Recruiting
Phase 1
Phase 2

Campath, Fludarabine, Melphalan

Sponsor:

Washington University School of Medicine

Code:

NCT00920972

Conditions

Metabolic Disorders

Hematologic, Immune, or Bone Marrow Disorders

Hemoglobinopathies

Non-malignant Disorders

Eligibility Criteria

Sex: All

Age: 0 - 20

Healthy Volunteers: Not accepted

Interventions

Treatment Plan 1: Stratum 1

Treatment Plan 2: Strata 2, 3, or 4

GVHD Regimen A: UCB Recipients

GVHD Regimen B: BM Recipients

Study Details

Brief summary:

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.

Conditions

Metabolic Disorders

Hematologic, Immune, or Bone Marrow Disorders

Hemoglobinopathies

Non-malignant Disorders

Study ID

NCT00920972

Start date

Dec, 2001

Status verified date

Mar, 2026

Completion date

Dec, 2031

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 20

Healthy Volunteers: Not accepted

Inclusion Criteria:

Stratum 1: Patient must have non-malignant disorder, excluding thalassemia. Must be receiving a 8/8 HLA-matched bone marrow, related or unrelated Stratum 2: Patient must have thalassemia receiving 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.

Stratum 3: Patient must have a hemoglobinopathy receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.

Stratum 4: Patient must have a non-malignant disorder (excluding hemoglobinopathy) receiving 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB. Related or unrelated.

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 <30%

Exclusion Criteria:

  • HIV positive
  • Invasive infection
  • Pregnancy/lactating

Study Design

Enrollment

220 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Stratum 1

Recipients with non-malignant disorders, excluding thalassemia. Related or unrelated 8/8 HLA-matched bone marrow

experimental: Stratum 2

Recipient with transfusion dependent thalassemia. Related or unrelated. 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB

experimental: Stratum 3

Recipient with hemoglobinopathy Related or unrelated. 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB

experimental: Stratum 4

Recipient with non-malignant disorder, excluding hemoglobinopathy Related or unrelated. 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB

Interventions

Treatment Plan 1: Stratum 1

Day -50 to -21: Hydroxyurea 30mg/kg PO q day 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...

Treatment Plan 2: Strata 2, 3, or 4

Day -50 to -21: Hydroxyurea 30mg/kg PO q day… 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 Regimen A: UCB Recipients

Day -3: Begin Tacrolimus or cyclosporine Begin MMF Day -1: Abatacept 10mg/kg IV Day +5: Abatacept 10mg/kg IV Day +14: Abatacept 10mg/kg IV Day +28: Abatacept 10mg/kg IV Day +60: Abatacept 10mg/kg IV Day +100: Abatacept 10mg/kg IV

GVHD Regimen B: BM Recipients

Day -3: Begin Tacrolimus or cyclosporine Begin MMF Day -1: Abatacept 10mg/kg IV Day +1: Methotrexate 7.5mg/m2 IV Day +3: Methotrexate 7.5mg/m2 IV Day +5: Abatacept 10mg/kg IV Day +6: Methotrexate 7.5mg/m2 IV Day +14: Abatacept 10mg/kg IV Day +28: Abatacept 10mg/kg IV Day +60: Abatacept 10mg/kg IV Day +100: Abatacept 10mg/kg IV Day +180: Abatacept 10mg/kg IV Day +270: Abatacept 10mg/kg IV Day +365: Abatacept 10mg/kg IV

Primary outcome measure

  • Donor engraftment as measured by chimerism [ Time Frame: 100 days post-transplant ]
  • Major toxicities as graded by the CTC v4 [ Time Frame: 100 days post-transplant ]

Central Contacts and Locations

Central contacts

Lissy Keller, CCRP

3142861168kellerl@wustl.edu

Ian Snyder, CCRP

3142735953ian.s@wustl.edu

Locations

Washington University School of Medicine (in St. Louis)

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Shalini Shenoy, MD

3144546018shalinishenoy@wustl.edu

Lissy L Keller, CCRP, BS

3142861168kellerl@wustl.edu

More Information

Sponsor

Washington University School of Medicine

Last update posted

Sep 16, 2026

Last verified

Mar, 2026

Keywords

  • Bone marrow
  • Transplant
  • Transplantation
  • Hematopoietic
  • Umbilical cord
  • Related
  • Unrelated
  • Reduced
  • Non-myeloablative
  • Nonmyeloablative
  • Non-malignant
  • Nonmalignant

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-07. This information was provided to ClinicalTrials.gov by Washington University School of Medicine on 2026-09-16. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.