Transfusion-dependent ThalassemiaPhase 1NCT06098014
Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia
A Phase 1 study testing Thalidomide Capsules in people with Transfusion-dependent Thalassemia, run by Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh at 1 site in Bangladesh.
Trial summary
Transfusion Dependent Thalassemia (TDT) is emerging as a global public health concern. Hemopoietic stem cell transplantation (HSCT) is the only curative treatment. But its adoption is limited due to lack of Human leukocyte antigen (HLA) matched donor, experienced centers and high initial cost. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Transfusion-dependent Thalassemia
- What is being tested?
- Thalidomide Capsules
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteBangladesh
- Study dates
- 1 Mar 2023to 28 Feb 2024
- 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients diagnosed as transfusion dependent thalassemia
- Age ranged from 3-18 years
- Blood transfusion for more than 1 year.
Who cannot join
- Active systemic illness,
- Abnormal liver and kidney functions;
- Severe cardiopulmonary or cerebrovascular diseases;
- Recent fracture or recent major surgery
- Use of drugs that might affect Hb levels 3 months before enrollment;
- Any bleeding disorder .
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia: a Quasi Randomized Control Trial in a Tertiary Care Hospital in Bangladesh
Transfusion Dependent Thalassemia (TDT) is emerging as a global public health concern. Hemopoietic stem cell transplantation (HSCT) is the only curative treatment. But its adoption is limited due to lack of Human leukocyte antigen (HLA) matched donor, experienced centers and high initial cost. So, researches are going on in search of an effective, safe, easily available treatment option. Thalidomide a Fetal Hemoglobin (HbF) inducing drug shown to be effective in treatment of TDT patients in few case reports and small scale prospective and retrospective studies. However, most of these researches were done in adolescent and adult population. No randomized control trial was done to determine the safety and efficacy of Thalidomide in TDT children. So, this study will predict the safety and efficacy of Thalidomide in TDT children and will play an important role in planning a cost effective and affordable treatment option for TDT children.
This single centered non blinded quasi randomized clinical trial will be conducted at the Department of Pediatric Hematology and Oncology in Bangabandhu Sheikh Mujib Medical University (BSMMU), Bangladesh for one year of period. The objective of this study is to assess the safety and efficacy of Thalidomide in TDT children 30 transfusion dependent thalassemia children of 3-18 years old will be included.
Read more
This study will involve minimum physical risk to the patient. Written informed consent will be taken from parents or study subjects after brief explanation of the purpose and procedure. They will also be informed about the freedom to participate or not to participate at any time. Privacy and confidentiality will be safe guarded. History regarding age, sex, height, weight of these patients will be taken. Through physical examinations and laboratory investigations including complete blood count (CBC), Hb electrophoresis, serum Ferritin, serum creatinine, serum glutamic pyruvic transaminase (SGPT), serum lactate dehydrogenase (LDH) will be done.
Data will be collected in a predesigned questionnaire and will be kept confidential. Statistical analysis will be done using the statistical package for social science (SPSS) software .
Read the detailed description
Study design: Quasi-randomized controlled tria
Period of Study: From March 2023 to February 2023
Place of study:
Department of Pediatric Hematology \& Oncology, Bangabandhu Sheikh Mujib Medical University (BSMMU), Dhaka, Bangladesh.
Study Population:
TDT patients of either gender and 3-18 years of age attending in the Department of Pediatric Hematology and Oncology, Bangabandhu Sheikh Mujib Medical University, Dhaka and will receive Thalidomide during the study period.
Sample size will be calculated by using following formula:
((u+v)^2 (σ₁²+σ₀²))/((μ₁-μ₀)²) Here, u = .842 (if power= 80%) v = 1.96 (if significance level= 5%) μ₀ (mean Hb level before treatment with Thalidomide) = 71 μ₁ (mean Hb level in after treatment with Thalidomide) = 95 σ₀ = assumed population standard deviation for Placebo group = 30 σ₁ = assumed population standard deviation for Thalidomide group = 30
Calculated sample size is:
((u+v)^2 (σ₁²+σ₀²))/((μ₁-μ₀)²)
- (1.96+0.842)2 ( 30² +30²)/ (24)2
= 24.5 ~25
Group sample sizes of 25 in each group, considering 10% drop out it may increase up to 30 as some cases may be lost to follow up during study period.
