Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome

NCT00475020 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: 2005-0726NCI-2012-01477
• Study Type: interventional
• Target: 63
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical research study is to learn if using a combination of fludarabine, busulfan, and antithymocyte globulin (ATG) can help to control myelofibrosis or myelodysplastic syndrome in patients receiving a bone marrow or blood stem cell transplant. The safety of these drugs will also be studied.

Conditions

Myelofibrosis

Keywords

Idiopathic Myelofibrosis; Myelofibrosis; Essential Thrombocythemia; Polycythemia Vera; Busulfan; Antithymocyte Globulin; ATG; Fludarabine; Thymoglobulin

Outcome Measures

Primary Outcomes (1)

• Rate of Non-relapse Mortality at 100 Days Post-transplant

  To evaluate the safety of Fludarabine/Busulfan as conditioned regimen for allogeneic stem cell transplantation in patients with myelofibrosis/myelodysplastic syndrome at 100 days post-transplant

  Non-relapse mortality at 100 days post-transplant

Secondary Outcomes (1)

• Efficacy of This Therapy 3 Years Post-transplant

  Up to 3 years post-transplant

Study Arms (1)

Fludarabine + Busulfan + Thymoglobulin

EXPERIMENTAL

Fludarabine 40 mg/m\^2 by vein daily over 1 hour x 4 days. Busulfan test dose = 32 mg/m\^2 by vein x 1 day; 100 mg/m\^2 by vein daily over 3 hours x 4 days. Thymoglobulin 2.5 mg/kg by vein over 6 hours x 3 days if there is an unrelated or a mismatched donor.

Drug: Busulfan; Drug: Fludarabine; Drug: Thymoglobulin (ATG)

Interventions

Busulfan DRUG

Test dose = 32 mg/m\^2 by vein x 1 day; 100 mg/m\^2 by vein daily over 3 hours x 4 days

Also known as: Busulfex, Myleran

Fludarabine + Busulfan + Thymoglobulin

Fludarabine DRUG

40 mg/m\^2 by vein daily over 1 hour x 4 days

Also known as: Fludarabine Phosphate

Fludarabine + Busulfan + Thymoglobulin

Thymoglobulin (ATG) DRUG

2.5 mg/kg by vein over 6 hours x 3 days if there is an unrelated or a mismatched donor

Also known as: Antithymocyte Globulin, Thymoglobulin, ATG

Fludarabine + Busulfan + Thymoglobulin

Eligibility Criteria

• Age: Up to 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Polycythemia Vera or Essential Thrombocythemia or Myelodysplastic syndrome with or without fibrosis.
• Patients 75 years or younger
• Patients must have an HLA matched or at least a 9/10 antigen (A, B, C, DQ or DR) matched related or unrelated donor.
• Patients must have a Zubrod PS 2 or less.
• Creatinine \< 1.6 mg/dl
• Ejection fraction \>/= 40%, unless cleared by cardiology
• Serum direct bilirubin \< 2 mg/dl (unless due to Gilbert's syndrome), serum glutamate pyruvate transaminase (SGPT) \</= 4 x normal values
• Forced expiratory volume at one second (FEV1), forced vital capacity (FVC), or diffusing capacity of lung for carbon monoxide (DLCO) \>/= 40% of expected.
• Negative Beta human chorionic gonadotropin (HCG) test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.

You may not qualify if:

• Uncontrolled life-threatening infections
• HIV positive
• Patients with Active viral hepatitis

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Primary Myelofibrosis; Thrombocythemia, Essential; Polycythemia Vera

Interventions

Busulfan; fludarabine; fludarabine phosphate; thymoglobulin; Antilymphocyte Serum

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Coagulation Disorders; Thrombocytosis; Blood Platelet Disorders; Hemorrhagic Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures

Results Point of Contact

• Title: Popat,Uday,M.D. / Stem Cell Transplantation
• Organization: UT MD Anderson Cancer Center

UPOPAT@MDANDERSON.ORG

713-745-3055

Study Officials

• Uday Popat, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2007
• First Posted: May 17, 2007
• Study Start: January 4, 2006
• Primary Completion: October 4, 2017
• Study Completion: October 4, 2017
• Last Updated: May 21, 2019
• Results First Posted: December 4, 2018

Record last verified: 2019-05

Locations

US (1)