Immune Thrombocytopenic PurpuraPhase 1NYMC207NCT04323748
Dose Dense Rituximab for High Risk Newly Diagnosed Acute Immune Thrombocytopenic Purpura
A Phase 1 study testing rituxan in people with Immune Thrombocytopenic Purpura, run by New York Medical College at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Immune Thrombocytopenic Purpura
- What is being tested?
- rituxan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Feb 2021to 31 Jul 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Show all 17Show fewer
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
New York Medical College
Vahalla, New York, 10595
Edo Schaefer, MD, eschafe@nymc.edu
About this study
Official title: 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.
- Conditions
- Immune Thrombocytopenic Purpura
- Being tested
- rituxan
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rituxanDrug
Also called rituximab
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
| Study group | Role | What they receive |
|---|---|---|
| rituximab | Experimental | 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 Receives: Drug: rituxan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2020
- Study start24 Feb 2021
- Primary completion31 Jul 2026 (estimated)
- Study completion31 Jul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune Thrombocytopenic 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Edo Scahefer, MD, Principal investigator, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04323748
- Sponsor's protocol ID
- 14124
- Acronym
- NYMC207
- Status last verified
- Apr 2026
- First submitted
- 14 Feb 2020
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Use of Dose Dense Rituximab for High Risk Patients With Newly Diagnosed Acute Immune Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT04323748
- Acronym / sponsor ID
- NYMC207 / 14124
- Conditions
- Immune Thrombocytopenic Purpura
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Study start
- 24 Feb 2021 (actual)
- Primary completion
- 31 Jul 2026 (estimated)
- Study completion
- 31 Jul 2027 (estimated)
- First posted
- 26 Mar 2020 (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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rituximab | Experimental | Drug: rituxanAll 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 |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- 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:
- 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
Key exclusion criteria (7 listed)
- 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
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 |
|---|---|---|
| rituxanAlso: rituximabThe 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 | Drug | rituximab |
Primary outcome
To determine the safety events: Number of participants with treatment-related Grade III or higher adverse events as assessed by CTCAE v5.0.
Time frame: 1 year
To determine the occurrence of any Grade ≥ 3 non hematologic toxicity (per CTCAE v.5) which is possibly, probably, or definitely related to rituximab.
To determine the Response Rate
Time frame: 1 year
To quantify remission rates for high-risk patients with acute ITP treated with a dose dense administration of rituximab.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- 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.
Edo Scahefer, MD principal investigator
New York Medical College
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing rituxan in people with Immune Thrombocytopenic Purpura, run by New York Medical College at 1 site in United States.
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 17 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 15 Apr 2026. 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 United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Erin Morris, RN (714-964-5359, erin_morris@nymc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine the safety events: Number of participants with treatment-related Grade III or higher adverse events as assessed by CTCAE v5.0. (time frame: 1 year). The study lists 2 primary outcomes in all. 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 15 Apr 2026.