NCT00466843

Brief Summary

Myelodysplastic syndrome (MDS) is a rare, potentially serious bone marrow disease. Currently available treatments for MDS have been only somewhat beneficial. The purpose of this study is to determine the effects of the medication antithymocyte globulin (ATG) in adults with MDS and to determine which individuals with MDS are most likely to benefit from treatment with ATG.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
54

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Apr 2007

Typical duration for phase_2

Geographic Reach
1 country

4 active sites

Status
unknown

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

April 1, 2007

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

April 25, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

April 27, 2007

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2010

Completed
Last Updated

June 2, 2009

Status Verified

June 1, 2009

Enrollment Period

2.8 years

First QC Date

April 25, 2007

Last Update Submit

June 1, 2009

Conditions

Keywords

Abnormal hematopoiesisLeukemiaAutoimmune Disease

Outcome Measures

Primary Outcomes (2)

  • Bone marrow response and hematologic improvement

    Measured at Week 16 or 24

  • Bone marrow cytogenetic response

    Measured at Week 16 or 24

Study Arms (1)

1

EXPERIMENTAL

Participants will be treated with ATG

Drug: Antithymocyte globulin (ATG)Drug: Prednisone

Interventions

ATG 2.5 mg/kg/day via IV will be given for 4 doses. Each participant will receive only one cycle of therapy. The daily infusion will be administered over at least 6 hours and slowed as necessary to minimize infusion-related symptoms.

1

All participants will be pre-treated with prednisone (1 mg/kg/day by mouth) 2 days prior to the first ATG does and continuing for 14 days after the final dose to prevent serum sickness

1

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of MDS that meets International Prognostic Scoring System (IPSS) criteria for low risk, intermediate-1 risk, or intermediate-2 risk. More information about this criterion can be found in the protocol.
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
  • Willing and able to attend study visits
  • Willing to use acceptable forms of contraception prior to study entry and for the duration of the study

You may not qualify if:

  • Any serious medical illness that might limit survival to less than 2 years
  • Any other uncontrolled condition or illness. More information about this criterion can be found in the protocol.
  • Prior anti-lymphocyte serotherapy (received serum from an immunized animal)
  • Proliferative chronic myelomonocytic leukemia
  • MDS that is caused by radiotherapy, chemotherapy, and/or immunotherapy for cancerous or autoimmune diseases
  • Previous or current cancer. More information about this criterion can be found in the protocol.
  • Receiving any other investigational agents
  • Certain abnormal lab values. More information about this criterion can be found in the protocol.
  • History of a grade 2 National Cancer Institute common toxic criteria allergic reaction to rabbit proteins
  • Psychiatric illness that might interfere with study participation
  • HIV-1 infection
  • Pregnancy or breastfeeding

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

UCLA Oncology Center

Los Angeles, California, 90095, United States

NOT YET RECRUITING

H. Lee Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

Cleveland Clinic Foundation - Case Western University

Cleveland, Ohio, 44195, United States

RECRUITING

Penn State University

Hershey, Pennsylvania, 17033, United States

RECRUITING

Related Publications (4)

  • Kochenderfer JN, Kobayashi S, Wieder ED, Su C, Molldrem JJ. Loss of T-lymphocyte clonal dominance in patients with myelodysplastic syndrome responsive to immunosuppression. Blood. 2002 Nov 15;100(10):3639-45. doi: 10.1182/blood-2002-01-0155. Epub 2002 Jul 5.

    PMID: 12393644BACKGROUND
  • Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS. Relationship between bone marrow failure syndromes and the presence of glycophosphatidyl inositol-anchored protein-deficient clones. Br J Haematol. 2001 Dec;115(4):1015-22. doi: 10.1046/j.1365-2141.2001.03191.x.

    PMID: 11843844BACKGROUND
  • Molldrem JJ, Leifer E, Bahceci E, Saunthararajah Y, Rivera M, Dunbar C, Liu J, Nakamura R, Young NS, Barrett AJ. Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med. 2002 Aug 6;137(3):156-63. doi: 10.7326/0003-4819-137-3-200208060-00007.

    PMID: 12160363BACKGROUND
  • Komrokji RS, Mailloux AW, Chen DT, Sekeres MA, Paquette R, Fulp WJ, Sugimori C, Paleveda-Pena J, Maciejewski JP, List AF, Epling-Burnette PK. A phase II multicenter rabbit anti-thymocyte globulin trial in patients with myelodysplastic syndromes identifying a novel model for response prediction. Haematologica. 2014 Jul;99(7):1176-83. doi: 10.3324/haematol.2012.083345. Epub 2014 Jan 31.

MeSH Terms

Conditions

Myelodysplastic SyndromesLeukemiaAutoimmune Diseases

Interventions

Antilymphocyte SerumPrednisone

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesNeoplasms by Histologic TypeNeoplasmsImmune System Diseases

Intervention Hierarchy (Ancestors)

Immune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex MixturesPregnadienediolsPregnadienesPregnanesSteroidsFused-Ring CompoundsPolycyclic Compounds

Study Officials

  • Alan List, MD

    H. Lee Moffitt Cancer Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH

Study Record Dates

First Submitted

April 25, 2007

First Posted

April 27, 2007

Study Start

April 1, 2007

Primary Completion

February 1, 2010

Study Completion

February 1, 2010

Last Updated

June 2, 2009

Record last verified: 2009-06

Locations