So, sample size will be 30+30 = 60.
Sampling technique:
From the date of start of study, patient will be selected purposively by inclusion and exclusion criteria during the desired study period. The patients will be randomized 1:1 to receive placebo or thalidomide. Block or restricted randomization will be used to select the intervention group and control. All odd number patients (1,3,7…) will be considered as block \& will be assigned to intervention group by lottery and then all even number patients (2,4,6…) will be assigned to the other group.
Data collection procedure:
This study will be conducted in the Department of Pediatric Hematology and Oncology, Bangabandhu Sheikh Mujib Medical University (BSMMU). Children aged 3 to 18 years of both sexes diagnosed as TDT visiting the hematology out patient department (OPD) between March 2023 to February 2024 will be included in this study. Informed written consent from the patients or parents or guardians will be obtained at the time of study enrollment.
After recruitment, subjects will be randomly divided into two groups according to the Thalidomide therapy as follows:
- Experimental Group: Patients who will receive Thalidomide along with regular drugs.
- Control Group: Patients who will receive placebo with other regular drugs are considered as in the control group.
Data will be collected using a preformed data collection sheet (questionnaire). Demographic data such as age, sex, socio-economic status, family history of thalassemia will be collected from guardian or parents. Medical data regarding age, sex, age at first transfusion, red cell transfusion in last 1 year will be compiled. Clinical information such as pallor, pulse, blood pressure, respiratory rate, spleen size and other systemic clinical parameters will be taken.
Thalidomide and placebo will be given to randomly selected patients as per inclusion criteria attending in the outpatient department. The instruction will be given by the principal investigator according to study design.
Patients diagnosed with transfusion-dependent thalassemia will be randomized to receive placebo or Thalidomide treatment at a dose of 2-5 mg/kg/day.
The initial evaluation will be performed before the patients started Thalidomide. Patients will get transfusion with red blood cells at 10 ml/kg at any time during the study period if their Hb level < 70 g/L. The patients are followed up every 14 days for the first 12 weeks, and every 3 months thereafter. The efficacy will be evaluated after 12 weeks of treatment. Additionally, in patients with an obvious elevation in Hb who are free from blood transfusions for at least 6 weeks, the original treatment will be continuously administered for further evaluation. When the last patient is enrolled and completes their first 12 weeks of follow-up, the placebo-controlled period of the study will end.
Each evaluation will be included in a data collection sheet that will record crucial information such as the evaluation number, age, sex, type of diagnosis, Hb level, transfusion requirement etc. All participants will be asked to report any adverse reactions and will be questioned about adverse events during study visit.
- Conditions
- Transfusion-dependent Thalassemia
- Being tested
- Thalidomide Capsules, Placebo
- Keywords
- Thalidomide,, Thalassemia, Children
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thalidomide CapsulesDrug
To see safety and efficacy of Thalidomide in Transfusion depended thalassemia
PlaceboDrug
compare with Thalidomide
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide group | Active comparator | Thalidomide group Receives: Drug: Thalidomide Capsules |
| Placebo group | Placebo comparator | Placebo group Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
change in Hb level
gm/dl
Measured: 12 weeks after treatment initiation.
Secondary outcomes (4)
blood transfusion frequency
time
Measured: 3 months from initiation of treatment
change in Hb F
Measured: 3 months after initiation of treatment
Change in s. Ferritin level
ng/mL
Measured: 3 months after initiation of treatment
Adverse effect
Measured: 3 months after initiation of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2023
- Study start1 Mar 2023
- Primary completion28 Feb 2024
- Study completion28 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (20)Papers the sponsor has linked to this study.
- Other trials for Transfusion-dependent 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr. Md. Mehedi Hasan, Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06098014
- Sponsor's protocol ID
- 4280
- Status last verified
- Apr 2024
- First submitted
- 12 Oct 2023
- Last updated
- 9 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
Fard AD, Hosseini SA, Shahjahani M, Salari F, Jaseb K. Evaluation of Novel Fetal Hemoglobin Inducer Drugs in Treatment of beta-Hemoglobinopathy Disorders. Int J Hematol Oncol Stem Cell Res. 2013;7(3):47-54.
PubMed 24505535Farmakis D, Porter J, Taher A, Domenica Cappellini M, Angastiniotis M, Eleftheriou A. 2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia. Hemasphere. 2022 Jul 29;6(8):e732. doi: 10.1097/HS9.0000000000000732. eCollection 2022 Aug.
PubMed 35928543Fozza C, Pardini S, Giannico DB, Targhetta C, Di Tucci AA, Dessalvi P, Angelucci E, Dore F. Dramatic erythroid response to low-dose thalidomide in two patients with transfusion independent thalassemia and severe post-transfusional alloimmune hemolysis. Am J Hematol. 2015 Jul;90(7):E141. doi: 10.1002/ajh.24030. No abstract available.
PubMed 25850682Hossain MS, Raheem E, Sultana TA, Ferdous S, Nahar N, Islam S, Arifuzzaman M, Razzaque MA, Alam R, Aziz S, Khatun H, Rahim A, Morshed M. Thalassemias in South Asia: clinical lessons learnt from Bangladesh. Orphanet J Rare Dis. 2017 May 18;12(1):93. doi: 10.1186/s13023-017-0643-z.
PubMed 28521805Jain M, Chakrabarti P, Dolai TK, Ghosh P, Mandal PK, Baul SN, De R. Comparison of efficacy and safety of thalidomide vs hydroxyurea in patients with Hb E-beta thalassemia - a pilot study from a tertiary care Centre of India. Blood Cells Mol Dis. 2021 May;88:102544. doi: 10.1016/j.bcmd.2021.102544. Epub 2021 Feb 3.
PubMed 33610115Lu Y, Wei Z, Yang G, Lai Y, Liu R. Investigating the Efficacy and Safety of Thalidomide for Treating Patients With ss-Thalassemia: A Meta-Analysis. Front Pharmacol. 2022 Jan 11;12:814302. doi: 10.3389/fphar.2021.814302. eCollection 2021.
PubMed 35087410Masera N, Tavecchia L, Capra M, Cazzaniga G, Vimercati C, Pozzi L, Biondi A, Masera G. Optimal response to thalidomide in a patient with thalassaemia major resistant to conventional therapy. Blood Transfus. 2010 Jan;8(1):63-5. doi: 10.2450/2009.0102-09. No abstract available.
PubMed 20104280Rachmilewitz EA, Giardina PJ. How I treat thalassemia. Blood. 2011 Sep 29;118(13):3479-88. doi: 10.1182/blood-2010-08-300335. Epub 2011 Aug 2.
PubMed 21813448Shah FT, Sayani F, Trompeter S, Drasar E, Piga A. Challenges of blood transfusions in beta-thalassemia. Blood Rev. 2019 Sep;37:100588. doi: 10.1016/j.blre.2019.100588. Epub 2019 Jul 6.
PubMed 31324412Taher A, Vichinsky E, Musallam K, Cappellini MD, Viprakasit V, authors. Weatherall D, editor. Guidelines for the Management of Non Transfusion Dependent Thalassaemia (NTDT) [Internet]. Nicosia (Cyprus): Thalassaemia International Federation; 2013. Available from http://www.ncbi.nlm.nih.gov/books/NBK190453/
PubMed 24672826Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarre C, Beuzard Y, Chretien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342.
PubMed 29669226Weatherall DJ. The Evolving Spectrum of the Epidemiology of Thalassemia. Hematol Oncol Clin North Am. 2018 Apr;32(2):165-175. doi: 10.1016/j.hoc.2017.11.008.
PubMed 29458724Wolman IJ, Ortolani M. Some clinical features of Cooley's anemia patients as related to transfusion schedules. Ann N Y Acad Sci. 1969 Nov 20;165(1):407-14. doi: 10.1111/j.1749-6632.1969.tb27811.x. No abstract available.
PubMed 5260163Yassin AK. Promising Response to Thalidomide in Symptomatic beta-Thalassemia. Indian J Hematol Blood Transfus. 2020 Apr;36(2):337-341. doi: 10.1007/s12288-019-01231-5. Epub 2019 Nov 18.
