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NCT06430788RECRUITINGIRB Ready

A Study of Emapalumab for Pediatric Aplastic Anemia

Sponsored by Memorial Sloan Kettering Cancer Center

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
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1 of 35 participants interested
3% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options.

Who Can Participate

Inclusion Criteria

Patients undergoing workup for suspected newly diagnosed sAA:
Patients with severe cytopenias and a hypocellular marrow concerning for sAA
Patients that meet the definition for suspected sAA (Camitta Criteria) as follows: Marrow Cellularity: \<25%, or 25-50% with \<30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): \<500 x 10\^9/L Platelets: \<20 x 10\^9/L Absolute Reticulocyte Count: \<60 x 10\^9/L
Patients that do not have evidence of leukemia or MDS
Patients \< 25 years of age at time of diagnosis
Able to tolerate emapalumab and IST (with standard institutional organ function criteria)

Exclusion Criteria

Uncontrolled infection at presentation.
Patients who have undergone previous treatment for sAA.
Patients with known inherited bone marrow failure
Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicabl

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

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Study Type

INTERVENTIONAL

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Target Enrollment

35 participants

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Age Range

0 Years - 25 Years

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Gender

ALL

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Sponsor

Memorial Sloan Kettering Cancer Center

Study Locations

Choose your preferred location or select flexible during enrollment

RECRUITING

New York

New York

Location available
View New York location page
RECRUITING

Cincinnati

Ohio

Location available
View Cincinnati location page
RECRUITING

Philadelphia

Pennsylvania

Location available
View Philadelphia location page
RECRUITING

Richmond

Virginia

Location available
View Richmond location page
RECRUITING

Milwaukee

Wisconsin

Location available
View Milwaukee location page
NOT_YET_RECRUITING

Milwaukee

Wisconsin

Location available
View Milwaukee location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for Aplastic Anemia Treatment in New York?

Join others in New York exploring innovative treatment options through clinical research

Aplastic Anemia Treatment Options in New York, New York

If you're searching for Aplastic Anemia treatment in New York, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in New York, Cincinnati, Philadelphia and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Aplastic Anemia. All study-related care is provided at no cost to participants.

Local Sites
3 locations in New York
Now Enrolling
Up to 35 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Aplastic Anemia?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Aplastic Anemia

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Aplastic Anemia Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT06430788. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.