High dose Rituximab for newly diagnosed acute immune thrombocytopenic purpura
The Use of Dose Dense Rituximab for High Risk Patients With Newly Diagnosed Acute Immune Thrombocytopenic Purpura
This study is testing if a higher dose of Rituximab can help people with newly diagnosed acute immune thrombocytopenic purpura feel better and reduce the chances of their condition coming back.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 1 Year to 21 Years |
| Sex | All |
| Sponsor | New York Medical College Academic / other |
| Drugs / interventions | rituximab, prednisone |
| Locations | 1 site (Vahalla, New York) |
| Trial ID | NCT04323748 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the effectiveness of a dose dense administration of Rituximab in treating patients with newly diagnosed acute immune thrombocytopenic purpura (ITP). It will assess the relapse rate following this treatment and include correlative studies as outlined in the protocol. Additionally, the quality of life of participants will be measured using the KIT assessment tool. The study focuses on high-risk patients to determine if this approach can improve outcomes.
Who should consider this trial
Good fit: Ideal candidates are children and young adults aged 1 to 21 years with newly diagnosed ITP and specific high-risk features.
Not a fit: Patients who have received prior treatment for ITP or those with a platelet count above the specified threshold may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce relapse rates in young patients with high-risk ITP.
How similar studies have performed: While this approach is novel in the context of high-risk ITP, similar studies with Rituximab in other conditions have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age: Subjects must be ≥ 1 year and ≤ 21 years of age. * Diagnosis: Patients must have newly diagnosed ITP and a platelet count of ≤ 20 x 109 per Liter. Bone marrow aspirate and biopsy should be performed to rule out malignancy in the bone marrow. * High-risk features : In addition, patients must have one of more of the following high-risk criteria: * Age ≥ 10 years * Grade II-IV bleeding at diagnosis * ANA positivity * No history of preceding infection within 2 weeks prior to ITP diagnosis * Performance Status: Patients must have a performance status ≥ 50%. Use Karnofsky for patients \> 16 years of age and Lansky for patients less than or equal to 16 years of age. See Appendix I for performance score. * Prior Therapy * Patients may not have received any treatment for ITP prior to start of therapy. * Patients may not receive systemic steroids ≥ 0.5 mg/kg prednisone (or equivalent) within 2 weeks prior to diagnosis. * Concomitant Medications Restrictions: * Steroids are only warranted as premedication prior to rituximab. * Patients who receive thrombopoetic agonists, eltrombopag or romiplostim will be taken off protocol. * Organ Function Requirements * Adequate Renal Function Defined As: estimated CrCl \> 60 mL/min or \>30% of GFR for age based on the Schwartz formula * Adequate Liver Function Defined As: AST and/or ALT less than 5 times the upper limit of normal, and/or direct bilirubin less than the 2 times of the upper limit of normal Exclusion Criteria * Patients with a history of Grade III-IV allergic reaction to rituximab * Patients with bone marrow neoplastic infiltration * Patients with a history of hepatitis B infection * Pregnancy and Breast Feeding * Female patients who are pregnant are ineligible (insert the reason: "due to risks of fetal and teratogenic adverse events as seen in animal/human studies" or "since there is yet no available information regarding human fetal or teratogenic toxicities"). * Lactating females are not eligible unless they have agreed not to breastfeed their infants. * Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained.
Where this trial is running
Vahalla, New York
- New York Medical College — Vahalla, New York, United States (Recruiting)
Study contacts
- Principal investigator: Edo Scahefer, MD — New York Medical College
- Study coordinator: Erin Morris, RN
- Email: erin_morris@nymc.edu
- Phone: 714-964-5359
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.