Beta-ThalassemiaPhase 3NCT07884773
Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia
A Phase 3 study testing Thalidomide, Danazol and Prednisolone in people with Beta-Thalassemia and Thalassemia, run by Khyber Medical University Peshawar at 1 site in Pakistan.
Trial summary
This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone. The other group will receive thalidomide plus danazol and prednisolone. Read more about this study
- Who is it for?
- Ages 5 to 15 years
- Conditions the study is for
- Beta-Thalassemia, Thalassemia
- What is being tested?
- Thalidomide, Danazol and Prednisolone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 172 participants
- Where
- 1 sitePakistan
- Study dates
- 12 Oct 2026to 20 May 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 5 to 15 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 5 to 15 years with confirmed transfusion-dependent beta-thalassemia major.
- Documented non-response to adequately dosed thalidomide therapy for at least 6 months.
- Pre-transfusion hemoglobin less than 9.0 g/dL with continued transfusion dependence (more than 2 units per month).
- Adequate baseline clinical and laboratory assessment for safe study treatment.
- Stable transfusion and iron-chelation regimen according to the protocol.
- Written informed consent from parent/legal guardian and age-appropriate assent from the participant, with willingness to comply with study procedures and follow-up.
Who cannot join
- Previous allogeneic hematopoietic stem cell or bone marrow transplantation.
- Diabetes mellitus or clinically significant uncontrolled metabolic disease.
- Active severe infection or uncontrolled hepatic, renal, or cardiovascular disease.
- Significant pre-existing peripheral neuropathy or history of clinically significant thromboembolic disease.
- Known hypersensitivity to any study medication.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Pakistan1 site, 1 recruiting
- Recruiting
Pak International Medical College
Peshawar, Khyber Pakhtunkhwa, 25000
Syeda Nayab Imtiaz, MBBS, +923369966014, dr.nayabimtiaz@gmail.com
Tariq Masood, MBBS, +923005888867, drtariqmsd@gmail.com
About this study
Official title: Safety and Efficacy of Combination Therapy (Danazol + Prednisolone + Thalidomide) in Thalidomide-Refractory Transfusion-Dependent β-Thalassemia Patients: A Prospective Randomized Controlled Clinical Trial
This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone. The other group will receive thalidomide plus danazol and prednisolone. The study will check whether the combination treatment reduces the need for blood transfusions, raises hemoglobin levels, and is safe. About 172 children aged 5 to 15 will be assigned by chance to one of the two groups. They will be followed for 9 to 12 months.
Read the detailed description
This is a two-arm, parallel-group, open-label, randomized controlled superiority trial. It will enroll 172 participants aged 5 to 15 years with transfusion-dependent beta-thalassemia major who have had an inadequate response to at least 6 months of adequately dosed thalidomide. Participants will be randomly assigned 1:1 to thalidomide monotherapy or thalidomide plus danazol plus prednisolone. The primary outcomes are transfusion independence, defined as no red blood cell transfusion for at least 8 consecutive weeks with hemoglobin at least 9.0 g/dL; mean change in pre-transfusion hemoglobin from baseline to 6 months; and safety and tolerability graded by CTCAE v5.0. Secondary outcomes include transfusion burden, hematological improvement, health-related quality of life using TranQOL, and exploratory associations of HBB mutation class and HbF-modifier genotypes (XmnI, BCL11A, HBS1L-MYB) with treatment response. Follow-up will be 9 to 12 months. The trial will be conducted at the Blood Care Clinic, Pak International Medical College, and Peshawar General Hospital, Hayatabad, Phase 5, Peshawar, Pakistan. The trial will be registered before the first participant is enrolled.
- Conditions
- Beta-Thalassemia, Thalassemia
- Being tested
- Thalidomide, Danazol, Prednisolone
- Keywords
- Beta-thalassemia, Transfusion-dependent, Thalidomide-refractory, Combination therapy, Randomized controlled trial
- Medical subject headings
- beta-Thalassemia, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThalidomideDrug
Also called Thalomid, alpha-(N-phthalimido)glutarimide
Initial oral capsule dose is 1 to 5 mg/kg taken once daily at night, with adjustments made every 2 to 4 weeks based on response and tolerance. The treatment is administered in both arms for a duration of 9 to 12 months.
DanazolDrug
Also called Danocrine
Oral capsule of 100 mg taken three times daily, continuously for 30 days per cycle. This medication is administered only in the combination arm.
PrednisoloneDrug
Also called Pediapred, Orapred
Oral tablet of 5 mg twice daily for 10 days each month, only administered in the combination arm.
