Sickle Cell Anemia, Thalassemia
Conditions
Keywords
sickle cell disease, sickle cell anemia, L-glutamine, Sickle Cell Anemia (homozygous), Sickle β°-Thalassemia
Brief summary
The primary purpose is to evaluate the effect of L-glutamine therapy on exercise endurance and breath by breath exercise response of sickle cell anemia patients The secondary purpose is to assess the effect of L-glutamine on incidence of painful crises; level of chronic pain, and amount of daily requirement for narcotics.
Detailed description
This is a phase II, prospective, randomized, double-blind, placebo-controlled, parallel-group,study to evaluate the safety and efficacy of oral L-glutamine therapy for patients with sickle cell anemia or sickle β°-thalassemia who are at least 18 years old with focus on the aspect of exercise endurance. In this study, the patients will orally take L-glutamine or placebo twice daily, and clinical and hematological parameters will be monitored. The adverse events,especially those attributable to L-glutamine therapy, will also be monitored. The data obtained will be essential in understanding the role of L-glutamine in therapy for sickle cell anemia and sickle β°-thalassemia.
Interventions
DRUGL-Glutamine
L-Glutamine at: 5 g 2X daily (17-33.3 Kg) 10 g 2x daily (33.4-66.6 Kg) 15 g 2x daily (\>66.7 kg)
DRUGPlacebo
Placebo (75% Maltodextrin, 24.5 % Starch, 0.5% Tricalcium Phosphate) - given at the same dosage as L-glutamine
Sponsors
Emmaus Medical, Inc.
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
To be eligible to participate in the study, a patient must meet all of the following inclusion criteria: * Patient is at least 18 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. * Patient is able to perform exercise tolerance test
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients | Baseline, Weeks 8 and 12 | Exercise endurance will be examined at each visit. Change from baseline will be reported at weeks 8 and 12 |
| Effect of L-glutamine Therapy on Breath by Breath Exercise of Sickle Cell Patients | Baseline, Weeks 18 and 12 | Breath by breath exercise will be examined at each visit. Change from baseline will be reported at weeks 8 and 12 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Effect of Oral L-glutamine on Incidence of Painful Crises | From Week 0 through Week 12 | Incidence of panful crises will be assessed at each visit. |
| Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics | From Week 0 through Week 20 | Amount of daily requirement for narcotics will assessed at each visit. |
| Effect of Oral L-glutamine on Level of Chronic Pain | From Week 0 to Week 12 | Level of chronic pain will be assessed at each visit. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| L-glutamine L-glutamine group will be given at the following dosage:
17-33.3 kg at 5 g 2x daily 33.4-66.6 kg at 10 g 2X daily \>66.7 at 15 g 2X daily
L-Glutamine: L-Glutamine at: 5 g 2X daily (17-33.3 Kg) 10 g 2x daily (33.4-66.6 Kg) 15 g 2x daily (\>66.7 kg) | 5 |
| Placebo Maltodextrin group will be given at the following dosage:
17-33.3 kg at 5 g 2x daily 33.4-66.6 kg at 10 g 2X daily \>66.7 at 15 g 2X daily
Placebo: Placebo (75% Maltodextrin, 24.5 % Starch, 0.5% Tricalcium Phosphate) - given at the same dosage as L-glutamine | 10 |
| Total | 15 |
Baseline characteristics
| Characteristic | L-glutamine | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants | 10 Participants | 15 Participants |
| Region of Enrollment United States | 5 participants | 10 participants | 15 participants |
| Sex: Female, Male Female | 3 Participants | 5 Participants | 8 Participants |
| Sex: Female, Male Male | 2 Participants | 5 Participants | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 5 | 0 / 10 |
| other Total, other adverse events | 4 / 5 | 7 / 10 |
| serious Total, serious adverse events | 2 / 5 | 5 / 10 |
Outcome results
Primary
Effect of L-glutamine Therapy on Breath by Breath Exercise of Sickle Cell Patients
Breath by breath exercise will be examined at each visit. Change from baseline will be reported at weeks 8 and 12
Time frame: Baseline, Weeks 18 and 12
Population: The study was terminated by the Sponsor's business decision prior to achieving the planned enrollment. Due to this the data was not collected and not analyzed. However safety data was collected and analyzed to gather data on the safety of L-glutamine in sickle cell patients.
Primary
Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients
Exercise endurance will be examined at each visit. Change from baseline will be reported at weeks 8 and 12
Time frame: Baseline, Weeks 8 and 12
Population: The study was terminated by the Sponsor's business decision prior to achieving the planned enrollment. Due to this the data was not collected and not analyzed. However safety data was collected and analyzed to gather data on the safety of L-glutamine in sickle cell patients.
Secondary
Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics
Amount of daily requirement for narcotics will assessed at each visit.
Time frame: From Week 0 through Week 20
Population: The study was terminated by the Sponsor's business decision prior to achieving the planned enrollment. Due to this the data was not collected and not analyzed. However safety data was collected and analyzed to gather data on the safety of L-glutamine in sickle cell patients.
Secondary
Effect of Oral L-glutamine on Incidence of Painful Crises
Incidence of panful crises will be assessed at each visit.
Time frame: From Week 0 through Week 12
Population: The study was terminated by the Sponsor's business decision prior to achieving the planned enrollment. Due to this the data was not collected and not analyzed. However safety data was collected and analyzed to gather data on the safety of L-glutamine in sickle cell patients.
Secondary
Effect of Oral L-glutamine on Level of Chronic Pain
Level of chronic pain will be assessed at each visit.
Time frame: From Week 0 to Week 12
Population: The study was terminated by the Sponsor's business decision prior to achieving the planned enrollment. Due to this the data was not collected and not analyzed. However safety data was collected and analyzed to gather data on the safety of L-glutamine in sickle cell patients.