Blood DiseaseNCT03881917
Cystatin c and Beta 2 Microglobulin in Thalassemic Children.
An observational study of Blood Disease, run by Assiut University.
Trial summary
Beta thalassemia has many complications on many systems as the renal system.So early detection of renal impairment is required in those children to decrease complications of this nephropathy. Read more about this study
- Who is it for?
- Ages 1 year to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Blood Disease
- What is being tested?
- Cystatin c and beta 2 microglobulin kits
- How many people
- 150 participants
- Where
- No sites listed yet
- Study dates
- 1 Nov 2019to 1 Mar 2021
- 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 1 year to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Children with beta thalassemia from the age of 1 year to 18 years.
Who may be able to join
- Children with beta thalassemia from the age of 1 year to 18 years.
Who cannot join
- Children who have other hematological or chronic disease.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Cystatin C and Beta 2 Microglobulin as Biochemical Markers for Early Detection of Renal Impairment in Children With Beta Thalassemia
Beta thalassemia has many complications on many systems as the renal system.So early detection of renal impairment is required in those children to decrease complications of this nephropathy.
Read the detailed description
Thalassemia syndromes are the most common single gene disorders worldwide especially in developing countries. The use of regular and frequent blood transfusions in patients with beta thalassemia major has improved patients' life spans and quality of life, but can lead to chronic iron overload. Many factors contribute to the functional abnormalities found in beta thalassemia patients such as decreased red cell life span, rapid iron turnover, tissue deposition of excess iron and also, specific iron chelators can affect kidneys. The success in management of patients of beta thalassemia has led to chronic hemosiderosis in different organs like liver and heart and long-term complications in other organs like pancreas and kidneys have recently been studied. The evidence of proximal tubular damage has been observed in beta thalassemia patients. Also, low-molecular-weight proteinuria has been found in almost all patients. Unlike other organs, it is unclear whether kidney damage results solely from intravascular haemolysis, chronic transfusion or as a complication of iron chelation therapy. Although the early identification of patients at high risk of renal impairment is of great importance as it may allow specific measures to be taken to delay renal impairment, there are limited studies about renal dysfunction in pediatric thalassemic patients. Thus, in this study we will use different measurements for early detection of renal impairment even if the patients have no symptoms to handle with the disease in its reversible stage before being irreversible. Beside the usual investigations of renal function we will measure cystatin c and beta2 microglobulin as early markers of renal impairment.
- Conditions
- Blood Disease
- Being tested
- Cystatin c and beta 2 microglobulin kits
- Keywords
- renal impairment
- Medical subject headings
- Hematologic Diseases, Renal Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cystatin c and beta 2 microglobulin kitsDiagnostic test
Kits for measurement concentration
What is being measured
The primary outcome is the main question the study is designed to answer.
mean difference of cystatin c and beta 2 microglobulin concentrations with normal range
Analysis of the results to differentiate the affected from non affected patients
Measured: baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2019
- Study start1 Nov 2019 (estimated)
- Primary completion1 Nov 2020 (estimated)
- Study completion1 Mar 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for Blood DiseaseStudies 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
- Prospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mohamed Naguib Khairy, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03881917
- Sponsor's protocol ID
- Renal markers in thalassemia.
- Status last verified
- Mar 2019
- First submitted
- 18 Mar 2019
- Last updated
- 20 Mar 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
den Bakker E, Gemke RJBJ, Bokenkamp A. Endogenous markers for kidney function in children: a review. Crit Rev Clin Lab Sci. 2018 May;55(3):163-183. doi: 10.1080/10408363.2018.1427041. Epub 2018 Feb 1.
PubMed 29388463Annayev A, Karakas Z, Karaman S, Yalciner A, Yilmaz A, Emre S. Glomerular and Tubular Functions in Children and Adults with Transfusion-Dependent Thalassemia. Turk J Haematol. 2018 Mar 1;35(1):66-70. doi: 10.4274/tjh.2017.0266. Epub 2017 Jul 28.
PubMed 28753129Behairy OG, Abd Almonaem ER, Abed NT, Abdel Haiea OM, Zakaria RM, AbdEllaty RI, Asr EH, Mansour AI, Abdelrahman AM, Elhady HA. Role of serum cystatin-C and beta-2 microglobulin as early markers of renal dysfunction in children with beta thalassemia major. Int J Nephrol Renovasc Dis. 2017 Sep 11;10:261-268. doi: 10.2147/IJNRD.S142824. eCollection 2017.
PubMed 28979155Deveci B, Kurtoglu A, Kurtoglu E, Salim O, Toptas T. Documentation of renal glomerular and tubular impairment and glomerular hyperfiltration in multitransfused patients with beta thalassemia. Ann Hematol. 2016 Feb;95(3):375-81. doi: 10.1007/s00277-015-2561-2. Epub 2015 Nov 23.
PubMed 26596972Hashemieh M, Radfar M, Azarkeivan A, Hosseini Tabatabaei SMT, Nikbakht S, Yaseri M, Sheibani K. Renal Hemosiderosis among Iranian Transfusion Dependent beta-Thalassemia Major Patients. Int J Hematol Oncol Stem Cell Res. 2017 Apr 1;11(2):133-138.
PubMed 28875008Ali BA, Mahmoud AM. Frequency of glomerular dysfunction in children with Beta thalassaemia major. Sultan Qaboos Univ Med J. 2014 Feb;14(1):e88-94. doi: 10.12816/0003341. Epub 2014 Jan 27.
PubMed 24516760Bakr A, Al-Tonbary Y, Osman G, El-Ashry R. Renal complications of beta-thalassemia major in children. Am J Blood Res. 2014 Sep 5;4(1):1-6. eCollection 2014.
PubMed 25232499Uzun E, Balci YI, Yuksel S, Aral YZ, Aybek H, Akdag B. Glomerular and tubular functions in children with different forms of beta thalassemia. Ren Fail. 2015;37(9):1414-8. doi: 10.3109/0886022X.2015.1077314. Epub 2015 Sep 12.
PubMed 26365703Nickavar A, Qmarsi A, Ansari S, Zarei E. Kidney Function in Patients With Different Variants of Beta-Thalassemia. Iran J Kidney Dis. 2017 Mar;11(2):132-137.
PubMed 28270645
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cystatin C and Beta 2 Microglobulin as Biochemical Markers for Early Detection of Renal Impairment in Children With Beta Thalassemia
- ClinicalTrials.gov ID
- NCT03881917
- Acronym / sponsor ID
- Renal markers in thalassemia.
- Conditions
- Blood Disease
- Keywords
- renal impairment
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 1 Nov 2019 (estimated)
- Primary completion
- 1 Nov 2020 (estimated)
- Study completion
- 1 Mar 2021 (estimated)
- First posted
- 20 Mar 2019 (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.
Beta thalassemia has many complications on many systems as the renal system.So early detection of renal impairment is required in those children to decrease complications of this nephropathy.
Detailed description
Thalassemia syndromes are the most common single gene disorders worldwide especially in developing countries. The use of regular and frequent blood transfusions in patients with beta thalassemia major has improved patients' life spans and quality of life, but can lead to chronic iron overload. Many factors contribute to the functional abnormalities found in beta thalassemia patients such as decreased red cell life span, rapid iron turnover, tissue deposition of excess iron and also, specific iron chelators can affect kidneys. The success in management of patients of beta thalassemia has led to chronic hemosiderosis in different organs like liver and heart and long-term complications in other organs like pancreas and kidneys have recently been studied. The evidence of proximal tubular damage has been observed in beta thalassemia patients. Also, low-molecular-weight proteinuria has been found in almost all patients. Unlike other organs, it is unclear whether kidney damage results solely from intravascular haemolysis, chronic transfusion or as a complication of iron chelation therapy. Although the early identification of patients at high risk of renal impairment is of great importance as it may allow specific measures to be taken to delay renal impairment, there are limited studies about renal dysfunction in pediatric thalassemic patients. Thus, in this study we will use different measurements for early detection of renal impairment even if the patients have no symptoms to handle with the disease in its reversible stage before being irreversible. Beside the usual investigations of renal function we will measure cystatin c and beta2 microglobulin as early markers of renal impairment.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Children with beta thalassemia from the age of 1 year to 18 years.
