Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692 | Completed Phase 1 Results

• 1 other identifier: Pro00008753
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.

Conditions

Non Malignant Disorders; Immunodeficiencies; Congenital Marrow Failures; Hemoglobinopathies; Inborn Errors of Metabolism; Sickle Cell; Thalassemia; Lysosomal Storage Disease

Keywords

Immunodeficiencies; Congenital Marrow Failures; Hemoglobinopathies; Inborn Errors of Metabolism; SCIDS; Wiskott Aldrich; FEL; HLH; IPEX; LAD; Sickle Cell; Thalassemia; Omenn's Syndrome; Hurler's Syndrome; MLD; ALD; Sanfilippo; Krabbe; Hunter's syndrome; TaySachs; Diamond Blackfan Anemia; transplant; MPS; Gaucher

Outcome Measures

Primary Outcomes (1)

• Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.

  Determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor cells at 180 days) following reduced intensity conditioning regimens in children \< 21 years receiving cord blood transplant for non-malignant disorders.

  180 days post transplant

Secondary Outcomes (8)

• To Describe the Pace of Neutrophil Recovery

  42 days post transplant

• To Evaluate the Pace of Immune Reconstitution.

  1 year post transplant

• To Determine the Overall Survival at day180 Post-transplant

  180 days

• To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)

  100 days post transplant

• To Describe the Incidence of Grade 3-4 Organ Toxicity

  2 years post transplant

Study Arms (1)

RIC Cord Blood Transplant

EXPERIMENTAL

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant

Biological: Unrelated Umbilical Cord Blood Transplant; Drug: Reduced Intensity Conditioning

Interventions

Unrelated Umbilical Cord Blood Transplant BIOLOGICAL

Reduced Intensity Conditioning for unrelated umbilical cord blood transplant

RIC Cord Blood Transplant

Reduced Intensity Conditioning DRUG

Also known as: Campath, Hydroxyurea, Fludarabine, Melphalan, Thiotepa

RIC Cord Blood Transplant

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• years of age with a diagnosis of a immunodeficiency, congenital marrow failure syndrome, inborn error of metabolism, or hereditary anemia
• Appropriately matched related or unrelated umbilical cord blood unit with a cell dose ≥ 3 x 10e7cells/kg
• Performance score (lansky or karnofsky) greater than or equal to 70
• Adequate organ function (Creatinine ≤ 2.0 mg/dl and creatinine clearance ≥ 50 ml/min/1.73 m2; Hepatic transaminases (ALT/AST) ≤ 4 x normal; Shortening fraction \>26% or ejection fraction \>40% or \> 80% of normal value for age; Pulmonary function tests demonstrating CVC or FEV1/FVC of \>60% of predicted for age.)
• Informed consent
• Not pregnant or breast feeding
• Minimum life expectancy of at least 6 months
• HIV negative
• No uncontrolled infections at the time of cytoreduction

You may not qualify if:

• Patients with hemoglobinopathies \> 3 years of age
• UCB unit with a total nucleated cell count \< 3 x 10e7/kg or \> 2 antigen mismatching
• Available HLA-matched related living donor able to donate without previous UCB donation
• Allogeneic hematopoietic stem cell transplant within the previous 6 months
• Any active malignancy, MDS, or any history of malignancy
• Severe acquired aplastic anemia
• DLCO \< 60% of normal value for age; requirement for supplemental oxygen
• Uncontrolled bacterial, viral or fungal infection (currently taking medication and progression of clinical symptoms)
• Pregnancy or nursing mother
• HIV/HTLV seropositive, Hep B surface antigen positive, or HCV RNA positive by PCR
• Any condition that precludes serial follow-up

Sponsors & Collaborators

• Duke University: lead

Study Sites (1)

Duke University Medical Center Pediatric Blood and Marrow Transplant Program

Durham, North Carolina, 27705, United States

Location

Related Publications (1)

• Parikh SH, Mendizabal A, Benjamin CL, Komanduri KV, Antony J, Petrovic A, Hale G, Driscoll TA, Martin PL, Page KM, Flickinger K, Moffet J, Niedzwiecki D, Kurtzberg J, Szabolcs P. A novel reduced-intensity conditioning regimen for unrelated umbilical cord blood transplantation in children with nonmalignant diseases. Biol Blood Marrow Transplant. 2014 Mar;20(3):326-36. doi: 10.1016/j.bbmt.2013.11.021. Epub 2013 Dec 1.

  PMID: 24296492 DERIVED

MeSH Terms

Conditions

Immunologic Deficiency Syndromes; Hemoglobinopathies; Metabolism, Inborn Errors; Thalassemia; Lysosomal Storage Diseases; Severe Combined Immunodeficiency; Mucopolysaccharidosis I; Mucopolysaccharidosis II; Anemia, Diamond-Blackfan

Interventions

Alemtuzumab; Hydroxyurea; fludarabine; Melphalan; Thiotepa

Condition Hierarchy (Ancestors)

Immune System Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Primary Immunodeficiency Diseases; Infant, Newborn, Diseases; DNA Repair-Deficiency Disorders; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System; Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Red-Cell Aplasia, Pure; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Urea; Amides; Organic Chemicals; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Phosphoramides; Organophosphorus Compounds; Triethylenephosphoramide; Aziridines; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Dr. Suhag Parikh
• Organization: Duke University Medical Center

suhag.parikh@dm.duke.edu

919-668-1100

Study Officials

• Suhag Parikh, MD

  Duke Pediatric Blood and Marrow Transplant

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 28, 2008
• First Posted: September 1, 2008
• Study Start: October 1, 2008
• Primary Completion: December 1, 2012
• Study Completion: April 1, 2014
• Last Updated: August 13, 2014
• Results First Posted: August 13, 2014

Record last verified: 2014-07

Locations

US (1)