Carnitine Deficiency Due to HemodialysisEarly Phase 1NCT05948124
Carnitine Supplementation in Pediatric Hemodialysis Patients
A Early Phase 1 study testing L-carnitine and isotonic saline in people with Carnitine Deficiency Due to Hemodialysis, run by Ain Shams University at 1 site in Egypt.
Trial summary
The goal of this study is: 1. To determine the prevalence of carnitine deficiency among pediatric patients on hemodialysis. 2. To evaluate the efficiency of carnitine supplementation in children on regular hemodialysis with carnitine deficiency in the treatment of renal anemia, cardiac dysfunction, dyslipidemia… Read more about this study
- Who is it for?
- Ages Up to 16 years
- Conditions the study is for
- Carnitine Deficiency Due to Hemodialysis
- What is being tested?
- L-carnitine and isotonic saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteEgypt
- Study dates
- 1 Aug 2023to 1 Mar 2024
- Phase
- Early Phase 1
- 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 Up to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- All patients on regular hemodialysis for more than three months with L-carnitine deficiency.
- Anemia (hemoglobin [Hb] < 11 g/dl; hematocrit [Hct] < 30%) resistant to erythropoietin defined as Anemia that require recombinant human erythropoietin (rHuEPO) doses >300 units/kg/week intravenously in spite of adequate iron stores (transferrin saturation >20%, ferritin >100 ng/mL), and without any other identifiable cause of anemia.
- Recurrent intradialytic complications (cramping, muscular pain, hypotension)
- Cardiomyopathy with reduced left ventricular ejection fraction.
- Sex: both males and females.
- Age: 16 years old or less.
Who cannot join
- Patients known to be allergic to L-carnitine.
- Patients with inborn error of metabolism.
- Patients on lipid lowering therapy.
- Patients with Diabetes mellitus.
- Patients with Associated congenital heart disease.
- Patients with Thyroid disorder, or malignancy.
- Patients received L-carnitine within the past 6 months.
- Patients received Blood transfusion 4 weeks prior to study.
Trial locations and contacts
1 site in 1 country.
Egypt1 site, 1 recruiting
- Recruiting
Ain Shams university
Cairo
Ainshams university Hospitals
About this study
Official title: Carnitine Deficiency and Benefits of Its Supplementation in Pediatric Hemodialysis Patients
The goal of this study is:
- To determine the prevalence of carnitine deficiency among pediatric patients on hemodialysis.
- To evaluate the efficiency of carnitine supplementation in children on regular hemodialysis with carnitine deficiency in the treatment of renal anemia, cardiac dysfunction, dyslipidemia, intradialytic muscle cramps and hypotension and their quality of life.
Read the detailed description
Patients on hemodialysis (HD) often have carnitine deficiency due to multiple factors; dietary intake of carnitine is decreased due to falls in appetite, total energy levels, and protein intake. In addition, accumulating evidence has linked inflammation to malnutrition, and chronic inflammation might also interrupt carnitine transfer in the intestine. Carnitine biosynthesis can also fall in patients on dialysis due to reduced biosynthesis in the kidney and limited compensation by the liver. Furthermore, because of the low molecular weight of carnitine and its high hydrophilicity and absence of protein binding, carnitine is significantly removed by the dialyzer. As in the healthy population, carnitine deficiency in patients receiving maintenance dialysis is most commonly defined as a serum free carnitine level less than 20 μmol/L .
Intravenous levocarnitine is commonly used to treat patients receiving maintenance hemodialysis who are diagnosed with carnitine deficiency since it has 100%bioavailability and does not break down into toxic metabolites. A common dose used for carnitine supplementation is 10-20 mg/kg administered after each hemodialysis session, which produces the supraphysiologic serum levels of carnitine that are required to adequately drive carnitine from the serum into skeletal muscles.
There are four principal indications for levocarnitine treatment in dialysis patients with carnitine deficiency according to the American National Kidney Foundation: (1) erythropoiesis stimulating agents resistant anemia that has not responded to the standard erythropoiesis stimulating agent dosage; (2) recurrent symptomatic hypotension during hemodialysis;(3) symptomatic cardiomyopathy or confirmed cardiomyopathy with reduced left ventricular ejection fraction and(4) fatigability and muscle weakness that undermine the quality of life.
- Conditions
- Carnitine Deficiency Due to Hemodialysis
- Being tested
- L-carnitine, isotonic saline
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L-carnitineDrug
L-carnitine supplementation
isotonic salineOther
Intravenous 5 ml of isotonic saline
| Study group | Role | What they receive |
|---|---|---|
| L-Carnitine Group | Experimental | Enrolled patients in this group who are on regular hemodialysis fulfilling our study's inclusion criteria with secondary carnitine deficiency(based on clinical manifestations and decreased serum level of L-carnitine ;serum free carnitine level less than 20 μmol/L) will receive intravenous L-carnitine (20 mg/kg dry body weight) after each dialysis session three times weekly for 6 months. Receives: Drug: L-carnitine |
| Placebo Group | Placebo comparator | enrolled patients in this group who will receive 5 ml intravenous isotonic saline after each dialysis session three times weekly for 6 months. Receives: Other: isotonic saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
change in serum free carnitine level
The investigator will measure serum free carnitine level in blood using ELISA technique.
