Fetal HemoglobinPhase 2ThalidomideNCT06490601
Long Term Beta Thalassemia Treatment: Findings From The Extension Period
A Phase 2 study testing thalidomide and hydroxyurea and Thalidomide in people with Fetal Hemoglobin and Beta-Thalassemia, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan at 1 site in Pakistan.
Trial summary
The project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone. Read more about this study
- Who is it for?
- Ages 8 to 35 years
- Conditions the study is for
- Fetal Hemoglobin, Beta-Thalassemia, Hemoglobinopathies
- What is being tested?
- thalidomide and hydroxyurea and Thalidomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 sitePakistan
- Study dates
- 1 Apr 2022to 1 Jul 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 8 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Known case of beta thalassemia major/intermediate (transfusion dependent)
- Willing to give informed consent
Who cannot join
- Patients with comorbidities such as liver dysfunction
- Married patients
- Lactating mothers
- History of thrombosis and fits
Trial locations and contacts
1 site in 1 country.
Pakistan1 site
National Institute of blood disease and bone marrow transplant
Karachi, Sindh
About this study
Official title: Long Term Comparison of Combination Therapy (Thalidomide and Hydroxyurea) vs Thalidomide Alone in Beta Thalassemia.
The project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone. The study, lasting three years, is a Phase 2 single-center, open-label interventional study with a sample size of 30 participants aged 8-35 years. It includes specific inclusion and exclusion criteria for participant selection. Data will be collected through clinical interviews and medical records and analyzed using(Statistical Package for the Social Sciences. This project aims to enhance beta thalassemia treatment strategies, focusing on reducing transfusion dependency and improving patient quality of life.
Read the detailed description
Study titled Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial, conducted at the National Institute of Blood Disease \& Bone Marrow Transplantation (NIBD \& BMT).
This study focuses on the long-term comparison of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone in treating beta thalassemia. The objective is to evaluate the efficacy and safety of the combination therapy compared to thalidomide alone, with the hypothesis that the combination will be more effective. Beta thalassemia is defined as an autosomal recessive disorder affecting beta-globin production, influenced by genetic modifiers. Key variables include hemoglobin, red blood cells, leukocyte count, reticulocyte count, platelets, lactate dehydrogenase, nucleated red blood cells, ferritin, bilirubin, Serum Glutamate Pyruvate Transaminase, creatinine, transfusion frequency, spleen and liver size, hemoglobin subunit beta [ Homo sapiens (human) ] mutation, and certain polymorphism in gamma globin gene . The study took place at NIBD hospital over three years, designed as a Phase 2 single-center, two-arm open-label interventional study with a sample size of 30 participants using simple randomized sampling. Inclusion criteria are beta thalassemia major/intermediate patients aged 8-35 years, while exclusion criteria include patients with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis and fits. Data will be collected through clinical interviews and medical record reviews and analyzed using (Statistical Package for the Social Sciences.
- Conditions
- Fetal Hemoglobin, Beta-Thalassemia, Hemoglobinopathies
- Being tested
- thalidomide and hydroxyurea, Thalidomide
- Keywords
- thalidomide, safety and efficacy, thalassemia, fetal hemoglobin inducer, hydroxyurea
- Medical subject headings
- beta-Thalassemia, Hemoglobinopathies, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
thalidomide and hydroxyureaDrug
Also called combination therapy in beta thalassemia
Thalidomide:
Thalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor- alpha Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine.
Hydroxyurea:
Hydroxyurea or hydroxycarbamide (HU) lies in the category of antimetabolite. Mechanism of fetal hemoglobin induction includes increase in erythropoietin and nitric oxide production, apoptosis induction and potentiating in granulocyte cycling activity.
ThalidomideDrug
Also called fetal hemoglobin inducer
Thalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor -alpha , Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine.