PubMed 32425386Aerbajinai W, Zhu J, Gao Z, Chin K, Rodgers GP. Thalidomide induces gamma-globin gene expression through increased reactive oxygen species-mediated p38 MAPK signaling and histone H4 acetylation in adult erythropoiesis. Blood. 2007 Oct 15;110(8):2864-71. doi: 10.1182/blood-2007-01-065201. Epub 2007 Jul 9.
PubMed 17620452
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia: a Quasi Randomized Control Trial in a Tertiary Care Hospital in Bangladesh
- ClinicalTrials.gov ID
- NCT06098014
- Acronym / sponsor ID
- 4280
- Conditions
- Transfusion-dependent Thalassemia
- Keywords
- Thalidomide,, Thalassemia, Children
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Bangabandhu Sheikh Mujib Medical University, Dhaka, BangladeshUniversity, hospital or other
- Study start
- 1 Mar 2023 (actual)
- Primary completion
- 28 Feb 2024 (actual)
- Study completion
- 28 Feb 2024 (actual)
- First posted
- 24 Oct 2023 (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.
Transfusion Dependent Thalassemia (TDT) is emerging as a global public health concern. Hemopoietic stem cell transplantation (HSCT) is the only curative treatment. But its adoption is limited due to lack of Human leukocyte antigen (HLA) matched donor, experienced centers and high initial cost. So, researches are going on in search of an effective, safe, easily available treatment option. Thalidomide a Fetal Hemoglobin (HbF) inducing drug shown to be effective in treatment of TDT patients in few case reports and small scale prospective and retrospective studies. However, most of these researches were done in adolescent and adult population. No randomized control trial was done to determine the safety and efficacy of Thalidomide in TDT children. So, this study will predict the safety and efficacy of Thalidomide in TDT children and will play an important role in planning a cost effective and affordable treatment option for TDT children.
This single centered non blinded quasi randomized clinical trial will be conducted at the Department of Pediatric Hematology and Oncology in Bangabandhu Sheikh Mujib Medical University (BSMMU), Bangladesh for one year of period. The objective of this study is to assess the safety and efficacy of Thalidomide in TDT children 30 transfusion dependent thalassemia children of 3-18 years old will be included.
This study will involve minimum physical risk to the patient. Written informed consent will be taken from parents or study subjects after brief explanation of the purpose and procedure. They will also be informed about the freedom to participate or not to participate at any time. Privacy and confidentiality will be safe guarded. History regarding age, sex, height, weight of these patients will be taken. Through physical examinations and laboratory investigations including complete blood count (CBC), Hb electrophoresis, serum Ferritin, serum creatinine, serum glutamic pyruvic transaminase (SGPT), serum lactate dehydrogenase (LDH) will be done.
Data will be collected in a predesigned questionnaire and will be kept confidential. Statistical analysis will be done using the statistical package for social science (SPSS) software .
Detailed description
Study design: Quasi-randomized controlled tria
Period of Study: From March 2023 to February 2023
Place of study:
Department of Pediatric Hematology \& Oncology, Bangabandhu Sheikh Mujib Medical University (BSMMU), Dhaka, Bangladesh.
Study Population:
TDT patients of either gender and 3-18 years of age attending in the Department of Pediatric Hematology and Oncology, Bangabandhu Sheikh Mujib Medical University, Dhaka and will receive Thalidomide during the study period.
Sample size will be calculated by using following formula:
((u+v)^2 (σ₁²+σ₀²))/((μ₁-μ₀)²) Here, u = .842 (if power= 80%) v = 1.96 (if significance level= 5%) μ₀ (mean Hb level before treatment with Thalidomide) = 71 μ₁ (mean Hb level in after treatment with Thalidomide) = 95 σ₀ = assumed population standard deviation for Placebo group = 30 σ₁ = assumed population standard deviation for Thalidomide group = 30
Calculated sample size is:
((u+v)^2 (σ₁²+σ₀²))/((μ₁-μ₀)²)
- (1.96+0.842)2 ( 30² +30²)/ (24)2
= 24.5 ~25
Group sample sizes of 25 in each group, considering 10% drop out it may increase up to 30 as some cases may be lost to follow up during study period.
So, sample size will be 30+30 = 60.
