- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00003336
Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders
RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: This phase II trial is studying how well umbilical cord blood transplantation works in treating patients with severe aplastic anemia, malignant thymoma, or myelodysplasia.
Study Overview
Status
Detailed Description
OBJECTIVES:
- Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders, refractory to medical management, who are undergoing high-dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation.
- Evaluate the rate and quality of immunologic reconstitution in this patient population.
OUTLINE: Patients are stratified according to weight (under 45 kg vs over 45 kg).
Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). The regimen varies according to the underlying cause of the anemia, but could include busulfan, cyclophosphamide or melphalan, anti-thymocyte globulin or methylprednisolone, and/or radiation therapy. One day after the conditioning regimen is completed, patients receive the UCBT.
Patients are followed weekly for 3 months, at 6 months, then every 6 months for 2.5 years, then annually thereafter.
PROJECTED ACCRUAL: A total of 4-90 patients will be accrued for this study within 5 years.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Ohio
-
Cleveland, Ohio, United States, 44106-7284
- Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
DISEASE CHARACTERISTICS:
- Histologically confirmed diagnosis of severe aplastic anemia based on bone marrow cellularity of less than 20%
Must meet at least two of the following criteria:
- Granulocyte count less than 500/mm^3
- Platelet count less than 20,000/mm^3
- Reticulocyte count less than 50,000/mm^3
Following etiologies eligible:
- Fanconi's anemia
- Hypoplastic leukemia
- Monosomy 7
- Drug exposure (chloramphenicol, NSAIDS)
- Viral exposure (EBV, hepatitis, parvovirus, HIV)
- Nutritional deficiencies
- Thymoma
- Paroxysmal nocturnal hemoglobinuria
- Amegakaryocytic thrombocytopenia OR
- Histologically confirmed myelodysplastic syndrome (MDS) that is refractory to medical management or with cytogenic abnormalities predictive of transformation into acute leukemia, including 5q-, 7q-, monosomy 7, or trisomy 8
The following etiologies only are eligible:
- Refractory anemia
- Refractory anemia with ringed sideroblasts
- De novo primary MDS
- Therapy-related secondary MDS OR
- Confirmed diagnosis of inherited hematopoietic disorder that is refractory to medical management
Following etiologies eligible:
- Severe combined immunodeficiency
- Familial erythrophagocytic lymphohistiocytosis
- Wiskott-Aldrich syndrome
- Kostmann's syndrome (infantile histiocytosis)
- Chronic granulomatous disease
- Leukocytic adhesion deficiency
- Chediak-Higashi syndrome
- Paroxysmal nocturnal hemoglobinuria
- Fanconi's anemia
- Dyskeratosis congenita
- Diamond-Blackfan anemia
- Amegakaryocytic thrombocytopenia
- Osteopetrosis
- Gaucher's disease
- Lesch-Nyhan syndrome
- Mucopolysaccharidoses
- Lipodoses
- Autologous or haploidentical related peripheral blood stem cells available as backup
- Serologically matched umbilical cord blood unit available in the New York Blood Center's Placental Blood Project, or other acceptable umbilical cord blood registry
PATIENT CHARACTERISTICS:
Age:
- 55 and under
Performance status:
- Zubrod 0-1
- Karnofsky 80-100%
Life expectancy:
- At least 3 months
Hematopoietic:
- See Disease Characteristics
Hepatic:
- ALT/AST no greater than 4 times normal
- Bilirubin no greater than 2.0 mg/dL
Renal:
- Creatinine no greater than 2.0 mg/dL
- Creatinine clearance at least 50 mL/min
Cardiovascular:
- Normal cardiac function by echocardiogram or radionuclide scan
- Shortening fraction or ejection fraction at least 80% normal for age
- Non-Fanconi patients with acquired or congenital cardiomyopathy may receive melphalan as a substitute for cyclophosphamide
Pulmonary:
- FVC and FEV_1 at least 60% of predicted for age
- DLCO at least 60% of predicted in adult patients
Other:
- No active concurrent malignancy
- No active infection
- Not pregnant or nursing
- HIV negative
- Must have an available serologic matched Umbilical Cord Blood Unit (UCBU) in the New York Blood Center's Placental Blood Project, or other acceptable Umbilical Cord Blood (UCB) registry
PRIOR CONCURRENT THERAPY:
Biologic therapy:
- Not specified
Chemotherapy:
- No concurrent cytotoxic chemotherapy
Endocrine therapy:
- No concurrent immunosuppressive medications
Radiotherapy:
- No concurrent radiotherapy
Surgery:
- Not specified
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Event-free survival by disease assessment
Time Frame: at 100 days and at 6, 9, 12, 18, and 24 months
|
at 100 days and at 6, 9, 12, 18, and 24 months
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Umbilical cord blood donor engraftment by chimerism and complete blood count (CBC) at time of myeloid recovery.
Time Frame: 100 days and at 6, 9, 12, 18, and 24 months
|
100 days and at 6, 9, 12, 18, and 24 months
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Mary J. Laughlin, MD, Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
- refractory anemia
- refractory anemia with ringed sideroblasts
- de novo myelodysplastic syndromes
- previously treated myelodysplastic syndromes
- secondary myelodysplastic syndromes
- childhood myelodysplastic syndromes
- atypical chronic myeloid leukemia
- myelodysplastic/myeloproliferative disease, unclassifiable
Additional Relevant MeSH Terms
- Pathologic Processes
- Neoplasms
- Disease
- Bone Marrow Diseases
- Hematologic Diseases
- Precancerous Conditions
- Syndrome
- Myelodysplastic Syndromes
- Preleukemia
- Myeloproliferative Disorders
- Myelodysplastic-Myeloproliferative Diseases
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Autonomic Agents
- Peripheral Nervous System Agents
- Anti-Inflammatory Agents
- Antirheumatic Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Antiemetics
- Gastrointestinal Agents
- Glucocorticoids
- Hormones
- Hormones, Hormone Substitutes, and Hormone Antagonists
- Neuroprotective Agents
- Protective Agents
- Antineoplastic Agents, Alkylating
- Alkylating Agents
- Myeloablative Agonists
- Methylprednisolone
- Cyclophosphamide
- Melphalan
- Busulfan
- Antilymphocyte Serum
Other Study ID Numbers
- CWRU5Y97
- P30CA043703 (U.S. NIH Grant/Contract)
- CASE-CWRU-5Y97
- NCI-G98-1431
- CASE-5Y97
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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