Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT06288932 |
| Other study ID # |
NIBD/IRB-263/08-2023 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
Phase 3
|
| First received |
|
| Last updated |
|
| Start date |
February 2024 |
| Est. completion date |
January 2025 |
Study information
| Verified date |
February 2024 |
| Source |
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan |
| Contact |
Rukh-e-Zainub |
| Phone |
03478542981 |
| Email |
rukhezainubnibd[@]gmail.com |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical 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. 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.
Description:
Immune thrombocytopenia Purpura is an acquired immune mediated state characterized by an
extreme reduction in peripheral thrombocyte count <100 x 109/L causing mucocutaneous
bleeding.Our main objective is to analyze and assess the addition of MMF along with steroids
to the first line treatment of ITP resulted in greater response and a lower risk of
refractory and chronic ITP, with decreased financial burden and improved outcome. This
prospective single-center ,Randomized Interventional study is scheduled to take place at NIBD
and BMT Hospital in Karachi. The study has received approval from the institute's ethical
review board. The study will comprise the newly diagnosed patients of Immune thrombocytopenia
purpura aged 5-60years after detailed counseling regarding the study and explaining the
adverse effects of use drug. 104 patients will be included in this trial, with a 1:1 ratio
among the two groups 52 patients will be in each group. Based on the findings of a prior
study, there was a notable difference of 22% in prevalence between the treatment and control
groups. This calculated difference informed the determination of the sample size, which is
set at 104. The study is designed with a statistical power of 80% and a margin of error of
0.05. The sample size calculations were performed using open epi version 3. One group will
receive steroids alone and the other will receive steroids and MMF. Exclusions criteria
includes patients having a history of hepatitis B and C infection, HIV infection, lactating
mothers, pregnancy and allergic to drug. Patients will be assessed for confirmatory clinical
diagnosis for ITP by monitoring parameters comprising of immature platelet fraction (IPF),
platelet count <100x109/L, clinical significant indication to start therapy. The other
possibilities of thrombocytopenia will be ruled out such as autoimmune profile, H-pylori
antigen and viral markers. Patients will be followed up for a period of 12 months. Detailed
history and examination will be done at baseline and during each follow-up visit initially at
2weeks of interval followed by 4weeks. At follow up visit, enquiry will be done regarding
adverse events related to MMF.
Before adding the experimental drug (MMF) biochemical markers will also be assessed at
baseline such as CBC, LFTs, Urea and creatinine.CBC will be repeated monthly and LFTs, urea,
creatinine after every 3months to ensure the drug safety profile. The drug pharmacovigilance
will also be checked by adverse drug events Performa on every follow-up visit. The
prednisolone dose will be given at 2mg/kg with PPI or H2 antagonist. MMF dose will be start
from 500mg twice daily, if the drug does not achieve complete or partial response then
increased to 750mg twice daily after 2 weeks of treatment. If the drug is well tolerated and
does not achieved response then increase the dose to 1gtwice daily after 4weeks after
starting the treatment. The pediatric dose for MMF is 15mg/kg twice daily. Attending
physician will be allowed to reduce the dose if the patient suffered from any adverse event.
The data collection procedure will be Performa based. The relationships between the groups
were assessed using different statistical methods based on the type of data under
investigation. The associations between the groups were measured by the Chi-square for
categorical variables and for quantitative variables ANOVA will be applied for the
determination of significance difference between the treatments. Kaplan-Meier curves will be
applied to measure the proportion of survival. The difference between the treatment groups
will be measured by log-rank test. Cox-regression methods will be applied for the hazard
ratios with different variables. SPSS version 25 and STATA version 15 will be used for the
analysis. The p-value of 0.05 will be considered statistically significant. The purpose of
our study is to ensure the efficacy and pharmacovigilance of mycophenolate mofetil by
introducing it as a first line treatment preference as well as reducing the economical burden
and risk of relapse ITP.
