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Completed

NCT Number: NCT00125788

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

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.

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Key information

Conditions

Sickle Cell Anemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Age range

5 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Kaiser Permanente, Bellflower, California, United States

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About this study

The primary purpose of this study is to evaluate the effectiveness of oral L-glutamine in the therapy of sickle cell anemia and sickle ß0-thalassemia.

The secondary purpose is to assess the effect of L-glutamine frequency of hospitalizations for sickle cell pain, frequency of emergency room visits for sickle cell pain; energy and appetite levels; narcotics usage.

Methodology:

By site, patients will be randomized to L-glutamine or placebo in a 1:1 ratio after a 4-week screening period. Patients will undergo 48 weeks of treatment with dosing BID orally, with dose calculated according to patient weight. Patient visits will occur every 4 weeks. After 48 weeks of treatment, dose will be tapered to zero within 3 weeks. A final evaluation visit will occur 2 weeks after last dose.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

To be eligible to participate in the study, a patient must meet all of the following inclusion criteria:

  • 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.

Exclusion criteria

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.

Treatment and study plan

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.

Other names: oral L-glutamine

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.

Other names: maltodextrin

Primary outcomes

  1. Number of Occurrences of Painful Sickle Cell Crises

    Time frame: From Week 0 through Week 48 (cumulative)

    The mean number of painful sickle crisis through week 48

Secondary outcomes

  1. Frequency of Hospitalizations for Sickle Cell Pain

    Time frame: From Week 0 through Week 48 (cumulative)

    The mean number of hospitalizations through week 48

  2. Frequency of Emergency Room Visits for Sickle Cell Pain

    Time frame: From Week 0 through Week 48 (cumulative)

    The mean number of emergency room visits through week 48

  3. The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin

    Time frame: Baseline, Weeks 4, 24 and 40

    Patient's hemoglobin will be collected at each visit.Change from Baseline will be reported at Weeks 4, 24 and 40.

  4. The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit

    Time frame: Baseline, Weeks 4, 24, and 40

    Patient's hematocrit will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40

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

    Time frame: Baseline, Weeks 0, 4, 24, 40

    Patient's reticulocyte count will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24 and 40

  6. Number of Participants With Narcotic Usage

    Time frame: Week 24, Week 48

    Analysis of narcotic usage was performed for the subset of patients with any narcotic use who completed the study. Changes in narcotic usage were determined by an independent consultant prior to database lock using morphine equivalents to determine relative use.

  7. Energy Level (11-point Scale)

    Time frame: Collected at Week 0, 8, 16, 24, 32, 40, 48

    The patient's energy level was evaluated at each visit using an 11 point scale from 0=extremely tired to 10=extremely energetic

  8. Patient Appetite (3-point Scale)

    Time frame: Collected at Week 0, 8, 16, 24, 32, 40, 48

    Patient's appetite level was evaluated at each visit using a 3 point scale: above average, average and below average. The parentages of patient at each visit whose appetite level was below, normal or above average were compared using CMH test (row mean scores) controlling for study center.

  9. The Effect of Oral L-glutamine on Vital Signs - Blood Pressure

    Time frame: Baseline, Weeks 4, 24, and 48

    Patient's blood pressure will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48

  10. The Effect of Oral L-glutamine on Vital Signs - Temperature

    Time frame: Baseline, Weeks 4, 24, and 48

    Patient's temperature will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48

  11. The Effect of Oral L-glutamine on Vital Signs - Respiration

    Time frame: Baseline, Weeks 4, 24, and 48

    Respiration will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48

  12. The Effect of Oral L-glutamine on Vital Signs - Pulse Rate

    Time frame: Baseline, Weeks 4, 24, and 48

    Patient's pulse rate will be collected at each visit, Change from Baseline will be reported at Weeks 4, 24, and 48

  13. Effect of L-glutamine on Alcohol Use

    Time frame: Weeks 0, 8,16, 24, 32, 40 and 48

    The patient's alcohol usage will be assessed at each visit. Alcohol usage will be reported at Weeks 0, 8,16, 24, 32, 40 and 48

  14. Effect of L-glutamine on Tobacco Use

    Time frame: Weeks 0, 8,16, 24, 32, 40 and 48

    Patient's alcohol usage will be assessed at each visit. Alcohol usage will be reported at Weeks 0, 8,16, 24, 32, 40 and 48

  15. The Effect of Oral L-glutamine on the Number of Days Patient's Daily Activities Are Interrupted Due to Sickle Cell Pain

    Time frame: Weeks 0, 8,16, 24, 32, 40 and 48

    Percentage of days a patient's daily activities were interrupted due to sickle pain calculated at each visit. Day's interrupted will be reported at Weeks 0, 8,16, 24, 32, 40 and 48

  16. The Effect of Oral L-glutamine on Subjective Quality of Life

    Time frame: Baseline and Week 24 (or at time of discontinuation)

    The subjective quality of life was evaluated using the scoring of the RAND 36-Item Health Survey Questionnaire. The subjective quality of life (Physical functioning, Physical health, Emotional problems, Energy/Fatigue, Emotional well being, Social functioning, Pain, General health) will be reported at Baseline and Week 24 (or at time of discontinuation). The range for Physical functioning, Physical health, Emotional problems, Emotional well being and Social functioning is 0-100, with a high score denotes a better quality of life. For Energy/Fatigue, Pain and General health the range is 0-100, with a lower score denotes better quality of life.

  17. Effect of Oral L--glutamine on Height

    Time frame: Baseline, Weeks 4, 24, and 48

    Height will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48

  18. Effect of Oral L--glutamine on Weight

    Time frame: Baseline, Weeks 4, 24 and 48

    Weight will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48

  19. Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Walk Without Rest

    Time frame: Baseline, Weeks 4, 24, and 48.

    Minutes patient could walk without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.

  20. Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Run Without Rest

    Time frame: Baseline, Weeks 4, 24, and 48.

    Minutes patient could run without rest will be measured at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.

  21. Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Walk Without Rest

    Time frame: Baseline, Weeks 4, 24, and 48.

    Distance patient could walk without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.

  22. Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Run Without Rest

    Time frame: Baseline, Weeks 4, 24, and 48.

    Distance patient could run without rest will be collected at each visit. Change from Baseline will be reported at Weeks 4, 24, and 48.

Sponsors and collaborators

Lead sponsor

Emmaus Medical, Inc.

Industry

Collaborators

  • FDA Office of Orphan Products Development

Registry information

Official study title

A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia

Important dates

Study start
2004
Primary completion
2008
Study completion
2008
First posted
Aug 2, 2005
Registry last updated
Jan 29, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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