CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 55 enrolled
Drug / intervention
leucinedrug
Likely dose
leucine 700 mg/m2from record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01362595
NCT01362595Phase 2Completed
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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

Northwell Health·interventional·Posted May 30, 2011·Updated Dec 2, 2022

In Brief

A Phase 2 clinical trial evaluating leucine for Diamond Blackfan Anemia and 4 related conditions. Completed, enrolled 55 participants across 11 sites.

Detailed Summary

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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDiamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
2011201220132014201520162017201820192020202120222023202420252026
First PostedMay 30, 2011
Enrollment StartJun 1, 2013
Primary CompletionJun 30, 2018
Study CompletionNov 30, 2020
TodayJul 2, 2026
Enrollment to primary: 5.1 yearsPosted 15.1 years ago

Interventions

leucinedrug

Dosage of leucine will be dependent on body surface area (BSA): leucine 700 mg/m2/dose by mouth three times a day