Clinical trials: XSCID
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)
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
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)
Saccular and Fusiform Aneurysms, Pseudoaneurysm
Targeting Real World Usage In Stroke Treatment
TRUST Registry is an observational, prospective, long-term, post-market surveillance registry of subjects treated with WallabyPhenox flow modulation devices, stent systems, bifurcation aneurysm implants including their HPC…
- Conditions
- Saccular and Fusiform Aneurysms, Pseudoaneurysm, Vascular Dissection, Perforation
- Trial name
- TRUST (NCT07474935)
- Testing
- Aneurysm Treatment with a Neurovascular Flow Diverter, Dissection Treatment with a Neurovascular Flow Diverter, Perforation Treatment with a Neurovascular Flow Diverter
- Where
- 13 sites in France, Germany and 3 more
- Volunteers
- 5,000 people
- Sponsor
- Phenox GmbH
X-linked Severe Combined Immunodeficiency
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys.
- Trial name
- NCT01175239
- Testing
- Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
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)
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
Intracranial Aneurysm
Diversion-p64 Post Market Clinical Follow-Up Study to Assess Safety and Effectiveness of the p64 Flow Modulation Device
The purpose of this study is to assess safety and effectiveness of the p64 Flow Modulation Device.
- Trial name
- NCT02600364
- Where
- 26 sites in Argentina, Belgium and 8 more
- Volunteers
- 450 people
- Sponsor
- Phenox GmbH
Severe Combined Immunodeficiency Disease, X-linked
Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required for the normal development of the human immune system.
- Trial name
- LVXSCID-ND (NCT01512888)
- Testing
- CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs
- Where
- 3 sites in United States
- Volunteers
- 28 people
- Sponsor
- St. Jude Children's Research Hospital
Severe Combined Immunodeficiency, X Linked, Gene Therapy
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the…
- Trial name
- NCT03311503
- Testing
- autologous CD34+ cell transduced with G2SCID vector
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- David Williams
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)
X-linked Severe Combined Immunodeficiency
Gene Therapy for X-linked Severe Combined Immunodeficiency
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys.
- Trial name
- SCID2 (NCT01410019)
- Testing
- Gene transfer
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
X-linked Severe Combined Immunodeficiency (XSCID)
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
This is a Phase I/II non-randomized clinical trial of ex vivo hematopoietic stem cell (HSC) gene transfer treatment for X-linked severe combined immunodeficiency (XSCID, also known as SCID-X1) using a self-inactivating lentiviral…
- Trial name
- NCT01306019
- Testing
- Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1alpha-hgammac-OPT vector, Busulfan, Palifermin
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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
Immune System Diseases
Developing Newborn Screening for Infants With Primary Immunodeficiency
This study will explore screening for immunodeficiency diseases (diseases that cause problems in fighting infections). There is no method at present to screen all babies at birth for immunodeficiency.
- Trial name
- NCT00113464
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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)
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Severe Combined Immunodeficiency Disease
Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID
Severe combined immunodeficiency (SCID) is a rare disease caused by a group of genetic disorders that leads to early death from recurrent infections in affected children.The only curative therapy for SCID is allogeneic…
- Trial name
- NCT04172181
- Testing
- cord blood stem cell transplantation
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Fudan University
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)