Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

This study is currently recruiting participants.
Verified April 2014 by Washington University School of Medicine
Sponsor:
Collaborator:
St. Louis Children's Hospital
Information provided by (Responsible Party):
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT00920972
First received: June 15, 2009
Last updated: April 15, 2014
Last verified: April 2014

June 15, 2009
April 15, 2014
December 2001
December 2018   (final data collection date for primary outcome measure)
  • Donor engraftment as measured by chimerism [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
  • Major toxicities as graded by the CTC v4 [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT00920972 on ClinicalTrials.gov Archive Site
  • Time to neutrophil and platelet engraftment as measured by complete blood counts [ Time Frame: Notapplicable ] [ Designated as safety issue: Yes ]
  • Incidence of acute graft-versus-host disease as measured by protocol grading scale [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]
  • Incidence of chronic graft-versus-host disease as measured by protocol grading scale [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
  • Long-term donor engraftment by donor chimerism [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
  • Immune reconstitution by laboratory evaluations [ Time Frame: 1 year post-transplant ] [ Designated as safety issue: Yes ]
  • Overall and disease free survival [ Time Frame: 2 years post-transplant ] [ Designated as safety issue: Yes ]
Same as current
Not Provided
Not Provided
 
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime

The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is also to determine major toxicities from these conditioning regimens, within the first 100 days after transplantation.

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Metabolic Disorders
  • Hematologic, Immune, or Bone Marrow Disorders
  • Hemoglobinopathies
  • Non-malignant Disorders
  • Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan

    Day -22 Campath-1H 3 mg IV or SQ... Day -21 Campath-1H 10 mg IV or SQ... Day -20 Campath-1H 15 mg IV or SQ... Day -19 Campath-1H 20 mg IV or SQ... Day -8 Fludarabine 30mg/m2 IV... Day -7 Fludarabine 30mg/m2 IV... Day -6 Fludarabine 30mg/m2 IV... Day -5 Fludarabine 30mg/m2 IV... Day -4 Fludarabine 30mg/m2 IV... Day -3 Melphalan 140 mg/m2 IV... (dose modifications for patients <10 kgs) Procedure/Surgery: Hematopoietic stem cell infusion on Day 0...

    GVHD Prophylaxis:

    Starting day -3: Tacrolimus or cyclosporine... Days +1, +3, +6: Methotrexate

    Other Names:
    • Alemtuzumab
    • Campath
  • Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan

    Day -50 to -21 Hydroxyurea 30mg/kg PO… Day -22 Campath-1H 3 mg IV or SQ... Day -21 Campath-1H 10 mg IV or SQ... Day -20 Campath-1H 15 mg IV or SQ... Day -19 Campath-1H 20 mg IV or SQ... Day -8 Fludarabine 30mg/m2 IV... Day -7 Fludarabine 30mg/m2 IV... Day -6 Fludarabine 30mg/m2 IV... Day -5 Fludarabine 30mg/m2 IV... Day -4 Fludarabine 30mg/m2 IV... Day -4 Thiotepa 8mg/kg IV… Day -3 Melphalan 140 mg/m2 IV... (dose modifications for patients <10 kgs) Procedure/Surgery: Hematopoietic stem cell infusion on day 0...

    GVHD Prophylaxis Options:

    Regimen 1= Starting day -3: Tacrolimus or cyclosporine... Days +1, +3, +6: Methotrexate.... Starting on day +7: Prednisone...

    OR Regimen 2 = Starting day -3: tacrolimus or cyclosporine Starting day -3: mycophenolate mofetil

    Other Names:
    • Alemtuzumab
    • Campath
  • Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan

    Day -50 to -21 Hydroxyurea 30mg/kg PO… Day -22 Campath-1H 3 mg IVor SQ... Day -21 Campath-1H 10 mg IV or SQ... Day -20 Campath-1H 15 mg IV or SQ... Day -19 Campath-1H 20 mg IV or SQ... Day -8 Fludarabine 30mg/m2 IV... Day -7 Fludarabine 30mg/m2 IV... Day -6 Fludarabine 30mg/m2 IV... Day -5 Fludarabine 30mg/m2 IV... Day -4 Fludarabine 30mg/m2 IV... Day -4 Thiotepa 8mg/kg IV… Day -3 Melphalan 140 mg/m2 IV... (dose modifications for patients <10 kgs) Procedure/Surgery: Hematopoietic stem cell infusion on day 0...

