L-leucine in Diamond Blackfan Anemia Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 16 March 2015
• Main ID: NCT02386267
• Date of registration: 18/02/2015
• Prospective Registration: No
• Primary sponsor: Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache
• Public title: L-leucine in Diamond Blackfan Anemia Patients
• Scientific title: Therapeutic Use of the Amino Acid Leucine in the Treatment of Transfusion-Dependent Diamond Blackfan Anemia Patients
• Date of first enrolment: September 2014
• Target sample size: 30
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT02386267
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Russian Federation

Contacts

• Name: Natalia - SMETANINA, MD, PhD
• Affiliation: FSCCPHOI, Outpatient Department

• Name: Natalia - SMETANINA, MD, PhD

• Name: Natalia - SMETANINA, MD, PhD

Key inclusion & exclusion criteria

Inclusion Criteria:

- signed Informed Consent Form

- diagnosed Diamond Blackfan Anemia

- transfusion dependen?e

- adequate renal function

- adequate liver function

- negative B-HCG and adequate contraception

Exclusion Criteria:

- known hypersensitivity to branched chain amino acids

- diagnosed AA metabolism disorder

- prior HSCT

- pregnancy or planning to become pregnant



Age minimum: 1 Year
Age maximum: 20 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Diamond Blackfan Anemia

Intervention(s)

Drug: L-leucine

Primary Outcome(s)

Hemoglobin level [Time Frame: every 4 weeks for 12 months]

Secondary Outcome(s)

Side effects of L-leucine in transfusion-dependent DBA patients for one year [Time Frame: every 4 weeks for 12 moths]

Secondary ID(s)

Ru0001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors