Clinical trial NCT00125788: L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Anemia
Thalassemia
Keywords: sickle cell disease
sickle cell anemia
L-glutamine
Sickle Cell Anemia (homozygous)
Sickle ß0-Thalassemia
Lead sponsor: Emmaus Medical, Inc.
Sponsor class: INDUSTRY
Collaborators: name: FDA Office of Orphan Products Development
First posted: 2005-08-02
Enrollment: 70
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Detailed description: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Occurrences of Painful Sickle Cell Crises
timeFrame: From Week 0 through Week 48 (cumulative)
Secondary outcomes:
measure: Frequency of Hospitalizations for Sickle Cell Pain
timeFrame: From Week 0 through Week 48 (cumulative)
measure: Frequency of Emergency Room Visits for Sickle Cell Pain
timeFrame: From Week 0 through Week 48 (cumulative)
measure: The Effect of Oral L-glutamine on Hematological Parameters - Hemoglobin
timeFrame: Baseline, Weeks 4, 24 and 40
measure: The Effect of Oral L-glutamine on Hematological Parameters - Hematocrit
timeFrame: Baseline, Weeks 4, 24, and 40
measure: The Effect of Oral L-glutamine on Hematological Parameters - Reticulocyte Count
timeFrame: Baseline, Weeks 0, 4, 24, 40
measure: Number of Participants With Narcotic Usage
timeFrame: Week 24, Week 48
measure: Energy Level (11-point Scale)
timeFrame: Collected at Week 0, 8, 16, 24, 32, 40, 48
measure: Patient Appetite (3-point Scale)
timeFrame: Collected at Week 0, 8, 16, 24, 32, 40, 48
measure: The Effect of Oral L-glutamine on Vital Signs - Blood Pressure
timeFrame: Baseline, Weeks 4, 24, and 48
measure: The Effect of Oral L-glutamine on Vital Signs - Temperature
timeFrame: Baseline, Weeks 4, 24, and 48
measure: The Effect of Oral L-glutamine on Vital Signs - Respiration
timeFrame: Baseline, Weeks 4, 24, and 48
measure: The Effect of Oral L-glutamine on Vital Signs - Pulse Rate
timeFrame: Baseline, Weeks 4, 24, and 48
measure: Effect of L-glutamine on Alcohol Use
timeFrame: Weeks 0, 8,16, 24, 32, 40 and 48
measure: Effect of L-glutamine on Tobacco Use
timeFrame: Weeks 0, 8,16, 24, 32, 40 and 48
measure: The Effect of Oral L-glutamine on the Number of Days Patient's Daily Activities Are Interrupted Due to Sickle Cell Pain
timeFrame: Weeks 0, 8,16, 24, 32, 40 and 48
measure: The Effect of Oral L-glutamine on Subjective Quality of Life
timeFrame: Baseline and Week 24 (or at time of discontinuation)
measure: Effect of Oral L--glutamine on Height
timeFrame: Baseline, Weeks 4, 24, and 48
measure: Effect of Oral L--glutamine on Weight
timeFrame: Baseline, Weeks 4, 24 and 48
measure: Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Walk Without Rest
timeFrame: Baseline, Weeks 4, 24, and 48.
measure: Effect of L-glutamine on Subjective Exercise Tolerance - Minutes Patient Could Run Without Rest
timeFrame: Baseline, Weeks 4, 24, and 48.
measure: Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Walk Without Rest
timeFrame: Baseline, Weeks 4, 24, and 48.
measure: Effect of L-glutamine on Subjective Exercise Tolerance - Distance Patient Could Run Without Rest
timeFrame: Baseline, Weeks 4, 24, and 48.
Other outcomes:
Locations:
city: Bellflower
country: United States
facility: Kaiser Permanente
state: California
city: Torrance
country: United States
facility: Harbor-UCLA Medical Center
state: California
city: Atlanta
country: United States
facility: Grady Memorial Hospital
state: Georgia
city: New Brunswick
country: United States
facility: University of Medicine and Dentistry, New Jersey
state: New Jersey
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
Study officials:
affiliation: CEO, Emmaus Medical, Inc
name: Yutaka Niihara, MD
role: PRINCIPAL_INVESTIGATOR
References: