Pulmonary HypertensionPhase 2NCT01048905
Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
A Phase 2 study testing L-Glutamine in people with Pulmonary Hypertension and Sickle Cell Disease, run by UCSF Benioff Children's Hospital Oakland at 1 site in United States.
Trial summary
The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease (SCD) and thalassemia (Thal) patients with PH. Read more about this study
- Who is it for?
- Ages 4 years and olderHealthy volunteers can take part
- Conditions the study is for
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- What is being tested?
- L-Glutamine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2009to Mar 2014
- 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 4 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Established diagnosis of SCD (Hb SS, SC or SBeta- thalassemia) or Thal
- PH documented by echocardiography, defined as at TRV greater than 2.5 m/s
- Age greater than or equal to 4 years
Who cannot join
- Inability to take or tolerate oral medication
- Acute crisis or hospitalization within 1 month of enrollment
- Hepatic dysfunction (SGPT greater than 3X normal)
- Renal dysfunction (Creatinine greater than 2X normal)
- Allergy to glutamine
- Pregnancy or breastfeeding
- Patients on sildenafil (Viagra), calcium channel blockers, or amino acid/protein supplements (other therapies acceptable if stable more than 3 months)
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital & Research Center Oakland
Oakland, California, 94608
About this study
Official title: Phase 2 Trial for Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease (SCD) and thalassemia (Thal) patients with PH. PH is defined as a tricuspid regurgitant jet velocity (TRV) on Doppler echocardiography > 2.5 m/s. We also predict that glutamine therapy will increase arginine bioavailability and subsequently alter sickle red cell endothelial interaction that can be identified using endo-PAT technology through nitric oxide (NO) generation, leading to changes in biological markers, and clinical outcome. Specifically our second hypothesis is that oral glutamine will decrease biomarkers of hemolysis and adhesion molecules, and improve the imbalanced arginine-to-ornithine ratio that occurs in hemolytic anemias, leading to improved arginine bioavailability and clinical endpoints of endothelial dysfunction and PH in patients with SCD and Thal.
- Conditions
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Being tested
- L-Glutamine
- Keywords
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Medical subject headings
- Hypertension, Pulmonary, 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
Oral L-glutamine 10 grams TID or (0.1g/kg TID) for children < 15 years of age.
| Study group | Role | What they receive |
|---|---|---|
| Treatment: L-glutamine | Experimental | Patients will receive an 8-week course of oral L-glutamine 10 grams TID Receives: Drug: L-Glutamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks
Erythrocyte Glutamine/Glutamate Ratio: a novel biomarker of oxidative stress
Measured: 8 weeks
Secondary outcomes (5)
Plasma Glutamine
Measured: 8 weeks
Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography
Tricuspid Regurgitant Jet Velocity was measured using Doppler Echocardiography in meters per second.
Measured: 8 week
6 Minute Walk Distance
The six-minute walk test (6MWT) measures the distance in meters an individual is able to walk over a total of six minutes on a hard, flat surface.
Measured: 8 weeks
Liver Function Tests
Alanine aminotransferase (ALT) Aspartate aminotransferase (AST)
Measured: 8 weeks
Renal Function Tests
Creatinine Blood urea nitrogen (BUN)
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2010 (estimated)
- Study startMar 2009
- Primary completionMar 2014
- Study completionMar 2014
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 Pulmonary HypertensionStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Sponsor type
- University, hospital or other
- Collaborators
- Food and Drug Administration (FDA)
- Responsible party
- Sponsor
- Investigators
- Claudia Morris, MD, Principal investigator, Emory UniversityAugusta Saulys, MD, Principal investigator, Children's Hosptial & Research Center Oakland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01048905
- Sponsor's protocol ID
- 1R01FD003531-01
- Other IDs
- IRB 2008-059, 1R01FD003531-01
- Status last verified
- Jun 2021
- First submitted
- 12 Jan 2010
- Last updated
- 10 Jun 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 10 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Trial for Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
- ClinicalTrials.gov ID
- NCT01048905
- Acronym / sponsor ID
- 1R01FD003531-01
- Conditions
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Keywords
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- UCSF Benioff Children's Hospital OaklandUniversity, hospital or other
- Collaborators
- Food and Drug Administration (FDA)
- Study start
- Mar 2009
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 14 Jan 2010 (estimated)
- Results
- Posted 10 Jun 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 hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease (SCD) and thalassemia (Thal) patients with PH. PH is defined as a tricuspid regurgitant jet velocity (TRV) on Doppler echocardiography > 2.5 m/s. We also predict that glutamine therapy will increase arginine bioavailability and subsequently alter sickle red cell endothelial interaction that can be identified using endo-PAT technology through nitric oxide (NO) generation, leading to changes in biological markers, and clinical outcome. Specifically our second hypothesis is that oral glutamine will decrease biomarkers of hemolysis and adhesion molecules, and improve the imbalanced arginine-to-ornithine ratio that occurs in hemolytic anemias, leading to improved arginine bioavailability and clinical endpoints of endothelial dysfunction and PH in patients with SCD and Thal.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment: L-glutamine | Experimental | Drug: L-GlutaminePatients will receive an 8-week course of oral L-glutamine 10 grams TID |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Established diagnosis of SCD (Hb SS, SC or SBeta- thalassemia) or Thal
- PH documented by echocardiography, defined as at TRV greater than 2.5 m/s
- Age greater than or equal to 4 years
Key exclusion criteria (7 listed)
- Inability to take or tolerate oral medication
- Acute crisis or hospitalization within 1 month of enrollment
- Hepatic dysfunction (SGPT greater than 3X normal)
- Renal dysfunction (Creatinine greater than 2X normal)
- Allergy to glutamine
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-GlutamineOral L-glutamine 10 grams TID or (0.1g/kg TID) for children < 15 years of age. | Drug | Treatment: L-glutamine |
Primary outcome
Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks
Time frame: 8 weeks
Erythrocyte Glutamine/Glutamate Ratio: a novel biomarker of oxidative stress
5 secondary outcomes
Plasma Glutamine
Time frame: 8 weeks
Tricuspid Regurgitant Jet Velocity on Doppler Echocardiography
Time frame: 8 week
6 Minute Walk Distance
Time frame: 8 weeks
Liver Function Tests
Time frame: 8 weeks
Renal Function Tests
Time frame: 8 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 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.
Claudia Morris, MD principal investigator
Emory University
Augusta Saulys, MD principal investigator
Children's Hosptial & Research Center Oakland
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 Pulmonary Hypertension and Sickle Cell Disease, run by UCSF Benioff Children's Hospital Oakland 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 3 inclusion and 7 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: finished, as of the sponsor's last update on 10 Jun 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. 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 10 Jun 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?
Erythrocyte Glutamine/Glutamate Ratio at 8 Weeks (time frame: 8 weeks). 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 10 Jun 2021.