Immune Thrombocytopenia PurpuraPhase 3NCT06288932
Outcome Study of Conventional Steroids Vs. Steroids Combined with Mycopehnolate in Newly Diagnosed Immune Thrombocytopenia Purpura.
A Phase 3 study testing Mycophenolate Mofetil (MMF) Treatment in people with Immune Thrombocytopenia Purpura, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan at 1 site in Pakistan.
Trial summary
Immune thrombocytopenia Purpura (ITP) is an autoimmune condition delineated by humoral as well as cell mediated immune response against thrombocyte surface proteins GPIIb/IIIa receptors, affecting primary homeostasis leading to mucocutaneous bleeding.ITP is characterized by platelet count <100 x 109/L. Read more about this study
- Who is it for?
- Ages 5 to 60 years
- Conditions the study is for
- Immune Thrombocytopenia Purpura
- What is being tested?
- Mycophenolate Mofetil (MMF) Treatment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 118 participants
- Where
- 1 sitePakistan
- Study dates
- 18 Aug 2023to 18 Aug 2024
- Phase
- Phase 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 5 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: 5-60 years • Number of plates: <30x109
Who cannot join
- Pregnancy; lactation; hepatitis B and C; HIV-reactive individuals; and medication allergies
Trial locations and contacts
1 site in 1 country.
Pakistan1 site
National Institute of Blood Diseases and Bone Marrow Transplantation
Karachi, Sindh, 75300
About this study
Official title: An Outcome Study of Conventional Steroids Vs. Steroids Combined with Mycophenolate for the Management of Newly Diagnosed Immune Thrombocytopenia Purpura: a Cost Effective Measure in Developing Nations.
Immune thrombocytopenia Purpura (ITP) is an autoimmune condition delineated by humoral as well as cell mediated immune response against thrombocyte surface proteins GPIIb/IIIa receptors, affecting primary homeostasis leading to mucocutaneous bleeding.ITP is characterized by platelet count <100 x 109/L. The conventional line of treatment for newly diagnosed ITP is steroids but significant disadvantages have been associated with long term use and a high risk of relapse when reducing the dose. The addition of MMF to the first line treatment of ITP resulted in substantial response and a lower risk of refractory ITP with decreased financial burden and improved outcome.
Read the detailed description
Immune Thrombocytopenia Purpura (ITP) is an acquired, immune-mediated disorder characterized by a severe decrease in peripheral thrombocyte count to less than 100 x 10⁹/L, leading to mucocutaneous bleeding. Our primary objective is to evaluate the efficacy of adding Mycophenolate Mofetil (MMF) alongside steroids in the first-line treatment of ITP, aiming for an enhanced therapeutic response, lower risk of refractory and chronic ITP, reduced financial burden, and improved overall outcomes.
The study aims to validate the efficacy and safety of MMF as a first-line treatment option for ITP while reducing the economic burden and risk of relapse associated with the condition
- Conditions
- Immune Thrombocytopenia Purpura
- Being tested
- Mycophenolate Mofetil (MMF) Treatment
- Keywords
- ITP, MMF
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mycophenolate Mofetil (MMF) TreatmentDrug
Also called Suprimun
The first-line treatment of immune thrombocytopenia purpura with Mycophenolate Mofetil produced a significant response, a lowered likelihood of refractory ITP, a better outcome, and less financial burden.
| Study group | Role | What they receive |
|---|---|---|
| Mycophenolate Mofetil (MMF) Treatment | Experimental | Patients in this arm will receive Mycophenolate Mofetil (MMF) at a starting dose of 500mg twice daily along with Prednisolone.
The pediatric dose of MMF will be calculated by 30mg/kg/day. Dose adjustment will be based on treatment response, with potential increases to 750mg twice daily or 1g twice daily.
MMF will be administered for a specific duration, as determined by the study design. Receives: Drug: Mycophenolate Mofetil (MMF) Treatment |
| Standard Treatment Control | No intervention | Patients in this arm will receive the standard treatment for ITP, which may include corticosteroids (prednisolone) commonly used for ITP management.The treatment regimen will be determined based on current standard guidelines and clinical practice. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet count monitoring
Platelet count improvement, as assessed through regular monitoring and according to response criteria:
The response criteria to the treatment described as follow:
- Complete response: Platelet count ≥ 100 x 10^ 9 /L
- Partial response: Platelet count 30≥ 100 x 10 ^9 /L
- No response: Platelet count 30 x 10^9 /
Measured: assessed after 3 months and 6 months of treatment.
Secondary outcomes (2)
Assessment of Health-Related Quality of Life questionnaire
Health-related quality of life questionnaire
Measured: 12 months
Incidence of adverse events
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2024
- Study start18 Aug 2023
- Primary completion18 Feb 2024
- Study completion18 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombocytopenia PurpuraStudies 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
- The study will comprise the newly diagnosed patients of Immune thrombocytopenia purpura aged 5-60 year. Patients will with a 1:1 ratio among the two group.
