NCT01816230

Brief Summary

A Study Evaluating the Safety and Efficacy of Transplantation of a single cord blood unit (CBU) of NiCord®, umbilical cord blood-derived Ex Vivo Expanded Stem and Progenitor Cells in Patients with Hematological Malignancies.

Trial Health

93
On Track

Trial Health Score

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

Enrollment
36

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Apr 2013

Longer than P75 for phase_1

Geographic Reach
5 countries

15 active sites

Status
completed

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

First Submitted

Initial submission to the registry

March 19, 2013

Completed
3 days until next milestone

First Posted

Study publicly available on registry

March 22, 2013

Completed
10 days until next milestone

Study Start

First participant enrolled

April 1, 2013

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2017

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2018

Completed
2.3 years until next milestone

Results Posted

Study results publicly available

September 21, 2020

Completed
Last Updated

October 22, 2020

Status Verified

October 1, 2020

Enrollment Period

4.2 years

First QC Date

March 19, 2013

Results QC Date

August 31, 2020

Last Update Submit

October 1, 2020

Conditions

Hematological MalignanciesAcute Lymphoblastic Leukemia (ALL)Acute Myeloid Leukemia (AML)Myelodysplastic Syndrome (MDS)

Outcome Measures

Primary Outcomes (1)

  • Engraftment

    Assess the cumulative incidence of patients with NiCord®-derived neutrophil engraftment at 42 days following transplantation.

    42 days

Study Arms (1)

NiCord®

EXPERIMENTAL

NiCord® is a stem/progenitor cell based product composed of ex vivo expanded allogeneic UCB cells.

Drug: NiCord®

Interventions

NiCord®DRUG
NiCord®

Eligibility Criteria

Age12 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Applicable disease and eligible for myeloablative SCT
  • Patients must have two partially HLA-matched CBUs
  • Back-up stem cell source
  • Adequate Karnofsky Performance score or Lansky Play-Performance scale
  • Sufficient physiological reserves
  • Signed written informed consent

You may not qualify if:

  • HLA-matched donor able to donate
  • Prior allogeneic HSCT
  • Other active malignancy
  • Active or uncontrolled infection
  • Active/symptoms of central nervous system (CNS) disease
  • Pregnancy or lactation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

Location

Loyola University, Cardinal Bernardin Cancer Center

Maywood, Illinois, 60153, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Steven and Alexandra Cohen Children's Medical Center

New York, New York, 11040, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Vanderbilt

Nashville, Tennessee, 37240, United States

Location

Children Hospital Gaslini Scientific Institute

Genoa, 16147, Italy

Location

University of Turin

Turin, 10043, Italy

Location

University Medical Center Utrecht

Utrecht, 3503 AB, Netherlands

Location

National University Cancer Institute

Singapore, 119074, Singapore

Location

Singapore General Hospital

Singapore, 169608, Singapore

Location

University Hospital Vall d´Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario La Fe

Valencia, 46009, Spain

Location

Related Publications (1)

  • Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, Nierkens S, Jagasia M, Wagner JE, Kuball J, Koh LP, Majhail NS, Stiff PJ, Hanna R, Hwang WYK, Kurtzberg J, Cilloni D, Freedman LS, Montesinos P, Sanz G. Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374. doi: 10.1200/JCO.18.00053. Epub 2018 Dec 4.

    PMID: 30523748DERIVED

MeSH Terms

Conditions

Hematologic NeoplasmsPrecursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Myeloid, AcuteMyelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Neoplasms by SiteNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLeukemia, LymphoidLeukemiaNeoplasms by Histologic TypeLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLeukemia, MyeloidBone Marrow Diseases

Results Point of Contact

Title
Kelly Myers
Organization
Gamida Cell
kelly@gamida-cell.com
026595631

Study Officials

  • Mitchell Horwitz, MD

    Duke University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 19, 2013

First Posted

March 22, 2013

Study Start

April 1, 2013

Primary Completion

June 1, 2017

Study Completion

June 1, 2018

Last Updated

October 22, 2020

Results First Posted

September 21, 2020

Record last verified: 2020-10

Locations

ES(2)IT(2)SG(2)NL(1)US(8)