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

Primary Purpose

Thalassemia

Status
Completed
Phase
Phase 2
Locations
Pakistan
Study Type
Interventional
Intervention
Thalidomide and Hydroxyurea
Sponsored by
Blood Diseases Clinic
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thalassemia focused on measuring Thalidomide, Thalassemia

Eligibility Criteria

1 Year - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Blood Diseases Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Thalidomide in combination with hydroxyurea

Arm Description

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.

Outcomes

Primary Outcome Measures

Haemoglobin Level
mg/dL
Response Rate (Excellent, Good, Partial and No response)
Adverse Effects

Secondary Outcome Measures

Full Information

First Posted
August 24, 2018
Last Updated
March 10, 2022
Sponsor
Blood Diseases Clinic
Collaborators
University of Peshawar
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1. Study Identification

Unique Protocol Identification Number
NCT03651102
Brief Title
Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia
Acronym
Thal-Thalido
Official Title
Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan
Study Type
Interventional

2. Study Status

Record Verification Date
March 2022
Overall Recruitment Status
Completed
Study Start Date
January 1, 2018 (Actual)
Primary Completion Date
November 30, 2021 (Actual)
Study Completion Date
March 1, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Blood Diseases Clinic
Collaborators
University of Peshawar

4. Oversight

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
Data Monitoring Committee
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia
Keywords
Thalidomide, Thalassemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
654 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Thalidomide in combination with hydroxyurea
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Thalidomide and Hydroxyurea
Intervention Description
Dosage: Thalidomide 1-4mg/kg/day, Hydroxyurea 10-20mg/kg/day
Primary Outcome Measure Information:
Title
Haemoglobin Level
Description
mg/dL
Time Frame
6-24 months
Title
Response Rate (Excellent, Good, Partial and No response)
Time Frame
6-24 months
Title
Adverse Effects
Time Frame
6-24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Muhammad Tariq M Khan, MBBS, PhD
Organizational Affiliation
Blood Diseases Clinic
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Zahid Ali, PharmD, MPhil
Organizational Affiliation
Department of Pharmacy, University of Peshawar
Official's Role
Study Director
Facility Information:
Facility Name
Blood Diseases Clinic
City
Peshawar
State/Province
Khyber Pakhtukhwa
ZIP/Postal Code
0000
Country
Pakistan

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
28718348
Citation
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.
Results Reference
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PubMed Identifier
29931453
Citation
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.
Results Reference
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PubMed Identifier
20104280
Citation
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. No abstract available.
Results Reference
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PubMed Identifier
26810455
Citation
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.
Results Reference
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PubMed Identifier
18439858
Citation
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. No abstract available.
Results Reference
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PubMed Identifier
28850716
Citation
Chen 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.
Results Reference
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PubMed Identifier
24505535
Citation
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.
Results Reference
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PubMed Identifier
20104272
Citation
Gambari 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.
Results Reference
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Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia

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