ThalassemiaNCT06888232
Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients
A study testing Thalidomide in people with Thalassemia, run by Muhammad Aamir Latif at 1 site in Pakistan.
Trial summary
This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children. Read more about this study
- Who is it for?
- Ages 2 to 5 years
- Conditions the study is for
- Thalassemia
- What is being tested?
- Thalidomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 121 participants
- Where
- 1 sitePakistan
- Study dates
- 1 Aug 2023to 31 Dec 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 2 to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children of both genders
- Aged 2-5 years
- With transfusion-dependent thalassemia (TDT)
- Whose parents/guardians showed willingness to adhere to follow-up visits
Who cannot join
- Children with uncontrolled infections
- Significant hepatic or renal dysfunction
- Malignancy
- Known contraindications to thalidomide
Trial locations and contacts
1 site in 1 country.
Pakistan1 site
The Children's Hospital & Institute of Child Health
Multan, Punjab Province, 66000
About this study
Official title: Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients: A Single Center Experience From South Punjab, Pakistan
This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.
Read the detailed description
Given the significant burden of transfusion dependency, an effective and safe inducer is needed that could improve quality of life and reduce the healthcare costs associated with frequent transfusions and iron chelation therapy. If this study could establish the safety of thalidomide in terms of teratogenicity along with its efficacy, it would be a great support to multi-transfused thalassemic children.
- Conditions
- Thalassemia
- Being tested
- Thalidomide
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThalidomideDrug
Thalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols.
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide group | Experimental | Thalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols. Receives: Drug: Thalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of treatment
Efficacy was categorized as excellent response showing an increase in hemoglobin by 1-2 g/dL or freedom from blood transfusions, partial response as an increase in hemoglobin <1 g/dL or a decrease in transfusion frequency, or no response if there was no increase in hemoglobin or an increase in transfusion requirement within three months.
Measured: 6 months
Safety of treatment
Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2025
- Study start1 Aug 2023
- Primary completion31 Dec 2024
- Study completion31 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 121 participants (actual)
Sponsor and investigators
- Lead sponsor
- Muhammad Aamir Latif
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Muhammad Aamir Latif, RESnTEC, Institute of Research
- Investigators
- Muhammad Aslam, FCPS, Principal investigator, Children's Hospital and institute of Child Health Multan, Punjab, PakistanSara Rubab, FCPS, Principal investigator, Children's Hospital and institute of Child Health Multan, Punjab, Pakistan
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06888232
- Sponsor's protocol ID
- DRASLAMCHMULTAN
- Status last verified
- Mar 2025
- First submitted
- 15 Mar 2025
- Last updated
- 21 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Data can be shared on a reasonable request.
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients: A Single Center Experience From South Punjab, Pakistan
- ClinicalTrials.gov ID
- NCT06888232
- Acronym / sponsor ID
- DRASLAMCHMULTAN
- Conditions
- Thalassemia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 121 participants (actual)
- Lead sponsor
- Muhammad Aamir LatifUniversity, hospital or other
- Study start
- 1 Aug 2023 (actual)
- Primary completion
- 31 Dec 2024 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 21 Mar 2025 (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 aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.
Detailed description
Given the significant burden of transfusion dependency, an effective and safe inducer is needed that could improve quality of life and reduce the healthcare costs associated with frequent transfusions and iron chelation therapy. If this study could establish the safety of thalidomide in terms of teratogenicity along with its efficacy, it would be a great support to multi-transfused thalassemic children.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thalidomide group | Experimental | Drug: ThalidomideThalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols. |
Who may qualify
- Ages
- 2 to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Children of both genders
- Aged 2-5 years
- With transfusion-dependent thalassemia (TDT)
- Whose parents/guardians showed willingness to adhere to follow-up visits
Key exclusion criteria (4 listed)
- Children with uncontrolled infections
- Significant hepatic or renal dysfunction
- Malignancy
- Known contraindications to thalidomide
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 |
|---|---|---|
| ThalidomideThalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols. | Drug | Thalidomide group |
Primary outcome
Efficacy of treatment
Time frame: 6 months
Efficacy was categorized as excellent response showing an increase in hemoglobin by 1-2 g/dL or freedom from blood transfusions, partial response as an increase in hemoglobin <1 g/dL or a decrease in transfusion frequency, or no response if there was no increase in hemoglobin or an increase in transfusion requirement within three months.
Safety of treatment
Time frame: 6 months
Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2025.
- Individual participant dataSharing planned: No. Data can be shared on a reasonable request.
Related trials for Thalassemia
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Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
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. This study is not recruiting at the moment.
Muhammad Aslam, FCPS principal investigator
Children's Hospital and institute of Child Health Multan, Punjab, Pakistan
Sara Rubab, FCPS principal investigator
Children's Hospital and institute of Child Health Multan, Punjab, Pakistan
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Thalidomide in people with Thalassemia, run by Muhammad Aamir Latif 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 4 inclusion and 4 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: finished, as of the sponsor's last update on 21 Mar 2025. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of treatment (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Mar 2025.