Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia (LeucineDBA)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01362595 |
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Recruitment Status :
Active, not recruiting
First Posted : May 30, 2011
Last Update Posted : September 12, 2019
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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 or disease | Intervention/treatment | Phase |
|---|---|---|
| Diamond Blackfan Anemia Blackfan Diamond Syndrome DBA Congenital Hypoplastic Anemia Pure Red Cell Aplasia | Drug: leucine | Phase 1 Phase 2 |
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.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 50 participants |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance Using Bone Marrow Failure as a Model: a Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia |
| Actual Study Start Date : | June 2013 |
| Actual Primary Completion Date : | June 30, 2018 |
| Estimated Study Completion Date : | December 30, 2019 |
| Arm | Intervention/treatment |
|---|---|
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Leucine
No alternative treatment arm
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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 |
- 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. ]
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
- Side effects of leucine in transfusion-dependent DBA patients [ Time Frame: Total study 12-15 months ]Secondary outcomes include safety parameters such as type, frequency, and severity of adverse events and relationship to leucine.
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, Learn About Clinical Studies.
| Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01362595
| United States, Arizona | |
| Phoenix Children's Hospital | |
| Phoenix, Arizona, United States, 85016 | |
| United States, California | |
| Stanford University Medical Center | |
| Palo Alto, California, United States, 94304 | |
| United States, Indiana | |
| Riley Hospital for Children | |
| Indianapolis, Indiana, United States, 46202 | |
| United States, Kentucky | |
| University of Louisville | |
| Louisville, Kentucky, United States, 40202 | |
| United States, Massachusetts | |
| Boston Children's Hospital | |
| Boston, Massachusetts, United States, 02115 | |
| United States, Michigan | |
| University of Michigan C.S. Mott Children's Hospital | |
| Ann Arbor, Michigan, United States, 48109 | |
| United States, Missouri | |
| University of Missouri-Columbia Women's and Children's Hospital | |
| Columbia, Missouri, United States, 65201 | |
| United States, Nevada | |
| Children's Specialty Center of Nevada | |
| Las Vegas, Nevada, United States, 89109 | |
| United States, New York | |
| Cohen Children's Medical Center of New York | |
| New Hyde Park, New York, United States, 11040 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| United States, Texas | |
| UT Southwestern Medical Center | |
| Dallas, Texas, United States, 75390 | |
| Principal Investigator: | Adrianna Vlachos, MD | North Shore- Long Island Jewish Medical Center; Cohen Children's Medical Center of NY |
Additional Information:
Daniella Maria Arturi Foundation is a family sponsored group which facilitates research for DBA.
The DBA foundation is a family sponsored organization who raise funds for research and provides emotional support to families.
Publications:
| Responsible Party: | Adrianna Vlachos, MD, Principal Investigator, Northwell Health |
| ClinicalTrials.gov Identifier: | NCT01362595 History of Changes |
| Other Study ID Numbers: |
12-375B |
| First Posted: | May 30, 2011 Key Record Dates |
| Last Update Posted: | September 12, 2019 |
| Last Verified: | September 2019 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
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leucine administration red blood cell transfusion Diamond Blackfan anemia |
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Anemia Anemia, Diamond-Blackfan Red-Cell Aplasia, Pure Anemia, Hypoplastic, Congenital |
Anemia, Aplastic Hematologic Diseases Bone Marrow Diseases Genetic Diseases, Inborn |