- Masking
- None (open label)
- Enrolment
- 118 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Rukh-e- Zainub, Pharm-D,Mphil(enrolled), Principal investigator, National Institute of Blood Diseases and Bone Marrow TransplantationShafaq Abdul Samad, MBBS,FCPS, Study director, National Institute of Blood Diseases and Bone Marrow TransplantationDr.Iyad Naeem, Ph.D, Study chair, KARACHI UNIVERSITY
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06288932
- Sponsor's protocol ID
- NIBD/IRB-263/08-2023
- Status last verified
- Oct 2024
- First submitted
- 16 Feb 2024
- Last updated
- 6 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Outcome Study of Conventional Steroids Vs. Steroids Combined with Mycophenolate for the Management of Newly Diagnosed Immune Thrombocytopenia Purpura: a Cost Effective Measure in Developing Nations.
- ClinicalTrials.gov ID
- NCT06288932
- Acronym / sponsor ID
- NIBD/IRB-263/08-2023
- Conditions
- Immune Thrombocytopenia Purpura
- Keywords
- ITP, MMF
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 118 participants (actual)
- Lead sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), PakistanOther government
- Study start
- 18 Aug 2023 (actual)
- Primary completion
- 18 Feb 2024 (actual)
- Study completion
- 18 Aug 2024 (actual)
- First posted
- 1 Mar 2024 (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.
Immune thrombocytopenia Purpura (ITP) is an autoimmune condition delineated by humoral as well as cell mediated immune response against thrombocyte surface proteins GPIIb/IIIa receptors, affecting primary homeostasis leading to mucocutaneous bleeding.ITP is characterized by platelet count <100 x 109/L. The conventional line of treatment for newly diagnosed ITP is steroids but significant disadvantages have been associated with long term use and a high risk of relapse when reducing the dose. The addition of MMF to the first line treatment of ITP resulted in substantial response and a lower risk of refractory ITP with decreased financial burden and improved outcome.
Detailed description
Immune Thrombocytopenia Purpura (ITP) is an acquired, immune-mediated disorder characterized by a severe decrease in peripheral thrombocyte count to less than 100 x 10⁹/L, leading to mucocutaneous bleeding. Our primary objective is to evaluate the efficacy of adding Mycophenolate Mofetil (MMF) alongside steroids in the first-line treatment of ITP, aiming for an enhanced therapeutic response, lower risk of refractory and chronic ITP, reduced financial burden, and improved overall outcomes.
The study aims to validate the efficacy and safety of MMF as a first-line treatment option for ITP while reducing the economic burden and risk of relapse associated with the condition
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The study will comprise the newly diagnosed patients of Immune thrombocytopenia purpura aged 5-60 year. Patients will with a 1:1 ratio among the two group.
| Arm | Type | Receives |
|---|---|---|
| Mycophenolate Mofetil (MMF) Treatment | Experimental | Drug: Mycophenolate Mofetil (MMF) TreatmentPatients in this arm will receive Mycophenolate Mofetil (MMF) at a starting dose of 500mg twice daily along with Prednisolone. The pediatric dose of MMF will be calculated by 30mg/kg/day. Dose adjustment will be based on treatment response, with potential increases to 750mg twice daily or 1g twice daily. MMF will be administered for a specific duration, as determined by the study design. |
| Standard Treatment Control | No intervention | –Patients in this arm will receive the standard treatment for ITP, which may include corticosteroids (prednisolone) commonly used for ITP management.The treatment regimen will be determined based on current standard guidelines and clinical practice. |
Who may qualify
- Ages
- 5 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Age: 5-60 years • Number of plates: <30x109
Key exclusion criteria (1 listed)
- Pregnancy; lactation; hepatitis B and C; HIV-reactive individuals; and medication allergies
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 |
|---|---|---|
| Mycophenolate Mofetil (MMF) TreatmentAlso: SuprimunThe first-line treatment of immune thrombocytopenia purpura with Mycophenolate Mofetil produced a significant response, a lowered likelihood of refractory ITP, a better outcome, and less financial burden. | Drug | Mycophenolate Mofetil (MMF) Treatment |
Primary outcome
Platelet count monitoring
Time frame: assessed after 3 months and 6 months of treatment.
Platelet count improvement, as assessed through regular monitoring and according to response criteria: The response criteria to the treatment described as follow: 1. Complete response: Platelet count ≥ 100 x 10^ 9 /L 2. Partial response: Platelet count 30≥ 100 x 10 ^9 /L 3. No response: Platelet count 30 x 10^9 /
2 secondary outcomes
Assessment of Health-Related Quality of Life questionnaire
Time frame: 12 months
Incidence of adverse events
Time frame: 12 months
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 6 Nov 2024.
- 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.
Rukh-e- Zainub, Pharm-D,Mphil(enrolled) principal investigator
National Institute of Blood Diseases and Bone Marrow Transplantation
Shafaq Abdul Samad, MBBS,FCPS study director
National Institute of Blood Diseases and Bone Marrow Transplantation
Dr.Iyad Naeem, Ph.D study chair
KARACHI UNIVERSITY
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 Mycophenolate Mofetil (MMF) Treatment in people with Immune Thrombocytopenia Purpura, run by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan 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 1 inclusion and 1 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 6 Nov 2024. 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?
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?
Platelet count monitoring (time frame: assessed after 3 months and 6 months of treatment.). 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 6 Nov 2024.