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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT01821781
Other study ID # 201301135
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date March 2013
Est. completion date March 2027

Study information

Verified date August 2023
Source Washington University School of Medicine
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Recruiting
Enrollment 20
Est. completion date March 2027
Est. primary completion date March 2027
Accepts healthy volunteers No
Gender All
Age group N/A to 21 Years
Eligibility 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

Study Design


Related Conditions & MeSH terms

  • Agammaglobulinemia
  • Autoimmune Lymphoproliferative Syndrome
  • Chediak-Higashi Syndrome
  • Chronic Granulomatous Disease
  • Common Variable Immune Deficiency
  • Common Variable Immunodeficiency
  • DiGeorge Syndrome
  • Granulomatous Disease, Chronic
  • Hemophagocytic Lymphohistiocytosis
  • Hyper-IgM
  • Immune Deficiency Disorders
  • Immune Dysregulatory Disorders
  • Immunologic Deficiency Syndromes
  • IPEX
  • Lymphohistiocytosis, Hemophagocytic
  • Lymphoproliferative Disorders
  • Severe Combined Immunodeficiency
  • Syndrome
  • Wiskott-Aldrich Syndrome
  • X-linked Agammaglobulinemia
  • X-linked Lymphoproliferative Syndrome

Intervention

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

Locations

Country Name City State
United States Washington University Saint Louis Missouri

Sponsors (1)

Lead Sponsor Collaborator
Washington University School of Medicine

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of participants with donor engraftment 1 year post transplant
Secondary Major Transplant Related Toxicities 1 years post transplant
Secondary Time to neutrophil recovery within 100 days post transplant
Secondary Number of patient with acute GVHD 180 days post transplant
Secondary Number of participants with infectious complications 2 years post transplant
Secondary Time to immune reconstitution 2 years post transplant
Secondary Overall survival 2 years post transplant
Secondary Time to platelet recovery within 100 days post transplant
Secondary Number of patients with chronic GVHD 2 years post transplant
Secondary Disease free survival 2 years post transplant
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