Beta-ThalassemiaPhase 2NCT06239389
Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
A Phase 2 study testing Thalidomide 100mg and Hydroxyurea in people with Beta-Thalassemia, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan at 1 site in Pakistan.
Trial summary
The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. Read more about this study
- Who is it for?
- Ages 10 to 30 years
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- Thalidomide 100mg and Hydroxyurea
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 sitePakistan
- Study dates
- 1 Sep 2021to 3 Mar 2022
- Phase
- Phase 2
- 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 10 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Know case of beta thalassemia age between 10-30 years
Who cannot join
- Patients on any other haemoglobin F inducer or erythropoietin.
- Patients with co-morbidities like cardiopulmonary and neurological disease
- Pregnant, lactating women.
- Patient did not willing to take contraceptive measures.
- Participants with history of thrombosis.
Trial locations and contacts
1 site in 1 country.
Pakistan1 site
National Institute of Blood Diseases and Bone Marrow Transplantation
Karachi, Sindh, 75300
About this study
The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. The Sample size was calculated by the World health organization sample size calculator. After screening 39 patients and 24 patients were eligible for enrollment in this study.
The main objective was to evaluate safety of both drugs in genetic disorder like thalassemia. for safety evaluation, hematological parameters were evaluated that includes total bilirubin , indirect bilirubin, Serum Glutamate Pyruvate Transaminase, serum glutamic-oxaloacetic transaminase, urea, creatinine and lactate dehydrogenase were monitored . Moreover to evaluate the efficacy of drug, hematological parameters that includes hemoglobin, red blood cells , nucleated red blood cells , reticulocytes count, Red blood cells indices ( mean corpuscular hemoglobin, mean corpuscular volume and mean corpuscular hemoglobin concentration) , white blood cells and platelets were done. Another important parameters to evaluate the efficacy of hemoglobin F inducer is transfusion frequency.
Test were done at baseline and after completion of study means after 06 months.
- Conditions
- Beta-Thalassemia
- Being tested
- Thalidomide 100mg, Hydroxyurea
- Keywords
- thalidomide,, hydroxyurea, hemoglobin F inducers, beta thalassemia
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thalidomide 100mgDrug
Also called thalido
Thalidomide is available in capsulated form in the strength of 100 mg and 50 mg. It was given according to the weight of the patient. Due to somnolence, it was advised to give at night only.
HydroxyureaDrug
Also called Hydrea
Hydroxyurea is available in capsulated form under the brand of hydrea. Its available strength is 500 mg . Dosing depends upon weight of patient, that is 15mg/kg/day. so patients unable to take full capsule due to low weight, was advised to take hydroxyurea syrup.
| Study group | Role | What they receive |
|---|---|---|
| hydroxyurea | Active comparator | Hydroxyurea is available in oral dosage form which is capsule. frequency: once a day duration: at every 24 hours dose: 15/mg/kg/day. Receives: Drug: Thalidomide 100mg |
| Thalidomide | Experimental | Thalidomide is available in oral dosage form which is capsule. frequency: once a day at night duration: at every 24 hours dose:50 mg / day (in patients >10-13 years) while the adult dose was 100 mg /day (age >13 Years) Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
comparison of hemoglobin
Hemoglobin
Measured: 06 months
comparison of red blood cells
Red blood cells
Measured: 06 months
comparison of Mean corpuscular volume
Mean corpuscular volume
Measured: 06 months
comparison of Mean Corpuscular Hemoglobin
Mean Corpuscular Hemoglobin
Measured: 06 months
comparison of Mean Corpuscular Hemoglobin concentration
Mean Corpuscular Hemoglobin concentration
Measured: 06 months
comparison of Indirect bilirubin
Indirect bilirubin
Measured: 06 months
comparison of Total Bilirubin
Total Bilirubin
Measured: 06 months
comparison of Serum glutamic pyruvic transaminase
Serum glutamic pyruvic transaminase
Measured: 06 months
comparison of glutamic-oxaloacetic transaminase
glutamic-oxaloacetic transaminase
Measured: 06 months
comparison of white blood cells
white blood cells
Measured: 06 months
comparison of platelets
Platelets
Measured: 06 months
comparison of urea
Urea
Measured: 06 months
comparison of creatinine
Creatinine
Measured: 06 months
comparison of Lactate dehydrogenase baseline and after the completion of study.
Lactate dehydrogenase
Measured: 06 months
comparison of tranfusion frequency baseline and after the completion of study.
Transfusion interval
Measured: 06 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2024
- Study start1 Sep 2021
- Primary completion1 Dec 2021
- Study completion3 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Patients were divided into 2 groups, one received hydroxyurea second group received thalidomide.
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Safia Mehmood khan, MPHIL, Principal investigator, National institute if blood diseases and bone marrow transplantation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06239389
- Sponsor's protocol ID
- NIBD/IRB-236/21-2021.