    GVHD Prophylaxis Regimen:

    Children < 6 years of age, BM recipients:

    Regimen 1 = Starting day -3: tacro or CsA... Days +1, +3, +6: MTX... Starting day +7: prednisone...

    Children >/=6 years, BM recipients:

    Regimen 3 = Starting day -3: tacro or CsA... Days +1, +3, +6: MTX... Days -1, +5, +14, +28: abatacept...

    UCB recipients:

    Regimen 1 (described above) OR Regimen 2 = Starting day -3: tacro or CsA... Starting day -3: MMF

    Other Names:
    • Alemtuzumab
    • Campath
  • Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan

    Day -50 to -21 Hydroxyurea 30mg/kg PO… Day -22 Campath-1H 3 mg IV or SQ... Day -21 Campath-1H 10 mg IV or SQ... Day -20 Campath-1H 15 mg IV or SQ... Day -19 Campath-1H 20 mg IV or SQ... Day -8 Fludarabine 30mg/m2 IV... Day -7 Fludarabine 30mg/m2 IV... Day -6 Fludarabine 30mg/m2 IV... Day -5 Fludarabine 30mg/m2 IV... Day -4 Fludarabine 30mg/m2 IV... Day -4 Thiotepa 8mg/kg IV… Day -3 Melphalan 140 mg/m2 IV... (dose modifications for patients <10 kgs) Procedure/Surgery: Hematopoietic stem cell infusion on day 0...

    GVHD Prophylaxis Regimen:

    Children < 6 years of age, BM recipients:

    Regimen 1 = Starting day -3: tacro or CsA... Days +1, +3, +6: MTX... Starting day +7: prednisone...

    Children >/=6 years, BM recipients:

    Regimen 3 = Starting day -3: tacro or CsA... Days +1, +3, +6: MTX... Days -1, +5, +14, +28: abatacept...

    UCB recipients:

    Regimen 1 (described above) OR Regimen 2 = Starting day -3: tacro or CsA... Starting day -3: MMF

    Other Names:
    • Alemtuzumab
    • Campath
  • Experimental: Matched related HSCT for hemoglobinopathies
    Intervention: Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
  • Experimental: Unrelated HSCT for thalassemia
    Intervention: Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan
  • Experimental: Minimally mismatched unrelated HSCT for hemoglobinopathies
    Intervention: Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan
  • Experimental: Minor mismatched unrelated HSCT for non-malignant disorders
    Intervention: Drug: Transplant conditioning regimen of hydroxyurea, campath, fludarabine, thiotepa, & melphalan

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
220
Not Provided
December 2018   (final data collection date for primary outcome measure)

Inclusion Criteria:

Stratum 1: Patient must have a hemoglobinopathy receiving a matched related transplant with bone marrow or peripheral blood stem cells.

Stratum 2: Patient must have thalassemia receiving an unrelated donor transplant with bone marrow or umbilical cord blood stem cells.

Stratum 3: Patient must have a hemoglobinopathy receiving a minimally mismatched unrelated donor transplant with bone marrow or single or double umbilical cord blood product.

Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving a minor mismatched unrelated donor transplant with bone marrow or single or double umbilical cord blood product.

All strata:

  • Recipient age < 21 years
  • Lansky/Karnofsky >/= 40
  • Adequate pulmonary, renal, liver, and other organ function as defined in protocol
  • Negative pregnancy test
  • Adequate total nucleated cell or CD34+ dose of product as defined in protocol
  • If sickle cell, Hemoglobin S <45%

Exclusion Criteria:

  • HIV positive
  • Invasive infection
  • Pregnancy/lactating
Both
up to 20 Years
No
Contact: Lisa Murray, MA, CCRP 3144544240 Murray_L@kids.wustl.edu
United States,   Canada
 
NCT00920972
01-0923
Yes
Washington University School of Medicine
Washington University School of Medicine
St. Louis Children's Hospital
Principal Investigator: Shalini Shenoy, MD Washington University School of Medicine (in St. Louis)
Washington University School of Medicine
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP