Thalassemia, BetaPhase 2/3NCT05132270
Clinical Experience of Thalidomide in Thalassemic Patients
A Phase 2/3 study testing hydroxyurea and thalidomide in people with Thalassemia, Beta, run by Children's Hospital Karachi at 1 site in Pakistan.
Trial summary
Objectives Primary objective: To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients. Secondary objective: To determine the change in liver and spleen size of beta-thalassemia patients on the combination therapy A single-arm non-randomized trial to… Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Thalassemia, Beta
- What is being tested?
- hydroxyurea and thalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 135 participants
- Where
- 1 sitePakistan
- Study dates
- 1 Jan 2020to 31 Jan 2021
- Phase
- Phase 2/3
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with clinical and genetic diagnosis of β-thalassemia major and intermedia
- Patients who showed partial response or a decline in response to hydroxyurea
- Patients who are not the candidates for the bone marrow transplant procedure.
Who cannot join
- Married Patients
- Patients with comorbidities such as liver, cerebrovascular, cardiovascular, or kidney diseases
- Patients allergic to the drug ingredients
- Patients with mental disorders
- Patients who are enrolled in other clinical trials
- Patients with a history of venous or arterial thrombosis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy and Safety of Combination of Hydroxyurea and Low-dose Thalidomide on Hemoglobin Synthesis in Thalassemia Patients
Objectives
Primary objective:
Read more
- To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
Secondary objective:
- To determine the change in liver and spleen size of beta-thalassemia patients on the combination therapy
A single-arm non-randomized trial to evaluate the efficacy and safety of combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. It was a twelve months study. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide for another six months. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during and end of the study.
Sample Size and Population This study included 135 Beta-thalassemia patients.
Read the detailed description
The purpose of the study was to evaluate the efficacy and safety of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. The participants were evaluated on the basis of eligibility criteria, consent was obtained and baseline investigations were performed on the screening visit. Participants were continued on Hydroxyurea (10-20mg/kg/day) for the first six months and then for the next six months thalidomide (2-5 mg/kg/day) was added to the intervention. Aspirin was also added (2-4mg/kg/day).
Efficacy:
Maintenance or rise in Hemoglobin (Hb) levels and changes in transfusion frequency before and after the use of combination therapy was used to evaluate the efficacy of the combination therapy. Good responders were the patients who were on transfusion and went off-transfusion after the combination therapy or individuals who were already off transfusion and after combination therapy demonstrated an increase in Hb of at least 1gm/dl. Responders were those who remained on transfusion on combination therapy but displayed a 50% reduction in blood requirements. Whereas, non-responders, were individuals who were off-transfusion before combination therapy and had improvement in Hb of <1gm/dl, or those who remained on transfusion during combination therapy and did not experience transfusion reduction of at least 50%.
Safety:
Safety of the drug was evaluated on the basis of the following parameters and intervention was discontinued or put on hold if:
- Creatinine >1.1mg/dl, Urea >43mg/dl),
- Liver function (SGPT >35mg/L)
- Absolute Neutrophil counts<2*109/L
- Platelets < 100*109/L
- Conditions
- Thalassemia, Beta
- Being tested
- hydroxyurea and thalidomide
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hydroxyurea and thalidomideDrug
Evaluation of hydroxyurea and thalidomide combination use in beta-thalassemia patients
| Study group | Role | What they receive |
|---|---|---|
| Combination of hydroxyurea and thalidomide | Experimental | Hydroxyurea was continued at a dose of 10-20 mg/kg/day for 6 months and then thalidomide was added orally at a dose of 2-5mg/kg/day for 6 months. Receives: Drug: hydroxyurea and thalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in transfusion requirements
Change in transfusion needs (measured as blood volume in ml) per month from baseline
Measured: 6 months on combination therapy
Change in Hemoglobin levels
Change in hemoglobin level of >1g/dl from baseline
Measured: 6 months on combination therapy
Change in the liver function test
Change in SGPT in U/l from baseline
Measured: 6 months on combination therapy
Change in liver function test
Change in total, direct, and indirect bilirubin in mg/dl from baseline
Measured: 6 months on combination therapy
Change in Kidney Function test
Change in urea and creatinine levels in mg/dl from baseline
Measured: 6 months on combination therapy
Secondary outcomes (2)
Change in Spleen size
Change in size of spleen (in centimeters) from baseline
Measured: 6 months on combination therapy
Change in liver size
Change in size of liver (in centimeters) from baseline
Measured: 6 months on combination therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2021
- Study start1 Jan 2020
- Primary completion31 Dec 2020
- Study completion31 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Thalassemia, BetaStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 135 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Karachi
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05132270
- Sponsor's protocol ID
- CH-0420
- Status last verified
- Nov 2021
- First submitted
- 21 Oct 2021
- Last updated
- 24 Nov 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ansari SH, Ansari I, Wasim M, Sattar A, Khawaja S, Zohaib M, Hussain Z, Adil SO, Ansari AH, Ansari UH, Farooq F, Masqati NU. Evaluation of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia. Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.
PubMed 35477175
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Combination of Hydroxyurea and Low-dose Thalidomide on Hemoglobin Synthesis in Thalassemia Patients
- ClinicalTrials.gov ID
- NCT05132270
- Acronym / sponsor ID
- CH-0420
- Conditions
- Thalassemia, Beta
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 135 participants (actual)
- Lead sponsor
- Children's Hospital KarachiUniversity, hospital or other
- Study start
- 1 Jan 2020 (actual)
- Primary completion
- 31 Dec 2020 (actual)
- Study completion
- 31 Jan 2021 (actual)
- First posted
- 24 Nov 2021 (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.
Objectives
Primary objective:
- To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
Secondary objective:
- To determine the change in liver and spleen size of beta-thalassemia patients on the combination therapy
A single-arm non-randomized trial to evaluate the efficacy and safety of combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. It was a twelve months study. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide for another six months. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during and end of the study.
Sample Size and Population This study included 135 Beta-thalassemia patients.
Detailed description
The purpose of the study was to evaluate the efficacy and safety of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. The participants were evaluated on the basis of eligibility criteria, consent was obtained and baseline investigations were performed on the screening visit. Participants were continued on Hydroxyurea (10-20mg/kg/day) for the first six months and then for the next six months thalidomide (2-5 mg/kg/day) was added to the intervention. Aspirin was also added (2-4mg/kg/day).
Efficacy:
Maintenance or rise in Hemoglobin (Hb) levels and changes in transfusion frequency before and after the use of combination therapy was used to evaluate the efficacy of the combination therapy. Good responders were the patients who were on transfusion and went off-transfusion after the combination therapy or individuals who were already off transfusion and after combination therapy demonstrated an increase in Hb of at least 1gm/dl. Responders were those who remained on transfusion on combination therapy but displayed a 50% reduction in blood requirements. Whereas, non-responders, were individuals who were off-transfusion before combination therapy and had improvement in Hb of <1gm/dl, or those who remained on transfusion during combination therapy and did not experience transfusion reduction of at least 50%.
Safety:
Safety of the drug was evaluated on the basis of the following parameters and intervention was discontinued or put on hold if:
- Creatinine >1.1mg/dl, Urea >43mg/dl),
- Liver function (SGPT >35mg/L)
- Absolute Neutrophil counts<2*109/L
- Platelets < 100*109/L
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Combination of hydroxyurea and thalidomide | Experimental | Drug: hydroxyurea and thalidomideHydroxyurea was continued at a dose of 10-20 mg/kg/day for 6 months and then thalidomide was added orally at a dose of 2-5mg/kg/day for 6 months. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients with clinical and genetic diagnosis of β-thalassemia major and intermedia
- Patients who showed partial response or a decline in response to hydroxyurea
- Patients who are not the candidates for the bone marrow transplant procedure.
Key exclusion criteria (6 listed)
- Married Patients
- Patients with comorbidities such as liver, cerebrovascular, cardiovascular, or kidney diseases
- Patients allergic to the drug ingredients
- Patients with mental disorders
- Patients who are enrolled in other clinical trials
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 |
|---|---|---|
| hydroxyurea and thalidomideEvaluation of hydroxyurea and thalidomide combination use in beta-thalassemia patients | Drug | Combination of hydroxyurea and thalidomide |
Primary outcome
Change in transfusion requirements
Time frame: 6 months on combination therapy
Change in transfusion needs (measured as blood volume in ml) per month from baseline
Change in Hemoglobin levels
Time frame: 6 months on combination therapy
Change in hemoglobin level of >1g/dl from baseline
Change in the liver function test
Time frame: 6 months on combination therapy
Change in SGPT in U/l from baseline
Change in liver function test
Time frame: 6 months on combination therapy
Change in total, direct, and indirect bilirubin in mg/dl from baseline
Change in Kidney Function test
Time frame: 6 months on combination therapy
Change in urea and creatinine levels in mg/dl from baseline
2 secondary outcomes
Change in Spleen size
Time frame: 6 months on combination therapy
Change in liver size
Time frame: 6 months on combination therapy
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 24 Nov 2021.
- Derived from this study: PubMed 35477175Ansari SH, Ansari I, Wasim M, Sattar A, Khawaja S, Zohaib M, Hussain Z, Adil SO, Ansari AH, Ansari UH, Farooq F, Masqati NU. Evaluation of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia. Blood Adv. 2022 Dec 27;6(24):6162-6168. doi: 10.1182/bloodadvances.2022007031.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing hydroxyurea and thalidomide in people with Thalassemia, Beta, run by Children's Hospital Karachi 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 3 inclusion and 6 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 24 Nov 2021. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
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?
Change in transfusion requirements (time frame: 6 months on combination therapy). The study lists 5 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 NCT05132270, which also lists 1 linked publication.
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 24 Nov 2021.