Severe combined immunodeficiency
MONDO:0015974Severe combined immunodeficiency (SCID) comprises a group of rare monogenic primary immunodeficiency disorders characterized by a lack of functional peripheral T lymphocytes resulting in early-onset severe respiratory infections and failure to thrive. They are classified according to immunological phenotype into SCID with absence of T cells but presence of B cells (T-B+ SCID) or SCID with absence of both (T-B- SCID). Both of these groups include several forms, with or without natural killer (NK) cells.
Also known as: SCID, severe combined immunodeficiency, severe combined immunodeficiency (disease), severe combined immunodeficiency disease
48 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
-
Gene therapy offers hope for babies with 'Bubble Boy' disease
⭐️ CURE ⭐️ Recruiting nowThis study tests a new gene therapy for children with a severe immune disorder called Artemis-SCID, where the body cannot fight infections. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and put the corrected cells back. The goal is to rebuild a work…
Phase: PHASE1, PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: ⭐️ CURE ⭐️
Last updated Jun 27, 2026 12:00 UTC
-
New gene therapy offers hope for older kids and adults with 'bubble boy' disease
Disease control Recruiting nowThis study tests a gene therapy for people aged 2 to 50 with X-linked severe combined immunodeficiency (SCID-X1), a condition that leaves them without a working immune system. Participants receive their own blood stem cells that are genetically corrected in a lab, then infused ba…
Phase: PHASE1, PHASE2 • Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
-
One-Time gene fix could rescue 'Bubble Boy' disease
Disease control Recruiting nowThis study tests a gene therapy given by IV for ADA-SCID, a rare and life-threatening immune disorder. The treatment uses a modified virus to deliver a working gene, aiming to restore the immune system. Ten participants will be monitored for safety and immune recovery over one ye…
Phase: NA • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
-
Bubble boy disease gene therapy trial launches
Disease control Recruiting nowThis trial tests a gene therapy given by IV for X-linked severe combined immunodeficiency (SCID-X1), a rare immune disorder. Ten participants with no matched donor will receive a lentiviral vector to correct the genetic defect. The goal is to restore immune function and improve s…
Phase: PHASE1, PHASE2 • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
-
New bone marrow transplant trial offers hope for kids with severe blood disorders
Disease control Recruiting nowThis study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…
Phase: PHASE1, PHASE2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
-
Stem cell transplant offers new hope for rare immune diseases
Disease control Recruiting nowThis study tests a stem cell transplant for people with common variable immunodeficiency (CVID) and other immune disorders. The goal is to replace the faulty immune system with a healthy donor's stem cells. Participants are aged 5 to 40 and will receive a reduced-intensity transp…
Phase: PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 12:33 UTC
-
Bubble boy disease: gene therapy trial offers hope for immune system repair
Disease control Recruiting nowThis trial tests a gene therapy for children with ADA-SCID, a severe immune disorder often called 'bubble boy disease.' Doctors take the child's own blood stem cells, add a working gene, and return them via infusion. The goal is to restore immune function and improve survival. Th…
Phase: PHASE1, PHASE2 • Sponsor: University of California, Los Angeles • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
-
Could longer or stronger antivirals beat COVID in vulnerable patients?
Disease control Recruiting nowThis study looks at whether giving a longer course (10 days instead of 5) or adding another antiviral (remdesivir) to the standard Paxlovid treatment helps immunocompromised people clear COVID-19 faster. About 256 adults with mild or no symptoms will be randomly assigned to diffe…
Phase: PHASE2 • Sponsor: ANRS, Emerging Infectious Diseases • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
-
New stem cell filter could make transplants safer for vulnerable patients
Disease control AVAILABLEThis expanded access program offers a special stem cell purification technique (CD34+ selection) for patients undergoing stem cell transplants. The method removes many T-cells from the donated stem cells, which lowers the risk of a serious complication called graft-versus-host di…
Sponsor: University of Florida • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
-
Gene therapy offers new hope for babies with rare 'Bubble Boy' disease
Disease control Recruiting nowThis study tests a gene therapy for Artemis-deficient severe combined immunodeficiency (ART-SCID), a life-threatening immune disorder. It involves taking the patient's own blood stem cells, adding a working copy of the faulty gene, and infusing them back after mild chemotherapy. …
Phase: PHASE1, PHASE2 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
-
Milder chemo before stem cell transplant shows promise for rare blood disorders
Disease control Recruiting nowThis study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…
Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
-
Milder transplant method aims to help young patients with rare blood diseases
Disease control Recruiting nowThis study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…
Phase: PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
-
Gene therapy offers hope for children with SCID-X1
Disease control Recruiting nowThis trial tests a gene therapy for children with X-linked severe combined immunodeficiency (SCID-X1), also known as 'bubble boy' disease. Participants receive a low dose of busulfan followed by an infusion of their own blood stem cells that have been genetically corrected with a…
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
-
Half-Matched stem cells give bone marrow a jump start for High-Risk patients
Disease control AVAILABLEThis program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…
Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC
-
Could a simple gas ease breathing for spinal cord injury patients?
Symptom relief Recruiting nowThis study tests whether daily treatments with slightly elevated carbon dioxide (hypercapnia) can improve breathing and reduce sleep apnea in Veterans with spinal cord injury. About 30 adults with chronic spinal cord injury or able-bodied people with sleep apnea will receive the …
Phase: NA • Sponsor: VA Office of Research and Development • Aim: Symptom relief
Last updated Jun 27, 2026 12:03 UTC
-
Massive gene hunt aims to unlock secrets of immune disorders
Knowledge-focused Recruiting nowThis study is looking for the genes that cause certain immune system problems. Researchers want to understand why some people get sick and others don't, even in the same family. Up to 5,000 people with immune disorders and their relatives will give blood or cheek cell samples for…
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC
-
Massive study aims to unravel mysteries of blood clots and vessel disease
Knowledge-focused Recruiting nowThis study aims to learn more about how diseases related to blood clots, the immune system, and blood vessels start and change over time. Researchers will enroll up to 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and healthy volun…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC
-
Scientists launch major study to track rare immune diseases
Knowledge-focused Recruiting nowThis study is observing up to 3,500 people with immune system problems, including rare genetic conditions like chronic granulomatous disease and severe combined immunodeficiency. Researchers will track how these diseases change over time through blood tests, imaging, and checkups…
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC
-
New study aims to spot lung trouble before it starts in transplant patients
Knowledge-focused Recruiting nowThis study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:01 UTC
-
Scientists hunt for genetic clues behind rare immune disorder
Knowledge-focused Recruiting nowThis study aims to find the genetic causes of severe combined immunodeficiency (SCID), a rare condition where the immune system doesn't work properly. Researchers will collect blood samples from 100 patients and their relatives to analyze DNA and other factors. The goal is to bet…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
-
Can a simple tool give kids a voice in their own transplant care?
Knowledge-focused Recruiting nowThis study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …
Phase: NA • Sponsor: Washington University School of Medicine • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:02 UTC
-
New study to reveal hidden weaknesses in vaccines for Immune-Compromised patients
Knowledge-focused Recruiting nowThis study looks at how well vaccines against respiratory viruses (like flu or COVID-19) work in people with weakened immune systems, including those with blood cancers. Researchers will measure both antibody and T cell responses after vaccination. The goal is to understand why v…
Sponsor: University of Cologne • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:32 UTC
-
Massive data hunt aims to unlock secrets of stem cell transplants
Knowledge-focused Recruiting nowThis study gathers basic health information from 2000 people receiving stem cell transplants for blood cancers or immune disorders. The goal is to track survival and complications to help doctors and researchers improve future treatments. Participants must be getting a standard t…
Sponsor: Wake Forest University Health Sciences • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:06 UTC
-
Can a simple questionnaire predict who will follow their treatment?
Knowledge-focused Recruiting nowThis study is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…
Phase: NA • Sponsor: Technical University of Madrid • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:01 UTC
-
Massive new registry aims to unlock secrets of biologic drugs
Knowledge-focused Recruiting nowThis study is creating a large registry of 800 people receiving biologic medications for immune conditions like autoimmune diseases and primary immune deficiencies. Researchers will collect blood samples and health data to better understand how these treatments work and who respo…
Sponsor: Icahn School of Medicine at Mount Sinai • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC
-
Gut virus linked to liver damage in immune-deficient patients?
Knowledge-focused Recruiting nowThis study looks at 120 people with various immune deficiencies to see if a chronic gut virus infection is tied to liver or digestive problems. Researchers will collect blood, stool, urine, and biopsy samples to screen for enteric viruses. The goal is to identify which patients a…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:00 UTC
-
New registry aims to unlock secrets of rare dwarfism disorders
Knowledge-focused Recruiting nowThis study creates a registry to collect health information from 200 people with rare forms of primordial dwarfism, such as MOPDII and Meier-Gorlin syndrome. Researchers hope to learn how these conditions change over a person's lifetime and improve future care. Participants provi…
Sponsor: Nemours Children's Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:52 UTC