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

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ClinicalTrials.gov Identifier: NCT03651102

Recruitment Status : Recruiting

First Posted : August 29, 2018

Last Update Posted : January 30, 2020

See Contacts and Locations

Sponsor:

Information provided by (Responsible Party):

Muhammad Tariq Masood Khan, Blood Diseases Clinic

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 younger thalassemia patients.

Condition or disease	Intervention/treatment	Phase
Thalassemia	Drug: Thalidomide Oral Product	Phase 2 Phase 3

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	500 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 :	January 1, 2018
Estimated Primary Completion Date :	August 30, 2020
Estimated Study Completion Date :	January 1, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Thalassemia

Drug Information available for: Thalidomide

Genetic and Rare Diseases Information Center resources: Thalassemia

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
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.	Drug: Thalidomide Oral Product Dosage: 1-3mg/kg/day

Outcome Measures

Primary Outcome Measures :

Haemoglobin Level [ Time Frame: 6-24 months ]
mg/dL

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	18 Months to 50 Years (Child, Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03651102

Contacts

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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

Locations

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Pakistan
Blood Diseases Clinic	Recruiting
Peshawar, Khyber Pakhtukhwa, Pakistan, 0000
Contact: Muhammad Tariq M Khan +92-300-5888867 drtariqmsd@gmail.com

Sponsors and Collaborators

Blood Diseases Clinic

Investigators

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

More Information

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.

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Responsible Party:	Muhammad Tariq Masood Khan, Consultant Haematologist; Research Associate, Blood Diseases Clinic
ClinicalTrials.gov Identifier:	NCT03651102
Other Study ID Numbers:	TI
First Posted:	August 29, 2018 Key Record Dates
Last Update Posted:	January 30, 2020
Last Verified:	January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	No

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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, Blood Diseases Clinic:


Thalidomide Thalassemia

Additional relevant MeSH terms:

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Thalassemia Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Thalidomide Immunosuppressive Agents Immunologic Factors	Physiological Effects of Drugs Leprostatic Agents Anti-Bacterial Agents Anti-Infective Agents Angiogenesis Inhibitors Angiogenesis Modulating Agents Growth Substances Growth Inhibitors Antineoplastic Agents

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