Key inclusion criteria (1 listed)
- Children with beta thalassemia from the age of 1 year to 18 years.
Key exclusion criteria (1 listed)
- Children who have other hematological or chronic disease.
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 |
|---|---|---|
| Cystatin c and beta 2 microglobulin kitsKits for measurement concentration | Diagnostic test | – |
Primary outcome
mean difference of cystatin c and beta 2 microglobulin concentrations with normal range
Time frame: baseline
Analysis of the results to differentiate the affected from non affected patients
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2019.
- Results publication: PubMed 29388463den Bakker E, Gemke RJBJ, Bokenkamp A. Endogenous markers for kidney function in children: a review. Crit Rev Clin Lab Sci. 2018 May;55(3):163-183. doi: 10.1080/10408363.2018.1427041. Epub 2018 Feb 1.
- Results publication: PubMed 28753129Annayev A, Karakas Z, Karaman S, Yalciner A, Yilmaz A, Emre S. Glomerular and Tubular Functions in Children and Adults with Transfusion-Dependent Thalassemia. Turk J Haematol. 2018 Mar 1;35(1):66-70. doi: 10.4274/tjh.2017.0266. Epub 2017 Jul 28.
- Results publication: PubMed 28979155Behairy OG, Abd Almonaem ER, Abed NT, Abdel Haiea OM, Zakaria RM, AbdEllaty RI, Asr EH, Mansour AI, Abdelrahman AM, Elhady HA. Role of serum cystatin-C and beta-2 microglobulin as early markers of renal dysfunction in children with beta thalassemia major. Int J Nephrol Renovasc Dis. 2017 Sep 11;10:261-268. doi: 10.2147/IJNRD.S142824. eCollection 2017.
- Results publication: PubMed 26596972Deveci B, Kurtoglu A, Kurtoglu E, Salim O, Toptas T. Documentation of renal glomerular and tubular impairment and glomerular hyperfiltration in multitransfused patients with beta thalassemia. Ann Hematol. 2016 Feb;95(3):375-81. doi: 10.1007/s00277-015-2561-2. Epub 2015 Nov 23.
- Results publication: PubMed 28875008Hashemieh M, Radfar M, Azarkeivan A, Hosseini Tabatabaei SMT, Nikbakht S, Yaseri M, Sheibani K. Renal Hemosiderosis among Iranian Transfusion Dependent beta-Thalassemia Major Patients. Int J Hematol Oncol Stem Cell Res. 2017 Apr 1;11(2):133-138.
- Results publication: PubMed 24516760Ali BA, Mahmoud AM. Frequency of glomerular dysfunction in children with Beta thalassaemia major. Sultan Qaboos Univ Med J. 2014 Feb;14(1):e88-94. doi: 10.12816/0003341. Epub 2014 Jan 27.
- Results publication: PubMed 25232499Bakr A, Al-Tonbary Y, Osman G, El-Ashry R. Renal complications of beta-thalassemia major in children. Am J Blood Res. 2014 Sep 5;4(1):1-6. eCollection 2014.
- Results publication: PubMed 26365703Uzun E, Balci YI, Yuksel S, Aral YZ, Aybek H, Akdag B. Glomerular and tubular functions in children with different forms of beta thalassemia. Ren Fail. 2015;37(9):1414-8. doi: 10.3109/0886022X.2015.1077314. Epub 2015 Sep 12.
- Results publication: PubMed 28270645Nickavar A, Qmarsi A, Ansari S, Zarei E. Kidney Function in Patients With Different Variants of Beta-Thalassemia. Iran J Kidney Dis. 2017 Mar;11(2):132-137.
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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.
Questions about this trial
What is this clinical trial studying?
An observational study of Blood Disease, run by Assiut University.
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 1 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 20 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mohamed Naguib Khairy, M.B.B.Ch (+201091921973, mohamed_ibrahim@med.au.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
mean difference of cystatin c and beta 2 microglobulin concentrations with normal range (time frame: baseline). 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 NCT03881917, which also lists 9 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 20 Mar 2019; AzTrials last refreshed its copy on 6 Nov 2026.