Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia (LeucineDBA)
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
Sponsor: Northwell Health
A PHASE1/PHASE2 clinical study on Blackfan Diamond Syndrome and Congenital Hypoplastic Anemia, this trial is completed. The trial is conducted by Northwell Health and has accumulated 15 data snapshots since 2013. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.
Study Description(click to expand)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.
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.
Status Flow
Change History
15 versions recorded-
Jan 2026 — Present [monthly]
Completed PHASE1/PHASE2
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Sep 2024 — Present [monthly]
Completed PHASE1/PHASE2
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Jul 2024 — Sep 2024 [monthly]
Completed PHASE1/PHASE2
Phase: PHASE1_PHASE2 → PHASE1/PHASE2
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Jan 2023 — Jul 2024 [monthly]
Completed PHASE1_PHASE2
Status: Unknown Status → Completed
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Mar 2022 — Jan 2023 [monthly]
Unknown Status PHASE1_PHASE2
Status: Active Not Recruiting → Unknown Status
▶ Show 10 earlier versions
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Jan 2021 — Mar 2022 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Mar 2020 — Jan 2021 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Jan 2020 — Mar 2020 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Oct 2019 — Jan 2020 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Sep 2019 — Oct 2019 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Apr 2019 — Sep 2019 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Jun 2018 — Apr 2019 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Feb 2018 — Jun 2018 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Feb 2017 — Feb 2018 [monthly]
Active Not Recruiting PHASE1_PHASE2
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Jan 2017 — Feb 2017 [monthly]
Active Not Recruiting PHASE1_PHASE2
First recorded
Jun 2013
Trial started
Per CT.gov start date — pre-dates our first snapshot
Eligibility Summary
No eligibility information available.
Contact Information
- Northwell Health
For direct contact, visit the study record on ClinicalTrials.gov .