Measured: before supplementation and after six months of supplementation.
change in cardiac functions
The investigator will assess ejection fraction percentage by Echocardiography.
Measured: before supplementation and after six months of supplementation.
change in hemoglobin level.
The investigator will measure hemoglobin level to assess effect of supplementation on renal anemia. and Erythropoietin dose
Measured: before supplementation and after six months of supplementation.
change in Body composition.
using (The Fresenius Medical Care Body Composition Monitor - BCM) to assess fat composition percentiles.
Measured: before supplementation and after six months of supplementation.
change in Body composition.
using (The Fresenius Medical Care Body Composition Monitor - BCM) to assess lean tissue index percentiles.
Measured: before supplementation and after six months of supplementation.
change in quality of life.
assessment of quality of life using 36 -item short form survey instrument (SF- 36) questionnaire, it is a perceived quality of life assessment tool which will be used to assess general health, physical functioning, bodily pain, and mental health with a scale 0 - 100 , The lower the score the more disability.
Measured: before supplementation and after six months of supplementation.
change in Cardiac functions
The investigator will measure fraction shortening percentage using Echocardiography
Measured: before supplementation and after six months of supplementation.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2023
- Study start1 Aug 2023 (estimated)
- Primary completion1 Feb 2024 (estimated)
- Study completion1 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Carnitine Deficiency Due to HemodialysisStudies of the same condition, open and under way.
- More trials run by Ain Shams 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
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fady georguos labib, Ain Shams University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05948124
- Sponsor's protocol ID
- Carnitine in Hemodialysis
- Status last verified
- Jul 2023
- First submitted
- 17 Jun 2023
- Last updated
- 17 Jul 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Eknoyan G, Latos DL, Lindberg J; National Kidney Foundation Carnitine Consensus Conference. Practice recommendations for the use of L-carnitine in dialysis-related carnitine disorder. National Kidney Foundation Carnitine Consensus Conference. Am J Kidney Dis. 2003 Apr;41(4):868-76. doi: 10.1016/s0272-6386(03)00110-0.
PubMed 12666074Evans A. Dialysis-related carnitine disorder and levocarnitine pharmacology. Am J Kidney Dis. 2003 Apr;41(4 Suppl 4):S13-26. doi: 10.1016/s0272-6386(03)00113-6.
PubMed 12751050KDOQI Work Group. KDOQI Clinical Practice Guideline for Nutrition in Children with CKD: 2008 update. Executive summary. Am J Kidney Dis. 2009 Mar;53(3 Suppl 2):S11-104. doi: 10.1053/j.ajkd.2008.11.017. No abstract available.
PubMed 19231749
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Carnitine Deficiency and Benefits of Its Supplementation in Pediatric Hemodialysis Patients
- ClinicalTrials.gov ID
- NCT05948124
- Acronym / sponsor ID
- Carnitine in Hemodialysis
- Conditions
- Carnitine Deficiency Due to Hemodialysis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- 1 Aug 2023 (estimated)
- Primary completion
- 1 Feb 2024 (estimated)
- Study completion
- 1 Mar 2024 (estimated)
- First posted
- 17 Jul 2023 (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 goal of this study is:
- To determine the prevalence of carnitine deficiency among pediatric patients on hemodialysis.
- To evaluate the efficiency of carnitine supplementation in children on regular hemodialysis with carnitine deficiency in the treatment of renal anemia, cardiac dysfunction, dyslipidemia, intradialytic muscle cramps and hypotension and their quality of life.
Detailed description
Patients on hemodialysis (HD) often have carnitine deficiency due to multiple factors; dietary intake of carnitine is decreased due to falls in appetite, total energy levels, and protein intake. In addition, accumulating evidence has linked inflammation to malnutrition, and chronic inflammation might also interrupt carnitine transfer in the intestine. Carnitine biosynthesis can also fall in patients on dialysis due to reduced biosynthesis in the kidney and limited compensation by the liver. Furthermore, because of the low molecular weight of carnitine and its high hydrophilicity and absence of protein binding, carnitine is significantly removed by the dialyzer. As in the healthy population, carnitine deficiency in patients receiving maintenance dialysis is most commonly defined as a serum free carnitine level less than 20 μmol/L .
Intravenous levocarnitine is commonly used to treat patients receiving maintenance hemodialysis who are diagnosed with carnitine deficiency since it has 100%bioavailability and does not break down into toxic metabolites. A common dose used for carnitine supplementation is 10-20 mg/kg administered after each hemodialysis session, which produces the supraphysiologic serum levels of carnitine that are required to adequately drive carnitine from the serum into skeletal muscles.
