ThalassemiaPhase 4NCT03402191
L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
A Phase 4 study testing L-arginine and Sildenafil in people with Thalassemia, run by Sherief Abd-Elsalam at 1 site in Egypt.
Trial summary
This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Thalassemia
- What is being tested?
- L-arginine and Sildenafil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 1 siteEgypt
- Study dates
- Jul 2016to Dec 2023
- Phase
- Phase 4
- 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 thalassemia and pulmonary hypertension.
Who cannot join
- Rheumatic heart diseases.
- Other comorbid disease
Trial locations and contacts
1 site in 1 country.
Egypt1 site, 1 recruiting
- Recruiting
Sherief Abd-Elsalam
Tanta
Sherief Abd-elsalam, lecturer, 00201000040794, Sherif_tropical@yahoo.com
About this study
Official title: Comparative Clinical Study Evaluating the Effect of L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
This study compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia
Read the detailed description
This study focuses on different lines of treatment for pulmonary hypertension in children with thalassemia as this study discusses the Effect of L-arginine Versus Sildenafil in Children with Beta Thalassemia Associated with Pulmonary Hypertension
- Conditions
- Thalassemia
- Being tested
- L-arginine, Sildenafil
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L-arginineDrug
l-arginine for thalassemia with pulmonary hypertension
SildenafilDrug
Also called Viagra
Sildenafil for thalassemia with pulmonary hypertension
| Study group | Role | What they receive |
|---|---|---|
| L-arginine | Active comparator | l-arginine for pulmonary hypertension in patients with thalassemia. Receives: Drug: L-arginine |
| Sildenafil | Active comparator | Sildenafil for pulmonary hypertension in patients with thalassemia. Receives: Drug: Sildenafil |
| Control | No intervention | No pulmonary hypertension |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with improvement of pulmonary hypertension
patients with improvement of pulmonary hypertension
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2018
- Study startJul 2016
- Primary completionDec 2023 (estimated)
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ThalassemiaStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sherief Abd-Elsalam
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Sherief Abd-Elsalam, Tanta University
- Investigators
- Sahar El-Haggar, Prof, Principal investigator, Tanta University - Faculty of PharmacyMohamed El-Shanshoury, Prof, Study director, Tanta University Pediatrics DepartmentOsama Abd-rab El-Rasol, Prof, Study chair, Tanta University Pediatrics DepartmentTarek Mostafa, Ass Prof, Study chair, Tanta University-Faculty of pharmacyEman El-Khateeb, Msc, Study chair, Tanta University-Faculty of pharmacy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03402191
- Sponsor's protocol ID
- Prof Elshanshoury
- Status last verified
- Aug 2018
- First submitted
- 9 Jan 2018
- Last updated
- 27 Aug 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparative Clinical Study Evaluating the Effect of L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension
- ClinicalTrials.gov ID
- NCT03402191
- Acronym / sponsor ID
- Prof Elshanshoury
- Conditions
- Thalassemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Sherief Abd-ElsalamUniversity, hospital or other
- Study start
- Jul 2016 (actual)
- Primary completion
- Dec 2023 (estimated)
- Study completion
- Dec 2023 (estimated)
- First posted
- 18 Jan 2018 (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 compares L-arginine Versus Sildenafil as treatment for pulmonary hypertension in Children with Beta Thalassemia
Detailed description
This study focuses on different lines of treatment for pulmonary hypertension in children with thalassemia as this study discusses the Effect of L-arginine Versus Sildenafil in Children with Beta Thalassemia Associated with Pulmonary Hypertension
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| L-arginine | Active comparator | Drug: L-argininel-arginine for pulmonary hypertension in patients with thalassemia. |
| Sildenafil | Active comparator | Drug: SildenafilSildenafil for pulmonary hypertension in patients with thalassemia. |
| Control | No intervention | –No pulmonary hypertension |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Children with thalassemia and pulmonary hypertension.
Key exclusion criteria (2 listed)
- Rheumatic heart diseases.
- Other comorbid 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-argininel-arginine for thalassemia with pulmonary hypertension | Drug | L-arginine |
| SildenafilAlso: ViagraSildenafil for thalassemia with pulmonary hypertension | Drug | Sildenafil |
Primary outcome
Number of patients with improvement of pulmonary hypertension
Time frame: 6 months
patients with improvement of pulmonary hypertension
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2018.
- Individual participant dataSharing planned: Undecided
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
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Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
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Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
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.
Sahar El-Haggar, Prof principal investigator
Tanta University - Faculty of Pharmacy
Mohamed El-Shanshoury, Prof study director
Tanta University Pediatrics Department
Osama Abd-rab El-Rasol, Prof study chair
Tanta University Pediatrics Department
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing L-arginine and Sildenafil in people with Thalassemia, run by Sherief Abd-Elsalam 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 1 inclusion and 2 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 27 Aug 2018. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 Sherief Abd-Elsalam, MD (00201095159522, sheriefabdelsalam@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of patients with improvement of pulmonary hypertension (time frame: 6 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 27 Aug 2018.