Sampling technique:
From the date of start of study, patient will be selected purposively by inclusion and exclusion criteria during the desired study period. The patients will be randomized 1:1 to receive placebo or thalidomide. Block or restricted randomization will be used to select the intervention group and control. All odd number patients (1,3,7…) will be considered as block \& will be assigned to intervention group by lottery and then all even number patients (2,4,6…) will be assigned to the other group.
Data collection procedure:
This study will be conducted in the Department of Pediatric Hematology and Oncology, Bangabandhu Sheikh Mujib Medical University (BSMMU). Children aged 3 to 18 years of both sexes diagnosed as TDT visiting the hematology out patient department (OPD) between March 2023 to February 2024 will be included in this study. Informed written consent from the patients or parents or guardians will be obtained at the time of study enrollment.
After recruitment, subjects will be randomly divided into two groups according to the Thalidomide therapy as follows:
- Experimental Group: Patients who will receive Thalidomide along with regular drugs.
- Control Group: Patients who will receive placebo with other regular drugs are considered as in the control group.
Data will be collected using a preformed data collection sheet (questionnaire). Demographic data such as age, sex, socio-economic status, family history of thalassemia will be collected from guardian or parents. Medical data regarding age, sex, age at first transfusion, red cell transfusion in last 1 year will be compiled. Clinical information such as pallor, pulse, blood pressure, respiratory rate, spleen size and other systemic clinical parameters will be taken.
Thalidomide and placebo will be given to randomly selected patients as per inclusion criteria attending in the outpatient department. The instruction will be given by the principal investigator according to study design.
Patients diagnosed with transfusion-dependent thalassemia will be randomized to receive placebo or Thalidomide treatment at a dose of 2-5 mg/kg/day.
The initial evaluation will be performed before the patients started Thalidomide. Patients will get transfusion with red blood cells at 10 ml/kg at any time during the study period if their Hb level < 70 g/L. The patients are followed up every 14 days for the first 12 weeks, and every 3 months thereafter. The efficacy will be evaluated after 12 weeks of treatment. Additionally, in patients with an obvious elevation in Hb who are free from blood transfusions for at least 6 weeks, the original treatment will be continuously administered for further evaluation. When the last patient is enrolled and completes their first 12 weeks of follow-up, the placebo-controlled period of the study will end.
Each evaluation will be included in a data collection sheet that will record crucial information such as the evaluation number, age, sex, type of diagnosis, Hb level, transfusion requirement etc. All participants will be asked to report any adverse reactions and will be questioned about adverse events during study visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Thalidomide group | Active comparator | Drug: Thalidomide CapsulesThalidomide group |
| Placebo group | Placebo comparator | Drug: PlaceboPlacebo group |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Patients diagnosed as transfusion dependent thalassemia
- Age ranged from 3-18 years
- Blood transfusion for more than 1 year.
Key exclusion criteria (6 listed)
- Active systemic illness,
- Abnormal liver and kidney functions;
- Severe cardiopulmonary or cerebrovascular diseases;
- Recent fracture or recent major surgery
- Use of drugs that might affect Hb levels 3 months before enrollment;
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 CapsulesTo see safety and efficacy of Thalidomide in Transfusion depended thalassemia | Drug | Thalidomide group |
| Placebocompare with Thalidomide | Drug | Placebo group |
Primary outcome
change in Hb level
Time frame: 12 weeks after treatment initiation.
gm/dl
4 secondary outcomes
blood transfusion frequency
Time frame: 3 months from initiation of treatment
change in Hb F
Time frame: 3 months after initiation of treatment
Change in s. Ferritin level
Time frame: 3 months after initiation of treatment
Adverse effect
Time frame: 3 months after initiation of treatment
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 9 Apr 2024.
- Background: PubMed 24505535Fard AD, Hosseini SA, Shahjahani M, Salari F, Jaseb K. Evaluation of Novel Fetal Hemoglobin Inducer Drugs in Treatment of beta-Hemoglobinopathy Disorders. Int J Hematol Oncol Stem Cell Res. 2013;7(3):47-54.
