Unrelated Donor Transplant for Malignant and Non-Malignant Disorders

Brief Title

Unrelated Donor Transplant for Malignant and Non-Malignant Disorders

Official Title

Unrelated Donor Stem Cell Transplant for Patients With Malignant and Non-Malignant Disorders

Brief Summary

      Unrelated matched donor (cord blood, bone marrow or peripheral blood) allogeneic stem cell
      transplantation (UDAlloSCT) with either myeloablative or reduced intensity conditioning will
      be well tolerated and result in a high degree of engraftment in patients with selected
      malignant and non malignant disorders.
    

Detailed Description

      This is a non-randomized study to determine the tolerability and degree of engraftment of
      unrelated matched donor allogeneic stem cell transplantation with either myeloablative or
      reduced intensity conditioning in patients with selected malignant and non malignant
      disorders. Patients will receive one of either full intensity or reduced intensity regimen
      based on the patient's disease status, organ function and performance and determined by the
      PI.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Incidence of toxicity related to myeloablative therapy

Secondary Outcome

 Incidence of toxicity related to reduced intensity therapy

Condition

Leukemia

Intervention

UDAlloSCT

Study Arms / Comparison Groups

 UDAlloSCT + Therapy
Description:  This is a non-randomized study to test the safety and response of unrelated matched donor allogeneic stem cell transplantation (UDAlloSCT) with either myleoablative (full intensity) or reduced intensity conditioning therapy in patients with selected malignant and non-malignant disorders. UDAlloSCT has been performed in both adults and children as an alternative transplant for patients who lack and HLA-matched family donor in both malignant and non-malignant disease with varying degrees of response.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Procedure

Estimated Enrollment

22

Start Date

November 2002

Completion Date

April 2011

Primary Completion Date

April 2011

Eligibility Criteria

        Inclusion Criteria:

          -  Adequate renal function defined as: serum creatinine 2.0 x normal, or creatinine
             clearance or radioisotope GFR > 40 ml/min/m2 or > 40 ml/min/1.73 m2 or an equivalent
             GFR as determined by the institutional normal range.

          -  Adequate liver function defined as: total bilirubin < 2.5 x normal; or SGOT (AST) or
             SGPT (ALT) < 5.0 x normal.

          -  Adequate cardiac function defined as: shortening fraction of > 25% by echocardiogram,
             or ejection fraction of > 40% by radionuclide angiogram or echocardiogram.

          -  Adequate pulmonary function defined as: DLCO > 35% by pulmonary function test. For
             children who are uncooperative, no evidence of dyspnea at rest, no exercise
             intolerance, and a pulse oximetry > 94% in room air.

          -  Diseases:

               -  CML (CP, AP or BC)

               -  AML/MDS/JCML

               -  ALL

               -  Lymphoma (Hodgkin's and non-Hodgkin's)

               -  Non-malignant disorders

          -  Bone Marrow Failure Syndromes: Patients with the following diagnoses are eligible:

               -  Severe Aplastic Anemia:

               -  Fanconi Anemia

               -  Severe Congenital Neutropenia (Kostmann's Syndrome)

               -  Amegakaryocytic Thrombocytopenia

               -  Diamond-Blackfan Anemia

               -  Infantile Osteopetrosis

               -  Schwachman-Diamond Syndrome

               -  Dyskeratosis Congenita

               -  Other bone marrow failure syndromes at discretion of Principal Investigator

          -  Immunodeficiencies:

               -  SCIDS, all subtypes

               -  Combined Immunodeficiency Syndrome

               -  Wiskott-Aldrich syndrome

               -  Chronic Granulomatous Disease

               -  Chediak-Higashi Syndrome

               -  Leukocyte Adhesion Deficiency

               -  Other immunodeficiencies at discretion of Principal Investigator

          -  Inborn Errors of Metabolism (IEOM):

               -  Transplant is recommended for the following disorders: Hurler syndrome
                  (L-iduronidase deficiency, MPS-I), Maroteaux-Lamy syndrome
                  (galactosamine-4-sulfatase deficiency, MP VI), Sly syndrome (glucuronidase
                  deficiency, MPS-VII), Globoid cell Leukodystrophy
                  (galactocerebrosidasedeficiency), Metachromatic leukodystrophy (arylsulfatase A
                  deficiency), Childhood-onset X-linked adrenoleukodystrophy (X-ALD), Fucosidosis
                  (fucosidase deficiency), Mannosidosis, Aspartylglucosaminuria, Niemann-Pick
                  Disease Type B (acid sphingomyelinase deficiency), Gaucher disease
                  (glucocerebrosidase deficiency) Type I (non neuropathic), Other diagnoses may be
                  considered at the discretion of the Principal Investigator

               -  For X-ALD patients greater than 5 years of age, IQ > 80 is required. For other
                  patients greater than 5 years of age, IQ > 70 is required.

               -  For patients less than 5 years of age, the developmental quotient or clinical
                  neurodevelopmental examination should demonstrate potential for stabilization at
                  a level of functioning where continuous life support (e.g. mechanical
                  ventilation) would not be predicted to be required in the year following
                  transplantation.

          -  Histiocytosis:

               -  Hemophagocytic Lymphohistiocytosis (HLH)

               -  Familial Erythrophagocytic Lymphohistiocytosis

               -  Langerhans Cell Histiocytosis

               -  Malignant Histiocytosis

          -  Other Malignant and non-malignant diseases: Other malignant and non-malignant diseases
             not listed above may be eligible if deemed appropriate by the Principal Investigator.

        Exclusion Criteria:

          -  Women who are pregnant and/or breast feeding are ineligible
      

Gender

All

Ages

N/A - 55 Years

Accepts Healthy Volunteers

No

Contacts

Mitchell S Cairo, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01050439

Organization ID

AAAB3095

Secondary IDs

CHNY-02-516

Responsible Party

Sponsor

Study Sponsor

Columbia University


Study Sponsor

Mitchell S Cairo, MD, Principal Investigator, Columbia University


Verification Date

April 2015