Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00003336
• Date of registration: 01/11/1999
• Prospective Registration: No
• Primary sponsor: Case Comprehensive Cancer Center
• Public title: Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions
• Scientific title: 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
• Date of first enrolment: January 1998
• Target sample size: 6
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00003336
• Study type: Interventional
• Study design: Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Mary J. Laughlin, MD
• Affiliation: Ireland Cancer Center at University Hospitals Case Medical Center, Case Comprehensive Cancer Center

Key inclusion & exclusion criteria

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



Age minimum: N/A
Age maximum: 55 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Myelodysplastic/Myeloproliferative Diseases

Leukemia

Myelodysplastic Syndromes

Intervention(s)

Drug: melphalan

Procedure: bone marrow ablation with stem cell support

Biological: anti-thymocyte globulin

Drug: cyclophosphamide

Radiation: radiation therapy

Drug: busulfan

Procedure: umbilical cord blood transplantation

Drug: methylprednisolone

Primary Outcome(s)

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

Secondary Outcome(s)

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]

Secondary ID(s)

CASE-5Y97

CASE-CWRU-5Y97

P30CA043703

CWRU5Y97

NCI-G98-1431

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Cancer Institute (NCI)