- Background: PubMed 35928543Farmakis D, Porter J, Taher A, Domenica Cappellini M, Angastiniotis M, Eleftheriou A. 2021 Thalassaemia International Federation Guidelines for the Management of Transfusion-dependent Thalassemia. Hemasphere. 2022 Jul 29;6(8):e732. doi: 10.1097/HS9.0000000000000732. eCollection 2022 Aug.
- Background: PubMed 25850682Fozza C, Pardini S, Giannico DB, Targhetta C, Di Tucci AA, Dessalvi P, Angelucci E, Dore F. Dramatic erythroid response to low-dose thalidomide in two patients with transfusion independent thalassemia and severe post-transfusional alloimmune hemolysis. Am J Hematol. 2015 Jul;90(7):E141. doi: 10.1002/ajh.24030. No abstract available.
- Background: PubMed 28521805Hossain MS, Raheem E, Sultana TA, Ferdous S, Nahar N, Islam S, Arifuzzaman M, Razzaque MA, Alam R, Aziz S, Khatun H, Rahim A, Morshed M. Thalassemias in South Asia: clinical lessons learnt from Bangladesh. Orphanet J Rare Dis. 2017 May 18;12(1):93. doi: 10.1186/s13023-017-0643-z.
- Background: PubMed 33610115Jain M, Chakrabarti P, Dolai TK, Ghosh P, Mandal PK, Baul SN, De R. Comparison of efficacy and safety of thalidomide vs hydroxyurea in patients with Hb E-beta thalassemia - a pilot study from a tertiary care Centre of India. Blood Cells Mol Dis. 2021 May;88:102544. doi: 10.1016/j.bcmd.2021.102544. Epub 2021 Feb 3.
- Background: PubMed 35087410Lu Y, Wei Z, Yang G, Lai Y, Liu R. Investigating the Efficacy and Safety of Thalidomide for Treating Patients With ss-Thalassemia: A Meta-Analysis. Front Pharmacol. 2022 Jan 11;12:814302. doi: 10.3389/fphar.2021.814302. eCollection 2021.
- Background: PubMed 20104280Masera N, Tavecchia L, Capra M, Cazzaniga G, Vimercati C, Pozzi L, Biondi A, Masera G. Optimal response to thalidomide in a patient with thalassaemia major resistant to conventional therapy. Blood Transfus. 2010 Jan;8(1):63-5. doi: 10.2450/2009.0102-09. No abstract available.
- Background: PubMed 21813448Rachmilewitz EA, Giardina PJ. How I treat thalassemia. Blood. 2011 Sep 29;118(13):3479-88. doi: 10.1182/blood-2010-08-300335. Epub 2011 Aug 2.
- Background: PubMed 31324412Shah FT, Sayani F, Trompeter S, Drasar E, Piga A. Challenges of blood transfusions in beta-thalassemia. Blood Rev. 2019 Sep;37:100588. doi: 10.1016/j.blre.2019.100588. Epub 2019 Jul 6.
- Background: PubMed 24672826Taher A, Vichinsky E, Musallam K, Cappellini MD, Viprakasit V, authors. Weatherall D, editor. Guidelines for the Management of Non Transfusion Dependent Thalassaemia (NTDT) [Internet]. Nicosia (Cyprus): Thalassaemia International Federation; 2013. Available from http://www.ncbi.nlm.nih.gov/books/NBK190453/
- Background: PubMed 29669226Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarre C, Beuzard Y, Chretien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Gene Therapy in Patients with Transfusion-Dependent beta-Thalassemia. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342.
- Background: PubMed 29458724Weatherall DJ. The Evolving Spectrum of the Epidemiology of Thalassemia. Hematol Oncol Clin North Am. 2018 Apr;32(2):165-175. doi: 10.1016/j.hoc.2017.11.008.
- Individual participant dataSharing planned: Yes
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Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
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
RecruitingPhase 3
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Transfusion-dependent Alpha-Thalassemia, 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.
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 1 study testing Thalidomide Capsules in people with Transfusion-dependent Thalassemia, run by Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh at 1 site in Bangladesh.
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 3 inclusion and 6 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 9 Apr 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 Bangladesh. 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. Some participants in this study may receive a placebo. 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?
change in Hb level (time frame: 12 weeks after treatment initiation.). 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 NCT06098014, which also lists 20 linked publications.
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 9 Apr 2024.