Beta Thalassemia With Increased Tricuspid Regurgitant Jet VelocityPhase 3NCT05584956
Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
A Phase 3 study testing L_carnitine and Sildenafil in people with Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity, run by Tanta University at 1 site in Egypt.
Trial summary
This study aims to investigate the possible efficacy and safety of L_Carnitine and Sildenafil on patient with Beta thalassemia complicated with increased Tricuspid Regurgitant Jet Velocity Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
- What is being tested?
- L_carnitine and Sildenafil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteEgypt
- Study dates
- 8 Jul 2022to 8 Jul 2024
- Phase
- Phase 3
- 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 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Children with Beta thalassemia major who have increased TRJV more than 2.5m/s.
- Children age from 6-18 years
Who cannot join
- Others hemolytic anemia
- Young age before 6 years of age
- Allergy to Sildenafil or L-carnitine
- Patient with documented causes of pulmonary hypertension rather than caused by Beta thalassemia.
- Hepatic dysfunction: serum Alanine Aminotransferase (ALT) 3X.
- Renal dysfunction: Creatinine level greater than or equal to 1.2 mg/dl.
Trial locations and contacts
1 site in 1 country.
Mostafa Zaki Zedan, Bsc of pharmaceutical science contact
Egypt1 site, 1 recruiting
- Recruiting
Tanta University Hospital
Tanta, Gharbia Governorate, 31511
Mohamed Ramadan El_shanshory, Professor
About this study
This study aims to investigate the possible efficacy and safety of L_Carnitine and Sildenafil on patient with Beta thalassemia complicated with increased Tricuspid Regurgitant Jet Velocity
- Being tested
- L_carnitine, Sildenafil
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L_carnitineDrug
l-carnitine stabilizes red blood cell membranes and thus improves the anemic state[
SildenafilDrug
selective and potent inhibitor of cGMP-specific phosphodiesterase 5 (PDE5), which promotes smooth muscle relaxation in lung vasculature, has been used successfully in the treatment of primary and secondary PH.
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Active comparator | n=22): Patients will receive traditional treatment and L- carnitine 50mg/kg/day orally (maximum dose 3g per day) Receives: Drug: L_carnitine |
| Group 2 | Active comparator | n=22): Patients will receive traditional treatment and Sildenafil 0.25mg/kg/dose every 6 h orally (maximum dose 60 mg per day) Receives: Drug: Sildenafil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cardiopulmonary functions
change in the studied cardiopulmonary functions at the baseline and 3 months after intervention
Measured: 3 months
Secondary outcomes (3)
Biochemical parameters
Changes in Vascular Endothelial Growth Factor level (VEGF) at baseline and 3 months after the intervention
Measured: 3 months
Biochemical parameters
Changes in Nitric oxide level (NO) at the baseline and 3 months after the intervention
Measured: 3 months
Biochemical parameters
Changes in ferritin serum level at the baseline and 3 months after the intervention
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2022
- Study start8 Jul 2022
- Primary completion8 Jul 2023 (estimated)
- Study completion8 Jul 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta Thalassemia With Increased Tricuspid Regurgitant Jet VelocityStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 44 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tanta University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mostafa Zaki Zedan, Tanta University
- Investigators
- Sahar Kamal Hegazy, Professor, Principal investigator, Tanta University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05584956
- Sponsor's protocol ID
- Beta thalassemia
- Status last verified
- Oct 2022
- First submitted
- 13 Oct 2022
- Last updated
- 18 Oct 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study to Evaluate the Possible Efficacy and Safety of L- Carnitine and Sildenafil in Children Having Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity
- ClinicalTrials.gov ID
- NCT05584956
- Acronym / sponsor ID
- Beta thalassemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- Tanta UniversityUniversity, hospital or other
- Study start
- 8 Jul 2022 (actual)
- Primary completion
- 8 Jul 2023 (estimated)
- Study completion
- 8 Jul 2024 (estimated)
- First posted
- 18 Oct 2022 (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.
This study aims to investigate the possible efficacy and safety of L_Carnitine and Sildenafil on patient with Beta thalassemia complicated with increased Tricuspid Regurgitant Jet Velocity
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Active comparator | Drug: L_carnitinen=22): Patients will receive traditional treatment and L- carnitine 50mg/kg/day orally (maximum dose 3g per day) |
| Group 2 | Active comparator | Drug: Sildenafiln=22): Patients will receive traditional treatment and Sildenafil 0.25mg/kg/dose every 6 h orally (maximum dose 60 mg per day) |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Children with Beta thalassemia major who have increased TRJV more than 2.5m/s.
- Children age from 6-18 years
Key exclusion criteria (6 listed)
- Others hemolytic anemia
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 stabilizes red blood cell membranes and thus improves the anemic state[ | Drug | Group 1 |
| Sildenafilselective and potent inhibitor of cGMP-specific phosphodiesterase 5 (PDE5), which promotes smooth muscle relaxation in lung vasculature, has been used successfully in the treatment of primary and secondary PH. | Drug | Group 2 |
Primary outcome
Cardiopulmonary functions
Time frame: 3 months
change in the studied cardiopulmonary functions at the baseline and 3 months after intervention
3 secondary outcomes
Biochemical parameters
Time frame: 3 months
Biochemical parameters
Time frame: 3 months
Biochemical parameters
Time frame: 3 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2022.
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.
Mostafa Zaki Zedan, Bsc of pharmaceutical science contact
Sahar Kamal Hegazy, Professor principal investigator
Tanta University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing L_carnitine and Sildenafil in people with Beta Thalassemia With Increased Tricuspid Regurgitant Jet Velocity, run by Tanta 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 6 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 18 Oct 2022. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 Mostafa Zaki Zedan, Bsc of pharmaceutical science (+0201554676330, mostaf150776@pharm.tanta.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cardiopulmonary functions (time frame: 3 months). 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 18 Oct 2022.