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Monoclonal Antibodies

Reduced-Intensity Stem Cell Transplant for Non-Malignant Disorders (HSCT+RIC Trial)

Q: What goals does this clinical trial hope to accomplish?

<p>The primary objective of this investigation over a post-<a href="https://www.withpower.com/clinical-trials/transplant">transplant</a> period of one year is to evaluate the attainment and maintenance of neurological milestones. Secondary objectives include determining normal enzyme levels, quantifying grade 3-4 organ toxicity, and assessing long-term ramifications such as endocrinopathy, sterility, and stunted growth.</p>

Phase 2

Recruiting

Led By Paul Szabolcs, MD

Research Sponsored by Paul Szabolcs

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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 matched unrelated donor bone marrow or peripheral blood progenitor graft

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year post-transplant

Awards & highlights

HSCT+RIC Trial Summary

This trial is testing whether a reduced-intensity conditioning regimen followed by umbilical cord blood transplant, double cord blood transplant, matched unrelated donor bone marrow transplant, or peripheral blood stem cell transplant is effective in treating patients with non-malignant disorders.

Who is the study for?

This trial is for patients with non-cancerous disorders like juvenile arthritis, bone marrow failure, primary immunodeficiency, anemia, and metabolic diseases. Participants must have proper organ function, no active malignancy or severe aplastic anemia, not be pregnant or nursing mothers, and should not have had a stem cell transplant in the last 6 months.Check my eligibility

What is being tested?

The study tests a reduced-intensity conditioning regimen before umbilical cord blood transplant (UCBT), bone marrow transplant (BMT) from unrelated donors or peripheral blood stem cell transplant (PBSCT). It aims to see how well these transplants work for treating various non-malignant disorders.See study design

What are the potential side effects?

Potential side effects may include reactions to medications like Melphalan and Alemtuzumab such as nausea, fatigue, mouth sores; Hydroxyurea can cause skin rashes; Fludarabine might lead to immune system suppression increasing infection risk.

HSCT+RIC Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have a suitable donor for my stem cell transplant.

HSCT+RIC Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post-transplant for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

GVHD occurrence

Immune Reconstitution

Neurodevelopmental milestones

+2 more

Secondary outcome measures

Donor cell engraftment

Grade 3-4 organ toxicity

Late graft failure

+3 more

HSCT+RIC Trial Design

2Treatment groups

Experimental Treatment

Group I: UCBT:transfusion dependent anemias or increased rejection riskExperimental Treatment5 Interventions

Day -21 to -19: Alemtuzumab + Hydroxyurea; Day -18 to -10: Hydroxyurea; Day -9 to -5: Fludarabine + Hydroxyurea; Day -4 to -3: Melphalan; Day -2: Thiotepa; Day -1: Rest; Day 0: Transplant

Group II: BMT, PBSCT and not transfusion dependent UCBTExperimental Treatment5 Interventions

Start of conditioning to Day -15: Hydroxyurea; Day -14 to -13: Alemtuzumab + Hydroxyurea; Day -12 to -10: Hydroxyurea; Day -9 to -5: Fludarabine + Hydroxyurea; Day -4 to -3: Melphalan; Day -2: Thiotepa; Day -1: Rest; Day 0: Transplant

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Alemtuzumab

2004

Completed Phase 4

~1890

Hydroxyurea

2006

Completed Phase 4

~3620

Fludarabine

2012

Completed Phase 3

~1100

Melphalan

2008

Completed Phase 3

~1500

Thiotepa

2008

Completed Phase 3

~2210

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Paul SzabolcsLead Sponsor

7 Previous Clinical Trials

132 Total Patients Enrolled

1 Trials studying Anemia

5 Patients Enrolled for Anemia

Paul Szabolcs, MDPrincipal Investigator - University of Pittsburgh

University of Pittsburgh

7 Previous Clinical Trials

129 Total Patients Enrolled

1 Trials studying Anemia

5 Patients Enrolled for Anemia

Media Library

Alemtuzumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT01962415 — Phase 2

Anemia Research Study Groups: BMT, PBSCT and not transfusion dependent UCBT, UCBT:transfusion dependent anemias or increased rejection risk

Anemia Clinical Trial 2023: Alemtuzumab Highlights & Side Effects. Trial Name: NCT01962415 — Phase 2

Alemtuzumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01962415 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many individuals have signed up for this clinical investigation?

"Indeed. According to the information made available on clinicaltrials.gov, this study is actively recruiting volunteers as of December 7th 2022, having been first posted on February 4th 2014. The trial requires 100 patients between a single research centre."

Answered by AI

What is the current governmental ruling on Melphalan?

"The safety rating of melphalan is 2, as the clinical trial has only demonstrated a level of safety but not efficacy."

Answered by AI

Is it possible for me to participate in this experiment?

"A total of 100 participants with congenital bone marrow failure syndromes aged between 2 Months and 55 are eligible to join this study. Eligibility criteria include being diagnosed with Omenn Syndrome, as well as a negative pregnancy test if the patient is in their puberty or menstruating."

Answered by AI

Is recruitment for this research still open to participants?

"Affirmative. Information sourced from clinicaltrials.gov details that this study, first advertised on February 4th 2014, is in need of participants and has recently been updated (12/7/2022). The trial requires 100 individuals to be recruited across one site for completion."

Answered by AI

What diseases or conditions is Melphalan typically administered to address?

"Melphalan is commonly prescribed to combat neoplasm metastasis, and there is evidence that it can also be used for the management of sickle cell anemia, inoperable ovarian cancer, and rejection; kidney transplant."

Answered by AI

What goals does this clinical trial hope to accomplish?

"The primary objective of this investigation over a post-transplant period of one year is to evaluate the attainment and maintenance of neurological milestones. Secondary objectives include determining normal enzyme levels, quantifying grade 3-4 organ toxicity, and assessing long-term ramifications such as endocrinopathy, sterility, and stunted growth."

Answered by AI

Is the minimum age requirement for this trial greater than 25 years?

"Those wishing to enroll in this medical trial must be between 2 months and 55 years of age. Meanwhile, on the same platform there are 719 trials for those below 18 and 1593 studies available for those above 65."

Answered by AI