ThalassemiaPhase 2/3Thal-ThalidoNCT03651102
Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
A Phase 2/3 study testing Thalidomide and Hydroxyurea in people with Thalassemia, run by Blood Diseases Clinic at 1 site in Pakistan.
Trial summary
Thalidomide is known to have hypnosedative, immuno-modulatory and anti-angiogenic effects. The drug is widely used in several neoplastic disorders (e.g. multiple myeloma and malignant melanoma), inflammatory conditions (e.g. Crohn's disease) and skin disorders (e.g. leprosy). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Thalassemia
- What is being tested?
- Thalidomide and Hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 654 participants
- Where
- 1 sitePakistan
- Study dates
- 1 Jan 2018to 1 Mar 2022
- Phase
- Phase 2/3
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Transfusion dependent thalassemia patients refractory to Hydroxyurea
Who cannot join
- Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan
Thalidomide is known to have hypnosedative, immuno-modulatory and anti-angiogenic effects. The drug is widely used in several neoplastic disorders (e.g. multiple myeloma and malignant melanoma), inflammatory conditions (e.g. Crohn's disease) and skin disorders (e.g. leprosy). Thalidomide has been successfully used in adult thalassemia patients. The current study explores its role in transfusion-dependent thalassemia patients.
- Conditions
- Thalassemia
- Being tested
- Thalidomide and Hydroxyurea
- Keywords
- Thalidomide, Thalassemia
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thalidomide and HydroxyureaDrug
Dosage: Thalidomide 1-4mg/kg/day, Hydroxyurea 10-20mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide in combination with hydroxyurea | Experimental | All the study patients refractory to hydroxyurea will be given thalidomide at an average dose of 2mg/kg/day (range 1-4mg/kg/day). Participants will also be continued on hydroxyurea at a dose of 10-20 mg/kg/day. Receives: Drug: Thalidomide and Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemoglobin Level
mg/dL
Measured: 6-24 months
Response Rate (Excellent, Good, Partial and No response)
Measured: 6-24 months
Adverse Effects
Measured: 6-24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2018
- Study start1 Jan 2018
- Primary completion30 Nov 2021
- Study completion1 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 654 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blood Diseases Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- University of Peshawar
- Responsible party
- Principal investigator, Muhammad Tariq Masood Khan, Blood Diseases Clinic
- Investigators
- Muhammad Tariq M Khan, MBBS, PhD, Principal investigator, Blood Diseases ClinicZahid Ali, PharmD, MPhil, Study director, Department of Pharmacy, University of Peshawar
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03651102
- Sponsor's protocol ID
- TDT
- Acronym
- Thal-Thalido
- Status last verified
- Mar 2022
- First submitted
- 24 Aug 2018
- Last updated
- 25 Mar 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Li Y, Ren Q, Zhou Y, Li P, Lin W, Yin X. Thalidomide has a significant effect in patients with thalassemia intermedia. Hematology. 2018 Jan;23(1):50-54. doi: 10.1080/10245332.2017.1354427. Epub 2017 Jul 18.
PubMed 28718348Ren Q, Zhou YL, Wang L, Chen YS, Ma YN, Li PP, Yin XL. Clinical trial on the effects of thalidomide on hemoglobin synthesis in patients with moderate thalassemia intermedia. Ann Hematol. 2018 Oct;97(10):1933-1939. doi: 10.1007/s00277-018-3395-5. Epub 2018 Jun 22.
PubMed 29931453Masera 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 20104280Ricchi P, Costantini S, Spasiano A, De Dominicis G, Di Matola T, Cinque P, Ammirabile M, Marsella M, Filosa A. The long-term and extensive efficacy of low dose thalidomide in a case of an untransfusable patient with Non-Transfusion-Dependent Thalassemia. Blood Cells Mol Dis. 2016 Mar;57:97-9. doi: 10.1016/j.bcmd.2016.01.003. Epub 2016 Jan 16.
PubMed 26810455Aguilar-Lopez LB, Delgado-Lamas JL, Rubio-Jurado B, Perea FJ, Ibarra B. Thalidomide therapy in a patient with thalassemia major. Blood Cells Mol Dis. 2008 Jul-Aug;41(1):136-7. doi: 10.1016/j.bcmd.2008.03.001. Epub 2008 Apr 24. No abstract available.
PubMed 18439858Chen J, Zhu W, Cai N, Bu S, Li J, Huang L. Thalidomide induces haematologic responses in patients with beta-thalassaemia. Eur J Haematol. 2017 Nov;99(5):437-441. doi: 10.1111/ejh.12955. Epub 2017 Sep 27.
PubMed 28850716Fard 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 24505535Gambari R. Foetal haemoglobin inducers and thalassaemia: novel achievements. Blood Transfus. 2010 Jan;8(1):5-7. doi: 10.2450/2009.0137-09. No abstract available.
