NCT01012492

Brief Summary

The primary objective of the study is to determine the safety and tolerability when adding abatacept to acute Graft versus Host Disease in transplants for malignant diseases using unrelated donor bone marrow or peripheral blood stem cell grafts.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
11

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Nov 2009

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2009

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

November 11, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 13, 2009

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2012

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2013

Completed
4.7 years until next milestone

Results Posted

Study results publicly available

September 15, 2017

Completed
Last Updated

November 21, 2019

Status Verified

October 1, 2019

Enrollment Period

3 years

First QC Date

November 11, 2009

Results QC Date

November 25, 2015

Last Update Submit

October 31, 2019

Conditions

AMLALLUndifferentiated LeukemiaBiphenotypic LeukemiaRefractory AnemiaRefractory Anemia With Ringed SideroblastsRefractory Cytopenia With Multilineage DysplasiaRef. Cytopenia w Multilineage Dysplasia & Ringed SideroblastsRefractory Anemia With Excess Blasts-1 (5-10% Blasts)Refractory Anemia With Excess Blasts-2 (10-20% Blasts)Myelodysplastic Syndrome, UnclassifiedMDS Associated With Isolated Del (5q)

Keywords

cancerleukemiatransplantbone marrow

Outcome Measures

Primary Outcomes (1)

  • Percentage of Participants With Grade III-IV Acute GVHD by Day 100.

    Grade III-IV Acute GVHD by Day 100. The incidence of Gr III-IV acute GVHD was measured by the modified Glucksburg scale.

    Day 100 post-transplant

Secondary Outcomes (3)

  • Percentage of Participants With Grades III-IV Acute GVHD at 2 Years

    2 years after transplant

  • Hematologic and Immunologic Reconstitution

    Day +100 post-transplant

  • Protective Immunity

    Day +365 post-transplant

Study Arms (1)

Abatacept

EXPERIMENTAL

Participants will receive one of two standard myeloablative conditioning regimens for their stem cell transplant, and will receive an aGvHD prophylaxis regimen including cyclosporine, methotrexate, and abatacept.

Drug: Abatacept

Interventions

AbataceptDRUG

Participants will receive one of two standard myeloablative conditioning regimens for their stem cell transplant, and will receive an aGvHD prophylaxis regimen including cyclosporine, methotrexate, and abatacept.

Also known as: orencia
Abatacept

Eligibility Criteria

Age12 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with AML, with or without a history of myelodysplastic syndrome in one of the following categories.
  • (a) Patients in first complete remission with high-risk features
  • Patients with ALL, in either of the following categories:
  • In 2nd or greater complete remission (complete remission is defined as \> 5% blasts in marrow)
  • Delayed 1st CR-Failure to achieve complete remission after a single round of induction therapy
  • Patients with undifferentiated or biphenotypic leukemia in 1st or greater complete remission.
  • Patients with Myelodysplastic Syndrome(s) with an IPSS score of \>1.5 and \<10% blasts in the bone marrow at the time of transplant. These conditions will include:
  • Refractory anemia
  • Refractory anemia with ringed sideroblasts
  • Refractory cytopenia with multilineage dysplasia
  • Refractory cytopenia with multilineage dysplasia and ringed sideroblasts
  • Refractory anemia with excess blasts-1 (5-10% blasts)
  • Refractory anemia with excess blasts-2 (10-20% blasts)
  • Myelodysplastic syndrome, unclassified
  • MDS associated with isolated del (5q)
  • +5 more criteria

You may not qualify if:

  • Age \<12 years old.
  • Patients requiring \>2 courses of induction chemotherapy to achieve remission status.
  • HIV infection
  • Tuberculosis Infection
  • Chronic Obstructive Pulmonary Disease
  • Pregnancy (positive serum b-HCG) or breastfeeding
  • Creatinine clearance or nuclear medicine GFR of \< 50 mL/min
  • Cardiac ejection fraction \< 50%
  • bilirubin \> 2 × upper limit of normal or ALT \> 4 × upper limit of normal or unresolved veno-occlusive disease.
  • Pulmonary disease with FVC, FEV1 or DLCO parameters \<45% predicted (corrected for hemoglobin) or O2 saturation \<92% on room air.
  • Karnofsky performance score or Lansky Play-Performance Scale \<80
  • Uncontrolled viral, bacterial, or fungal infection at the time of study enrollment
  • Availability of a willing and fully MHC-matched related donor.
  • Positive cytotoxic recipient-donor cross-match or positive HLA antibody screen against donor-disparate antigens.
  • Any active infection.
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Related Publications (2)

  • Koura DT, Horan JT, Langston AA, Qayed M, Mehta A, Khoury HJ, Harvey RD, Suessmuth Y, Couture C, Carr J, Grizzle A, Johnson HR, Cheeseman JA, Conger JA, Robertson J, Stempora L, Johnson BE, Garrett A, Kirk AD, Larsen CP, Waller EK, Kean LS. In vivo T cell costimulation blockade with abatacept for acute graft-versus-host disease prevention: a first-in-disease trial. Biol Blood Marrow Transplant. 2013 Nov;19(11):1638-49. doi: 10.1016/j.bbmt.2013.09.003. Epub 2013 Sep 15.

    PMID: 24047754RESULT

  • Suessmuth Y, Mukherjee R, Watkins B, Koura DT, Finstermeier K, Desmarais C, Stempora L, Horan JT, Langston A, Qayed M, Khoury HJ, Grizzle A, Cheeseman JA, Conger JA, Robertson J, Garrett A, Kirk AD, Waller EK, Blazar BR, Mehta AK, Robins HS, Kean LS. CMV reactivation drives posttransplant T-cell reconstitution and results in defects in the underlying TCRbeta repertoire. Blood. 2015 Jun 18;125(25):3835-50. doi: 10.1182/blood-2015-03-631853. Epub 2015 Apr 7.

    PMID: 25852054RESULT

MeSH Terms

Conditions

Anemia, RefractoryMyelodysplastic SyndromesNeoplasmsLeukemia

Interventions

Abatacept

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow DiseasesNeoplasms by Histologic Type

Intervention Hierarchy (Ancestors)

ImmunoconjugatesAntibodiesImmunoglobulinsSerum GlobulinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsGlobulins

Results Point of Contact

Title
Leslie S Kean
Organization
Emory University School of Medicine
Leslie.kean@emory.edu
404-376-0187

Study Officials

  • Leslie Kean, MD, PhD

    Boston Children's Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD

Study Record Dates

First Submitted

November 11, 2009

First Posted

November 13, 2009

Study Start

November 1, 2009

Primary Completion

November 1, 2012

Study Completion

January 1, 2013

Last Updated

November 21, 2019

Results First Posted

September 15, 2017

Record last verified: 2019-10

Locations

US(1)