Therapeutic Use of the Amino Acid, Leucine in the Treatment of Transfusion - Dependent Diamond Blackfan Anemia Patients (LeucineDBA)
This study is currently recruiting participants.
(see Contacts and Locations)
Verified March 2014 by North Shore Long Island Jewish Health System
Sponsor:
North Shore Long Island Jewish Health System
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: March 27, 2014
Last verified: March 2014
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
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:
Genetics Home Reference related topics:
Diamond-Blackfan anemia
Drug Information available for:
Leucine
Genetic and Rare Diseases Information Center resources:
Diamond-Blackfan Anemia
Pure Red Cell Aplasia
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:
- Complete response (CR): Hb > 9 gm/dL and transfusion-independence as defined in DBA
- 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.)
- 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)
- 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: | June 2013 |
| Estimated Study Completion Date: | April 2015 |
| Estimated Primary Completion Date: | April 2015 (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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01362595
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, Arizona | |
| Phoenix Children's Hospital | Not yet recruiting |
| Phoenix, Arizona, United States, 85016 | |
| Contact: Erica Olson, RN 602-933-0171 eolson1@phoenixchildrens.com | |
| Principal Investigator: Christine Knoll, MD | |
| Sub-Investigator: Sanjay Shah, MD | |
| United States, California | |
| Stanford University Medical Center | Recruiting |
| Palo Alto, California, United States, 94304 | |
| Contact: Kirsten Mouradian, RN, FNP 650-724-2448 KMouradian@stanfordchildrens.org | |
| Contact: Anu Narla, MD 650-725-7349 anunarla@stanford.edu | |
| Principal Investigator: Bertil Glader, MD, PhD | |
| Sub-Investigator: Anu Narla, MD | |
| United States, Indiana | |
| Riley Hospital for Children | Not yet recruiting |
| Indianapolis, Indiana, United States, 46202 | |
| Contact: Anne Bubnick, CCRP 317-948-0101 abubnick@iu.edu | |
| Principal Investigator: Gregorz Nalepa, MD, PhD | |
| United States, Massachusetts | |
| Boston Children's Hospital | Not yet recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Contact: Jesse K McKenna, MPH 617-355-3748 Jesse.McKenna@childrens.harvard.edu | |
| Principal Investigator: Colin Sieff, MBBCh | |
| Sub-Investigator: Ellis Neufeld, MD, PhD | |
| Sub-Investigator: Vijay Sankaran, MD, PhD | |
| United States, Michigan | |
| University of Michigan C.S. Mott Children's Hospital | Recruiting |
| Ann Arbor, Michigan, United States, 48109 | |
| Contact: Angela Stovall 734-232-3978 astovall@med.umich.edu | |
| Principal Investigator: Kelly Walkovich, MD | |
| Sub-Investigator: James Connelly, MD | |
| United States, Missouri | |
| University of Missouri-Columbia Women's and Children's Hospital | Not yet recruiting |
| Columbia, Missouri, United States, 65201 | |
| Contact: Mihaela Popescu, MS, CCRP 573-882-1960 popescumi@health.missouri.edu | |
| Principal Investigator: Thomas W Loew, MD | |
| Sub-Investigator: Barbara Gruner, MD | |
| United States, New York | |
| Cohen Children's Medical Center of New York | 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: Lawrence C Wolfe, MD | |
| Sub-Investigator: Ellen Muir, MSN, RN, CNS | |
| Principal Investigator: Adrianna Vlachos, MD | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Contact: Swapna Pamu 267-426-6765 pamus@email.chop.edu | |
| Principal Investigator: Monica Bessler, MD, PhD | |
| Sub-Investigator: Helge Hartung, MD | |
| United States, Texas | |
| UT Southwestern Medical Center | Recruiting |
| Dallas, Texas, United States, 75390 | |
| Contact: Zora R Rogers, MD 214-648-3896 Zora.Rogers@UTSouthwestern.edu | |
| Contact: Leah M Adix, CCRP 214-456-2888 leah.adix@childrens.com | |
| Principal Investigator: Zora R Rogers, MD | |
| Sub-Investigator: George R Buchanan, MD | |
| Sub-Investigator: Timothy L McCavit, 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:
Provides facts about Diamond Blackfan Anemia for purposes of surveillance, awareness and prevention.
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: | March 27, 2014 |
| 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 Anemia, Diamond-Blackfan Anemia, Hypoplastic, Congenital Red-Cell Aplasia, Pure Anemia, Aplastic |
Bone Marrow Diseases Genetic Diseases, Inborn Hematologic Diseases Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on March 03, 2015