PubMed 20104272Ali Z, Ismail M, Rehman IU, Rani GF, Ali M, Khan MTM. Long-term clinical efficacy and safety of thalidomide in patients with transfusion-dependent beta-thalassemia: results from Thal-Thalido study. Sci Rep. 2023 Aug 21;13(1):13592. doi: 10.1038/s41598-023-40849-4.
PubMed 37604857
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan
- ClinicalTrials.gov ID
- NCT03651102
- Acronym / sponsor ID
- Thal-Thalido / TDT
- Conditions
- Thalassemia
- Keywords
- Thalidomide, Thalassemia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 654 participants (actual)
- Lead sponsor
- Blood Diseases ClinicUniversity, hospital or other
- Collaborators
- University of Peshawar
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 30 Nov 2021 (actual)
- Study completion
- 1 Mar 2022 (actual)
- First posted
- 29 Aug 2018 (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.
Thalidomide is known to have hypnosedative, immuno-modulatory and anti-angiogenic effects. The drug is widely used in several neoplastic disorders (e.g. multiple myeloma and malignant melanoma), inflammatory conditions (e.g. Crohn's disease) and skin disorders (e.g. leprosy). Thalidomide has been successfully used in adult thalassemia patients. The current study explores its role in transfusion-dependent thalassemia patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thalidomide in combination with hydroxyurea | Experimental | Drug: Thalidomide and HydroxyureaAll the study patients refractory to hydroxyurea will be given thalidomide at an average dose of 2mg/kg/day (range 1-4mg/kg/day). Participants will also be continued on hydroxyurea at a dose of 10-20 mg/kg/day. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Transfusion dependent thalassemia patients refractory to Hydroxyurea
Key exclusion criteria (1 listed)
- Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery
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 HydroxyureaDosage: Thalidomide 1-4mg/kg/day, Hydroxyurea 10-20mg/kg/day | Drug | Thalidomide in combination with hydroxyurea |
Primary outcome
Haemoglobin Level
Time frame: 6-24 months
mg/dL
Response Rate (Excellent, Good, Partial and No response)
Time frame: 6-24 months
Adverse Effects
Time frame: 6-24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2022.
- Background: PubMed 28718348Li Y, Ren Q, Zhou Y, Li P, Lin W, Yin X. Thalidomide has a significant effect in patients with thalassemia intermedia. Hematology. 2018 Jan;23(1):50-54. doi: 10.1080/10245332.2017.1354427. Epub 2017 Jul 18.
- Background: PubMed 29931453Ren Q, Zhou YL, Wang L, Chen YS, Ma YN, Li PP, Yin XL. Clinical trial on the effects of thalidomide on hemoglobin synthesis in patients with moderate thalassemia intermedia. Ann Hematol. 2018 Oct;97(10):1933-1939. doi: 10.1007/s00277-018-3395-5. Epub 2018 Jun 22.
- 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 26810455Ricchi P, Costantini S, Spasiano A, De Dominicis G, Di Matola T, Cinque P, Ammirabile M, Marsella M, Filosa A. The long-term and extensive efficacy of low dose thalidomide in a case of an untransfusable patient with Non-Transfusion-Dependent Thalassemia. Blood Cells Mol Dis. 2016 Mar;57:97-9. doi: 10.1016/j.bcmd.2016.01.003. Epub 2016 Jan 16.
- Background: PubMed 18439858Aguilar-Lopez LB, Delgado-Lamas JL, Rubio-Jurado B, Perea FJ, Ibarra B. Thalidomide therapy in a patient with thalassemia major. Blood Cells Mol Dis. 2008 Jul-Aug;41(1):136-7. doi: 10.1016/j.bcmd.2008.03.001. Epub 2008 Apr 24. No abstract available.
- Background: PubMed 28850716Chen J, Zhu W, Cai N, Bu S, Li J, Huang L. Thalidomide induces haematologic responses in patients with beta-thalassaemia. Eur J Haematol. 2017 Nov;99(5):437-441. doi: 10.1111/ejh.12955. Epub 2017 Sep 27.
- 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 20104272Gambari R. Foetal haemoglobin inducers and thalassaemia: novel achievements. Blood Transfus. 2010 Jan;8(1):5-7. doi: 10.2450/2009.0137-09. No abstract available.
- Derived from this study: PubMed 37604857Ali Z, Ismail M, Rehman IU, Rani GF, Ali M, Khan MTM. Long-term clinical efficacy and safety of thalidomide in patients with transfusion-dependent beta-thalassemia: results from Thal-Thalido study. Sci Rep. 2023 Aug 21;13(1):13592. doi: 10.1038/s41598-023-40849-4.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Muhammad Tariq M Khan, MBBS, PhD principal investigator
Blood Diseases Clinic
Zahid Ali, PharmD, MPhil study director
Department of Pharmacy, University of Peshawar
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Thalidomide and Hydroxyurea in people with Thalassemia, run by Blood Diseases Clinic 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 1 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 25 Mar 2022. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Haemoglobin Level (time frame: 6-24 months). The study lists 3 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 NCT03651102, which also lists 9 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 25 Mar 2022.