Combined Immunodeficiency
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Genetic Basis of Immunodeficiency
This update describes an NHLBI-funded study investigating the genetic basis of severe combined immunodeficiency (SCID) that is currently rec…
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CLINICAL_TRIALS trial_result · 2026-04-21Clinical Trial Update: Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector [Phase 1, Phase 2]
Phase 1/2 clinical trial results for gene transfer in X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating gammaret…
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CLINICAL_TRIALS research · 2026-02-19Clinical Trial Update: Immune Disorder HSCT Protocol [Phase 2]
Phase 2 clinical trial update on a hematopoietic stem cell transplant protocol for several inherited immune deficiency disorders; the study …
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts
This clinical trial update describes expanded access to CD34+ selection using the Miltenyi CliniMACS Prodigy for patients receiving peripher…
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CLINICAL_TRIALS trial_result · 2026-02-17Clinical Trial Update: Conditioning SCID Infants Diagnosed Early [Phase 2]
A Phase 2 clinical trial for conditioning in infants with severe combined immunodeficiency diagnosed early is currently active but not recru…
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CLINICAL_TRIALS research · 2026-02-13Clinical Trial Update: Autologous Gene Therapy for Artemis-Deficient SCID [Phase 1, Phase 2]
Clinical trial update for autologous Artemis-deficient SCID gene therapy, currently recruiting in Phase 1/Phase 2.
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CLINICAL_TRIALS trial_result · 2026-01-15Clinical Trial Update: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies [NA]
This clinical trial investigates allogeneic hematopoietic stem cell transplantation as a treatment for primary immune deficiencies, includin…
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EMA approval · 2025-12-18EMA Authorised: Strimvelis (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells) — Fondazione Telethon ETS
EMA authorised Strimvelis for the treatment of severe combined immunodeficiency due to ADA deficiency (ADA-SCID); the marketing authorizatio…
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CLINICAL_TRIALS trial_result · 2025-12-15Clinical Trial Update: Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases [Phase 1, Phase 2]
This clinical trial update describes an early-phase study evaluating sequential cadaveric lung transplantation followed by bone marrow trans…
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CLINICAL_TRIALS withdrawal · 2025-12-01Clinical Trial Update: Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating busulfan-based conditioning with h-ATG, radiation, and sirolimus for matched related/unrelated donor s…
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CLINICAL_TRIALS research · 2025-12-01Clinical Trial Update: Study of Populations at Risk of Developing Chronic Hepatitis Linked to Chronic Enteric Virus Infection in Patients With Primary Immunodeficiency and Secondary Humoral Deficiency
This clinical trial update indicates the study is currently recruiting to evaluate populations at risk of developing chronic hepatitis linke…
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CLINICAL_TRIALS trial_result · 2025-10-23Clinical Trial Update: Methadone as an Alternative Treatment for Children Underdoing HSCT [Phase 3]
Phase 3 trial evaluating methadone as an alternative treatment for children undergoing hematopoietic stem cell transplantation; the study is…
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CLINICAL_TRIALS trial_result · 2025-09-09Clinical Trial Update: SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating an intravenous lentiviral gene therapy (Ivlv-X1) for X-linked severe combined immunodeficiency (SCI…
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CLINICAL_TRIALS trial_result · 2025-07-28Clinical Trial Update: JSP191 Antibody Targeting Conditioning in SCID Patients [Phase 1, Phase 2]
Jasper Therapeutics' JSP-191 (anti-CD117 antibody) used as conditioning for hematopoietic stem cell transplantation in severe combined immun…
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CLINICAL_TRIALS research · 2025-07-01Clinical Trial Update: Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating a haploidentical transplant approach using TCRα/β depletion followed by CD45RA-depleted donor lymphocy…
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CLINICAL_TRIALS trial_result · 2024-10-31Clinical Trial Update: Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases [Phase 2, Phase 3]
Clinical trial update reports Phase 2 and Phase 3 results for risk-adapted allogeneic stem cell transplantation in non-malignant diseases wi…
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CLINICAL_TRIALS trial_result · 2024-10-30Clinical Trial Update: Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Long-term follow-up results are reported for patients who received gene therapy or gene-marked products across several conditions, including…
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CLINICAL_TRIALS research · 2024-08-21Clinical Trial Update: Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation [NA]
This ClinicalTrials.gov update reports a completed feasibility study evaluating an adapted physical activity program for children, adolescen…
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CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating conditioning regimens for allogeneic hematopoietic stem-cell transplantation has completed recruitment…
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CLINICAL_TRIALS trial_result · 2022-09-27Clinical Trial Update: Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients [Phase 1, Phase 2]
Clinical trial update on HTLP injection after partially HLA-compatible HSCT in SCID patients, encompassing Phase 1 and Phase 2, with unknown…
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CLINICAL_TRIALS trial_result · 2020-04-16Clinical Trial Update: EZN-2279 in Patients With ADA-SCID [Phase 3]
Phase 3 trial update for EZN-2279 in ADA-SCID has completed; results posted in 2020.
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CLINICAL_TRIALS trial_result · 2017-09-18Clinical Trial Update: SCID Bu/Flu/ATG Study With T Cell Depletion [Phase 1, Phase 2]
Phase 1/2 trial update for SCID using Bu/Flu/ATG with T cell depletion; study terminated with posted results.
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) [Phase 1]
Phase 1 clinical trial update for a stem cell gene therapy targeting X-linked severe combined immunodeficiency. The study status indicates t…
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID
Clinical trial update on a NHGRI-funded study evaluating how female adolescents decide whether to pursue carrier testing for X-linked severe…
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CLINICAL_TRIALS trial_result · 2016-06-17Clinical Trial Update: Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells [Phase 2]
Phase 2 study evaluating related hematopoietic stem cell transplantation for several genetic blood disorders; the trial was terminated and r…