There are four principal indications for levocarnitine treatment in dialysis patients with carnitine deficiency according to the American National Kidney Foundation: (1) erythropoiesis stimulating agents resistant anemia that has not responded to the standard erythropoiesis stimulating agent dosage; (2) recurrent symptomatic hypotension during hemodialysis;(3) symptomatic cardiomyopathy or confirmed cardiomyopathy with reduced left ventricular ejection fraction and(4) fatigability and muscle weakness that undermine the quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| L-Carnitine Group | Experimental | Drug: L-carnitineEnrolled patients in this group who are on regular hemodialysis fulfilling our study's inclusion criteria with secondary carnitine deficiency(based on clinical manifestations and decreased serum level of L-carnitine ;serum free carnitine level less than 20 μmol/L) will receive intravenous L-carnitine (20 mg/kg dry body weight) after each dialysis session three times weekly for 6 months. |
| Placebo Group | Placebo comparator | Other: isotonic salineenrolled patients in this group who will receive 5 ml intravenous isotonic saline after each dialysis session three times weekly for 6 months. |
Who may qualify
- Ages
- Up to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- All patients on regular hemodialysis for more than three months with L-carnitine deficiency.
- Anemia (hemoglobin [Hb] < 11 g/dl; hematocrit [Hct] < 30%) resistant to erythropoietin defined as Anemia that require recombinant human erythropoietin (rHuEPO) doses >300 units/kg/week intravenously in spite of adequate iron stores (transferrin saturation >20%, ferritin >100 ng/mL), and without any other identifiable cause of anemia.
- Recurrent intradialytic complications (cramping, muscular pain, hypotension)
- Cardiomyopathy with reduced left ventricular ejection fraction.
- Sex: both males and females.
Key exclusion criteria (8 listed)
- Patients known to be allergic to L-carnitine.
- Patients with inborn error of metabolism.
- Patients on lipid lowering therapy.
- Patients with Diabetes mellitus.
- Patients with Associated congenital heart 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 |
|---|---|---|
| L-carnitineL-carnitine supplementation | Drug | L-Carnitine Group |
| isotonic salineIntravenous 5 ml of isotonic saline | Other | Placebo Group |
Primary outcome
change in serum free carnitine level
Time frame: before supplementation and after six months of supplementation.
The investigator will measure serum free carnitine level in blood using ELISA technique.
change in cardiac functions
Time frame: before supplementation and after six months of supplementation.
The investigator will assess ejection fraction percentage by Echocardiography.
change in hemoglobin level.
Time frame: before supplementation and after six months of supplementation.
The investigator will measure hemoglobin level to assess effect of supplementation on renal anemia. and Erythropoietin dose
change in Body composition.
Time frame: before supplementation and after six months of supplementation.
using (The Fresenius Medical Care Body Composition Monitor - BCM) to assess fat composition percentiles.
change in Body composition.
Time frame: before supplementation and after six months of supplementation.
using (The Fresenius Medical Care Body Composition Monitor - BCM) to assess lean tissue index percentiles.
change in quality of life.
Time frame: before supplementation and after six months of supplementation.
assessment of quality of life using 36 -item short form survey instrument (SF- 36) questionnaire, it is a perceived quality of life assessment tool which will be used to assess general health, physical functioning, bodily pain, and mental health with a scale 0 - 100 , The lower the score the more disability.
change in Cardiac functions
Time frame: before supplementation and after six months of supplementation.
The investigator will measure fraction shortening percentage using Echocardiography
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2023.
- Background: PubMed 12666074Eknoyan G, Latos DL, Lindberg J; National Kidney Foundation Carnitine Consensus Conference. Practice recommendations for the use of L-carnitine in dialysis-related carnitine disorder. National Kidney Foundation Carnitine Consensus Conference. Am J Kidney Dis. 2003 Apr;41(4):868-76. doi: 10.1016/s0272-6386(03)00110-0.
- Background: PubMed 12751050Evans A. Dialysis-related carnitine disorder and levocarnitine pharmacology. Am J Kidney Dis. 2003 Apr;41(4 Suppl 4):S13-26. doi: 10.1016/s0272-6386(03)00113-6.
- Background: PubMed 19231749KDOQI Work Group. KDOQI Clinical Practice Guideline for Nutrition in Children with CKD: 2008 update. Executive summary. Am J Kidney Dis. 2009 Mar;53(3 Suppl 2):S11-104. doi: 10.1053/j.ajkd.2008.11.017. No abstract available.
- Individual participant dataSharing planned: Yes
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing L-carnitine and isotonic saline in people with Carnitine Deficiency Due to Hemodialysis, run by Ain Shams 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 6 inclusion and 8 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 17 Jul 2023. 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. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Fady LAbib (+201023811345/201282657642, Fadygeorguos@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
change in serum free carnitine level (time frame: before supplementation and after six months of supplementation.). The study lists 7 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 NCT05948124, which also lists 3 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 17 Jul 2023.