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Not listed Phase 1Phase 2 Interventional

Study of ASC-101 in Patients With Hematologic Malignancies Who Receive Dual-cord Umbilical Cord Blood Transplantation

ClinicalTrials.gov ID: NCT01983761

Public ClinicalTrials.gov record NCT01983761. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 16, 2026, 7:45 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase I/IIa, Open-label, Non-randomized, Study of ASC-101 in Patients With Hematologic Malignancies or Myelodysplastic Syndrome (MDS) Who Are Candidates for Dual-cord Umbilical Cord Blood Transplantation (UCBT)

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The goal of this clinical research study is to learn if it is safe and feasible to transplant patients with one of two units of cord blood that has been changed in the laboratory before it is given. Only patients with leukemia, lymphoma or myelodysplastic syndrome will be allowed on this study. The secondary goal is to obtain the preliminary efficacy outcome. Researchers also want to learn if using cord blood that has been changed can help to control the disease. One cord blood unit will not be changed before it is administered to you. The cord blood unit that will be altered will be changed to use sugar that is found in small amounts in blood cells. It plays a role in telling transplanted cells where they should go in the body. Adding more sugars to the cord blood cells in the laboratory helps the cord blood cells find their way to the bone marrow faster. This process is called fucosylation. "Conditioning" is the chemo and other medicines and will be given to patients to prepare to receive cord blood transplant cells. This prevents immune system from rejecting the cells. Conditioning will be started before the transplant. ATG is a protein that removes immune cells that cause damage to the body. Clofarabine is designed to interfere with the growth and development of cancer cells. Fludarabine is designed to interfere with the DNA of cancer cells, which may cause the cancer cells to die. This chemotherapy is also designed to block your body's ability to reject the donor's bone marrow cells. Melphalan and busulfan are designed to bind to the DNA of cells, which may cause cancer cells to die. MMF and tacrolimus are designed to block the donor cells from growing and spreading in a way that could cause graft versus host disease (GVHD -- a condition in which transplanted tissue attacks the recipient's body). This may help to prevent GVHD. Rituximab is designed to attach to cancer cells, which may cause them to die. A Phase I study for treatment of patients (N=25) with hematologic malignancies and MDS who are candidates for dual-cord UCBT is ongoing at M.D. Anderson Cancer Center under an Investigator-initiated IND Application, E.J. Shpall, MD, PI. Since August, 2012, Preliminary results indicate that ASC-101 UCBT is well-tolerated and no ASC-101 related untoward adverse events have been observed. To date, the median time to neutrophil engraftment (N=9) is 15 days, and the median time to platelet engraftment (N=9) is 33 days. The trial remains ongoing.

Study identification

NCT ID
NCT01983761
Recruitment status
Not listed
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
Targazyme, Inc.
Industry
Enrollment
25 participants

Conditions and interventions

Eligibility (public fields only)

Age range
1 Year to 80 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Oct 31, 2013
Primary completion
Feb 28, 2014
Completion
Not listed
Last update posted
Nov 17, 2013

Started 2013

United States locations

U.S. sites
4
U.S. states
3
U.S. cities
4
Facility City State ZIP Site status
Scripps Green Hospital La Jolla California 92037
University Hospitals of Cleveland Cleveland Ohio 44106
M.D. Anderson Cancer Center Houston Texas 77030
Texas Transplant Institute San Antonio Texas 78229

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01983761, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Nov 17, 2013 · Synced Sep 16, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01983761 live on ClinicalTrials.gov.

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