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Trial record 11 of 1805 for:    "Anemia"
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Therapeutic Use of the Amino Acid, Leucine in the Treatment of Transfusion - Dependent Diamond Blackfan Anemia Patients (LeucineDBA)

This study is not yet open for participant recruitment.
Verified December 2012 by North Shore Long Island Jewish Health System
Sponsor:
Information provided by (Responsible Party):
Adrianna Vlachos, North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier:
NCT01362595
First received: May 20, 2011
Last updated: December 12, 2012
Last verified: December 2012
History of Changes
  Purpose

This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions.

The leucine is expected to produce a response in patients with DBA to the point where red blood cell production is increased. Red cell transfusions can then be less frequent or possibly discontinued.

The investigators will study the side effects, if any, of giving leucine to DBA patients. Leucine levels of leucine will be obtained at baseline and during the study.

The drug leucine will be provided in capsule form and taken 3 times a day for a total of 9 months.


Condition Intervention Phase
Diamond Blackfan Anemia
Blackfan Diamond Syndrome
DBA
Congenital Hypoplastic Anemia
Pure Red Cell Aplasia
 Drug: leucine
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Therapeutic Use of the Amino Acid, Leucine in the Treatment of Transfusion - Dependent Diamond Blackfan Anemia Patients: A Study in Collaboration With the Diamond Blackfan Anemia Registry

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by North Shore Long Island Jewish Health System:

Primary Outcome Measures:
  • Response to Leucine in Transfusion dependent patients with Diamond Blackfan Anemia [ Time Frame: Patients will take leucine for 9 months. The study is expected to take 12-15 months to complete. ] [ Designated as safety issue: Yes ]

    The primary outcome is the type of response observed at 9 months (and 6 months). Response to treatment can be one of the following:

    1. Complete response (CR): Hb > 9 gm/dL and transfusion-independence as defined in DBA
    2. Partial response (PR): Hb < 9 gm/dL and increased reticulocyte count to greater than 1% and any increase in transfusion interval from baseline. (Baseline reticulocytes range from 0.1 to 0.5 and transfusions are usually performed every 3 weeks. An increase of reticulocyte counts to over 1 to 1.5% and any increase in transfusion interval will be considered a PR.)
    3. No response (NR): no change in transfusion requirements and no significant change in Hb or reticulocytes (or any response that does not satisfy the conditions of either a PR or CR)
    4. Progression: worsening of disease as defined by the need for more frequent transfusions


Secondary Outcome Measures:
  • Side effects of leucine in transfusion-dependent DBA patients [ Time Frame: Total study 12-15 months ] [ Designated as safety issue: Yes ]
    Secondary outcomes include safety parameters such as type, frequency, and severity of adverse events and relationship to leucine.


Estimated Enrollment: 50
Study Start Date: January 2013
Estimated Study Completion Date: June 2014
Estimated Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Leucine
No alternative treatment arm
 Drug: leucine

Dosage of leucine will be dependent on body surface area (BSA):

leucine 700 mg/m2/dose by mouth three times a day

Other Name: leucine, L-leucine

Detailed Description:

Leucine will be provided to participants in the form of a capsule and will be taken three times daily.

Blood hemoglobin levels will be monitored every 3-4 weeks for 9 months.

The entire study will last 12-15 months in length.

Subjects must be two years of age or older and on transfusion for more than six months prior to enrollment.

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • diagnosed with Diamond Blackfan anemia as published in British Journal of Hematology
  • transfusion dependent
  • age 2 years and older
  • adequate renal function
  • adequate liver function
  • negative B-HCG if patient is a menstruating female and documentation of adequate contraception
  • signed informed consent

Exclusion Criteria:

  • Known hypersensitivity to branched chain amino acids
  • Diagnosis of an inborn error of amino acid metabolism disorder
  • Prior hematopoietic stem cell transplantation
  • Pregnancy, or plans to become pregnant during duration of trial
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01362595

Contacts
Contact: Ellen Muir, MSN, RN, CNS 516-562-1505 emuir@nshs.edu
Contact: Eva Atsidaftos, MA 516-562-1504 eatsidaf@nshs.edu

Locations
United States, New York
Cohen Children's Medical Center of New York Not yet recruiting
New Hyde Park, New York, United States, 11040
Contact: Ellen Muir, MSN     516-562-1505     emuir@nshs.edu    
Contact: Eva Atsidaftos, MA     516-562-1504     eatsidaf@nshs.edu    
Sub-Investigator: Jeffrey M Lipton, MD, PhD            
Sub-Investigator: Johnson Liu, MD            
Sub-Investigator: Eva Atsidaftos, MA            
Sub-Investigator: Sharon Singh, MD            
Sub-Investigator: Lawrence C Wolfe, MD            
Sub-Investigator: Ellen Muir, MSN, RN, CNS            
Principal Investigator: Adrianna Vlachos, MD            
Sponsors and Collaborators
North Shore Long Island Jewish Health System
Investigators
Principal Investigator: Adrianna Vlachos, MD North Shore- Long Island Jewish Medical Center; Cohen Children's Medical Center of NY
  More Information

Additional Information:
Diamond Blackfan Anemia Registry - a registry for patients with DBA in North America  This link exits the ClinicalTrials.gov site
Daniella Maria Arturi Foundation is a family sponsored group which facilitates research for DBA.  This link exits the ClinicalTrials.gov site
The DBA foundation is a family sponsored organization who raise funds for research and provides emotional support to families.  This link exits the ClinicalTrials.gov site
Provides facts about Diamond Blackfan Anemia for purposes of surveillance, awareness and prevention.  This link exits the ClinicalTrials.gov site

Publications:
AMA Department of Drugs; AMA Drug Evaluations, 6th ed. American Medical Association, Chicago, IL,1986.

Responsible Party: Adrianna Vlachos, MD, Principal Investigator, North Shore Long Island Jewish Health System
ClinicalTrials.gov Identifier: NCT01362595     History of Changes
Other Study ID Numbers: Vlachos-1
Study First Received: May 20, 2011
Last Updated: December 12, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by North Shore Long Island Jewish Health System:
leucine administration
red blood cell transfusion
Diamond Blackfan anemia

Additional relevant MeSH terms:
Anemia
Red-Cell Aplasia, Pure
Anemia, Aplastic
Anemia, Diamond-Blackfan
 Anemia, Hypoplastic, Congenital
Hematologic Diseases
Bone Marrow Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on June 18, 2013