← Back to Search

Alkylating agents

Cord Blood Transplant for Blood Diseases

Phase 1

Recruiting

Led By Jane L Liesveld, MD

Research Sponsored by University of Rochester

Eligibility Criteria Checklist

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

Must have

Performance Status Karnofsky or Lansky score ≥ 70%

Patients must have a disease or syndrome amenable to therapy with hematopoietic stem cell transplantation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

Awards & highlights

Study Summary

This trial is testing different ways to prepare for and receive stem cell transplants from cord blood, to see which is best and safest.

Who is the study for?

This trial is for patients with various blood diseases, immune disorders, and cancers like leukemia, lymphoma, and solid tumors. Participants need to have a certain level of physical fitness (Karnofsky or Lansky score ≥ 70%) and good heart, lung, kidney, and liver function. They must not be pregnant or breastfeeding, HIV positive, or have had a recent autologous HSCT. A suitable HLA-matched donor should not be available.Check my eligibility

What is being tested?

The study tests umbilical cord blood stem cell transplantation using one of four preparative regimens that include Melphalan, Mesna, Fludarabine among others. It aims to validate the transplantation process at the institution conducting the research.See study design

What are the potential side effects?

Potential side effects may include reactions from chemotherapy drugs like nausea and hair loss; organ inflammation due to radiation; increased risk of infections post-transplantation; possible graft-versus-host disease where transplanted cells attack the body.

Eligibility Criteria

Inclusion Criteria

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

Select...

I am mostly able to care for myself but may not be able to do active work.

Select...

My condition can be treated with a stem cell transplant.

Select...

My treatment includes confirmed cord blood products before starting conditioning therapy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months. for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Engraftment of ANC and Platelets

Secondary outcome measures

Disease-free survival

Incidence of acute graft-versus-host disease

Incidence of chronic graft-versus-host disease

+1 more

Trial Design

4Treatment groups

Experimental Treatment

Group I: Reduced Intensity ChemotherapyExperimental Treatment3 Interventions

Reduced Intensity Chemotherapy Fludarabine 30 mg/m2/day x 5 doses days -6 to -2 Melphalan 140 mg/m2/day x 1 dose day -2 Cord Blood Infusion Other names: Flu/Mel

Group II: Non-Myeloablative ConditioningExperimental Treatment4 Interventions

Fludarabine 40 mg/m2/day x 5 doses days -6 to -2 Cyclophosphamide 50 mg/kg/day x 1 dose day -6 Mesna 50 mg/kg/day with 20% loading dose with Cyclophosphamide dose followed by continuous infusion over 24 hours x 1 dose [to be completed 24 hours after Cyclophosphamide dose] Total Body Irradiation 200 cGy in a single fraction day -1 Cord Blood Infusion Other names: Flu/Cy/TBI

Group III: Full Intensity, TBI-based ConditioningExperimental Treatment4 Interventions

Full Intensity TBI-based Conditioning Total Body Irradiation 1200 cGy in fractions of 150 cGy days -8 or -7 to -4 Cyclophosphamide 60 mg/kg/day x 2 doses days -3 and -2 Mesna 60 mg/kg/day with 20% loading dose with first Cyclophosphamide followed by continuous infusion over 24 hours x 2 doses [to be completed 24 hours after final Cyclophosphamide dose] followed by Cord Blood Infusion Other names: TBI/Cy

Group IV: Full Intensity, Chemo-based ConditioningExperimental Treatment4 Interventions

Full Intensity, Chemotherapy Conditioning Busulfan days -7 to -4 Recipients <5 years - 1 mg/kg/dose x 16 doses every 6 hours Recipients >/= 5 years - 0.8 mg/kg/dose x 16 doses every 6 hours Cyclophosphamide 60 mg/kg/day x 2 doses days -3 and -2 Mesna 60 mg/kg/day with 20% loading dose with first Cyclophosphamide followed by continuous infusion over 24 hours x 2 doses [to be completed 24 hours after final Cyclophosphamide dose] followed by Cord Blood Infusion Other names: Bu/Cy

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Melphalan

2008

Completed Phase 3

~1500

Mesna

2003

Completed Phase 2

~1380

Busulfan

2008

Completed Phase 3

~1120

Fludarabine

2012

Completed Phase 3

~1100

Cord Blood Infusion

2012

Completed Phase 2

~320

Total Body Irradiation 1200 cGy

2015

Completed Phase 1

~80

Cyclophosphamide

1995

Completed Phase 3

~3770

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of RochesterLead Sponsor

840 Previous Clinical Trials

534,564 Total Patients Enrolled

13 Trials studying Leukemia

1,292 Patients Enrolled for Leukemia

Jane L Liesveld, MDPrincipal InvestigatorMedical Director, Blood & Marrow Transplant Unit

Media Library

Busulfan (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT03016806 — Phase 1

Leukemia Research Study Groups: Full Intensity, TBI-based Conditioning, Non-Myeloablative Conditioning, Full Intensity, Chemo-based Conditioning, Reduced Intensity Chemotherapy

Leukemia Clinical Trial 2023: Busulfan Highlights & Side Effects. Trial Name: NCT03016806 — Phase 1

Busulfan (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03016806 — Phase 1

Frequently Asked Questions

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

What criteria must participants meet to be eligible for this research?

"For this trial, 30 patients aged between 2 Months and 75 with lymphoma must be found. Additionally, these individuals should have immunodeficiencies (like Severe Combined Immunodeficiency or Wiskott-Aldrich Syndrome), inherited stem cell defects (e.g Chediak-Higashi Syndrome or Osteogenesis imperfecta), metabolic disorders (including Hurler's Syndrome), aplastic anemia, treatment refractory Acute Myelogenous Leukemia that has either evolved from myelodysplastic syndrome or contains FLT3 abnormalities are also eligible to apply for the study."

Answered by AI

Has this therapeutic approach received sanction from the FDA?

"Given the preliminary nature of this medical trial, safety was ranked a 1 on our scale. This is due to restricted data corroborating its efficacy and security."

Answered by AI

To what maladies is this therapeutic approach typically applied?

"This medication is frequently employed to manage multiple sclerosis, but it can also be beneficial for patients with leukemia, myelocytic acute, allogeneic hematopoietic stem cell transplant, chronic myelogenous leukemia and thrombocythemia."

Answered by AI

Is the age threshold for this study higher than thirty-five years old?

"The requirements for entry into this trial dictate that an individual must be between 2 Months and 75 years old. Additionally, there are 1364 available clinical trials specifically designated for minors, as well as 5130 research initiatives open to seniors."

Answered by AI

Could you summarize any other trials that have evaluated this therapeutic approach?

"Currently, 964 studies related to this treatment are still in progress. Out of these trials, 171 have advanced to the clinical trial's third and final phase. Although many of those research projects take place in Philadelphia, Pennsylvania, there are a grand total of 29 566 medical centres actively engaged with such investigations."

Answered by AI

Does this research project currently offer enrolment opportunities to potential participants?

"Affirmative. Per the information on clinicaltrials.gov, this experiment is actively recruiting participants as of today. The initial posting was made on June 1st 2015 and the most recent edit occurred on June 20th 2022. 30 individuals are needed at one site for this endeavor to be successful."

Answered by AI

How many participants have volunteered to be part of this clinical experiment?

"Affirmative. Clinicaltrials.gov has documented that this experiment is currently looking for volunteers, with the original posting date being June 1st 2015 and a most recent update on June 20th 2022. The project's objective is to collect data from 30 patients at one medical location."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Umbilical Cord Blood Transplantation From Unrelated Donors

Jane Liesveld 2015. "Umbilical Cord Blood Transplantation From Unrelated Donors". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03016806.

All > Aplastic Anemia > Myelodysplastic Syndrome