Thal-Thalido: Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
Sponsor
Blood Diseases Clinic (Other)
Overall Status
Completed
CT.gov ID
NCT03651102
Collaborator
University of Peshawar (Other)
654
1
1
49.9
13.1
Study Details
Study Description
Brief 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). Thalidomide has been successfully used in adult thalassemia patients. The current study explores its role in transfusion-dependent thalassemia patients.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2/Phase 3 |
Study Design
Study Type:
Interventional
Actual Enrollment
:
654 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan
Actual Study Start Date
:
Jan 1, 2018
Actual Primary Completion Date
:
Nov 30, 2021
Actual Study Completion Date
:
Mar 1, 2022
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Thalidomide in combination with hydroxyurea 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. |
Drug: Thalidomide and Hydroxyurea
Dosage: Thalidomide 1-4mg/kg/day, Hydroxyurea 10-20mg/kg/day
|
Outcome Measures
Primary Outcome Measures
- Haemoglobin Level [6-24 months]
mg/dL
- Response Rate (Excellent, Good, Partial and No response) [6-24 months]
- Adverse Effects [6-24 months]
Eligibility Criteria
Criteria
Ages Eligible for Study:
1 Year
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
- Transfusion dependent thalassemia patients refractory to Hydroxyurea
Exclusion Criteria:
- Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Blood Diseases Clinic | Peshawar | Khyber Pakhtukhwa | Pakistan | 0000 |
Sponsors and Collaborators
- Blood Diseases Clinic
- University of Peshawar
Investigators
- Principal Investigator: Muhammad Tariq M Khan, MBBS, PhD, Blood Diseases Clinic
- Study Director: Zahid Ali, PharmD, MPhil, Department of Pharmacy, University of Peshawar
Study Documents (Full-Text)
None provided.More Information
Publications
- Aguilar-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.
- Chen J, Zhu W, Cai N, Bu S, Li J, Huang L. Thalidomide induces haematologic responses in patients with β-thalassaemia. Eur J Haematol. 2017 Nov;99(5):437-441. doi: 10.1111/ejh.12955. Epub 2017 Sep 27.
- Fard AD, Hosseini SA, Shahjahani M, Salari F, Jaseb K. Evaluation of Novel Fetal Hemoglobin Inducer Drugs in Treatment of β-Hemoglobinopathy Disorders. Int J Hematol Oncol Stem Cell Res. 2013;7(3):47-54. Review.
- Gambari R. Foetal haemoglobin inducers and thalassaemia: novel achievements. Blood Transfus. 2010 Jan;8(1):5-7. doi: 10.2450/2009.0137-09. Review.
- 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.
- Masera 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.
- Ren 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.
- Ricchi 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.
Responsible Party:
Muhammad Tariq Masood Khan,
Consultant Haematologist; Research Associate,
Blood Diseases Clinic
ClinicalTrials.gov Identifier:
NCT03651102
Other Study ID Numbers:
- TDT
First Posted:
Aug 29, 2018
Last Update Posted:
Mar 25, 2022
Last Verified:
Mar 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Product Manufactured in and Exported from the U.S.:
No
Keywords provided by Muhammad Tariq Masood Khan,
Consultant Haematologist; Research Associate,
Blood Diseases Clinic
Additional relevant MeSH terms: