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Metabolic DisordersPhase 1/2NCT00920972

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

A Phase 1/2 study testing Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4 and GVHD Regimen A: UCB Recipients in people with Metabolic Disorders and Hematologic, Immune, or Bone Marrow Disorders, run by Washington University School of Medicine at 28 sites in 2 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages Up to 20 years
What is being tested?
Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4 and GVHD Regimen A: UCB Recipients
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
220 participants
Where
28 sitesin 2 countries
Study dates
Dec 2001to Dec 2031
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 20 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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
Show all 10Show fewer
  • 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%

Who cannot join

  • HIV positive
  • Invasive infection
  • Pregnancy/lactating

Trial locations and contacts

28 sites in 2 countries. Contact the study team or a site near you to ask about taking part.

Lissy Keller, CCRP contact

3142861168

kellerl@wustl.edu

Ian Snyder, CCRP contact

3142735953

ian.s@wustl.edu

28 sites in 2 countries

United States25 sites, 1 recruiting

  • University of North Carolina

    Chapel Hill, North Carolina, 27514

    Active, not recruiting

  • Carolinas Medical Center

    Charlotte, North Carolina, 28232

    Completed

  • Children's Memorial Hospital

    Chicago, Illinois, 60614

    Completed

  • Duke Children's Hospital

    Durham, North Carolina, 27705

    Completed

  • Cook Children's Hospital

    Forth Worth, Texas, 76104

    Completed

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

    Completed

  • Indiana University School of Medicine

    Indianapolis, Indiana, 46202

    Active, not recruiting

  • Nemours Children's Health

    Jacksonville, Florida, 32207

    Completed

  • Children's Mercy

    Kansas City, Missouri, 64108

    Completed

  • University of Miami

    Miami, Florida, 33136

    Active, not recruiting

  • Miami Children's Hospital

    Miami, Florida, 33155

    Completed

  • Yale School of Medicine

    New Haven, Connecticut, 06510

    Active, not recruiting

  • Children's Hospital of New Orleans

    New Orleans, Louisiana, 70118

    Completed

  • Columbia University Medical Center

    New York, New York, 10032

    Active, not recruiting

  • The University of Oklahoma

    Oklahoma City, Oklahoma, 73104

    Completed

  • Children's Hospital of Orange County

    Orange, California, 92868

    Active, not recruiting

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

    Active, not recruiting

  • University of Pittsburg

    Pittsburgh, Pennsylvania, 15224

    Completed

  • University of Utah

    Salt Lake City, Utah, 84132

    Active, not recruiting

  • Texas Transplant Institute

    San Antonio, Texas, 78229

    Completed

  • University of California

    San Diego, California, 92123

    Active, not recruiting

  • St. Louis University

    St Louis, Missouri, 63104

    Active, not recruiting

  • Washington University School of Medicine (in St. Louis)

    St Louis, Missouri, 63110

    Shalini Shenoy, MD, 3144546018, shalinishenoy@wustl.edu

    Lissy L Keller, CCRP, BS, 3142861168, kellerl@wustl.edu

    Recruiting

  • All Children's Hospital

    St. Petersburg, Florida, 33701

    Completed

  • George Washington University School of Medicine

    Washington D.C., District of Columbia, 20010

    Active, not recruiting

Other trials in United States

Canada3 sites

  • University of Calgary

    Calgary

    Completed

  • BC Children's Hospital

    Vancouver, British Columbia, V6H 3N1

    Completed

  • University of Manitoba

    Winnipeg, Manitoba, MB R3E 0T4

    Completed

Other trials in Canada

About this study

Official title: 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.

Read the detailed description

The study uses reduced intensity conditioning that is immune suppressive to achieve donor cell engraftment without exposure to radiation or high dose chemotherapy in children with non-malignant disorders. The intent is to minimize early and late regimen related toxicities in the context of a reduced intensity regimen.

In addition to maximizing opportunity for donor cell engraftment, the trial seeks to minimize toxicities associated with transplant such as graft versus host disease and employs GVHD prophylaxis that seeks to decrease rates of acute and chronic GVHD in the setting of matched and mismatched donor stem cell transplants from marrow and cord blood sources.

Being tested
Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4, GVHD Regimen A: UCB Recipients, GVHD Regimen B: BM Recipients
Keywords
Bone marrow, Transplant, Transplantation, Hematopoietic, Umbilical cord, Related, Unrelated, Reduced, Non-myeloablative, Nonmyeloablative, Non-malignant, Nonmalignant
Medical subject headings
Metabolic Diseases, Hemoglobinopathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Treatment Plan 1: Stratum 1Drug

    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 4Drug

    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 RecipientsDrug

    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 RecipientsDrug

    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

Study groups in NCT00920972
Study groupRoleWhat they receive
Stratum 1ExperimentalRecipients with non-malignant disorders, excluding thalassemia. Related or unrelated 8/8 HLA-matched bone marrow
Receives: Drug: Treatment Plan 1: Stratum 1; Drug: GVHD Regimen B: BM Recipients
Stratum 2ExperimentalRecipient with transfusion dependent thalassemia. Related or unrelated. 8/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB
Receives: Drug: Treatment Plan 2: Strata 2, 3, or 4; Drug: GVHD Regimen A: UCB Recipients; Drug: GVHD Regimen B: BM Recipients
Stratum 3ExperimentalRecipient with hemoglobinopathy Related or unrelated. 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB
Receives: Drug: Treatment Plan 2: Strata 2, 3, or 4; Drug: GVHD Regimen A: UCB Recipients; Drug: GVHD Regimen B: BM Recipients
Stratum 4ExperimentalRecipient with non-malignant disorder, excluding hemoglobinopathy Related or unrelated. 7/8 HLA-matched bone marrow or 5-8/8 HLA-matched UCB
Receives: Drug: Treatment Plan 2: Strata 2, 3, or 4; Drug: GVHD Regimen A: UCB Recipients; Drug: GVHD Regimen B: BM Recipients

