Fetal HemoglobinPhase 2BTMNCT06490627
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
A Phase 2 study testing Thalidomide in people with Fetal Hemoglobin and Thalassemia Major, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan at 1 site in Pakistan.
Trial summary
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Read more about this study
- Who is it for?
- Ages 8 to 35 years
- Conditions the study is for
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- What is being tested?
- Thalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 54 participants
- Where
- 1 sitePakistan
- Study dates
- 22 Apr 2024to 22 Apr 2026
- 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
- Know 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 mother
- H/O thrombosis and fits
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Pakistan1 site, 1 recruiting
- Recruiting
National Institute of blood disease and bone marrow transplant
Karachi, Sindh
Safia Mehmood khan, PhD, 03342231644, rphsafia.nibd@gmail.com
About this study
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Conducted over two years at NIBD hospital, the study involves 54 transfusion-dependent patients aged 8-35. The primary objective is to correlate thalidomide doses with disease severity, adverse effects, and treatment response, aiming to optimize treatment strategies and reduce side effects.
Data will be collected through clinical interviews and medical record reviews and analyzed using SPSS. Key variables include hemoglobin levels, leukocyte and reticulocyte counts, platelets, liver and spleen size, genetic modifiers, and transfusion frequency. Inclusion criteria are specific to beta thalassemia patients, while exclusion criteria rule out those with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis or fits.
Read the detailed description
Thalassemia is an inherited monogenic blood disorder caused by improper synthesis of the hemoglobin chain, inherited in an autosomal recessive pattern. Hemoglobin is essential for oxygen transport from the lungs to body tissues. Initially observed in individuals of Italian descent, thalassemia is characterized by anemia, enlarged spleen, and bone abnormalities. It affects approximately 1.5% of the global population, with 60,000 infants born annually with severe forms such as homozygous alpha thalassemia, beta-thalassemia, and HbH disease. Patients with thalassemia major require frequent blood transfusions and iron chelation therapy to manage iron overload, which can lead to complications like cirrhosis, heart failure, and growth retardation. Iron chelators such as deferasirox, deferiprone, and deferoxamine are used in Pakistan either as solo or combination therapy based on iron levels. Bone marrow transplantation from HLA-identical siblings offers a curative option with high success rates, but non-HLA identical cases are less promising. Emerging therapies like HbF production reactivation, cell therapy, and gene therapy show potential for better management of thalassemia.
Beta thalassemia is a prevalent genetic disorder, especially in the Mediterranean, Middle East, and Southeast Asia. It causes reduced hemoglobin production, severe anemia, and dependence on regular blood transfusions, which lead to iron overload and associated complications. Thalidomide, initially marketed as a sedative in 1954 and later withdrawn due to teratogenic effects, has shown efficacy in hematologic disorders. Its potential in beta thalassemia, particularly for reducing transfusion requirements and managing iron overload, remains underexplored. Preliminary studies suggest thalidomide could reduce transfusion needs, but comprehensive dose-dependent research is lacking. This study aims to evaluate the effects of thalidomide at various doses in transfusion-dependent beta thalassemia patients, hypothesizing that optimal dosing can improve disease management and quality of life.
Preliminary research indicates thalidomide might reduce transfusion frequency and manage iron overload in beta thalassemia patients. However, detailed dose-dependent studies are necessary. This research aims to fill the gap by exploring thalidomide's benefits and safety profiles at diverse doses, potentially revolutionizing the therapeutic approach to beta thalassemia.
The study aims to evaluate the impact of diverse thalidomide doses on reducing transfusion dependency in beta thalassemia patients. Primary objectives include assessing the efficacy of thalidomide in reducing transfusion needs. Secondary objectives involve evaluating the impact on complete blood count, liver function, spleen size, serum ferritin levels, and iron overload, alongside monitoring safety profiles and adverse events. Impact of genetic modifiers on thalidomide and beta thalassemia phenotype will also monitor in this study.
The study will be a single-center randomized controlled clinical trial at the National Institute of Blood Disease and Bone Marrow Transplant Hospital in Karachi, Pakistan, specializing in genetic disorders and hematology-oncology. Participants will be divided into treatment (with two dose groups) and control groups, with a total sample size of 54 calculated using OpenEpi. The study will span two years, from May 2024 to May 2026, involving data collection through medical records, interviews, and questionnaires. Ethical approval and informed consent will be obtained, ensuring patient confidentiality and adherence to ethical standards.
Data will include patient demographics (age, gender, ethnicity, marital status, weight, height), clinical variables (type of thalassemia, comorbidities, previous treatment), laboratory variables (hemoglobin, leukocyte count, reticulocyte count, platelets, lactate dehydrogenase, ferritin, d-dimer, bilirubin levels, SGPT), genetic modifiers (HBB mutation, XMN polymorphism, BCL11A polymorphism, alpha chain co-inheritance), and others (spleen and liver size, fibroscan, T2 star, transfusion frequency).
- Conditions
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- Being tested
- Thalidomide
- Keywords
- thalidomide, safety and efficacy, thalassemia, fetal hemoglobin inducer
- Medical subject headings
- beta-Thalassemia, Genetic Diseases, Inborn, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThalidomideDrug
Thalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients.
Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 5-6mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period.
ThalidomideDrug
Thalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients.
Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 7-8mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period.
| Study group | Role | What they receive |
|---|---|---|
| Control | No intervention | in this group, patient will not receive any interventional drug or treatment drug so simply they will blood transfusion dependent. |
| Group 1 Thalidomide | Experimental | In this group , thalassemia patients will receive thalidomide at the dose of 5-6mg/kg/day at night with aspirin. Receives: Drug: Thalidomide |
| Group 2 Thalidomide | Active comparator | In this group , thalassemia patients will receive thalidomide at the dose of 7-8mg/kg/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.
transfusion dependency
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: 1 year
Secondary outcomes (16)
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: 2 years
total 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: 2 years
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: 2 years
Platelets
Platelet 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 number of platelets.
Measured: 2 years
impact of genetic modifiers (e.g., HBB mutation, XMN polymorphism, BCL11A polymorphism, and co-inheritance of alpha chain) on the treatment outcomes with thalidomide.
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: 2 years
Spleen Size
Spleen size will be assessed using imaging techniques such as ultrasound, computed tomography (CT) scan, or magnetic resonance imaging (MRI). Measurements will be reported in centimeters (cm).
Measured: 2 years
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.
Measured: 2 years
Serum Ferritin Levels
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: 2 years
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: 2 years
Nucleated red blood cells
Nucleated red blood cells (NRBCs) will be assessed using a complete blood count (CBC) test with a differential, measured as the number of NRBCs per 100 white blood cells (WBCs) or as an absolute number per microliter (µL) of blood. This test involves analyzing a venous blood sample with an automated hematology analyzer, which can differentiate and count NRBCs to provide insights into bone marrow function and potential hematologic disorders.
Measured: 2 years
D-dimer
D-dimer levels will be assessed through a blood test, typically measured in nanograms per milliliter (ng/mL) or micrograms per liter (µg/L). This test analyzes a venous blood sample to determine the concentration of D-dimer, a fibrin degradation product.
Measured: 2 years
Total bilirubin
Total bilirubin (TBIL) levels will be assessed through a blood test, measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the total concentration of bilirubin, including both direct (conjugated) and indirect (unconjugated) bilirubin.
Measured: 2 years
Indirect bilirubin
Indirect bilirubin (IBIL) levels will be assessed through a blood test, measured in milligrams per deciliter (mg/dL). This test analyzes a venous blood sample to determine the concentration of unconjugated bilirubin, which is a byproduct of red blood cell breakdown before it is processed by the liver.
Measured: 2 years
Fibroscan
FibroScan will be used to assess liver stiffness and steatosis, which can be indicative of liver iron overload. Elevated serum ferritin levels may suggest iron accumulation in the liver, and FibroScan provides a non-invasive method to evaluate the extent of liver fibrosis and fat content.
Measured: 2 years
T2 star imaging
T2* (T2 star) magnetic resonance imaging (MRI) will be used to assess liver iron concentration in relation to serum ferritin levels. This imaging technique measures the rate of signal decay in liver tissue, which is inversely correlated with iron content. A shorter T2* value indicates higher iron concentration. T2* MRI provides a non-invasive, quantitative assessment of liver iron overload.
Measured: 2 years
transfusion dependency
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: 2 year
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 start22 Apr 2024
- Primary completion22 Apr 2025
- Study completion22 Apr 2026 (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
- in this study three groups have been made. one is control group which received no intervention and on transfusion only. whereas other two groups are interventional both received thalidomide. one received thalidomide at 5-6mg/kg/day and second at 7-8 mg/kg/day.