Investigating the impact of thalidomide on transfusion-dependent beta thalassemia patients is essential for discerning its therapeutic efficacy and safety profile.
| Study group | Role | What they receive |
|---|---|---|
| combination therapy (thalidomide and hydroxyurea) | Experimental | In this group, participants were treated with combination therapy that includes thalidomide and hydroxyurea at the dose of 100mg/day at night with aspirin and 500mg /day respectively. Receives: Drug: thalidomide and hydroxyurea |
| thalidomide alone | Active comparator | In this group, participants were treated with thalidomide at the dose of 100mg/day at night with aspirin. Receives: Drug: Thalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
hemoglobin levels
Measure the improvement in hemoglobin levels Hemoglobin levels will be assessed using a complete blood count (CBC) test, measured in grams per deciliter (g/dL) of blood. This test is conducted through a venous blood sample, which is then analyzed using an automated hematology analyzer to determine the hemoglobin concentration.
Measured: 3 years
red blood cell count.
Measure the improvement in red blood cell count. Red blood cell count will be assessed using a complete blood count (CBC) test, measured in millions of cells per microliter (million cells/µL) of blood. This test is conducted through a venous blood sample, analyzed using an automated hematology analyzer to determine the number of red blood cells present.
Measured: 3 years
leukocyte count
Measure the effect on in leukocyte count Leukocyte count will be assessed using a complete blood count (CBC) test, measured in thousands of cells per microliter (thousand cells/µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer to determine the total number of white blood cells present.
Measured: 3 years
reticulocyte count
Measure the effect on reticulocyte count Reticulocyte count will be assessed using a complete blood count (CBC) test, measured as a percentage of the total red blood cells or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which identifies and quantifies reticulocytes using specific staining techniques.
Measured: 3 years
Transfusion Frequency:
Document and compare the frequency of blood transfusions required by patients in both treatment arms over the study period.
Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event.
Measured: 3 years
Secondary outcomes (7)
Spleen and Liver Size
Measure changes in spleen and liver size as a response to the treatments. Spleen and liver size will be assessed using imaging techniques such as ultrasound.. Measurements will be reported in centimeters (cm), capturing the dimensions of each organ to evaluate any enlargement or abnormalities.
Measured: 3 years
Serum Ferritin Levels
Evaluate the effectiveness of the combination therapy in reducing serum ferritin levels, indicating decreased iron overload.
Serum ferritin levels will be assessed using a blood test, measured in nanograms per milliliter (ng/mL). A venous blood sample will be analyzed in a clinical laboratory using immunoassay techniques to determine the concentration of ferritin, providing an indicator of the body's iron stores.
Measured: 3 years
Genetic Modifiers:
Analyze the influence of genetic modifiers. Genetic modifiers will be assessed through genetic testing,e.g thalassemia genetic profile focusing on specific genes or genetic variations known to influence the expression or function of proteins related to the condition under study. This analysis helps identify how genetic factors may modify disease progression, treatment response, or other relevant outcomes.
Measured: 3 years
bilirubin
Monitor and compare changes in biochemical parameters such as bilirubin. Bilirubin levels will be assessed through a blood test, typically measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the concentration of bilirubin, a pigment produced from the breakdown of red blood cells, providing insights into liver function and potential health conditions such as jaundice or liver disease.
Measured: 3 years
lactate dehydrogenase.
Monitor and compare changes in biochemical parameters such as lactate dehydrogenase.
Lactate dehydrogenase (LDH) levels will be assessed through a blood test, measured in units per liter (U/L). This test analyzes a venous blood sample to determine the concentration of LDH, an enzyme involved in cellular metabolism. Elevated LDH levels may indicate tissue damage or disease.
Measured: 3 years
Serum Glutamate Pyruvate Transaminase,
Monitor and compare changes in biochemical parameters such as Serum Glutamate Pyruvate Transaminase. Serum Glutamate Pyruvate Transaminase, also known as alanine aminotransferase (ALT), will be assessed through a blood test, measured in units per liter (U/L). This test analyzes a venous blood sample to determine the concentration of ALT, an enzyme primarily found in the liver cells. Elevated ALT levels may indicate liver damage or disease.
Measured: 3 years
creatinine
Monitor and compare changes in biochemical parameters such as creatinine. Creatinine levels will be assessed through a blood test, typically measured in milligrams per deciliter (mg/dL) or micromoles per liter (µmol/L). This test involves analyzing a venous blood sample to determine the concentration of creatinine, a waste product from muscle metabolism filtered by the kidneys. Monitoring creatinine levels helps evaluate kidney function and detect conditions such as kidney disease or impaired renal function.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2024
- Study start1 Apr 2022
- Primary completion1 Apr 2024
- Study completion1 Jul 2025 (estimated)
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 Fetal HemoglobinStudies 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
- Randomised
- Intervention model
- Parallel groups
- Model description
- study participants were divided into 2 groups. one was treated with combination therapy that includes thalidomide and hydroxyurea whereas another group was treated with thalidomide alone.