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Donor engraftment as measured by chimerism

    Engraftment is measured in myeloid and lymphoid lineage cells

    Measured: 100 days post-transplant

  2. Major toxicities as graded by the CTC v4

    Toxicity monitoring includes unanticipated side effects (new) and all severe irreversible toxicities Grade 3 and above unexpected Grade 4 and above - all toxicities that are possibly, probably or definitely related to protocol therapy All deaths irrespective of attribution

    Measured: 100 days post-transplant

Secondary outcomes (6)

  1. Time to neutrophil and platelet engraftment as measured by complete blood counts

    Defined as an ANC >500/microliter and platelets >20,000 or 50,000/microliter depending on disorder

    Measured: Post transplant

  2. Incidence of acute graft-versus-host disease as measured by protocol grading scale

    aGVHD - involving the skin, gut and liver. Classified according to grading described by Thomas et al. NEJM 1975; 292:895-902

    Measured: 100 days post-transplant

  3. Incidence of chronic graft-versus-host disease as measured by protocol grading scale

    cGVHD classified per Schulman et al. Am J Med 69: 204-17, 1980.

    Measured: 2 years post-transplant

  4. Long-term donor engraftment by donor chimerism

    Donor chimerism is determined by PCR analysis after cell separation into lymphoid and myeloid lineage cells using antibodies. Can also be detected by FISH analysis in the event of donor and recipient sex discrepancy.

    Measured: 2 years post-transplant

  5. Immune reconstitution by laboratory evaluations

    Immune reconstitution detected by absolute numbers of T cell phenotypes, B cells and NK cells. T cell function determined by proliferative response to mitogens. B cell function determined by evaluating anti-tetanus antibody titers.

    Measured: 1 year post-transplant

  6. Overall and disease free survival

    Overall survival is defined as survival with or without disease Event free survival is defined as disease free, severe GVHD free survival, monitoring quality of life and relevant parameters.

    Measured: 2 years post-transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Jun 2009 (estimated)
  2. Study startDec 2001
  3. Primary completionDec 2026 (estimated)
  4. Study completionDec 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
220 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00920972
Sponsor's protocol ID
201110144
Status last verified
Mar 2026
First submitted
15 Jun 2009
Last updated
16 Sep 2026
Results posted
No
Data monitoring committee
Yes

Publications

  • Bhatla D, Davies SM, Shenoy S, Harris RE, Crockett M, Shoultz L, Smolarek T, Bleesing J, Hansen M, Jodele S, Jordan M, Filipovich AH, Mehta PA. Reduced-intensity conditioning is effective and safe for transplantation of patients with Shwachman-Diamond syndrome. Bone Marrow Transplant. 2008 Aug;42(3):159-65. doi: 10.1038/bmt.2008.151. Epub 2008 May 26.

    PubMed 18500373

  • Hansen MD, Filipovich AH, Davies SM, Mehta P, Bleesing J, Jodele S, Hayashi R, Barnes Y, Shenoy S. Allogeneic hematopoietic cell transplantation (HCT) in Hurler's syndrome using a reduced intensity preparative regimen. Bone Marrow Transplant. 2008 Feb;41(4):349-53. doi: 10.1038/sj.bmt.1705926. Epub 2007 Nov 19.

    PubMed 18026148

  • Rao A, Kamani N, Filipovich A, Lee SM, Davies SM, Dalal J, Shenoy S. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood. 2007 Jan 1;109(1):383-5. doi: 10.1182/blood-2006-05-025072. Epub 2006 Sep 21.

    PubMed 16990602

  • Shenoy S, Grossman WJ, DiPersio J, Yu LC, Wilson D, Barnes YJ, Mohanakumar T, Rao A, Hayashi RJ. A novel reduced-intensity stem cell transplant regimen for nonmalignant disorders. Bone Marrow Transplant. 2005 Feb;35(4):345-52. doi: 10.1038/sj.bmt.1704795.

    PubMed 15592491

  • King AA, Kamani N, Bunin N, Sahdev I, Brochstein J, Hayashi RJ, Grimley M, Abraham A, Dioguardi J, Chan KW, Douglas D, Adams R, Andreansky M, Anderson E, Gilman A, Chaudhury S, Yu L, Dalal J, Hale G, Cuvelier G, Jain A, Krajewski J, Gillio A, Kasow KA, Delgado D, Hanson E, Murray L, Shenoy S. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015 Dec;90(12):1093-8. doi: 10.1002/ajh.24183. Epub 2015 Oct 6.

    PubMed 26348869

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Treatment Plan 1: Stratum 1, Treatment Plan 2: Strata 2, 3, or 4 and GVHD Regimen A: UCB Recipients in people with Metabolic Disorders and Hematologic, Immune, or Bone Marrow Disorders, run by Washington University School of Medicine at 28 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Lissy Keller, CCRP (3142861168, kellerl@wustl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Donor engraftment as measured by chimerism (time frame: 100 days post-transplant). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00920972, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.

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