- Masking
- None (open label)
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Safia mehmood khan, National Institute of Blood Disease Center, Pakistan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06490627
- Sponsor's protocol ID
- NIBD/IRB-266/01-2024
- Acronym
- BTM
- Status last verified
- May 2025
- First submitted
- 20 Jun 2024
- Last updated
- 30 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Maybe i will share the IPD upon request
Publications
Yang K, Wu Y, Zhou Y, Long B, Lu Q, Zhou T, Wang L, Geng Z, Yin X. Thalidomide for Patients with beta-Thalassemia: A Multicenter Experience. Mediterr J Hematol Infect Dis. 2020 May 1;12(1):e2020021. doi: 10.4084/MJHID.2020.021. eCollection 2020.
PubMed 32395210
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
- Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
- ClinicalTrials.gov ID
- NCT06490627
- Acronym / sponsor ID
- BTM / NIBD/IRB-266/01-2024
- Conditions
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- Keywords
- thalidomide, safety and efficacy, thalassemia, fetal hemoglobin inducer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), PakistanOther government
- Study start
- 22 Apr 2024 (actual)
- Primary completion
- 22 Apr 2025 (actual)
- Study completion
- 22 Apr 2026 (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 "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing anemia. Conducted over two years at NIBD hospital, the study involves 54 transfusion-dependent patients aged 8-35. The primary objective is to correlate thalidomide doses with disease severity, adverse effects, and treatment response, aiming to optimize treatment strategies and reduce side effects.
Data will be collected through clinical interviews and medical record reviews and analyzed using SPSS. Key variables include hemoglobin levels, leukocyte and reticulocyte counts, platelets, liver and spleen size, genetic modifiers, and transfusion frequency. Inclusion criteria are specific to beta thalassemia patients, while exclusion criteria rule out those with liver dysfunction, married patients, lactating mothers, and those with a history of thrombosis or fits.
Detailed description
Thalassemia is an inherited monogenic blood disorder caused by improper synthesis of the hemoglobin chain, inherited in an autosomal recessive pattern. Hemoglobin is essential for oxygen transport from the lungs to body tissues. Initially observed in individuals of Italian descent, thalassemia is characterized by anemia, enlarged spleen, and bone abnormalities. It affects approximately 1.5% of the global population, with 60,000 infants born annually with severe forms such as homozygous alpha thalassemia, beta-thalassemia, and HbH disease. Patients with thalassemia major require frequent blood transfusions and iron chelation therapy to manage iron overload, which can lead to complications like cirrhosis, heart failure, and growth retardation. Iron chelators such as deferasirox, deferiprone, and deferoxamine are used in Pakistan either as solo or combination therapy based on iron levels. Bone marrow transplantation from HLA-identical siblings offers a curative option with high success rates, but non-HLA identical cases are less promising. Emerging therapies like HbF production reactivation, cell therapy, and gene therapy show potential for better management of thalassemia.
Beta thalassemia is a prevalent genetic disorder, especially in the Mediterranean, Middle East, and Southeast Asia. It causes reduced hemoglobin production, severe anemia, and dependence on regular blood transfusions, which lead to iron overload and associated complications. Thalidomide, initially marketed as a sedative in 1954 and later withdrawn due to teratogenic effects, has shown efficacy in hematologic disorders. Its potential in beta thalassemia, particularly for reducing transfusion requirements and managing iron overload, remains underexplored. Preliminary studies suggest thalidomide could reduce transfusion needs, but comprehensive dose-dependent research is lacking. This study aims to evaluate the effects of thalidomide at various doses in transfusion-dependent beta thalassemia patients, hypothesizing that optimal dosing can improve disease management and quality of life.
Preliminary research indicates thalidomide might reduce transfusion frequency and manage iron overload in beta thalassemia patients. However, detailed dose-dependent studies are necessary. This research aims to fill the gap by exploring thalidomide's benefits and safety profiles at diverse doses, potentially revolutionizing the therapeutic approach to beta thalassemia.
The study aims to evaluate the impact of diverse thalidomide doses on reducing transfusion dependency in beta thalassemia patients. Primary objectives include assessing the efficacy of thalidomide in reducing transfusion needs. Secondary objectives involve evaluating the impact on complete blood count, liver function, spleen size, serum ferritin levels, and iron overload, alongside monitoring safety profiles and adverse events. Impact of genetic modifiers on thalidomide and beta thalassemia phenotype will also monitor in this study.
The study will be a single-center randomized controlled clinical trial at the National Institute of Blood Disease and Bone Marrow Transplant Hospital in Karachi, Pakistan, specializing in genetic disorders and hematology-oncology. Participants will be divided into treatment (with two dose groups) and control groups, with a total sample size of 54 calculated using OpenEpi. The study will span two years, from May 2024 to May 2026, involving data collection through medical records, interviews, and questionnaires. Ethical approval and informed consent will be obtained, ensuring patient confidentiality and adherence to ethical standards.
Data will include patient demographics (age, gender, ethnicity, marital status, weight, height), clinical variables (type of thalassemia, comorbidities, previous treatment), laboratory variables (hemoglobin, leukocyte count, reticulocyte count, platelets, lactate dehydrogenase, ferritin, d-dimer, bilirubin levels, SGPT), genetic modifiers (HBB mutation, XMN polymorphism, BCL11A polymorphism, alpha chain co-inheritance), and others (spleen and liver size, fibroscan, T2 star, transfusion frequency).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- in this study three groups have been made. one is control group which received no intervention and on transfusion only. whereas other two groups are interventional both received thalidomide. one received thalidomide at 5-6mg/kg/day and second at 7-8 mg/kg/day.
| Arm | Type | Receives |
|---|---|---|
| Control | No intervention | –in this group, patient will not receive any interventional drug or treatment drug so simply they will blood transfusion dependent. |
| Group 1 Thalidomide | Experimental | Drug: ThalidomideIn this group , thalassemia patients will receive thalidomide at the dose of 5-6mg/kg/day at night with aspirin. |
| Group 2 Thalidomide | Active comparator | Drug: ThalidomideIn this group , thalassemia patients will receive thalidomide at the dose of 7-8mg/kg/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)
- Know 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 mother
- H/O 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 |
|---|---|---|
| ThalidomideThalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients. Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 5-6mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period. | Drug | Group 1 Thalidomide |
| ThalidomideThalidomide is being investigated for its therapeutic efficacy and safety profile in transfusion-dependent beta thalassemia patients. Participants in this arm received thalidomide. Thalidomide was administered orally at a dosage of 7-8mg/kg/day. The medication was taken continuously or on a specified schedule (e.g., daily,) for the duration of the study period, which lasted 2 years. Participants were monitored for adherence to the medication regimen and for any adverse effects throughout the intervention period. | Drug | Group 2 Thalidomide |
Primary outcome
transfusion dependency
Time frame: 1 year
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
16 secondary outcomes
hemoglobin levels.
Time frame: 2 years
total leukocyte count.
Time frame: 2 years
reticulocyte count
Time frame: 2 years
Platelets
Time frame: 2 years
impact of genetic modifiers (e.g., HBB mutation, XMN polymorphism, BCL11A polymorphism, and co-inheritance of alpha chain) on the treatment outcomes with thalidomide.
Time frame: 2 years
Spleen Size
Time frame: 2 years
Serum Glutamate Pyruvate Transaminase,
Time frame: 2 years
Serum Ferritin Levels
Time frame: 2 years
lactate dehydrogenase
Time frame: 2 years
Nucleated red blood cells
Time frame: 2 years
D-dimer
Time frame: 2 years
Total bilirubin
Time frame: 2 years
Indirect bilirubin
Time frame: 2 years
Fibroscan
Time frame: 2 years
T2 star imaging
Time frame: 2 years
transfusion dependency
Time frame: 2 year
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.
- Results publication: PubMed 32395210Yang K, Wu Y, Zhou Y, Long B, Lu Q, Zhou T, Wang L, Geng Z, Yin X. Thalidomide for Patients with beta-Thalassemia: A Multicenter Experience. Mediterr J Hematol Infect Dis. 2020 May 1;12(1):e2020021. doi: 10.4084/MJHID.2020.021. eCollection 2020.
- Individual participant dataSharing planned: Undecided. Maybe i will share the IPD upon request
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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.
National Institute of blood disease and bone marrow transplant
Karachi, Pakistan
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 in people with Fetal Hemoglobin and Thalassemia Major, 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: looking for participants now, 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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
transfusion dependency (time frame: 1 year). 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 NCT06490627, 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.