- Status last verified
- Mar 2024
- First submitted
- 23 Dec 2023
- Last updated
- 12 Mar 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
- ClinicalTrials.gov ID
- NCT06239389
- Acronym / sponsor ID
- NIBD/IRB-236/21-2021.
- Conditions
- Beta-Thalassemia
- Keywords
- thalidomide,, hydroxyurea, hemoglobin F inducers, beta thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), PakistanOther government
- Study start
- 1 Sep 2021 (actual)
- Primary completion
- 1 Dec 2021 (actual)
- Study completion
- 3 Mar 2022 (actual)
- First posted
- 2 Feb 2024 (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 compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. The Sample size was calculated by the World health organization sample size calculator. After screening 39 patients and 24 patients were eligible for enrollment in this study.
The main objective was to evaluate safety of both drugs in genetic disorder like thalassemia. for safety evaluation, hematological parameters were evaluated that includes total bilirubin , indirect bilirubin, Serum Glutamate Pyruvate Transaminase, serum glutamic-oxaloacetic transaminase, urea, creatinine and lactate dehydrogenase were monitored . Moreover to evaluate the efficacy of drug, hematological parameters that includes hemoglobin, red blood cells , nucleated red blood cells , reticulocytes count, Red blood cells indices ( mean corpuscular hemoglobin, mean corpuscular volume and mean corpuscular hemoglobin concentration) , white blood cells and platelets were done. Another important parameters to evaluate the efficacy of hemoglobin F inducer is transfusion frequency.
Test were done at baseline and after completion of study means after 06 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Patients were divided into 2 groups, one received hydroxyurea second group received thalidomide.
| Arm | Type | Receives |
|---|---|---|
| hydroxyurea | Active comparator | Drug: Thalidomide 100mgHydroxyurea is available in oral dosage form which is capsule. frequency: once a day duration: at every 24 hours dose: 15/mg/kg/day. |
| Thalidomide | Experimental | Drug: HydroxyureaThalidomide is available in oral dosage form which is capsule. frequency: once a day at night duration: at every 24 hours dose:50 mg / day (in patients >10-13 years) while the adult dose was 100 mg /day (age >13 Years) |
Who may qualify
- Ages
- 10 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Know case of beta thalassemia age between 10-30 years
Key exclusion criteria (5 listed)
- Patients on any other haemoglobin F inducer or erythropoietin.
- Patients with co-morbidities like cardiopulmonary and neurological disease
- Pregnant, lactating women.
- Patient did not willing to take contraceptive measures.
- Participants with history of thrombosis.
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 |
|---|---|---|
| Thalidomide 100mgAlso: thalidoThalidomide is available in capsulated form in the strength of 100 mg and 50 mg. It was given according to the weight of the patient. Due to somnolence, it was advised to give at night only. | Drug | hydroxyurea |
| HydroxyureaAlso: HydreaHydroxyurea is available in capsulated form under the brand of hydrea. Its available strength is 500 mg . Dosing depends upon weight of patient, that is 15mg/kg/day. so patients unable to take full capsule due to low weight, was advised to take hydroxyurea syrup. | Drug | Thalidomide |
Primary outcome
comparison of hemoglobin
Time frame: 06 months
Hemoglobin
comparison of red blood cells
Time frame: 06 months
Red blood cells
comparison of Mean corpuscular volume
Time frame: 06 months
Mean corpuscular volume
comparison of Mean Corpuscular Hemoglobin
Time frame: 06 months
Mean Corpuscular Hemoglobin
comparison of Mean Corpuscular Hemoglobin concentration
Time frame: 06 months
Mean Corpuscular Hemoglobin concentration
comparison of Indirect bilirubin
Time frame: 06 months
Indirect bilirubin
comparison of Total Bilirubin
Time frame: 06 months
Total Bilirubin
comparison of Serum glutamic pyruvic transaminase
Time frame: 06 months
Serum glutamic pyruvic transaminase
comparison of glutamic-oxaloacetic transaminase
Time frame: 06 months
glutamic-oxaloacetic transaminase
comparison of white blood cells
Time frame: 06 months
white blood cells
comparison of platelets
Time frame: 06 months
Platelets
comparison of urea
Time frame: 06 months
Urea
comparison of creatinine
Time frame: 06 months
Creatinine
comparison of Lactate dehydrogenase baseline and after the completion of study.
Time frame: 06 months
Lactate dehydrogenase
comparison of tranfusion frequency baseline and after the completion of study.
Time frame: 06 months
Transfusion interval
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 12 Mar 2024.
- Individual participant dataSharing planned: No
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RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
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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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.
Safia Mehmood khan, MPHIL principal investigator
National institute if blood diseases and bone marrow transplantation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thalidomide 100mg and Hydroxyurea in people with Beta-Thalassemia, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan at 1 site in Pakistan.
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 5 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 12 Mar 2024. 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 Pakistan. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
comparison of hemoglobin (time frame: 06 months). The study lists 15 primary outcomes in all. 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 12 Mar 2024.