L-Glutamine Therapy for Sickle Cell Anemia
Study Details
Study Description
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.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
|
Phase 2 |
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.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: 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 |
Drug: 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)
|
Placebo Comparator: 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 |
Drug: Placebo
Placebo (75% Maltodextrin, 24.5 % Starch, 0.5% Tricalcium Phosphate) - given at the same dosage as L-glutamine
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- 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.
Eligibility Criteria
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 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.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | LA Biomed at Harbor-UCLA Medical Center | Torrance | California | United States | 90502 |
Sponsors and Collaborators
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Emmaus Medical, Inc.
Investigators
- Principal Investigator: Yutaka Niihara, MD, LaBiomed At Harbor-UCLA Medical Center
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 10511-01RY
Study Results
Participant Flow
Recruitment Details | |
---|---|
Pre-assignment Detail |
Arm/Group Title | L-glutamine | Placebo |
---|---|---|
Arm/Group Description | 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) | 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 |
Period Title: Overall Study | ||
STARTED | 5 | 10 |
COMPLETED | 5 | 10 |
NOT COMPLETED | 0 | 0 |
Baseline Characteristics
Arm/Group Title | L-glutamine | Placebo | Total |
---|---|---|---|
Arm/Group Description | 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) | 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 | Total of all reporting groups |
Overall Participants | 5 | 10 | 15 |
Age (Count of Participants) | |||
<=18 years |
0
0%
|
0
0%
|
0
0%
|
Between 18 and 65 years |
5
100%
|
10
100%
|
15
100%
|
>=65 years |
0
0%
|
0
0%
|
0
0%
|
Sex: Female, Male (Count of Participants) | |||
Female |
3
60%
|
5
50%
|
8
53.3%
|
Male |
2
40%
|
5
50%
|
7
46.7%
|
Region of Enrollment (participants) [Number] | |||
United States |
5
100%
|
10
100%
|
15
100%
|
Outcome Measures
Title | Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
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. |
Arm/Group Title | L-glutamine | Placebo |
---|---|---|
Arm/Group Description | 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) | 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 |
Measure Participants | 0 | 0 |
Title | Effect of L-glutamine Therapy on Breath by Breath Exercise of Sickle Cell Patients |
---|---|
Description | 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 |
Outcome Measure Data
Analysis Population Description |
---|
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. |
Arm/Group Title | L-glutamine | Placebo |
---|---|---|
Arm/Group Description | 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) | 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 |
Measure Participants | 0 | 0 |
Title | Effect of Oral L-glutamine on Incidence of Painful Crises |
---|---|
Description | Incidence of panful crises will be assessed at each visit. |
Time Frame | From Week 0 through Week 12 |
Outcome Measure Data
Analysis Population Description |
---|
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. |
Arm/Group Title | L-glutamine | Placebo |
---|---|---|
Arm/Group Description | 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) | 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 |
Measure Participants | 0 | 0 |
Title | Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics |
---|---|
Description | Amount of daily requirement for narcotics will assessed at each visit. |
Time Frame | From Week 0 through Week 20 |
Outcome Measure Data
Analysis Population Description |
---|
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. |
Arm/Group Title | L-glutamine | Placebo |
---|---|---|
Arm/Group Description | 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) | 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 |
Measure Participants | 0 | 0 |
Title | Effect of Oral L-glutamine on Level of Chronic Pain |
---|---|
Description | Level of chronic pain will be assessed at each visit. |
Time Frame | From Week 0 to Week 12 |
Outcome Measure Data
Analysis Population Description |
---|
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. |
Arm/Group Title | L-glutamine | Placebo |
---|---|---|
Arm/Group Description | 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) | 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 |
Measure Participants | 0 | 0 |
Adverse Events
Time Frame | Adverse events data were collected throughout the course of the study (53 weeks or about 1 year). | |||
---|---|---|---|---|
Adverse Event Reporting Description | The Safety population will include all patients who received at least one dose of study medication. | |||
Arm/Group Title | L-glutamine | Placebo | ||
Arm/Group Description | 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) | 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 | ||
All Cause Mortality |
||||
L-glutamine | Placebo | |||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 0/5 (0%) | 0/10 (0%) | ||
Serious Adverse Events |
||||
L-glutamine | Placebo | |||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 2/5 (40%) | 5/10 (50%) | ||
Congenital, familial and genetic disorders | ||||
Sickle cell anaemia with crisis | 1/5 (20%) | 1 | 4/10 (40%) | 4 |
Investigations | ||||
Haemoglobin decreased | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Vascular disorders | ||||
Hypertension | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Other (Not Including Serious) Adverse Events |
||||
L-glutamine | Placebo | |||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 4/5 (80%) | 7/10 (70%) | ||
Congenital, familial and genetic disorders | ||||
Sickle cell anaemia with crisis | 1/5 (20%) | 1 | 4/10 (40%) | 4 |
Gastrointestinal disorders | ||||
Constipation | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Diarrhoea | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Mouth Pain | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Nausea | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Vomiting | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
General disorders | ||||
Chest pain | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Infections and infestations | ||||
Clostridium difficile colitis | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Fungal infection | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Nasopharyngitis | 1/5 (20%) | 1 | 1/10 (10%) | 1 |
Investigations | ||||
Cardiac murmur | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Haemoglobin decreased | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Musculoskeletal and connective tissue disorders | ||||
Arthralgia | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Back pain | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Nervous system disorders | ||||
Headache | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Respiratory, thoracic and mediastinal disorders | ||||
Chronic obstructive pulmonary disease | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Cough | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Surgical and medical procedures | ||||
Endodontic procedure | 0/5 (0%) | 0 | 1/10 (10%) | 1 |
Vascular disorders | ||||
Hypertension | 1/5 (20%) | 1 | 0/10 (0%) | 0 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title | Yutaka Niihara, MD, MPH |
---|---|
Organization | Emmaus Medical, Inc. |
Phone | 310-214-0065 |
yniihara@emmausmedical.com |
- 10511-01RY