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide plus Danazol plus Prednisolone | Experimental | In this treatment regimen, Thalidomide and Danazol are administered at a dosage of 100 mg orally three times daily for 30 days each cycle. Prednisolone is given at a dosage of 5 mg orally twice daily for 10 days each month. Supportive care includes Famotidine, folic acid, and vitamin B12. This treatment plan is continued for a duration of 9 to 12 months. Receives: Drug: Thalidomide; Drug: Danazol; Drug: Prednisolone |
| Thalidomide Monotherapy | Active comparator | Thalidomide should be taken orally at a dose of 1 to 5 mg/kg at night, with dosage titrated upward every 2 to 4 weeks based on response and tolerance. Clopidogrel and aspirin should be taken together for thromboprophylaxis. This regimen should continue for 9 to 12 months. Receives: Drug: Thalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Transfusion independence
Proportion of participants who remain free from red blood cell transfusion for at least 8 consecutive weeks while maintaining hemoglobin greater than or equal to 9.0 g/dL.
Measured: Assessed continuously from randomization through final study visit (Month 9 to 12).
Hemoglobin response
Mean change in pre-transfusion hemoglobin (g/dL) from baseline to 6 months.
Measured: Baseline and Month 6.
Safety and tolerability
Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0.
Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0.
Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0.
Measured: Assessed at every scheduled visit (Months 1, 3, 6, 9, and final visit).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2026
- Study start12 Oct 2026
- Primary completion12 Apr 2027 (estimated)
- Study completion20 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (12)Papers the sponsor has linked to this study.
- Other trials for Beta-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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Two-arm, parallel-group, randomized controlled superiority trial. Participants are allocated 1:1 to thalidomide monotherapy or thalidomide plus danazol plus prednisolone. Open-label.
- Masking
- None (open label)
- Enrolment
- 172 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Khyber Medical University Peshawar
- Sponsor type
- University, hospital or other
- Collaborators
- Pak International Medical College and Hospital, Peshawar, Peshawar General Hospital
- Responsible party
- Sponsor
- Investigators
- Syeda Nayab Imtiaz, MBBS, Principal investigator, Institute of Pathology and Diagnostic Medicine, Khyber Medical University, PeshawarGulab Fatima Rani, MBBS, PhD, Study chair, Institute of Pathology and Diagnostic Medicine, Khyber Medical University, PeshawarYasar Mehmood Yousafzai, PhD, Study director, Institute of Pathology and Diagnostic Medicine, Khyber Medical University, Peshawar
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07884773
- Sponsor's protocol ID
- KMU/DIR/CTU/2026/14
- Status last verified
- Oct 2026
- First submitted
- 13 Oct 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual participant data (IPD), the full trial protocol, statistical analysis plan, informed consent form, and analytic code will be made available to other researchers upon reasonable request after publication of the primary results. Data sharing will comply with KMU data-sharing policy and patient confidentiality requirements.
Publications
Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ. 2008 Jun;86(6):480-7. doi: 10.2471/blt.06.036673.
PubMed 18568278Weatherall DJ, Clegg JB. Inherited haemoglobin disorders: an increasing global health problem. Bull World Health Organ. 2001;79(8):704-12. Epub 2001 Oct 24.
PubMed 11545326Montoto LG, Arregui L, Sanchez NM, Gomendio M, Roldan ER. Postnatal testicular development in mouse species with different levels of sperm competition. Reproduction. 2012 Mar;143(3):333-46. doi: 10.1530/REP-11-0245. Epub 2011 Dec 20.
PubMed 22187670Safdari KM, Converse C, Dong F, MacDougall NA, Hyer K, Runyon A, Ahlering H, Comunale ME. Hemodynamic Effects of Methamphetamine and General Anesthesia. Anesthesiol Res Pract. 2022 Feb 17;2022:7542311. doi: 10.1155/2022/7542311. eCollection 2022.
PubMed 35222639Ouzaid I, Kammerer-Jacquet SF, Khene Z, Ravaud A, Patard JJ, Bensalah K, Rioux-Leclercq N. Exploring Biological Predictive Factors of Progression After Surgery in High-Risk Renal Cell Carcinoma: Results From the French Cohort of the Randomized S-TRAC Trial Patients. Front Surg. 2020 Jun 5;7:26. doi: 10.3389/fsurg.2020.00026. eCollection 2020.
PubMed 32582758Bakathir AA, Moos KF, Ayoub AF, Bagg J. Factors Contributing to the Spread of Odontogenic Infections: A prospective pilot study. Sultan Qaboos Univ Med J. 2009 Dec;9(3):296-304. Epub 2009 Dec 19.
