Recruiting

Phase 1

Phase 2

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

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

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

3142861168 kellerl@wustl.edu

Ian Snyder, CCRP

3142735953 ian.s@wustl.edu

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85016

Principal Investigator:

Wilson A Vasconez Samaniego, MD

Children's Hospital of Orange County

Recruiting

Orange, California, United States, 92868

Principal Investigator:

David Buchbinder, MD

University of California

Recruiting

San Diego, California, United States, 92123

Principal Investigator:

Eric Anderson, MD

Yale School of Medicine

Recruiting

New Haven, Connecticut, United States, 06510

Principal Investigator:

Lakshmanan Krishnamurti, MD

George Washington University School of Medicine

Recruiting

Washington D.C., District of Columbia, United States, 20010

Principal Investigator:

David Jacobsohn, MD

University of Miami

Recruiting

Miami, Florida, United States, 33136

Principal Investigator:

Edward Dela Ziga, MD

Indiana University School of Medicine

Recruiting

Indianapolis, Indiana, United States, 46202

Principal Investigator:

Jodi Skiles, MD

St. Louis University

Recruiting

St Louis, Missouri, United States, 63104

Principal Investigator:

Deepika Bhatla, MD

Washington University School of Medicine (in St. Louis)

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Shalini Shenoy, MD

3144546018 shalinishenoy@wustl.edu

Lissy L Keller, CCRP, BS

3142861168 kellerl@wustl.edu

Columbia University Medical Center

Recruiting

New York, New York, United States, 10032

Principal Investigator:

Monica Bhatia, MD

University of North Carolina

Recruiting

Chapel Hill, North Carolina, United States, 27514

Principal Investigator:

Andrew Gilman, MD

University of Utah

Recruiting

Salt Lake City, Utah, United States, 84132

Principal Investigator:

Michael Pulsipher, MD

More Information

Sponsor

Washington University School of Medicine

Last update posted

Mar 25, 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 2026-09-09. This information was provided to ClinicalTrials.gov by Washington University School of Medicine on 2026-03-25.