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Completed Phase 1Phase 2 Interventional Results available

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

ClinicalTrials.gov ID: NCT01362595

Public ClinicalTrials.gov record NCT01362595. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Jul 20, 2026, 6:40 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

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

Study identification

NCT ID
NCT01362595
Recruitment status
Completed
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
Northwell Health
Other
Enrollment
55 participants

Conditions and interventions

Eligibility (public fields only)

Age range
2 Years and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
May 31, 2013
Primary completion
Jun 29, 2018
Completion
Nov 29, 2020
Last update posted
Dec 1, 2022

2013 – 2020

United States locations

U.S. sites
11
U.S. states
11
U.S. cities
11
Facility City State ZIP Site status
Phoenix Children's Hospital Phoenix Arizona 85016
Stanford University Medical Center Palo Alto California 94304
Riley Hospital for Children Indianapolis Indiana 46202
University of Louisville Louisville Kentucky 40202
Boston Children's Hospital Boston Massachusetts 02115
University of Michigan C.S. Mott Children's Hospital Ann Arbor Michigan 48109
University of Missouri-Columbia Women's and Children's Hospital Columbia Missouri 65201
Children's Specialty Center of Nevada Las Vegas Nevada 89109
Cohen Children's Medical Center of New York New Hyde Park New York 11040
Children's Hospital of Philadelphia Philadelphia Pennsylvania 19104
UT Southwestern Medical Center Dallas Texas 75390

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01362595, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Dec 1, 2022 · Synced Jul 20, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01362595 live on ClinicalTrials.gov.

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