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Severe Combined Immunodeficiency clinical trials

View clinical trials related to Severe Combined Immunodeficiency.

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NCT ID: NCT04959890 Enrolling by invitation - Clinical trials for Severe Combined Immunodeficiency Due to ADA Deficiency

Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Start date: April 23, 2021
Study type: Observational

To evaluate accuracy and precision of retroviral insertion site (RIS) analysis and its utility for investigating and predicting the potential for insertional oncogenesis in subjects treated with Strimvelis.

NCT ID: NCT04797260 Recruiting - Clinical trials for Severe Combined Immunodeficiency Due to RAG1 Deficiency

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID

Start date: November 1, 2021
Phase: N/A
Study type: Interventional

This study is a prospective, non-randomized, open-label, two-centre phase I/II intervention study designed to treat children up to 24 months of age with RAG1-deficient SCID with an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The study involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter called RAG1 LV CD34+ cells) in five patients with RAG1-deficient SCID.

NCT ID: NCT04370795 Enrolling by invitation - Clinical trials for Severe Combined Immunodeficiency (SCID)

Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus

Start date: September 24, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

Background: Severe combined immune deficiency (SCID) is a group of conditions where the immune system does not work properly. The only cure for most SCIDs is a stem cell transplant (getting cells from a donor). These transplants can have serious complications. Before the transplant, people often get high doses of drugs and radiation to prepare the body to accept the cells from the donor. Researchers want to see if low doses of drugs alone without radiation work just as well as low doses of drugs with radiation for SCID patients getting stem cell transplants. Objective: To test a set of drugs with or without radiation given before a stem cell transplant. Eligibility: People ages 3-40 who have SCID and who have a stem cell donor - either related or unrelated. Design: Participants will be admitted to the hospital 10 days before transplant. They will undergo: medical history medication review physical exam blood and urine tests (may include a 24-hour urine collection) heart, lung, and breathing tests imaging scans bone marrow sample nutrition assessment dental exam eye exam meeting with a social worker. Participants will get a plastic port called a central line. It is a hollow tube that is placed in the upper chest. It will be used to give medicines and take blood. All participants will take chemotherapy drugs. Some will get radiation. Participants will have a stem cell transplant. They will get the cells as an infusion through their central line. They will stay in the hospital for 30 days after transplant. Participants must stay within 1 hour of NIH for 3 months after transplant. During this time, they will have follow-up visits at NIH at least once a week. Then they will have follow-up visits once or twice a year for 5-6 years.

NCT ID: NCT04331483 Withdrawn - Clinical trials for Myelodysplastic Syndromes

A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts

Start date: December 8, 2018
Phase: N/A
Study type: Interventional

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, bone marrow aplasia, severe combined immunodeficiency). Despite the major therapeutic progress made over the last 50 years, particularly in terms of supportive care, post-transplant morbidity and mortality remains high. Infectious complications, whose incidence varies between 30 and 60%, are the first cause of mortality in the immediate post-transplant period. In order to protect the patient from the occurrence of severe infectious episodes, aHSCTmust be performed in a highly protected environment (positive pressure chambers). This has implications for the experience and impact of hospitalization on the patient and family. This is particularly true in paediatrics, whether in children, adolescents or young adults, where it is not only the patient's quality of life that is at stake, but also their emotional and psychomotor development. In these patients, prolonged hospitalization (at least 6 weeks) in a sterile room will be responsible for physical deconditioning accompanied by a decrease in muscle mass, itself concomitant with undernutrition, and an increase in sedentary lifestyle. This prolonged hospitalisation in a sterile room, associated with myeloablative treatments, is therefore the cause of social isolation of patients, but it is also often synonymous with physical inactivity leading to a rapid decrease in physical condition, quality of life and an increase in fatigue. Today, the benefits of physical activity (PA) during and after cancer treatment have been widely demonstrated. The objective is to evaluate the feasibility of an adapted physical activity program during the isolation phase for achieving aHSCT in children, adolescents and young adults. This is a prospective, interventional, monocentric cohort study conducted at the Institute of Paediatric Haematology and Oncology in Lyon. The intervention will take place during the isolation phase and consists of an adapted physical activity (APA) program defined at inclusion, integrating supervised sessions with an APA teacher, as well as autonomous sessions. The program is individualized according to age, aerobic capacity, and PA preferences. Sessions are also tailored to the biological, psychological, and social parameters of patients. The total duration of the intervention is 3 months. To date, no PA studies have been performed in patients under 21 years of age requiring aGCSH during the sterile isolation phase. EVAADE will therefore be the first study in this population to offer an innovative procedure with a connected device.

NCT ID: NCT04286815 Recruiting - Gene Therapy Clinical Trials

Gene Therapy for X Linked Severe Combined Immunodeficiency

Start date: May 1, 2020
Phase: N/A
Study type: Interventional

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 followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.

NCT ID: NCT04246840 Completed - Clinical trials for Severe Combined Immunodeficiency

Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow Allograft

Start date: January 13, 2020
Study type: Observational

To investigate the presence of seconday of lymphoid organs in patients who underwent allogenic hematopoietic stem cell transplantation 15 to 45 years ago. The goal aims to assess the development of seconday lymphoid organs given the fact that the absence of myeloablation these patients present a split chimerism between T lymphocytes and the other leucocytes. Thus, they may not be able to generate seconday lymphoid organs. Practically, whole body MRI is being used to visualise and quantify both mediastinal and intraabdominal lymph nodes. Delta will be compared with those obtained in healthy age-matched individuals. It is scheduled to include 15 patients and 15 controls.

NCT ID: NCT04172181 Active, not recruiting - Clinical trials for Severe Combined Immunodeficiency Disease

Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID

Start date: December 1, 2019
Study type: Observational

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 hematopoietic stem cell transplantation.Unrelated umbilical cord blood(UCB) is increasingly used as an alternative to bone marrow.

NCT ID: NCT04140539 Suspended - Clinical trials for Severe Combined Immunodeficiency Due to ADA Deficiency

A Clinical Study to Enable Process Validation of Commercial Grade OTL-101

Start date: October 15, 2019
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of the current study is to treat at least 3 ADA-SCID patients with OTL-101 prepared by the commercial manufacturing process.

NCT ID: NCT04049084 Enrolling by invitation - Clinical trials for Adenosine Deaminase Deficiency

An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID

Start date: September 26, 2019
Study type: Observational

This observational long-term follow-up study is designed to collect safety and efficacy data from ADA-SCID patients previously treated with autologous ex vivo gene therapy products based on the EFS-ADA LV encoding for human adenosine deaminase (ADA) gene (EFS-ADA LV), as part of the OTL-101 clinical development program. No investigational medicinal product will be administered to these patients as part of the OTL-101-6 study.

NCT ID: NCT03879876 Recruiting - Pediatric Patients Clinical Trials

Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients

HTLP Necker
Start date: May 13, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients.