene.
- Conditions
- X-linked Severe Combined Immunodeficiency, X-SCID, XSCID
- Trial name
- NCT06851767
- Testing
- Plerixafor, Filgrastim, Palifermin
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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.
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
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Severe Combined Immunodeficiency, Transplacental Maternal Engraftment
Sirolimus Prophylaxis for aGVHD in TME SCID
Study Design: SCID infants receiving an unconditioned haploidentical transplant will be started on Sirolimus (0.05 mg/kg/day) day -5 for Acute Graft-Versus-Host Disease (aGVHD) prophylaxis.
- Conditions
- Severe Combined Immunodeficiency, Transplacental Maternal Engraftment, Stem Cell Transplant
- Trial name
- NCT02177760
- Testing
- Sirolimus
- Where
- United States
- Sponsor
- University of California, San Francisco
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.
Epilepsy
Study to Evaluate the Safety and Efficacy of USL261 (Intranasal Midazolam) in Patients With Seizure Clusters
The purpose of this study is to examine the safety and effectiveness of USL261 for the outpatient treatment of seizure clusters.
- Trial name
- ARTEMIS1 (NCT01390220)
- Testing
- USL261
- Where
- 90 sites in United States, Australia and 9 more
- Volunteers
- 292 people
- Sponsor
- UCB Biopharma S.P.R.L.
Anemia, Aplastic, Fanconi Anemia
Cord Blood Stem Cell Transplantation Study (COBLT)
To evaluate if HLA-mismatched, unrelated-donor umbilical cord blood stem and progenitor cell units (UCBU) offered a clinically acceptable alternative to matched unrelated-donor allogeneic bone marrow for transplantation with…
- Conditions
- Anemia, Aplastic, Fanconi Anemia, Hematologic Diseases, Leukemia
- Trial name
- NCT00000603
- Testing
- stem cell transplantation
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID)
Evaluation of Patients With Immune Function Abnormalities
This study will evaluate patients with abnormal immune function that results in recurrent or unusual infections or chronic inflammation.
- Conditions
- Chronic Granulomatous Disease (CGD), X-Linked Severe Combined Immune Deficiency (XSCID), Leukocyte Adhesion Deficiency 1 (LAD), Graft Versus Host Disease (cGvHD)
- Trial name
- NCT00128973
- Where
- United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- 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
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
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)
Severe Combined Immunodeficiency
Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
This is a clinical trial of gene therapy for X-linked severe combined immunodeficiency (XSCID), a genetic disease caused by defects in a protein called the common gamma chain, which is normally on the surface of immune cells…
- Trial name
- NCT00028236
- Testing
- Gene-Transduced Autologous CD34+ Stem Cells
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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)
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Severe Combined Immunodeficiency, Malignancy, Hematologic
Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
This protocol (GENEFU) provides a mechanism for the 15-year followup period that the FDA requires for all participants in gene transfer protocols and assures that adequate followup can be maintained for a wide variety of…
- Conditions
- Severe Combined Immunodeficiency, Malignancy, Hematologic, Neuroblastoma, Neoplasm
- Trial name
- NCT00695279
- Testing
- Venipuncture
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- St. Jude Children's Research Hospital
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
Leukemia, Aplastic Anemia
Methadone as an Alternative Treatment for Children Underdoing HSCT
Mucositis is a normal side effect of stem cell transplant which happens as a result of chemotherapy being given prior to a new donor cell infusion (bone marrow transplant).
- Conditions
- Leukemia, Aplastic Anemia, Bone Marrow Failure Syndromes, SCID
- Trial name
- MATCH (NCT06940570)
- Testing
- PCA plus methadone (Experimental), PCA Alone (SOC)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of Texas Southwestern Medical Center