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06490601
- Sponsor's protocol ID
- NIBD/IRB-242/12-2022
- Acronym
- Thalidomide
- Status last verified
- May 2025
- First submitted
- 21 Jun 2024
- Last updated
- 30 May 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: it will decide later.
Publications
Ali S, Mumtaz S, Shakir HA, Khan M, Tahir HM, Mumtaz S, Mughal TA, Hassan A, Kazmi SAR, Sadia, Irfan M, Khan MA. Current status of beta-thalassemia and its treatment strategies. Mol Genet Genomic Med. 2021 Dec;9(12):e1788. doi: 10.1002/mgg3.1788. Epub 2021 Nov 5.
PubMed 34738740
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Comparison of Combination Therapy (Thalidomide and Hydroxyurea) vs Thalidomide Alone in Beta Thalassemia.
- ClinicalTrials.gov ID
- NCT06490601
- Acronym / sponsor ID
- Thalidomide / NIBD/IRB-242/12-2022
- Conditions
- Fetal Hemoglobin, Beta-Thalassemia, Hemoglobinopathies
- Keywords
- thalidomide, safety and efficacy, thalassemia, fetal hemoglobin inducer, hydroxyurea
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), PakistanOther government
- Study start
- 1 Apr 2022 (actual)
- Primary completion
- 1 Apr 2024 (actual)
- Study completion
- 1 Jul 2025 (estimated)
- First posted
- 8 Jul 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 project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone. The study, lasting three years, is a Phase 2 single-center, open-label interventional study with a sample size of 30 participants aged 8-35 years. It includes specific inclusion and exclusion criteria for participant selection. Data will be collected through clinical interviews and medical records and analyzed using(Statistical Package for the Social Sciences. This project aims to enhance beta thalassemia treatment strategies, focusing on reducing transfusion dependency and improving patient quality of life.
Detailed description
Study titled Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial, conducted at the National Institute of Blood Disease \& Bone Marrow Transplantation (NIBD \& BMT).
This study focuses on the long-term comparison of combination therapy (thalidomide and hydroxyurea) versus thalidomide alone in treating beta thalassemia. The objective is to evaluate the efficacy and safety of the combination therapy compared to thalidomide alone, with the hypothesis that the combination will be more effective. Beta thalassemia is defined as an autosomal recessive disorder affecting beta-globin production, influenced by genetic modifiers. Key variables include hemoglobin, red blood cells, leukocyte count, reticulocyte count, platelets, lactate dehydrogenase, nucleated red blood cells, ferritin, bilirubin, Serum Glutamate Pyruvate Transaminase, creatinine, transfusion frequency, spleen and liver size, hemoglobin subunit beta [ Homo sapiens (human) ] mutation, and certain polymorphism in gamma globin gene . The study took place at NIBD hospital over three years, designed as a Phase 2 single-center, two-arm open-label interventional study with a sample size of 30 participants using simple randomized sampling. Inclusion criteria are beta thalassemia major/intermediate patients aged 8-35 years, while exclusion criteria include patients with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis and fits. Data will be collected through clinical interviews and medical record reviews and analyzed using (Statistical Package for the Social Sciences.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- study participants were divided into 2 groups. one was treated with combination therapy that includes thalidomide and hydroxyurea whereas another group was treated with thalidomide alone.
