Clinical trial NCT00072579: Sargramostim in Treating Patients With Chronic Phase Chronic Myelogenous Leukemia Who Are Not in Complete Cytogenetic Remission Following Initial Treatment
Official title: Phase II Study of GM-CSF in Patients With Chronic Phase Chronic Myeloid Leukemia (CP-CML) Who Are Not in Complete Cytogenetic Remission After Initial Therapy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Leukemia
Keywords: chronic phase chronic myelogenous leukemia
chronic myelogenous leukemia, BCR-ABL1 positive
Lead sponsor: Wake Forest University Health Sciences
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2003-11-05
Sex: ALL
Minimum age (years): 18
Maximum age (years): 120
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: RATIONALE: Colony-stimulating factors, such as sargramostim, may increase the number of immune cells found in bone marrow or peripheral blood and may bring about complete remission in patients who have chronic phase chronic myelogenous leukemia.

PURPOSE: This phase II trial is studying sargramostim to see how well it works in treating patients with chronic phase chronic myelogenous leukemia that is not in complete cytogenetic remission after initial treatment.

Detailed description: OBJECTIVES:

* Determine the efficacy and safety of sargramostim (GM-CSF) by cytogenetic examination of the bone marrow in patients with chronic phase chronic myelogenous leukemia who are not in complete cytogenetic remission after initial therapy.

OUTLINE: Patients receive sargramostim (GM-CSF) subcutaneously daily for 3 months in the absence of disease progression or unacceptable toxicity. Patients achieving no response receive GM-CSF for an additional 3 months. Patients failing to achieve a partial response or better after the second course of GM-CSF are removed from the study. Patients achieving a partial response after the first or second course of GM-CSF continue to receive GM-CSF for an additional 9 months. Patients are then re-evaluated. Patients achieving a complete cytologic response at 9 months then receive GM-CSF 3 times weekly in the absence of disease progression or unacceptable toxicity.

Patients are followed every 2 weeks.

PROJECTED ACCRUAL: A total of 9-24 patients will be accrued for this study within 3 years.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed chronic phase chronic myelogenous leukemia (CML)

  * Presence of t(9;22)(q34;q11) with at least 20 cells examined in metaphase by cytogenetic examination of the bone marrow
* Complete hematologic remission during prior therapy\* as seen on 2 separate blood count analyses, defined by the following:

  * WBC no greater than 10,000/mm\^3 AND platelet count no greater than 450,000/mm\^3
  * Disappearance of all signs and symptoms of disease, including palpable splenomegaly
  * Normal differential counts (i.e., absence of blasts, promyelocytes, myelocytes, and metamyelocytes) NOTE: \*Continuation of therapy that led to complete hematologic remission is required during study participation
* Persistent cytogenetic disease despite 12 months of prior imatinib mesylate therapy, which may have included a trial dose-escalation OR intolerant of imatinib mesylate at a dose greater than 400 mg/day
* Not in complete cytogenetic remission within 30 days of study entry

  * Persistent Philadelphia chromosome by bone marrow exam

PATIENT CHARACTERISTICS:

Age

* 18 and over

Performance status

* ECOG 0-2

Life expectancy

* More than 6 months

Hematopoietic

* See Disease Characteristics

Hepatic

* Not specified

Renal

* Not specified

Other

* Not pregnant or nursing
* Fertile patients must use effective contraception
* No uncontrolled active infective
* No serious medical or psychiatric illness that would prevent giving informed consent or limit survival to less than 6 months
* No other malignancy not in remission except curatively treated basal cell skin cancer or carcinoma in situ of the cervix

PRIOR CONCURRENT THERAPY:

Biologic therapy

* Prior sargramostim (GM-CSF) allowed
* Prior interferon alfa for CML allowed
* No prior stem cell transplantation
* Concurrent interferon alfa\* for CML allowed NOTE: \*No dose increase during study participation

Chemotherapy

* At least 4 weeks since prior chemotherapy

Endocrine therapy

* Not specified

Radiotherapy

* At least 4 weeks since prior radiotherapy
* No concurrent radiotherapy

Surgery

* At least 4 weeks since prior surgery

Other

* Prior imatinib mesylate for CML allowed
* No other concurrent medication for CML
* Concurrent imatinib mesylate\* for CML allowed NOTE: \*No dose increase during study participation

Primary outcomes:
measure: Cytogenetic response (complete and partial)
Secondary outcomes:
measure: Toxicity as assessed by the Expanded Common Toxicity Criteria v2.0
measure: Time to progression
measure: Survival
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: CCOP - Western Regional, Arizona
state: Arizona
city: Oakland
country: United States
facility: CCOP - Bay Area Tumor Institute
state: California
city: Miami Beach
country: United States
facility: CCOP - Mount Sinai Medical Center
state: Florida
city: Rome
country: United States
facility: Regional Radiation Oncology Center at Rome
state: Georgia
city: Decatur
country: United States
facility: CCOP - Central Illinois
state: Illinois
city: Louisville
country: United States
facility: Kentuckiana Cancer Institute, PLLC
state: Kentucky
city: New Orleans
country: United States
facility: MBCCOP - LSU Health Sciences Center
state: Louisiana
city: Burlington
country: United States
facility: Alamance Cancer Center
state: North Carolina
city: Elkin
country: United States
facility: Hugh Chatham Memorial Hospital
state: North Carolina
city: Goldsboro
country: United States
facility: Southeastern Medical Oncology Center
state: North Carolina
city: Greenville
country: United States
facility: Brody School of Medicine at East Carolina University
state: North Carolina
city: Winston-Salem
country: United States
facility: Comprehensive Cancer Center at Wake Forest University
state: North Carolina
city: Columbus
country: United States
facility: CCOP - Columbus
state: Ohio
city: Greenville
country: United States
facility: Cancer Centers of the Carolinas - Eastside
state: South Carolina
city: Spartanburg
country: United States
facility: CCOP - Upstate Carolina
state: South Carolina
Study officials:
affiliation: Wake Forest University Health Sciences
name: Istvan Molnar, MD
role: STUDY_CHAIR
affiliation: Wake Forest University Health Sciences
name: Bayard L. Powell, MD
References: