Phase 2 N=26 Thymus Transplantation in DiGeorge Syndrome #668
DiGeorge Syndrome · Complete Typical DiGeorge Anomaly
Primary: Survival at 1 Year Post-Cultured Thymus Tissue Implantation (CTTI) — 72 % of participants who survive to 1 year
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Phase 2 N=7 Thymus Transplantation Dose in DiGeorge #932
DiGeorge Anomaly · DiGeorge Syndrome · Complete DiGeorge Anomaly
Primary: Survival at 1 Year Post-CTTI — 83 % of participants who survive to 1 year
N/A N=29 Thymus Transplantation Safety-Efficacy
Complete DiGeorge Anomaly · DiGeorge Syndrome · DiGeorge Anomaly
Primary: Survival Rate at End of 1 Year — 79.3 percentage of participants
Phase 2 N=14 Phase I/II Thymus Transplantation With Immunosuppression #950
DiGeorge Anomaly · Complete DiGeorge Anomaly · Complete Atypical DiGeorge Anomaly
Primary: Survival at 1 Year Post-CTTI — 71 % of participants who survive to 1 year
Phase 2 N=37 NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
22q11 Deletion Syndrome
Primary: Safety and Tolerability of NB-001 — 18; 24; 14; 20 Participants
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Phase 2 N=10 MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe Combined Immunodeficiency
Primary: Number of Participants With Adverse Events — 10; 9; 0; 10 participants
Phase 2 N=15 Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease
Chronic Granulomatous Disease
Primary: Percentage of Participants With Engraftment — 100 percentage of participants
Phase 2 N=19 Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
22q13 Deletion Syndrome · Phelan-McDermid Syndrome
Primary: Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1 — 15.78; 11.22; 7.61; 9.70 score on a scale
Phase 3 N=98 TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID
Primary Immune Deficiency Disorder · Hematopoietic Stem Cell Transplantation
Primary: Overall Survival — 86; 86; 87 percentage of survival probability — p=0.95
Phase 2 N=47 Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of participants
Phase 2 N=47 Tofacitinib for Immune Skin Conditions in Down Syndrome
Down Syndrome · Alopecia Areata · Atopic Dermatitis / Eczema
Primary: Number of Serious Adverse Events (SAE) Definitely Related to Tofacitinib Treatment. — 0 Serious Adverse Events
Phase 2 N=12 ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID
Immunologic Deficiency Syndromes
Primary: Survival — 12 Participants
Phase 2 N=48 Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
Myelodysplastic Syndrome · Aplastic Anemia
Primary: Overall Response — 8; 8; 4 participants
Phase 2 N=25 Effects Of Oral Alfacalcidol On Maturation Of Dendritic Cells In Graves' Disease Patients
Graves Disease
Primary: CD80 Level — 45.60; 54.51 nM — p=0.48
Phase 3 N=36 Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Dyskeratosis Congenita · Aplastic Anemia
Primary: Neutrophil Engraftment — 15; 20 Participants
Phase 2 N=6 Growth Hormone Treatment in Children With Phelan McDermid Syndrome
Phelan McDermid Syndrome
Primary: ABC - Social Withdrawal Subscale — 10.31; 4.6; 14.8; 6.2 score on a scale
Phase 3 N=37 Study of Efficacy of CDZ173 in Patients With APDS/PASLI
Common Variable Immunodeficiency (CVID), APDS / PASLI
Primary: Part I: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — 2; 0; 2; 0 Participants
Early Phase 1 N=48 Donor Stem Cell Transplantation for Congenital Immunodeficiencies
Inherited Immune Deficiencies
Primary: Stem Cell Transplant Engraftment — 7; 31; 3; 0 Participants
Phase 2 N=42 Antithymocyte Globulin and Cyclosporine to Treat Myelodysplasia
Myelodysplastic Syndrome
Primary: Red Blood Cell Transfusion Independence — 6; 10; 21 participants
Phase 2 N=89 ATG-GCSF in New Onset Type 1 Diabetes
Diabetes Mellitus, Type 1
Primary: Change in Area Under the Stimulated C-peptide Curve From Baseline to 12 Months. — 0.528; 0.646; 0.406 nmol/L
Phase 2 N=2 Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases
Bone Marrow Failure Syndromes · Immunodeficiencies · Immune Dysregulation Syndromes
Primary: Number of Participants With Engraftment — 2 Participants
Phase 2 N=46 Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene
ADA-SCID
Primary: Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year) — 100; 85.71; 100; 92.31 percentage of participants
N/A N=60 Auto-antibodies Prevalence and CD1 Role in Gaucher Disease
Gaucher Disease
Primary: Number of Patients With GD Diagnosis Confirmed by : Enzyme Testing of acidβ-glucosidase Activity Activity <15% in Blood Leucocytes Completed When Necsssary by GB1…
N/A N=257 Down Syndrome Metabolic Health Study
Down Syndrome · Trisomy 21
Primary: Non-HDL Cholesterol — 128; 107 mg/dl
Phase 2 N=8 Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Severe Combined Immunodeficiency
Primary: CD3 Cell Count Post Infusion — 5; 3 Participants
Phase 2 N=20 Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
Aplastic Anemia
Primary: Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months — 9 participants
Phase 2 N=19 Stem Cell Transplant for Immunologic or Histiocytic Disorders
Hemophagocytic Lymphohistiocytosis · X-Linked Lymphoproliferative Disorders · Chediak-Higashi Syndrome
Primary: Number of Subjects With Mixed Chimerism — 3; 8; 4 participants
Phase 3 N=205 Combination Chemotherapy in Treating Young Patients With Down Syndrome and Acute Myeloid Leukemia or Myelodysplastic Syndromes
Childhood Acute Basophilic Leukemia · Childhood Acute Eosinophilic Leukemia · Childhood Acute Erythroleukemia (M6)
Primary: Event-free Survival (EFS) at 3 Years — 90.1 percentage
Phase 1 N=16 Safety, Tolerability and Immunogenicity of ACI-24 Vaccine in Adults With Down Syndrome
Down Syndrome
Primary: Antibody Titer (Serum Anti-Aβ1-42 Free IgG) - Mean Absolute Value — 632.3; 335.4; 162.8; 751.5 AU/mL (AU: Arbitrary Unit)