L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia

NCT00125788 | Completed Phase 2 Results

• 1 other identifier: 10478
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 5

Brief Summary

The purpose of this research is to evaluate the effects of L-glutamine as a therapy for sickle cell anemia and sickle ß0-thalassemia. as evaluated by the number of occurrences of sickle cell crises.

Conditions

Sickle Cell Anemia; Thalassemia

Keywords

sickle cell disease; sickle cell anemia; L-glutamine; Sickle Cell Anemia (homozygous); Sickle ß0-Thalassemia

Outcome Measures

Primary Outcomes (1)

• Number of Occurrences of Painful Sickle Cell Crises

  The mean number of painful sickle crisis through week 48

  From Week 0 through Week 48 (cumulative)

Secondary Outcomes (22)

• Frequency of Hospitalizations for Sickle Cell Pain

  From Week 0 through Week 48 (cumulative)

• Frequency of Emergency Room Visits for Sickle Cell Pain

  From Week 0 through Week 48 (cumulative)

• The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin

  Baseline, Weeks 4, 24 and 40

• The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit

  Baseline, Weeks 4, 24, and 40

• The Effect of Oral L-glutamine on Hematological Parameters - Reticulocyte Count

  Baseline, Weeks 0, 4, 24, 40

Study Arms (2)

investigational product

EXPERIMENTAL

L-glutamine

Drug: L-glutamine

placebo

PLACEBO COMPARATOR

maltodextrin

Drug: Placebo

Interventions

L-glutamine DRUG

Approximately 0.3 g/kg total daily dose of L-glutamine will be orally administered (over two doses), with a maximum total daily dose of 30 grams.

Also known as: oral L-glutamine

investigational product

Placebo DRUG

Approximately 0.3 g/kg total daily dose of maltodextrin placebo will be orally administered (over two doses), with a maximum total daily dose of 30 grams.

Also known as: maltodextrin

placebo

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient is at least five years of age.
• Patient has been diagnosed with sickle cell anemia or sickle ß0-thalassemia (documented by hemoglobin electrophoresis).
• Patient has had at least two episodes of painful crises within 12 months of the screening visit.
• If the patient has been treated with an anti-sickling agent within three months of the screening visit, the therapy must have been continuous for at least three months with the intent to continue for the next 14 months.
• Patient or the patient's legally authorized representative has given written informed consent.
• If the patient is a female of child-bearing potential, she agrees to practice a recognized form of birth control during the course of the study.

You may not qualify if:

• If the patient meets any of the following criteria, the patient must not be enrolled:
• Patient has a significant medical condition that required hospitalization (other than sickle painful crisis) within two months of the screening visit.
• Patient has diabetes mellitus with untreated fasting blood sugar \>115 mg/dL.
• Patient has prothrombin time International Normalized Ratio (INR) \> 2.0.
• Patient has serum albumin \< 3.0 g/dl.
• Patient has received any blood products within three weeks of the screening visit.
• Patient has a history of uncontrolled liver disease or renal insufficiency.
• Patient is pregnant or lactating.
• Patient has been treated with an experimental anti-sickling medication/treatment (except hydroxyurea) within 30 days of the screening visit.
• Patient has been treated with an experimental drug within 30 days of the screening visit.
• There are factors that would, in the judgment of the investigator, make it difficult for the patient to comply with the requirements of the study.

Sponsors & Collaborators

• Emmaus Medical, Inc.: lead
• FDA Office of Orphan Products Development: collaborator

Study Sites (5)

Kaiser Permanente

Bellflower, California, 90706, United States

Location

Harbor-UCLA Medical Center

Torrance, California, 90502, United States

Location

Grady Memorial Hospital

Atlanta, Georgia, 30303, United States

Location

University of Medicine and Dentistry, New Jersey

New Brunswick, New Jersey, 08903, United States

Location

Jacobi Medical Center

The Bronx, New York, 10461, United States

Location

Related Publications (1)

• Bolarinwa AB, Oduwole O, Okebe J, Ogbenna AA, Otokiti OE, Olatinwo AT. Antioxidant supplementation for sickle cell disease. Cochrane Database Syst Rev. 2024 May 22;5(5):CD013590. doi: 10.1002/14651858.CD013590.pub2.

  PMID: 38775255 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell; Thalassemia

Interventions

Glutamine; maltodextrin

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Amino Acids, Basic; Amino Acids; Amino Acids, Peptides, and Proteins; Amino Acids, Diamino; Amino Acids, Neutral

Results Point of Contact

• Title: Yutaka Niihara, MD, MPH
• Organization: Emmaus Medical, Inc.

yniihara@emmausmedical.com

310-214-0065

Study Officials

• Yutaka Niihara, MD

  CEO, Emmaus Medical, Inc

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2005
• First Posted: August 2, 2005
• Study Start: March 1, 2004
• Primary Completion: July 1, 2008
• Study Completion: July 1, 2008
• Last Updated: January 29, 2021
• Results First Posted: January 29, 2021

Record last verified: 2020-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)