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OpenTrials
Active, Not Recruiting

NCT Number: NCT01821781

Immune Disorder HSCT Protocol

This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Immune Deficiency Disorders 22q11 Deletion Syndrome Abnormalities, Multiple Albinism Autoimmune Diseases Autoimmune Lymphoproliferative Syndrome Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Blood Protein Disorders Bruton type agammaglobulinemia Cardiovascular Abnormalities Cardiovascular Diseases Chediak-Higashi Syndrome Chromosome Disorders Chronic Disease Chronic Granulomatous Disease Common Variable Immune Deficiency Common Variable Immunodeficiency Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Craniofacial Abnormalities Cytopenia DNA Repair-Deficiency Disorders DiGeorge Syndrome Disease Attributes Dysgammaglobulinemia Endocrine System Diseases Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Genetic Diseases, X-Linked Granulomatous Disease, Chronic Heart Defects, Congenital Heart Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemophagocytic Lymphohistiocytosis Hemorrhagic Disorders Histiocytosis Histiocytosis, Non-Langerhans-Cell Hyper-IgM Hyper-IgM Immunodeficiency Syndrome Hypoparathyroidism IPEX Immune Dysregulatory Disorders Immune System Diseases Immunologic Deficiency Syndromes Immunoproliferative Disorders Infant, Newborn, Diseases Leukocyte Disorders Leukopenia Lymphatic Abnormalities Lymphatic Diseases Lymphohistiocytosis, Hemophagocytic Lymphopenia Lymphoproliferative Disorders Metabolic Diseases Musculoskeletal Abnormalities Musculoskeletal Diseases Nutritional and Metabolic Diseases Parathyroid Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Phagocyte Bactericidal Dysfunction Primary Immunodeficiency Diseases Severe Combined Immunodeficiency Wiskott-Aldrich Syndrome X-linked Agammaglobulinemia X-linked Lymphoproliferative Syndrome

Age range

Up to 21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Washington University

St Louis, Missouri, 63110, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • </= 28 years of age
  • Performance status >/= 40
  • DLCO >/= 40%
  • LVEF >/=40% or LVSF >/=26%
  • Serum creatinine < 2x ULN
  • Liver enzymes </= 5x ULN
  • Negative pregnancy test
  • Suitably matched donor (6/6 matched sib UCB, 8/8 matched sib BM or PBSC, 5-6/6 matched unrelated UCB, 7-8/8 matched unrelated BM, double cord)

Exclusion criteria

  • Known diagnosis of HIV I/II
  • Pregnant or breastfeeding
  • Uncontrolled invasive fungal or bacterial infections within 1 month prior to starting alemtuzumab
  • Uncontrolled viral infection within 1 week prior to starting alemtuzumab

Treatment and study plan

Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan

Drug

Between days -23 and -15: alemtuzumab test dose, 3mg IV or SQ Day -14: alemtuzumab, 10mg IV or SQ Day -13: alemtuzumab, 15mg IV or SQ Day -12: alemtuzumab, 20mg IV or SQ Days -8 to -4: fludarabine, 30mg/m2 IV Day -4: thiotepa 4mg/kg IV q 12 hours Day -3: melphalan, 140mg/m2 IV Day 0: stem cell infusion Day +7: G-CSF

Primary outcomes

  1. Number of participants with donor engraftment

    Time frame: 1 year post transplant

Secondary outcomes

  1. Major Transplant Related Toxicities

    Time frame: 1 years post transplant

  2. Time to neutrophil recovery

    Time frame: within 100 days post transplant

  3. Number of patient with acute GVHD

    Time frame: 180 days post transplant

  4. Number of participants with infectious complications

    Time frame: 2 years post transplant

  5. Time to immune reconstitution

    Time frame: 2 years post transplant

  6. Overall survival

    Time frame: 2 years post transplant

  7. Time to platelet recovery

    Time frame: within 100 days post transplant

  8. Number of patients with chronic GVHD

    Time frame: 2 years post transplant

  9. Disease free survival

    Time frame: 2 years post transplant

Sponsors and collaborators

Lead sponsor

Washington University School of Medicine

Other

Registry information

Official study title

A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime

Important dates

Study start
2013
Primary completion
2025
Study completion
2026
First posted
Apr 1, 2013
Registry last updated
Feb 19, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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