Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia (Thal-Thalido)

• ClinicalTrials.gov Identifier: NCT03651102
• Recruitment Status: Recruiting
• First Posted: August 29, 2018
• Last Update Posted: January 30, 2020
• Sponsor: Blood Diseases Clinic
• Information provided by (Responsible Party): Muhammad Tariq Masood Khan, Blood Diseases Clinic

Tracking Information

• First Submitted Date: August 24, 2018
• First Posted Date: August 29, 2018
• Last Update Posted Date: January 30, 2020
• Actual Study Start Date: January 1, 2018
• Estimated Primary Completion Date: August 30, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 26, 2020)

Haemoglobin Level [ Time Frame: 6-24 months ]

mg/dL

Original Primary Outcome Measures (submitted: August 27, 2018)

Haemoglobin Level [ Time Frame: 6 months ]

mg/dL

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
• Official Title: Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan
• 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 younger thalassemia patients.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2; Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Thalassemia

Intervention

Drug: Thalidomide Oral Product

Dosage: 1-3mg/kg/day

Study Arms

Experimental: Patients Receiving Thalidomide Therapy

All the study patients will be given thalidomide at an average dose of 2mg/kg/day (range 1-3mg/kg/day). The patients will be followed at 4 weeks interval by a haematologist for monitoring of potential side effects and for evaluation of clinical and laboratory response.

Intervention: Drug: Thalidomide Oral Product

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.
• 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.
• 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.
• 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.
• 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.

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 26, 2020): 500
• Original Estimated Enrollment (submitted: August 27, 2018): 100
• Estimated Study Completion Date: January 1, 2021
• Estimated Primary Completion Date: August 30, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Transfusion dependent thalassemia patients

Exclusion Criteria:

• Those with active systemic comorbidity, with past personal or family history of thrombophilia or history of splenectomy in the past 6 months

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Months to 50 Years (Child, Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Muhammad Tariq M Khan, MBBS, PhD; +92-300-5888867; drtariqmsd@gmail.com

Contact: Muhammad Tariq H Khan, MBBS, MPhil; +92-333-9355661; .tariq_razmian@yahoo.com

• Listed Location Countries: Pakistan

Administrative Information

• NCT Number: NCT03651102
• Other Study ID Numbers: TI
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• 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

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Muhammad Tariq Masood Khan, Blood Diseases Clinic
• Study Sponsor: Blood Diseases Clinic
• Collaborators: Not Provided

Investigators

• Study Chair: Muhammad Tariq M Khan, MBBS, PhD; Blood Diseases Clinic

• PRS Account: Blood Diseases Clinic
• Verification Date: January 2020