Dose Dense Rituximab for High Risk Newly Diagnosed Acute Immune Thrombocytopenic Purpura (NYMC207)

The Use of Dose Dense Rituximab for High Risk Patients With Newly Diagnosed Acute Immune Thrombocytopenic Purpura

The purpose of this study is to determine if a dose dense administration of Rituximab in newly diagnosed acute immune thrombocytopenic purpura (ITP) and determine relapse rate following this treatment.

Correlative studies will be performed as outlined in the appendices.

Quality of Life will be measured using the KIT as outlined in the protocol.

Study Overview

• Status: Recruiting
• Conditions: Immune Thrombocytopenic Purpura
• Intervention / Treatment: Drug: rituxan

• Study Type: Interventional
• Enrollment (Estimated): 20
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Erin Morris, RN; Phone Number: 714-964-5359; Email: erin_morris@nymc.edu
• Study Contact Backup: Name: Lauren Harrison, MSN; Phone Number: 617-285-7844; Email: lauren_harrison@nymc.edu

Study Locations

• United States
  • New York
    • Valhalla, New York, United States, 10595
      • Recruiting
      • New York Medical College
      • Contact: Jordan Milner, MD
      • Contact: Elizabeth Mintzer, CRA; Email: emintzer2@nymc.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: rituximab; All patients enrolled will receive the dose dense administration of rituximab. Five total doses will be administered on Days: 0, 2, 7 (± 2 days), 14 (± 2 days), and 21 (± 2 days); Dose: 375 mg/m2	Drug: rituxan; The dose dense administration of rituximab will consist of 5 doses total Days: 0, 2, 7 (± 2 days), 14 (± 2 days), and 21 (± 2 days); Dose: 375 mg/m2; Other Names: rituximab

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To determine the safety events: Number of participants with treatment-related Grade III or higher adverse events as assessed by CTCAE v5.0.	To determine the occurrence of any Grade ≥ 3 non hematologic toxicity (per CTCAE v.5) which is possibly, probably, or definitely related to rituximab.	1 year
To determine the Response Rate	To quantify remission rates for high-risk patients with acute ITP treated with a dose dense administration of rituximab.	1 year

Collaborators and Investigators

• Sponsor: New York Medical College
• Investigators: Principal Investigator: Jordan Milner, MD, New York Medical College

Study record dates

Study Major Dates

• Study Start (Actual): February 24, 2021
• Primary Completion (Estimated): July 31, 2024
• Study Completion (Estimated): July 31, 2025

Study Registration Dates

• First Submitted: February 14, 2020
• First Submitted That Met QC Criteria: March 25, 2020
• First Posted (Actual): March 26, 2020

Study Record Updates

• Last Update Posted (Actual): October 26, 2023
• Last Update Submitted That Met QC Criteria: October 24, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Thrombocytopenia; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Physiological Effects of Drugs; Antirheumatic Agents; Antineoplastic Agents; Immunologic Factors; Antineoplastic Agents, Immunological; Rituximab
• Other Study ID Numbers: 14124

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes