Immune Disorder HSCT Protocol

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Washington University School of Medicine
Sponsor:
Information provided by (Responsible Party):
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT01821781
First received: March 19, 2013
Last updated: April 16, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
Immune Deficiency Disorders:
Severe Combined Immunodeficiency
Chronic Granulomatous Disease
X-linked Agammaglobulinemia
Wiskott-Aldrich Syndrome
Hyper-IgM
DiGeorge Syndrome
Chediak-Higashi Syndrome
Common Variable Immune Deficiency
Immune Dysregulatory Disorder:
Hemophagocytic Lymphohistiocytosis
IPEX
Autoimmune Lymphoproliferative Syndrome
X-linked Lymphoproliferative Syndrome
Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime

Resource links provided by NLM:


Further study details as provided by Washington University School of Medicine:

Primary Outcome Measures:
  • Number of participants with donor engraftment [ Time Frame: 1 year post transplant ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Major Transplant Related Toxicities [ Time Frame: 1 years post transplant ] [ Designated as safety issue: Yes ]
  • Time to neutrophil recovery [ Time Frame: within 100 days post transplant ] [ Designated as safety issue: No ]
  • Number of patient with acute GVHD [ Time Frame: 180 days post transplant ] [ Designated as safety issue: No ]
  • Number of participants with infectious complications [ Time Frame: 2 years post transplant ] [ Designated as safety issue: No ]
  • Time to immune reconstitution [ Time Frame: 2 years post transplant ] [ Designated as safety issue: No ]
  • Overall survival [ Time Frame: 2 years post transplant ] [ Designated as safety issue: No ]
  • Time to platelet recovery [ Time Frame: within 100 days post transplant ] [ Designated as safety issue: No ]
  • Number of patients with chronic GVHD [ Time Frame: 2 years post transplant ] [ Designated as safety issue: No ]
  • Disease free survival [ Time Frame: 2 years post transplant ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: March 2013
Estimated Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Preparative 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

  Eligibility

Ages Eligible for Study:   up to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • </= 21 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01821781

Locations
United States, Missouri
Washington University Recruiting
St. Louis, Missouri, United States, 63110
Contact: Jeffrey Bednarski, MD    314-454-6018    Bednarski_J@kids.wustl.edu   
Contact: Lisa Murray, CCRP    314-454-4240    Murray_L@kids.wustl.edu   
Principal Investigator: Jeffrey Bednarski, MD         
Sponsors and Collaborators
Washington University School of Medicine
  More Information

No publications provided

Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01821781     History of Changes
Other Study ID Numbers: 201301135
Study First Received: March 19, 2013
Last Updated: April 16, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Washington University School of Medicine:
Immune deficiency
Immune disorders
Immune dysregulatory
Reduced Intensity
Alemtuzumab
Campath

Additional relevant MeSH terms:
Severe Combined Immunodeficiency
Agammaglobulinemia
Chediak-Higashi Syndrome
Common Variable Immunodeficiency
DiGeorge Syndrome
Granulomatous Disease, Chronic
Immunologic Deficiency Syndromes
Immune System Diseases
Lymphohistiocytosis, Hemophagocytic
Wiskott-Aldrich Syndrome
Genetic Diseases, X-Linked
Lymphoproliferative Disorders
Granuloma
Autoimmune Lymphoproliferative Syndrome
Blood Protein Disorders
Hematologic Diseases
Lymphatic Diseases
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
22q11 Deletion Syndrome
Craniofacial Abnormalities
Musculoskeletal Abnormalities
Musculoskeletal Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Lymphatic Abnormalities
Abnormalities, Multiple
Congenital Abnormalities

ClinicalTrials.gov processed this record on August 27, 2014