Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00054236
• Date of registration: 05/02/2003
• Prospective Registration: No
• Primary sponsor: Case Comprehensive Cancer Center
• Public title: Combination Chemotherapy Followed By Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Severe Aplastic Anemia
• Scientific title: Pilot Study Of Multiple Umbilical Cord Blood Unit Transplantation Following Non-Myeloablative Conditioning In Patients With Hematologic Disorders Or Severe Aplastic Anemia
• Date of first enrolment: May 2002
• Target sample size: 55
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00054236
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Brenda Cooper, MD
• Affiliation: Case Medical Center, University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center

Key inclusion & exclusion criteria

DISEASE CHARACTERISTICS:

- One of the following histologically confirmed diagnoses:

- Acquired severe aplastic anemia

- Meets at least 2 of the following criteria:

- Granulocyte count less than 500/mm^3

- Platelet count less than 20,000/mm^3

- Absolute reticulocyte count less than 20,000/mm^3 (after correction for
hematocrit)

- Unresponsive to OR recurrent disease after prior treatment with
anti-thymocyte globulin and/or cyclosporine

- Acute myeloid leukemia (AML), meeting 1 of the following criteria:

- Failed induction therapy

- In first complete remission (CR) with any of the following high-risk
features:

- Stem cell or biphenotype classification (M0)

- Erythroleukemia (M6)

- Acute megakaryocytic leukemia (M7)

- Cytogenetic markers indicative of poor prognosis

- t(15;17) translocation and failed first-line induction therapy OR there
is molecular evidence of persistent disease

- t(8;21) and inv(16) translocations and failed first-line induction
therapy

- In early relapse*

- In second or subsequent remission

- Recurrent disease after prior autologous stem cell transplantation (SCT)
NOTE: *No refractory relapse

- Acute lymphoblastic leukemia, meeting 1 of the following criteria:

- In early relapse*

- In second or subsequent remission

- In first CR with the following high-risk features:

- t(4;11) or t(9;22) translocation

- Hyperleukocytosis (initial WBC greater than 30,000/mm^3)

- Failed to achieve CR by day 28 of standard induction therapy

- Recurrent disease after prior autologous SCT NOTE: *No refractory relapse

- Chronic myelogenous leukemia

- Chronic or accelerated phase that has failed medical management

- Blastic phase allowed after reinduction chemotherapy induces chronic phase

- Myelodysplastic syndromes meeting 1 of the following criteria:

- Refractory to medical management

- Presence of cytogenetic abnormalities predictive of transformation to acute
leukemia, including the following:

= 5q- = 7q-

- Monosomy 7 and trisomy 8

- Evidence of evolution to AML (e.g., refractory anemia with excess blasts [RAEB], or
RAEB in transformation)

- Chronic lymphocytic leukemia

- Refractory to treatment including fludarabine-based therapy

- Recurrent disease after prior autologous SCT

- Multiple myeloma

- Recurrent disease after prior autologous SCT

- Beyond first CR or failed induction therapy

- Disease is sensitive to pretransplantation cytoreduction

- Hodgkin's lymphoma

- Beyond first CR or failed induction therapy

- Disease is sensitive to pretransplantation cytoreduction

- Non-Hodgkin's lymphoma (NHL)

- Recurrent disease after prior autologous SCT

- Beyond first CR or failed induction therapy

- Disease is sensitive to pretransplantation cytoreduction

- Mantle zone NHL allowed after induction therapy

- Myeloproliferative disorders

- Refractory to medical management

- Allografting required unless grade 3 or greater myelofibrosis by bone marrow
biopsy

- No HLA-matched sibling donor available

- Ineligible for a myeloablative conditioning regimen due to advanced age
(over 55), extensive prior therapy, and/or other comorbidities

- If under age 55, must meet at least 1 of the following criteria:

- Received extensive prior therapy

- Organ toxicity or infection precluding eligibility for allogeneic transplantation
with full ablation conditioning

- Availability of 2-5 umbilical cord blood units that are at least a 4/6 HLA
match

- No active CNS disease

- No primary or grade 3 or 4 myelofibrosis

PATIENT CHARACTERISTICS:

Age

- Any age

Performance status

- Karnofsky 70-100% (for patients 16 years of age and older)

- Lansky 50-100% (for patients under 16 years of age)

Life expectancy

- At least 3 months

Hematopoietic

- See Disease Characteristics

Hepatic

- ALT/AST less than 4 times normal

- Bilirubin less than 2.0 mg/dL (unless due to hepatic infiltration by primary
malignancy)

Renal

- Creatinine clearance greater than 40 mL/min

Cardiovascular

- Shortening fraction or ejection fraction greater than 40% of normal value for age by
echocardiogram or radionuclide scan

Pulmonary

- FVC and FEV_1 greater than 60% of predicted

- DLCO greater than 60% of predicted (adult patients)

- Clearance by pulmonologist required if patient cannot perform pulmonary function tests

Other

- Not pregnant or nursing

- No uncontrolled active infection (viral, bacterial, or fungal)

- HIV negative

PRIOR CONCURRENT THERAPY:

Biologic therapy

- See Disease Characteristics

- More than 3 months since prior autologous stem cell transplantation

Chemotherapy

- See Disease Characteristics

- At least 4 weeks since prior chemotherapy

Endocrine therapy

- Not specified

Radiotherapy

- Not specified

Surgery

- Not specified

Other

- Recovered from prior therapy

- No other concurrent investigational agents that would preclude study participation or
increase risk to patient

- Investigational diagnostic procedures allowed



Age minimum: N/A
Age maximum: 120 Years
Gender: All

Health Condition(s) or Problem(s) studied

Myelodysplastic/Myeloproliferative Diseases

Lymphoma

Chronic Myeloproliferative Disorders

Leukemia

Multiple Myeloma and Plasma Cell Neoplasm

Myelodysplastic Syndromes

Intervention(s)

Biological: anti-thymocyte globulin

Biological: filgrastim

Drug: cyclophosphamide

Procedure: umbilical cord blood transplantation

Drug: methylprednisolone

Drug: fludarabine phosphate

Primary Outcome(s)

Event-free survival by disease assessment [Time Frame: at 28 and 100 days and then at 6, 9, 12, 18, and 24 months]

Secondary Outcome(s)

Umbilical cord blood donor engraftment by chimerism and complete blood count (CBC) [Time Frame: monthly for 6 months and then at 9, 12, 18, and 24 months]

Secondary ID(s)

CWRU6Y01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors