RecruitingNCT00128973
Immune
Severe Combined Immunodeficiency
Also known as SCID, bubble boy disease, ADA-SCID, X-linked SCID
Severe Combined Immunodeficiency encompasses a heterogeneous group of inherited disorders marked by profound defects in both T-cell and B-cell immunity, rendering affected infants susceptible to life-threatening infections by bacteria, viru
ORPHA:183660 ↗Gene IL2RGGene ADAGene RAG1Gene RAG2 (multiple)Prevalence 1 in 50,000-100,000 live birthsOnset Neonatal to infancy (typically first 3-6 months of life)Combined primary immunodeficiency
12
studies recruiting now
as of 7 Sept 2026
116
studies registered in total
as of 7 Sept 2026
2
countries with a recruiting site
as of 7 Sept 2026
16 Dec 2025
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT00055172
Genetic Basis of Immunodeficiency
RecruitingPhase 1 / Phase 2NCT01306019
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
RecruitingPhase 2NCT07284641
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
RecruitingPhase 1 / Phase 2NCT05071222
Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE)
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 12 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Severe Combined Immunodeficiency
Severe Combined Immunodeficiency encompasses a heterogeneous group of inherited disorders marked by profound defects in both T-cell and B-cell immunity, rendering affected infants susceptible to life-threatening infections by bacteria, viruses, and fungi. Newborn screening programs have enabled earlier identification, dramatically improving outcomes when hematopoietic stem cell transplantation or gene therapy is initiated before infectious complications occur. Without definitive treatment, SCID is uniformly fatal in early childhood.
Common clinical features
Recurrent and severe infections beginning in infancyFailure to thrive and poor weight gainChronic diarrhea and oral candidiasisAbsent or minimal lymph nodes and tonsilsOpportunistic infections (Pneumocystis, CMV)Severe reactions to live viral vaccinesMaternal engraftment graft-versus-host disease
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
2 approved treatments and 8 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Autologous Cd34+ Enriched Cell Fraction That Contains Cd34+ Cells Transduced With Retroviral Vector That Encodes For The Human Ada Cdna Sequence (Strimvelis)Approved: Elapegademase (Revcovi)
Phase 3Simoladagene Autotemcel
Phase 2Busulfan (Busilvex)
Phase 2Sirolimus (Fyarro)
Phase 2Alemtuzumab (Campath mabcampath)
Phase 1/2Pegademase Bovine (Adagen)
Phase 1/2Briquilimab
Phase 1Filgrastim (Accofil)
Phase 1Zidovudine (Retrovir)
Before you apply
Things trial teams commonly ask about for Severe Combined Immunodeficiency. Not eligibility rules; those are set by each study.
- Gene therapy trials for ADA-SCID and X-linked SCID typically enroll infants without a matched sibling donor; confirm HLA typing results and donor search status before applying
- Most interventional trials require patients to be free of active infection at enrollment; timing of application relative to current infectious status is critical
- Newborn screening detection status (positive vs. incidental diagnosis) may affect eligibility in natural history studies — report NBS results accurately
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).