| Arm | Type | Receives |
|---|---|---|
| combination therapy (thalidomide and hydroxyurea) | Experimental | Drug: thalidomide and hydroxyureaIn this group, participants were treated with combination therapy that includes thalidomide and hydroxyurea at the dose of 100mg/day at night with aspirin and 500mg /day respectively. |
| thalidomide alone | Active comparator | Drug: ThalidomideIn this group, participants were treated with thalidomide at the dose of 100mg/day at night with aspirin. |
Who may qualify
- Ages
- 8 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Known case of beta thalassemia major/intermediate (transfusion dependent)
- Willing to give informed consent
Key exclusion criteria (4 listed)
- Patients with comorbidities such as liver dysfunction
- Married patients
- Lactating mothers
- History of thrombosis and fits
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 and hydroxyureaAlso: combination therapy in beta thalassemiaThalidomide: Thalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor- alpha Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine. Hydroxyurea: Hydroxyurea or hydroxycarbamide (HU) lies in the category of antimetabolite. Mechanism of fetal hemoglobin induction includes increase in erythropoietin and nitric oxide production, apoptosis induction and potentiating in granulocyte cycling activity. | Drug | combination therapy (thalidomide and hydroxyurea) |
| ThalidomideAlso: fetal hemoglobin inducerThalidomide glutarimide is derivation of glutamic acid. Potentiating of fetal hemoglobin expression occurs by up regulation of Erythroid transcription factor and Erythroid Krüppel-like factor expression Furthermore few studies also concluded that thalidomide hypomethylate 27th amino acid in Histone H3 in the gamma globin gene. Initiating cause of this process is suppression of Nuclear factor (kappa-light-chain-enhancer of activated B cells) activation by tumor necrosis factor -alpha , Vascular endothelial growth factor , Prostaglandin E2 and inflammatory cytokine. Investigating the impact of thalidomide on transfusion-dependent beta thalassemia patients is essential for discerning its therapeutic efficacy and safety profile. | Drug | thalidomide alone |
Primary outcome
hemoglobin levels
Time frame: 3 years
Measure the improvement in hemoglobin levels Hemoglobin levels will be assessed using a complete blood count (CBC) test, measured in grams per deciliter (g/dL) of blood. This test is conducted through a venous blood sample, which is then analyzed using an automated hematology analyzer to determine the hemoglobin concentration.
red blood cell count.
Time frame: 3 years
Measure the improvement in red blood cell count. Red blood cell count will be assessed using a complete blood count (CBC) test, measured in millions of cells per microliter (million cells/µL) of blood. This test is conducted through a venous blood sample, analyzed using an automated hematology analyzer to determine the number of red blood cells present.
leukocyte count
Time frame: 3 years
Measure the effect on in leukocyte count Leukocyte count will be assessed using a complete blood count (CBC) test, measured in thousands of cells per microliter (thousand cells/µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer to determine the total number of white blood cells present.
reticulocyte count
Time frame: 3 years
Measure the effect on reticulocyte count Reticulocyte count will be assessed using a complete blood count (CBC) test, measured as a percentage of the total red blood cells or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which identifies and quantifies reticulocytes using specific staining techniques.
Transfusion Frequency:
Time frame: 3 years
Document and compare the frequency of blood transfusions required by patients in both treatment arms over the study period. Transfusion frequency will be assessed by recording the number of blood transfusions a patient receives over a specified period, such as weekly, monthly, or annually. This data will be collected from patient medical records and/or transfusion logs, ensuring accurate tracking of each transfusion event.
7 secondary outcomes
Spleen and Liver Size
Time frame: 3 years
Serum Ferritin Levels
Time frame: 3 years
Genetic Modifiers:
Time frame: 3 years
bilirubin
Time frame: 3 years
lactate dehydrogenase.
Time frame: 3 years
Serum Glutamate Pyruvate Transaminase,
Time frame: 3 years
creatinine
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2025.
- Background: PubMed 34738740Ali S, Mumtaz S, Shakir HA, Khan M, Tahir HM, Mumtaz S, Mughal TA, Hassan A, Kazmi SAR, Sadia, Irfan M, Khan MA. Current status of beta-thalassemia and its treatment strategies. Mol Genet Genomic Med. 2021 Dec;9(12):e1788. doi: 10.1002/mgg3.1788. Epub 2021 Nov 5.
- Individual participant dataSharing planned: Undecided. it will decide later.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 and hydroxyurea and Thalidomide in people with Fetal Hemoglobin and 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 2 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: under way, not looking for participants, as of the sponsor's last update on 30 May 2025. 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?
This study is still under way. Results are not available in the source record yet.
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?
hemoglobin levels (time frame: 3 years). The study lists 5 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 NCT06490601, 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 30 May 2025.