NCT01590628

Brief Summary

Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies

Trial Health

87
On Track

Trial Health Score

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

Enrollment
16

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Sep 2012

Longer than P75 for phase_1

Geographic Reach
1 country

3 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

April 15, 2012

Completed
18 days until next milestone

First Posted

Study publicly available on registry

May 3, 2012

Completed
4 months until next milestone

Study Start

First participant enrolled

September 1, 2012

Completed
6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2018

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2019

Completed
2.6 years until next milestone

Results Posted

Study results publicly available

April 27, 2022

Completed
Last Updated

May 20, 2022

Status Verified

April 1, 2022

Enrollment Period

6 years

First QC Date

April 15, 2012

Results QC Date

May 19, 2021

Last Update Submit

April 27, 2022

Conditions

Sickle Cell Disease & Thalassemia

Keywords

Sickle Cell Disease & ThalassemiaGenetic disorder

Outcome Measures

Primary Outcomes (2)

  • Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity.

    Assessment of acute toxicity associated with the infusion of NiCord within 24 hours post-infusion.

    24 hours post-infusion

  • Assessment of Cumulative Incidence of Donor-derived Neutrophil Engraftment.

    Neutrophil engraftment is defined as achieving an absolute neutrophil count (ANC) of ≥500/ μL for 3 consecutive measurements on different days by Day 42 inclusive (the day of engraftment was defined as the first of these 3 days).

    By Day 42

Secondary Outcomes (3)

  • Proportion of Transplant-related Mortality.

    at 100 days

  • Event-free Survival

    100 days post-transplant

  • Overall Survival

    180 days

Study Arms (1)

NiCord

EXPERIMENTAL

NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.

Drug: NiCord

Interventions

NiCordDRUG

NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells

NiCord

Eligibility Criteria

Age2 Years - 45 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Must be 2 - 45 years of age and at least 10 kg
  • Must have clinically severe SCD (SS, SC or SBeta0 Thal) or thalassemia major and be eligible for myeloablative SCT
  • Must have two partially HLA-matched CBUs for part 1; and one partially HLA-matched CBU for part 2
  • Back-up autologous stem cells harvested from bone marrow
  • Adequate Karnofsky Performance score or Lansky Play-Performance scale
  • Sufficient physiological reserves
  • Signed written informed consent

You may not qualify if:

  • HLA-matched related donor able to donate
  • Severe alloimmunization with inability to guarantee a supply of adequate PRBC donors
  • Prior allogeneic hematopoietic SCT within the last 12 months or reduced-intensity transplant within the past 6 months
  • Human immunodeficiency virus (HIV) infection
  • Active or uncontrolled infection
  • Pregnancy or lactation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Steven & Alexandra Cohen Children's Medical Center, New York

New York, New York, 11040, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

The University Of Texas M. D. Anderson Cancer Center

Houston, Texas, 77030-4009, United States

Location

Related Publications (1)

  • Parikh S, Brochstein JA, Galamidi E, Schwarzbach A, Kurtzberg J. Allogeneic stem cell transplantation with omidubicel in sickle cell disease. Blood Adv. 2021 Feb 9;5(3):843-852. doi: 10.1182/bloodadvances.2020003248.

    PMID: 33560399DERIVED

Related Links

MeSH Terms

Conditions

Anemia, Sickle CellThalassemiaGenetic Diseases, Inborn

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

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

Study Officials

  • Joanne Kurtzberg, MD

    Duke University Medical Center, NC, USA

    PRINCIPAL INVESTIGATOR

  • Joel Brochstein, MD

    Steven & Alexandra Cohen Children's Medical Center, New York

    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

April 15, 2012

First Posted

May 3, 2012

Study Start

September 1, 2012

Primary Completion

September 1, 2018

Study Completion

October 1, 2019

Last Updated

May 20, 2022

Results First Posted

April 27, 2022

Record last verified: 2022-04

Locations

US(3)