PubMed 21509313O'Callaghan J. Russian Moon lander crash - what happened, and what's next? Nature. 2023 Aug 21. doi: 10.1038/d41586-023-02659-6. Online ahead of print. No abstract available.
PubMed 37604864Hou X, Zhang P, Du H, Chu W, Sun R, Qin S, Tian Y, Zhang Z, Xu F. Akkermansia Muciniphila Potentiates the Antitumor Efficacy of FOLFOX in Colon Cancer. Front Pharmacol. 2021 Sep 17;12:725583. doi: 10.3389/fphar.2021.725583. eCollection 2021.
PubMed 34603035Zywiel M, Ellis K, Veillette CJH, Skou ST, McGlasson R. Implementation of the Good Life with osteoArthritis in Denmark (GLA:D) Program across Canada for the Management of Hip and Knee Osteoarthritis. Healthc Q. 2021 Apr;24(1):54-59. doi: 10.12927/hcq.2021.26464.
PubMed 33864442Gelber PE, Drager J, Maheshwer B, Leyes M, Barenius B, Robinson J, Pujol N, Tischer T, Margheritini F, Fritsch B, Frosh KH, Chahla J. Large variability exists in the management of posterolateral corner injuries in the global surgical community. Knee Surg Sports Traumatol Arthrosc. 2020 Jul;28(7):2116-2123. doi: 10.1007/s00167-020-05922-9. Epub 2020 Apr 1.
PubMed 32239270Courpied JP, Caton JH. Total Hip Arthroplasty, state of the art for the 21st century. Int Orthop. 2011 Feb;35(2):149-50. doi: 10.1007/s00264-011-1207-9. Epub 2011 Jan 18. No abstract available.
PubMed 21243356Nahar R, Zhai W, Zhang T, Takano A, Khng AJ, Lee YY, Liu X, Lim CH, Koh TPT, Aung ZW, Lim TKH, Veeravalli L, Yuan J, Teo ASM, Chan CX, Poh HM, Chua IML, Liew AA, Lau DPX, Kwang XL, Toh CK, Lim WT, Lim B, Tam WL, Tan EH, Hillmer AM, Tan DSW. Elucidating the genomic architecture of Asian EGFR-mutant lung adenocarcinoma through multi-region exome sequencing. Nat Commun. 2018 Jan 15;9(1):216. doi: 10.1038/s41467-017-02584-z.
PubMed 29335443
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Combination Therapy (Danazol + Prednisolone + Thalidomide) in Thalidomide-Refractory Transfusion-Dependent β-Thalassemia Patients: A Prospective Randomized Controlled Clinical Trial
- ClinicalTrials.gov ID
- NCT07884773
- Acronym / sponsor ID
- KMU/DIR/CTU/2026/14
- Conditions
- Beta-Thalassemia, Thalassemia
- Keywords
- Beta-thalassemia, Transfusion-dependent, Thalidomide-refractory, Combination therapy, Randomized controlled trial
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 172 participants (estimated)
- Lead sponsor
- Khyber Medical University PeshawarUniversity, hospital or other
- Collaborators
- Pak International Medical College and Hospital, Peshawar, Peshawar General Hospital
- Study start
- 12 Oct 2026 (actual)
- Primary completion
- 12 Apr 2027 (estimated)
- Study completion
- 20 May 2027 (estimated)
- First posted
- 21 Oct 2026 (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.
This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone. The other group will receive thalidomide plus danazol and prednisolone. The study will check whether the combination treatment reduces the need for blood transfusions, raises hemoglobin levels, and is safe. About 172 children aged 5 to 15 will be assigned by chance to one of the two groups. They will be followed for 9 to 12 months.
Detailed description
This is a two-arm, parallel-group, open-label, randomized controlled superiority trial. It will enroll 172 participants aged 5 to 15 years with transfusion-dependent beta-thalassemia major who have had an inadequate response to at least 6 months of adequately dosed thalidomide. Participants will be randomly assigned 1:1 to thalidomide monotherapy or thalidomide plus danazol plus prednisolone. The primary outcomes are transfusion independence, defined as no red blood cell transfusion for at least 8 consecutive weeks with hemoglobin at least 9.0 g/dL; mean change in pre-transfusion hemoglobin from baseline to 6 months; and safety and tolerability graded by CTCAE v5.0. Secondary outcomes include transfusion burden, hematological improvement, health-related quality of life using TranQOL, and exploratory associations of HBB mutation class and HbF-modifier genotypes (XmnI, BCL11A, HBS1L-MYB) with treatment response. Follow-up will be 9 to 12 months. The trial will be conducted at the Blood Care Clinic, Pak International Medical College, and Peshawar General Hospital, Hayatabad, Phase 5, Peshawar, Pakistan. The trial will be registered before the first participant is enrolled.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Two-arm, parallel-group, randomized controlled superiority trial. Participants are allocated 1:1 to thalidomide monotherapy or thalidomide plus danazol plus prednisolone. Open-label.
| Arm | Type | Receives |
|---|---|---|
| Thalidomide plus Danazol plus Prednisolone | Experimental | Drug: Thalidomide, Drug: Danazol, Drug: PrednisoloneIn this treatment regimen, Thalidomide and Danazol are administered at a dosage of 100 mg orally three times daily for 30 days each cycle. Prednisolone is given at a dosage of 5 mg orally twice daily for 10 days each month. Supportive care includes Famotidine, folic acid, and vitamin B12. This treatment plan is continued for a duration of 9 to 12 months. |
| Thalidomide Monotherapy | Active comparator | Drug: ThalidomideThalidomide should be taken orally at a dose of 1 to 5 mg/kg at night, with dosage titrated upward every 2 to 4 weeks based on response and tolerance. Clopidogrel and aspirin should be taken together for thromboprophylaxis. This regimen should continue for 9 to 12 months. |
Who may qualify
- Ages
- 5 to 15 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Age 5 to 15 years with confirmed transfusion-dependent beta-thalassemia major.
- Documented non-response to adequately dosed thalidomide therapy for at least 6 months.
- Pre-transfusion hemoglobin less than 9.0 g/dL with continued transfusion dependence (more than 2 units per month).
- Adequate baseline clinical and laboratory assessment for safe study treatment.
- Stable transfusion and iron-chelation regimen according to the protocol.
Key exclusion criteria (5 listed)
- Previous allogeneic hematopoietic stem cell or bone marrow transplantation.
- Diabetes mellitus or clinically significant uncontrolled metabolic disease.
- Active severe infection or uncontrolled hepatic, renal, or cardiovascular disease.
- Significant pre-existing peripheral neuropathy or history of clinically significant thromboembolic disease.
- Known hypersensitivity to any study medication.
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 |
|---|---|---|
| ThalidomideAlso: Thalomid, alpha-(N-phthalimido)glutarimideInitial oral capsule dose is 1 to 5 mg/kg taken once daily at night, with adjustments made every 2 to 4 weeks based on response and tolerance. The treatment is administered in both arms for a duration of 9 to 12 months. | Drug | Thalidomide Monotherapy, Thalidomide plus Danazol plus Prednisolone |
| DanazolAlso: DanocrineOral capsule of 100 mg taken three times daily, continuously for 30 days per cycle. This medication is administered only in the combination arm. | Drug | Thalidomide plus Danazol plus Prednisolone |
| PrednisoloneAlso: Pediapred, OrapredOral tablet of 5 mg twice daily for 10 days each month, only administered in the combination arm. | Drug | Thalidomide plus Danazol plus Prednisolone |
Primary outcome
Transfusion independence
Time frame: Assessed continuously from randomization through final study visit (Month 9 to 12).
Proportion of participants who remain free from red blood cell transfusion for at least 8 consecutive weeks while maintaining hemoglobin greater than or equal to 9.0 g/dL.
Hemoglobin response
Time frame: Baseline and Month 6.
Mean change in pre-transfusion hemoglobin (g/dL) from baseline to 6 months.
Safety and tolerability
Time frame: Assessed at every scheduled visit (Months 1, 3, 6, 9, and final visit).
Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0. Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0. Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2026.
- Background: PubMed 18568278Modell B, Darlison M. Global epidemiology of haemoglobin disorders and derived service indicators. Bull World Health Organ. 2008 Jun;86(6):480-7. doi: 10.2471/blt.06.036673.
- Background: PubMed 11545326Weatherall DJ, Clegg JB. Inherited haemoglobin disorders: an increasing global health problem. Bull World Health Organ. 2001;79(8):704-12. Epub 2001 Oct 24.
- Background: PubMed 22187670Montoto LG, Arregui L, Sanchez NM, Gomendio M, Roldan ER. Postnatal testicular development in mouse species with different levels of sperm competition. Reproduction. 2012 Mar;143(3):333-46. doi: 10.1530/REP-11-0245. Epub 2011 Dec 20.
- Background: PubMed 35222639Safdari KM, Converse C, Dong F, MacDougall NA, Hyer K, Runyon A, Ahlering H, Comunale ME. Hemodynamic Effects of Methamphetamine and General Anesthesia. Anesthesiol Res Pract. 2022 Feb 17;2022:7542311. doi: 10.1155/2022/7542311. eCollection 2022.
