Thalassemia MajorPhase 1NCT04260516
The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major
A Phase 1 study testing N-acetyl cysteine in people with Thalassemia Major, run by Beni-Suef University at 1 site in Egypt.
Trial summary
The effect of N-acetylcysteine as antioxidant and its effect on pretransfusion hemoglobin and iron overload in patients with thalassemia were compared to patients who didn't receive n-acetylcysteine after 3 months of study duration Read more about this study
- Who is it for?
- Ages 3 to 14 years
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- N-acetyl cysteine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteEgypt
- Study dates
- 3 Jun 2019to 30 Dec 2019
- Phase
- 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 3 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Thalassemia major children on regular blood transfusion and iron chelation therapy
Who cannot join
- Renal or hepatic impairments
- Complicated thalassemia major
- Non adherence to therapy or follow up visits
- Change in the iron chelation dose within 3 months before enrollment or during study period
Trial locations and contacts
1 site in 1 country.
About this study
The effect of N-acetylcysteine as antioxidant and its effect on pretransfusion hemoglobin and iron overload in patients with thalassemia were compared to patients who didn't receive n-acetylcysteine after 3 months of study duration
Read the detailed description
N-acetylcysteine( NAC) is a widely used mucolytic drug and several studies had reported its efficacy as anti oxidant. Patients with thalassemia major are at great risk for oxidative stress due to excess of b-globin chains and iron overload. In this study the investigators compared the effect of NAC on oxidative status , pretransfusion hemoglobin levels and iron overload in thalassemia patients before and after treatment with NAC for 3 months and with that of patients who served as controls.
- Conditions
- Thalassemia Major
- Being tested
- N-acetyl cysteine
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetyl cysteineDrug
Mucolytic and antioxidant drug
| Study group | Role | What they receive |
|---|---|---|
| N-acetylcysteine group | Active comparator | Patients received oral n-acetylcysteine syrup on dose of 10 mg/kg/day as single dose for 3 months Receives: Drug: N-acetyl cysteine |
| Non n-acetylcysteine group | No intervention | Thalassemia major patients on regular chelation therapy who didn't receive n-acetylcysteine and served as controls |
What is being measured
The primary outcome is the main question the study is designed to answer.
Oxidative stress index (pg/mmol)
Oxidative stress index is calculated as the ratio of total oxidative status (pg/dl) to total anti oxidative capacity (mmol/dl)
Measured: 3 months
Serum ferritin level ( mg/dl)
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 posted7 Feb 2020
- Study start3 Jun 2019
- Primary completion3 Dec 2019
- Study completion30 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Beni-Suef 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Two groups are followed up for study parameters for 3 months one group is the intervention group and the other is the control group
- Masking
- None (open label)
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Beni-Suef University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Heba Mostafa Ahmed, Beni-Suef University
- Investigators
- Heba M Ahmed, Principal investigator, Beni-Suef University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04260516
- Sponsor's protocol ID
- FM-BSU REC
- Status last verified
- Feb 2020
- First submitted
- 4 Feb 2020
- Last updated
- 7 Feb 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ozdemir ZC, Koc A, Aycicek A, Kocyigit A. N-Acetylcysteine supplementation reduces oxidative stress and DNA damage in children with beta-thalassemia. Hemoglobin. 2014;38(5):359-64. doi: 10.3109/03630269.2014.951890. Epub 2014 Sep 15.
PubMed 25222041
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of N-acetylcysteine on Oxidative Stress Status and Iron Overload in Thalassemia Major
- ClinicalTrials.gov ID
- NCT04260516
- Acronym / sponsor ID
- FM-BSU REC
- Conditions
- Thalassemia Major
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Beni-Suef UniversityUniversity, hospital or other
- Study start
- 3 Jun 2019 (actual)
- Primary completion
- 3 Dec 2019 (actual)
- Study completion
- 30 Dec 2019 (actual)
- First posted
- 7 Feb 2020 (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 effect of N-acetylcysteine as antioxidant and its effect on pretransfusion hemoglobin and iron overload in patients with thalassemia were compared to patients who didn't receive n-acetylcysteine after 3 months of study duration
Detailed description
N-acetylcysteine( NAC) is a widely used mucolytic drug and several studies had reported its efficacy as anti oxidant. Patients with thalassemia major are at great risk for oxidative stress due to excess of b-globin chains and iron overload. In this study the investigators compared the effect of NAC on oxidative status , pretransfusion hemoglobin levels and iron overload in thalassemia patients before and after treatment with NAC for 3 months and with that of patients who served as controls.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Two groups are followed up for study parameters for 3 months one group is the intervention group and the other is the control group
| Arm | Type | Receives |
|---|---|---|
| N-acetylcysteine group | Active comparator | Drug: N-acetyl cysteinePatients received oral n-acetylcysteine syrup on dose of 10 mg/kg/day as single dose for 3 months |
| Non n-acetylcysteine group | No intervention | –Thalassemia major patients on regular chelation therapy who didn't receive n-acetylcysteine and served as controls |
Who may qualify
- Ages
- 3 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Thalassemia major children on regular blood transfusion and iron chelation therapy
Key exclusion criteria (4 listed)
- Renal or hepatic impairments
- Complicated thalassemia major
- Non adherence to therapy or follow up visits
- Change in the iron chelation dose within 3 months before enrollment or during study period
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 |
|---|---|---|
| N-acetyl cysteineMucolytic and antioxidant drug | Drug | N-acetylcysteine group |
Primary outcome
Oxidative stress index (pg/mmol)
Time frame: 3 months
Oxidative stress index is calculated as the ratio of total oxidative status (pg/dl) to total anti oxidative capacity (mmol/dl)
Serum ferritin level ( mg/dl)
Time frame: 3 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 7 Feb 2020.
- Results publication: PubMed 25222041Ozdemir ZC, Koc A, Aycicek A, Kocyigit A. N-Acetylcysteine supplementation reduces oxidative stress and DNA damage in children with beta-thalassemia. Hemoglobin. 2014;38(5):359-64. doi: 10.3109/03630269.2014.951890. Epub 2014 Sep 15.
- Individual participant dataSharing planned: No
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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.
Heba M Ahmed principal investigator
Beni-Suef University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing N-acetyl cysteine in people with Thalassemia Major, run by Beni-Suef 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 1 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 7 Feb 2020. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Oxidative stress index (pg/mmol) (time frame: 3 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 NCT04260516, which also lists 1 linked publication.
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 7 Feb 2020.