Clinical trial NCT06288932: Outcome Study of Conventional Steroids Vs. Steroids Combined with Mycopehnolate in Newly Diagnosed Immune Thrombocytopenia Purpura.
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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Immune Thrombocytopenia Purpura
Keywords: ITP
MMF
Lead sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sponsor class: OTHER_GOV
First posted: 2024-03-01
Enrollment: 118
Sex: ALL
Minimum age (years): 5
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention 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.
Primary purpose: TREATMENT

Brief 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.

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

Eligibility criteria: Inclusion Requirements:

• Age: 5-60 years • Number of plates: \<30x109

Exclusion Standards:

Pregnancy; lactation; hepatitis B and C; HIV-reactive individuals; and medication allergies

Primary outcomes:
measure: Platelet count monitoring
timeFrame: assessed after 3 months and 6 months of treatment.
Secondary outcomes:
measure: Assessment of Health-Related Quality of Life questionnaire
timeFrame: 12 months
measure: Incidence of adverse events
timeFrame: 12 months
Other outcomes:
Locations:
city: Karachi
country: Pakistan
facility: National Institute of Blood Diseases and Bone Marrow Transplantation
state: Sindh
Study officials:
affiliation: National Institute of Blood Diseases and Bone Marrow Transplantation
name: Rukh-e- Zainub, Pharm-D,Mphil(enrolled)
role: PRINCIPAL_INVESTIGATOR
affiliation: National Institute of Blood Diseases and Bone Marrow Transplantation
name: Shafaq Abdul Samad, MBBS,FCPS
role: STUDY_DIRECTOR
affiliation: KARACHI UNIVERSITY
name: Dr.Iyad Naeem, Ph.D
role: STUDY_CHAIR
References: