Reduced Intensity Conditioning for Non-Malignant Disorders

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Recruiting Phase 2 Interventional Study
Primary Immunodeficiency (PID) Congenital Bone Marrow Failure Syndromes Inherited Metabolic Disorders (IMD) Hereditary Anemias Inflammatory Conditions Systemic Juvenile Idiopathic Arthritis (sJIA) Juvenile Rheumatoid Arthritis (JRA)
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
2 Months – 55
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
100 (estimated)
Sponsor
Paul Szabolcs · Other
Who this trial is looking for

This trial is looking for patients with certain non-cancerous disorders who may benefit from a special type of stem cell transplant. Participants will receive treatment and be monitored for how well the transplant works and any long-term effects.

Are You a Good Fit for This Trial?

You may be able to join if

  • I have a non-malignant disorder that can be treated with a stem cell transplant.
  • I have a matching umbilical cord blood or donor bone marrow.
  • I have signed a consent form to participate.
  • I am HIV negative.
  • I have good organ function.
  • I am not pregnant or nursing.
  • I can attend follow-up appointments.

You may not be able to join if

  • I had a stem cell transplant in the last 6 months.
  • I have an active cancer.
  • I have a severe infection that is not controlled.
  • I have poorly controlled pulmonary hypertension.
  • I have severe acquired aplastic anemia.
  • I cannot follow up for appointments.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

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About This Trial
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). …
Trial Locations
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Eligibility Criteria
Inclusion: 1. A 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit available that will deliver a pre-cryopreservation total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double unit grafts, each cord blood unit delivering at least 2 x 10e7 cells/kg OR an 8 of 8 or 7 of 8 HLA allele level ma…
Contacts

Paul Szabolcs, MD

412-692-5427

paul.szabolcs@chp.edu

CONTACT

Shawna McIntyre, RN

412-692-5552

mcintyresm@upmc.edu

CONTACT