- Background: PubMed 32582758Ouzaid I, Kammerer-Jacquet SF, Khene Z, Ravaud A, Patard JJ, Bensalah K, Rioux-Leclercq N. Exploring Biological Predictive Factors of Progression After Surgery in High-Risk Renal Cell Carcinoma: Results From the French Cohort of the Randomized S-TRAC Trial Patients. Front Surg. 2020 Jun 5;7:26. doi: 10.3389/fsurg.2020.00026. eCollection 2020.
- Background: PubMed 21509313Bakathir AA, Moos KF, Ayoub AF, Bagg J. Factors Contributing to the Spread of Odontogenic Infections: A prospective pilot study. Sultan Qaboos Univ Med J. 2009 Dec;9(3):296-304. Epub 2009 Dec 19.
- Background: PubMed 37604864O'Callaghan J. Russian Moon lander crash - what happened, and what's next? Nature. 2023 Aug 21. doi: 10.1038/d41586-023-02659-6. Online ahead of print. No abstract available.
- Background: PubMed 34603035Hou X, Zhang P, Du H, Chu W, Sun R, Qin S, Tian Y, Zhang Z, Xu F. Akkermansia Muciniphila Potentiates the Antitumor Efficacy of FOLFOX in Colon Cancer. Front Pharmacol. 2021 Sep 17;12:725583. doi: 10.3389/fphar.2021.725583. eCollection 2021.
- Background: PubMed 33864442Zywiel M, Ellis K, Veillette CJH, Skou ST, McGlasson R. Implementation of the Good Life with osteoArthritis in Denmark (GLA:D) Program across Canada for the Management of Hip and Knee Osteoarthritis. Healthc Q. 2021 Apr;24(1):54-59. doi: 10.12927/hcq.2021.26464.
- Background: PubMed 32239270Gelber PE, Drager J, Maheshwer B, Leyes M, Barenius B, Robinson J, Pujol N, Tischer T, Margheritini F, Fritsch B, Frosh KH, Chahla J. Large variability exists in the management of posterolateral corner injuries in the global surgical community. Knee Surg Sports Traumatol Arthrosc. 2020 Jul;28(7):2116-2123. doi: 10.1007/s00167-020-05922-9. Epub 2020 Apr 1.
- Background: PubMed 21243356Courpied JP, Caton JH. Total Hip Arthroplasty, state of the art for the 21st century. Int Orthop. 2011 Feb;35(2):149-50. doi: 10.1007/s00264-011-1207-9. Epub 2011 Jan 18. No abstract available.
- Background: PubMed 29335443Nahar R, Zhai W, Zhang T, Takano A, Khng AJ, Lee YY, Liu X, Lim CH, Koh TPT, Aung ZW, Lim TKH, Veeravalli L, Yuan J, Teo ASM, Chan CX, Poh HM, Chua IML, Liew AA, Lau DPX, Kwang XL, Toh CK, Lim WT, Lim B, Tam WL, Tan EH, Hillmer AM, Tan DSW. Elucidating the genomic architecture of Asian EGFR-mutant lung adenocarcinoma through multi-region exome sequencing. Nat Commun. 2018 Jan 15;9(1):216. doi: 10.1038/s41467-017-02584-z.
- Individual participant dataSharing planned: Yes. De-identified individual participant data (IPD), the full trial protocol, statistical analysis plan, informed consent form, and analytic code will be made available to other researchers upon reasonable request after publication of the primary results. Data sharing will comply with KMU data-sharing policy and patient confidentiality requirements.
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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.
Syeda Nayab Imtiaz, MBBS principal investigator
Institute of Pathology and Diagnostic Medicine, Khyber Medical University, Peshawar
Gulab Fatima Rani, MBBS, PhD study chair
Institute of Pathology and Diagnostic Medicine, Khyber Medical University, Peshawar
Yasar Mehmood Yousafzai, PhD study director
Institute of Pathology and Diagnostic Medicine, Khyber Medical University, Peshawar
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Thalidomide, Danazol and Prednisolone in people with Beta-Thalassemia and Thalassemia, run by Khyber Medical University Peshawar 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 6 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 21 Oct 2026. 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 phase mean?
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Syeda Nayab Imtiaz, MBBS (+923369966014, dr.nayabimtiaz@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Transfusion independence (time frame: Assessed continuously from randomization through final study visit (Month 9 to 12).). 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 NCT07884773, which also lists 12 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 21 Oct 2026.