NCT03173937

Brief Summary

Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective. Objective: To test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective. Eligibility: Recipients ages 4-60 with SAA or MDS Donors ages 4-75 Design: Recipients will be screened with:

  • Blood, lung, and heart tests
  • Bone marrow biopsy
  • CT scan Recipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose. Recipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits:
  • First 3-4 months: 1-2 times weekly
  • Then every 6 months for 5 years Donors will be screened with:
  • Physical exam
  • Medical history
  • Blood tests Donors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein. Donors will get Filgrastim or biosimilar (G-CSF) injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later. ...

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
37

participants targeted

Target at P50-P75 for phase_1

Timeline
71mo left

Started Jun 2017

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress60%
Jun 2017Mar 2032

First Submitted

Initial submission to the registry

May 31, 2017

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 2, 2017

Completed
11 days until next milestone

Study Start

First participant enrolled

June 13, 2017

Completed
9.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 28, 2027

Expected
4.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2032

Last Updated

March 31, 2026

Status Verified

March 25, 2026

Enrollment Period

9.9 years

First QC Date

May 31, 2017

Last Update Submit

March 28, 2026

Conditions

Severe Aplastic AnemiaHypo-Plastic MDSMyelodysplastic Syndrome (MDS)

Keywords

HaploidenticalNonmyeloablative

Outcome Measures

Primary Outcomes (1)

  • Cord engraftment

    Cord engraftment

    At or before day 100

Secondary Outcomes (1)

  • Treatment related mortality (TRM), and standard transplant outcome variables such as non-hematologic toxicity, incidence and severity of acute and chronic GVHD, and relapse of disease

    100 day and 200 day

Study Arms (1)

1

EXPERIMENTAL

CordIn is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells.

Biological: Omidubicel (former CordIn)Device: Miltenyi CliniMACS(R) CD34 Reagent System

Interventions

Omidubicel (former CordIn)BIOLOGICAL

Stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic umbilical/unrelated cord blood (UCB) cells. CordIn(TM) comprises: 1) cord blood-derived ex vivo expanded CD133+ cells (CordIn (TM) cultured fraction (CF)); and 2) the non-cultured cell fraction (CD133-) of the same CBU (CordIn(TM) Non-cultured Fraction (NF)). Both fractions, i.e. CordIn(TM) CF and CordIn(TM) NF are kept frozen until they are infused on the day of transplantation.

1
Miltenyi CliniMACS(R) CD34 Reagent SystemDEVICE

The CliniMACS CD34 Reagent System is a medical device system that is designed for the in vitro enrichment of CD34+ target cells from heterogeneous hematologic cell populations. The process of CD34+ cell selection results in simultaneous passive depletion of donor lymphocytes, potentially obviating the need for immunosuppressive drugs to prevent GVHD.

1

Eligibility Criteria

Age4 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosed with severe aplastic anemia with bone marrow cellularity \<30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and/or neutropenia (absolute neutrophil count \<=1000 cells/ uL or for patients receiving granulocyte transfusions, absolute neutrophil count \<=1000 cells/ uL before beginning granulocyte transfusions).
  • History of severe aplastic anemia transformed to MDS that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \< 5% myeloblasts and \< 30% of cellularity in the bone marrow on screening morphologic analysis.
  • Intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.
  • Identification of either a) at least one alternative donor (i.e. HLA- haploidentical related donor (i.e. \>=5/10 HLA match: HLA-A, B, C, DR, and DQ loci) or \>=9/10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the CordIn(TM) unit has been rejected or b) umbilical cord blood unit/s that can be used for a salvage cord blood transplant in the event that the CordIn(TM) unit has been rejected.
  • Availability of at least one \>=4/8 HLA matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).
  • The cord blood unit must contain a minimum TNC of at least 1.8 x 10\^9 and at least 1.5x10\^7/kg TNC and at least 8 x 10\^6 CD34+ cells (all doses prior to thawing).
  • Exception: Cord units containing at least 8 x 10\^6 CD34+ cells but less than 1.8 x 10\^9 TNC may be eligible for use on this trial if
  • the pre-expansion CD34/kg recipient cell number is at least 1.5 x 10\^5 /kg
  • AND
  • approval for use of this cord unit for expansion is granted by Gamida Cell.
  • the final cell counts on the cultured and non-cultured fractions meet the IND specified minimal release criteria.
  • The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation. All CBUs should be procured from public banks that meet local applicable regulations.
  • Ages 4-60 years inclusive.
  • Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.

You may not qualify if:

  • Availability of an HLA identical (12/12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.
  • ECOG performance status of 2 or more.
  • Major anticipated illness or organ failure incompatible with survival from transplant.
  • Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.
  • HIV positive.
  • Diagnosis of Fanconi s anemia (by chromosome breakage study).
  • Diffusion capacity of carbon monoxide (DLCO) \<40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).
  • Left ventricular ejection fraction \< 40% (evaluated by ECHO).
  • Transaminases \> 5x upper limit of normal.
  • Serum bilirubin \>4 mg/dl.
  • Creatinine clearance \< 50 cc/min/BSAm2 by 24-hour urine collection adjusted by body surface area.
  • Serum creatinine \> 2.5 mg/dl
  • Presence of an active infection not adequately responding to appropriate therapy.
  • History of a malignant disease liable to relapse or progress within 5 years.
  • Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Anemia, AplasticMyelodysplastic Syndromes

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Study Officials

  • Richard W Childs, M.D.

    National Heart, Lung, and Blood Institute (NHLBI)

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Melissa M Spencer, R.N.

CONTACT

(301) 402-5609melissa.spencer@nih.gov

Richard W Childs, M.D.

CONTACT

(301) 768-9433childsr@nhlbi.nih.gov

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 31, 2017

First Posted

June 2, 2017

Study Start

June 13, 2017

Primary Completion (Estimated)

April 28, 2027

Study Completion (Estimated)

March 1, 2032

Last Updated

March 31, 2026

Record last verified: 2026-03-25

Locations

US(1)