Using PTCy and Ruxolitinib to prevent GVHD after stem cell transplants in children with immune disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the effectiveness of a combined regimen for preventing graft-versus-host disease (GVHD) in patients with inborn errors of immunity undergoing hematopoietic stem cell transplantation (HSCT). It utilizes post-transplant cyclophosphamide (PTCY) and ruxolitinib alongside thymoglobulin in the conditioning regimen. The study aims to determine how well this combination works in reducing the incidence of GVHD, which is a significant risk in these patients. Participants will receive specific doses of cyclophosphamide and ruxolitinib at designated times post-transplant.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Patients aged ≥ 0 months and \< 21 years
2. Patients diagnosed with NBS eligible for an allogeneic HSCT
3. Signed written informed consent signed by a parent or legal guardian

Exclusion Criteria:

Concomitant severe somatic disease associated with an additional risk of severe complications

Where it's running

• HSCT department
  Moscow, Russia Recruiting

Study contacts

• Study coordinator: Dmitry Balashov, MD, PhD
• Email: bala8@yandex.ru
• Phone: +74956647091

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Ruxolitinib and venetoclax added to chemotherapy for children and young adults with relapsed or refractory ALL/LBL

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• Graft-versus-host disease after stem cell transplant: testing LBP-MARCO with ruxolitinib

  We are testing whether combining LBP-MARCO with ruxolitinib is safe for adults with GVHD after a stem cell transplant.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.