Thalassemia in ChildrenNCT03133169
Erythrocyte Glutamine Level Relation to Pulmonary Hypertension Risk in Beta Thalassemia Major Children
An observational study of Thalassemia in Children and Pulmonary Hypertension, run by Assiut University at 1 site in Egypt.
Trial summary
The study will investigate the relation between erythrocyte glutamine/glutamate ratio and pulmonary hypertension risk in Egyptian thalassemic children in Assiut University Children Hospital Read more about this study
- Who is it for?
- Ages 10 to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Thalassemia in Children, Pulmonary Hypertension, Hemolysis
- What is being tested?
- blood sample and Tricuspid regurge velocity
- Type of treatment
- Pill or oral medicine
- How many people
- 80 participants
- Where
- 1 siteEgypt
- Study dates
- 1 Jun 2017to 26 Aug 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
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 10 to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Upper Egypt thalassemic children aged 10-18 years old attending Assiut University Children Hospital.
Who may be able to join
- Established diagnosis of Thalassemia.
- PH risk documented by doppler echocardiography, defined as tricuspid regurge velocity (TRV) equal to or greater than 2.5 m/s
Who cannot join
- Acute crisis or hospitalization within 1 month of enrollment
- Hepatic dysfunction (SGPT greater than 3X normal)
- Renal dysfunction (Creatinine greater than 2X normal)
- Patients on sildenafil (Viagra), calcium channel blockers or other drugs for the control of PH.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Erythrocyte Glutamine Level Relation to Pulmonary Hypertension Risk in Beta Thalassemia Major Children in Assiut University Children Hospital
The study will investigate the relation between erythrocyte glutamine/glutamate ratio and pulmonary hypertension risk in Egyptian thalassemic children in Assiut University Children Hospital
- Conditions
- Thalassemia in Children, Pulmonary Hypertension, Hemolysis
- Being tested
- blood sample, Tricuspid regurge velocity
- Keywords
- Thalassemia, Glutamine/glutamate ratio, Pulmonary hypertension
- Medical subject headings
- Hypertension, Pulmonary, Hemolysis, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
blood sampleDiagnostic test
blood sample for measuring liver and kidney function, CBC, ferritin level and erythrocyte glutamine level
Tricuspid regurge velocityDiagnostic test
Tricuspid regurge velocity will be measured by doppler echocardiography denoting the pulmonary hypertension risk in children
| Study group | Role | What they receive |
|---|---|---|
| on chelation | patients with B-thalassemia major samples of which will be examined for renal and liver function, Erythrocyte Glutamine level, ferritin level and complete blood picture. Also Echo will be done for measuring the Tricuspid regurge velocity.
group 1: cases on chelation: deferasirox 500mg oral tablet with initial dose 20 mg/kg guided by ferritin level
Diagnostic Test: blood sample
Diagnostic Test: Tricuspid regurge velocity Receives: Diagnostic Test: blood sample; Diagnostic Test: Tricuspid regurge velocity | |
| No chelation | group 2: cases without chelation
Diagnostic Test: blood sample
Diagnostic Test: Tricuspid regurge velocity Receives: Diagnostic Test: blood sample; Diagnostic Test: Tricuspid regurge velocity | |
| splenectomy | group 3: cases with splenectomy
Diagnostic Test: blood sample
Diagnostic Test: Tricuspid regurge velocity Receives: Diagnostic Test: blood sample; Diagnostic Test: Tricuspid regurge velocity | |
| no splenectomy | group 4: cases without splenectomy Diagnostic Test: blood sample Diagnostic Test: Tricuspid regurge velocity Receives: Diagnostic Test: blood sample; Diagnostic Test: Tricuspid regurge velocity |
What is being measured
The primary outcome is the main question the study is designed to answer.
Erythrocyte glutamine level
marker for oxidative stress
Measured: 2 months
Tricuspid regurge velocity
Measures the risk of pulmonary hypertension
Measured: 2 months
Secondary outcomes (4)
Plasma glutamine level
Assess the glutamine level at each visit
Measured: 2 months
Liver function tests
Evaluates the state of liver
Measured: 2 months
Renal function tests
Evaluates the state of kidney
Measured: 2 months
Ferritin level
measuring the iron overload
Measured: 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2017
- Study start1 Jun 2017
- Primary completion25 Aug 2019
- Study completion26 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (18)Papers the sponsor has linked to this study.
- Other trials for Thalassemia in ChildrenStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fatma Sami, Assiut University
- Investigators
- Fahim M Fahim, PhD, Principal investigator, Children Hospital, Assiut UniversityFatma S AbdElshafi, bachelor's, Principal investigator, Children Hospital, Assiut UniversityEman F. Mohamed, PhD, Principal investigator, Children Hospital, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03133169
- Sponsor's protocol ID
- GlnThalassemia
- Status last verified
- Aug 2019
- First submitted
- 15 Apr 2017
- Last updated
- 28 Aug 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Gladwin MT, Sachdev V, Jison ML, Shizukuda Y, Plehn JF, Minter K, Brown B, Coles WA, Nichols JS, Ernst I, Hunter LA, Blackwelder WC, Schechter AN, Rodgers GP, Castro O, Ognibene FP. Pulmonary hypertension as a risk factor for death in patients with sickle cell disease. N Engl J Med. 2004 Feb 26;350(9):886-95. doi: 10.1056/NEJMoa035477.
PubMed 14985486Morris CR, Kuypers FA, Kato GJ, Lavrisha L, Larkin S, Singer T, Vichinsky EP. Hemolysis-associated pulmonary hypertension in thalassemia. Ann N Y Acad Sci. 2005;1054:481-5. doi: 10.1196/annals.1345.058.
PubMed 16339702Morris CR, Vichinsky EP. Pulmonary hypertension in thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:205-13. doi: 10.1111/j.1749-6632.2010.05580.x.
PubMed 20712794Walter PB, Fung EB, Killilea DW, Jiang Q, Hudes M, Madden J, Porter J, Evans P, Vichinsky E, Harmatz P. Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease. Br J Haematol. 2006 Oct;135(2):254-63. doi: 10.1111/j.1365-2141.2006.06277.x.
PubMed 17010049Morris CR. Mechanisms of vasculopathy in sickle cell disease and thalassemia. Hematology Am Soc Hematol Educ Program. 2008:177-85. doi: 10.1182/asheducation-2008.1.177.
PubMed 19074078Kuypers FA. Membrane lipid alterations in hemoglobinopathies. Hematology Am Soc Hematol Educ Program. 2007:68-73. doi: 10.1182/asheducation-2007.1.68.
PubMed 18024611Fung EB, Harmatz P, Milet M, Ballas SK, De Castro L, Hagar W, Owen W, Olivieri N, Smith-Whitley K, Darbari D, Wang W, Vichinsky E; Multi-Center Study of Iron Overload Research Group. Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload. Am J Hematol. 2007 Apr;82(4):255-65. doi: 10.1002/ajh.20809.
PubMed 17094096Machado RF, Gladwin MT. Pulmonary hypertension in hemolytic disorders: pulmonary vascular disease: the global perspective. Chest. 2010 Jun;137(6 Suppl):30S-38S. doi: 10.1378/chest.09-3057.
PubMed 20522578Janda S, Shahidi N, Gin K, Swiston J. Diagnostic accuracy of echocardiography for pulmonary hypertension: a systematic review and meta-analysis. Heart. 2011 Apr;97(8):612-22. doi: 10.1136/hrt.2010.212084. Epub 2011 Feb 25.
PubMed 21357375De Castro LM, Jonassaint JC, Graham FL, Ashley-Koch A, Telen MJ. Pulmonary hypertension associated with sickle cell disease: clinical and laboratory endpoints and disease outcomes. Am J Hematol. 2008 Jan;83(1):19-25. doi: 10.1002/ajh.21058.
PubMed 17724699Anthi A, Machado RF, Jison ML, Taveira-Dasilva AM, Rubin LJ, Hunter L, Hunter CJ, Coles W, Nichols J, Avila NA, Sachdev V, Chen CC, Gladwin MT. Hemodynamic and functional assessment of patients with sickle cell disease and pulmonary hypertension. Am J Respir Crit Care Med. 2007 Jun 15;175(12):1272-9. doi: 10.1164/rccm.200610-1498OC. Epub 2007 Mar 22.
PubMed 17379852Hebbel RP. Reconstructing sickle cell disease: a data-based analysis of the "hyperhemolysis paradigm" for pulmonary hypertension from the perspective of evidence-based medicine. Am J Hematol. 2011 Feb;86(2):123-54. doi: 10.1002/ajh.21952.
PubMed 21264896Morris CR. Vascular risk assessment in patients with sickle cell disease. Haematologica. 2011 Jan;96(1):1-5. doi: 10.3324/haematol.2010.035097. No abstract available.
PubMed 21193426Mok E, Hankard R. Glutamine supplementation in sick children: is it beneficial? J Nutr Metab. 2011;2011:617597. doi: 10.1155/2011/617597. Epub 2011 Nov 14.
PubMed 22175008Xu H, Pearl RG. Effect of l-glutamine on pulmonary hypertension in the perfused rabbit lung. Pharmacology. 1994 Apr;48(4):260-4. doi: 10.1159/000139188.
PubMed 8177911
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Erythrocyte Glutamine Level Relation to Pulmonary Hypertension Risk in Beta Thalassemia Major Children in Assiut University Children Hospital
- ClinicalTrials.gov ID
- NCT03133169
- Acronym / sponsor ID
- GlnThalassemia
- Conditions
- Thalassemia in Children, Pulmonary Hypertension, Hemolysis
- Keywords
- Thalassemia, Glutamine/glutamate ratio, Pulmonary hypertension
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 1 Jun 2017 (actual)
- Primary completion
- 25 Aug 2019 (actual)
- Study completion
- 26 Aug 2019 (actual)
- First posted
- 28 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will investigate the relation between erythrocyte glutamine/glutamate ratio and pulmonary hypertension risk in Egyptian thalassemic children in Assiut University Children Hospital
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| on chelation | – | Diagnostic Test: blood sample, Diagnostic Test: Tricuspid regurge velocitypatients with B-thalassemia major samples of which will be examined for renal and liver function, Erythrocyte Glutamine level, ferritin level and complete blood picture. Also Echo will be done for measuring the Tricuspid regurge velocity. group 1: cases on chelation: deferasirox 500mg oral tablet with initial dose 20 mg/kg guided by ferritin level Diagnostic Test: blood sample Diagnostic Test: Tricuspid regurge velocity |
| No chelation | – | Diagnostic Test: blood sample, Diagnostic Test: Tricuspid regurge velocitygroup 2: cases without chelation Diagnostic Test: blood sample Diagnostic Test: Tricuspid regurge velocity |
| splenectomy | – | Diagnostic Test: blood sample, Diagnostic Test: Tricuspid regurge velocitygroup 3: cases with splenectomy Diagnostic Test: blood sample Diagnostic Test: Tricuspid regurge velocity |
| no splenectomy | – | Diagnostic Test: blood sample, Diagnostic Test: Tricuspid regurge velocitygroup 4: cases without splenectomy Diagnostic Test: blood sample Diagnostic Test: Tricuspid regurge velocity |
Who may qualify
- Ages
- 10 to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Upper Egypt thalassemic children aged 10-18 years old attending Assiut University Children Hospital.
Key inclusion criteria (2 listed)
- Established diagnosis of Thalassemia.
- PH risk documented by doppler echocardiography, defined as tricuspid regurge velocity (TRV) equal to or greater than 2.5 m/s
Key exclusion criteria (4 listed)
- Acute crisis or hospitalization within 1 month of enrollment
- Hepatic dysfunction (SGPT greater than 3X normal)
- Renal dysfunction (Creatinine greater than 2X normal)
- Patients on sildenafil (Viagra), calcium channel blockers or other drugs for the control of PH.
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 |
|---|---|---|
| blood sampleblood sample for measuring liver and kidney function, CBC, ferritin level and erythrocyte glutamine level | Diagnostic test | No chelation, no splenectomy, on chelation, splenectomy |
| Tricuspid regurge velocityTricuspid regurge velocity will be measured by doppler echocardiography denoting the pulmonary hypertension risk in children | Diagnostic test | No chelation, no splenectomy, on chelation, splenectomy |
Primary outcome
Erythrocyte glutamine level
Time frame: 2 months
marker for oxidative stress
Tricuspid regurge velocity
Time frame: 2 months
Measures the risk of pulmonary hypertension
4 secondary outcomes
Plasma glutamine level
Time frame: 2 months
Liver function tests
Time frame: 2 months
Renal function tests
Time frame: 2 months
Ferritin level
Time frame: 2 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Aug 2019.
- Background: PubMed 14985486Gladwin MT, Sachdev V, Jison ML, Shizukuda Y, Plehn JF, Minter K, Brown B, Coles WA, Nichols JS, Ernst I, Hunter LA, Blackwelder WC, Schechter AN, Rodgers GP, Castro O, Ognibene FP. Pulmonary hypertension as a risk factor for death in patients with sickle cell disease. N Engl J Med. 2004 Feb 26;350(9):886-95. doi: 10.1056/NEJMoa035477.
- Background: PubMed 16339702Morris CR, Kuypers FA, Kato GJ, Lavrisha L, Larkin S, Singer T, Vichinsky EP. Hemolysis-associated pulmonary hypertension in thalassemia. Ann N Y Acad Sci. 2005;1054:481-5. doi: 10.1196/annals.1345.058.
- Background: PubMed 20712794Morris CR, Vichinsky EP. Pulmonary hypertension in thalassemia. Ann N Y Acad Sci. 2010 Aug;1202:205-13. doi: 10.1111/j.1749-6632.2010.05580.x.
- Background: PubMed 17010049Walter PB, Fung EB, Killilea DW, Jiang Q, Hudes M, Madden J, Porter J, Evans P, Vichinsky E, Harmatz P. Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease. Br J Haematol. 2006 Oct;135(2):254-63. doi: 10.1111/j.1365-2141.2006.06277.x.
- Background: PubMed 19074078Morris CR. Mechanisms of vasculopathy in sickle cell disease and thalassemia. Hematology Am Soc Hematol Educ Program. 2008:177-85. doi: 10.1182/asheducation-2008.1.177.
- Background: PubMed 18024611Kuypers FA. Membrane lipid alterations in hemoglobinopathies. Hematology Am Soc Hematol Educ Program. 2007:68-73. doi: 10.1182/asheducation-2007.1.68.
- Background: PubMed 17094096Fung EB, Harmatz P, Milet M, Ballas SK, De Castro L, Hagar W, Owen W, Olivieri N, Smith-Whitley K, Darbari D, Wang W, Vichinsky E; Multi-Center Study of Iron Overload Research Group. Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload. Am J Hematol. 2007 Apr;82(4):255-65. doi: 10.1002/ajh.20809.
- Background: PubMed 20522578Machado RF, Gladwin MT. Pulmonary hypertension in hemolytic disorders: pulmonary vascular disease: the global perspective. Chest. 2010 Jun;137(6 Suppl):30S-38S. doi: 10.1378/chest.09-3057.
- Background: PubMed 21357375Janda S, Shahidi N, Gin K, Swiston J. Diagnostic accuracy of echocardiography for pulmonary hypertension: a systematic review and meta-analysis. Heart. 2011 Apr;97(8):612-22. doi: 10.1136/hrt.2010.212084. Epub 2011 Feb 25.
- Background: PubMed 17724699De Castro LM, Jonassaint JC, Graham FL, Ashley-Koch A, Telen MJ. Pulmonary hypertension associated with sickle cell disease: clinical and laboratory endpoints and disease outcomes. Am J Hematol. 2008 Jan;83(1):19-25. doi: 10.1002/ajh.21058.
- Background: PubMed 17379852Anthi A, Machado RF, Jison ML, Taveira-Dasilva AM, Rubin LJ, Hunter L, Hunter CJ, Coles W, Nichols J, Avila NA, Sachdev V, Chen CC, Gladwin MT. Hemodynamic and functional assessment of patients with sickle cell disease and pulmonary hypertension. Am J Respir Crit Care Med. 2007 Jun 15;175(12):1272-9. doi: 10.1164/rccm.200610-1498OC. Epub 2007 Mar 22.
- Background: PubMed 21264896Hebbel RP. Reconstructing sickle cell disease: a data-based analysis of the "hyperhemolysis paradigm" for pulmonary hypertension from the perspective of evidence-based medicine. Am J Hematol. 2011 Feb;86(2):123-54. doi: 10.1002/ajh.21952.
- Individual participant dataSharing planned: Undecided
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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.
Fahim M Fahim, PhD principal investigator
Children Hospital, Assiut University
Fatma S AbdElshafi, bachelor's principal investigator
Children Hospital, Assiut University
Eman F. Mohamed, PhD principal investigator
Children Hospital, Assiut University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thalassemia in Children and Pulmonary Hypertension, run by Assiut University at 1 site in Egypt.
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 2 inclusion and 4 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 28 Aug 2019. 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 Egypt. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 level (time frame: 2 months). 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?
On the ClinicalTrials.gov record for NCT03133169, which also lists 18 linked publications.
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 28 Aug 2019.