Sickle Cell AnemiaPhase 2NCT00586209
L-Glutamine Therapy for Sickle Cell Anemia
A Phase 2 study testing L-Glutamine in people with Sickle Cell Anemia and Thalassemia, run by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Anemia, Thalassemia
- What is being tested?
- L-Glutamine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Feb 2004to Nov 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
LA Biomed at Harbor-UCLA Medical Center
Torrance, California, 90502
About this study
Official title: A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia
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.
Read the 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.
- Conditions
- Sickle Cell Anemia, Thalassemia
- Being tested
- L-Glutamine, Placebo
- Keywords
- sickle cell disease, sickle cell anemia, L-glutamine, Sickle Cell Anemia (homozygous), Sickle β°-Thalassemia
- Medical subject headings
- Anemia, Sickle Cell, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L-GlutamineDrug
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)
PlaceboDrug
Placebo (75% Maltodextrin, 24.5 % Starch, 0.5% Tricalcium Phosphate) - given at the same dosage as L-glutamine
| Study group | Role | What they receive |
|---|---|---|
| L-glutamine | Experimental | 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 Receives: Drug: L-Glutamine |
| Placebo | Placebo comparator | 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 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline, Weeks 8 and 12
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
Measured: Baseline, Weeks 18 and 12
Secondary outcomes (3)
Effect of Oral L-glutamine on Incidence of Painful Crises
Incidence of panful crises will be assessed at each visit.
Measured: From Week 0 through Week 12
Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics
Amount of daily requirement for narcotics will assessed at each visit.
Measured: From Week 0 through Week 20
Effect of Oral L-glutamine on Level of Chronic Pain
Level of chronic pain will be assessed at each visit.
Measured: From Week 0 to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2008 (estimated)
- Study start6 Feb 2004
- Primary completion27 Aug 2008
- Study completionNov 2009
Why it stopped: Inadequate clinical supplies and Emmaus business decision.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Emmaus Medical, Inc.
- Responsible party
- Principal investigator, yutaka niihara, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Investigators
- Yutaka Niihara, MD, Principal investigator, LaBiomed At Harbor-UCLA Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00586209
- Sponsor's protocol ID
- 10511-01RY
- Status last verified
- Feb 2021
- First submitted
- 21 Dec 2007
- Last updated
- 2 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Study of L Glutamine Therapy for Sickle Cell Anemia and Sickle ß0-Thalassemia
- ClinicalTrials.gov ID
- NCT00586209
- Acronym / sponsor ID
- 10511-01RY
- Conditions
- Sickle Cell Anemia, Thalassemia
- Keywords
- sickle cell disease, sickle cell anemia, L-glutamine, Sickle Cell Anemia (homozygous), Sickle β°-Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Inadequate clinical supplies and Emmaus business decision.
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterUniversity, hospital or other
- Collaborators
- Emmaus Medical, Inc.
- Study start
- 6 Feb 2004 (actual)
- Primary completion
- 27 Aug 2008 (actual)
- Study completion
- Nov 2009 (actual)
- First posted
- 4 Jan 2008 (estimated)
- Results
- Posted 2 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| L-glutamine | Experimental | Drug: L-GlutamineL-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 |
| Placebo | Placebo comparator | Drug: PlaceboMaltodextrin 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| L-GlutamineL-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) | Drug | L-glutamine |
| PlaceboPlacebo (75% Maltodextrin, 24.5 % Starch, 0.5% Tricalcium Phosphate) - given at the same dosage as L-glutamine | Drug | Placebo |
Primary outcome
Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients
Time frame: 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
Time frame: 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
3 secondary outcomes
Effect of Oral L-glutamine on Incidence of Painful Crises
Time frame: From Week 0 through Week 12
Effect of Oral L-glutamine on Amount of Daily Requirement for Narcotics
Time frame: From Week 0 through Week 20
Effect of Oral L-glutamine on Level of Chronic Pain
Time frame: From Week 0 to Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yutaka Niihara, MD principal investigator
LaBiomed At Harbor-UCLA Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing L-Glutamine in people with Sickle Cell Anemia and Thalassemia, run by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of L-glutamine Therapy on Exercise Endurance of Sickle Cell Anemia Patients (time frame: Baseline, Weeks 8 and 12). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Feb 2021; AzTrials last refreshed its copy on 1 Nov 2026.