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Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies

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ClinicalTrials.gov Identifier: NCT01221857
Recruitment Status : Completed
First Posted : October 15, 2010
Results First Posted : February 26, 2019
Last Update Posted : April 23, 2019
Sponsor:
Information provided by (Responsible Party):
Gamida Cell ltd

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Acute Lymphoblastic Leukemia (ALL)
Acute Myelogenous Leukemia (AML)
Myelodysplastic Syndrome (MDS)
Non-Hodgkin's Lymphoma
Hodgkin's Disease
Intervention Drug: NiCord®
Enrollment 12
Recruitment Details  
Pre-assignment Details 12 patients were enrolled/transplanted; efficacy results were summarized for 11 patients treated with NiCord. 1 patient was transplanted with unmanipulated cord only. Safety results were summarized for all patients.
Arm/Group Title NiCord
Hide Arm/Group Description NiCord: NiCord is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Period Title: Overall Study
Started 12
Completed [1] 12
Not Completed 0
[1]
1 patient received unmanipulated cord only.
Arm/Group Title NiCord
Hide Arm/Group Description NiCord®: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 12 participants
45
(21 to 61)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
<=18 years
0
   0.0%
Between 18 and 65 years
12
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
6
  50.0%
Male
6
  50.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 12 participants
12
 100.0%
1.Primary Outcome
Title Acute Toxicity Associated With the Infusion of NiCord
Hide Description Acute toxicity associated with the infusion of NiCord will be measured by adverse events within 24 hours post-infusion, defined as the acute toxicity period. Known adverse events associated with myeloablation and cord blood transplant were specifically monitored including fever, chills, allergic reaction/hypersensitivity, anaphylaxis, sinus bradycardia, sinus tachycardia, hypertension, hypotension, nausea, vomiting, diarrhea, dyspnea, hypoxia, hemoglobinuria, infection, flank pain and any other skin, CNS, cardiac, pulmonary or other toxicity manifestations.
Time Frame 180 days post-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
Patients transplanted with NiCord
Arm/Group Title NiCord
Hide Arm/Group Description:
Analysis population is patients transplanted with NiCord plus unmanipulated cord blood unit.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
0
2.Primary Outcome
Title Proportion of Patients With Neutrophil Engraftment
Hide Description Neutrophil engraftment was defined as achieving an Absolute Neutrophil Count (ANC) of ≥500 mm3 for 3 consecutive measurements on different days by day 42 inclusive (the day of engraftment was defined as the first of these 3 days). The ANC recovery must be of donor origin documented by peripheral blood chimerism assays indicating less than or equal to 10% host cells in peripheral blood.
Time Frame 42 days
Hide Outcome Measure Data
Hide Analysis Population Description
Patients transplanted with NiCord
Arm/Group Title Neutrophil Engraftment
Hide Arm/Group Description:
Patients transplanted with NiCord plus unmanipulated cord blood unit.
Overall Number of Participants Analyzed 11
Measure Type: Number
Unit of Measure: proportion of patients
0.91
3.Secondary Outcome
Title Proportion of Patients Who Developed Acute GvHD Grade II-IV and III-IV
Hide Description

Acute GvHD was assessed from transplantation (day 0) until day 99 post-transplant or more frequently as clinically indicated. GvHD was classified according to the Glucksberg Classification (Glucksberg, Storb et al. 1974).

The overall grade of GvHD, however, was determined by an assessment of skin disease, liver disease and gastrointestinal manifestations.

Time Frame 180 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Grade II-IV GvHD Grade III-IV GvHD
Hide Arm/Group Description:
All patients transplanted were assessed for acute GvHD grade II-IV.
All patients transplanted were assessed for acute GvHD grade III-IV.
Overall Number of Participants Analyzed 11 11
Measure Type: Number
Unit of Measure: proportion of patients
0.45 0
4.Secondary Outcome
Title Non-relapse Mortality
Hide Description Proportion of patients who had non-relapse mortality at 100 days.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Non-relapse Mortality
Hide Arm/Group Description:
proportion of patients with non-relapse mortality at 100 days
Overall Number of Participants Analyzed 11
Measure Type: Number
Unit of Measure: proportion of patients
0.09
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title NiCord
Hide Arm/Group Description NiCord®: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
All-Cause Mortality
NiCord
Affected / at Risk (%)
Total   3/12 (25.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
NiCord
Affected / at Risk (%) # Events
Total   8/12 (66.67%)    
Blood and lymphatic system disorders   
Graft rejection   1/12 (8.33%)  1
Cardiac disorders   
Hypertension   2/12 (16.67%)  2
Supraventricular tachycardia   1/12 (8.33%)  1
Gastrointestinal disorders   
Gastrointestinal Disorder   2/12 (16.67%)  2
Infections and infestations   
Pneumonia   1/12 (8.33%)  1
Investigations   
Disease relapse   1/12 (8.33%)  1
Psychiatric disorders   
Depression   1/12 (8.33%)  1
Altered mental status   1/12 (8.33%)  1
Renal and urinary disorders   
Hematuria   1/12 (8.33%)  1
Acute renal failure   1/12 (8.33%)  1
Respiratory, thoracic and mediastinal disorders   
Hemoptysis   1/12 (8.33%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
NiCord
Affected / at Risk (%) # Events
Total   11/12 (91.67%)    
Cardiac disorders   
Hypertension   6/12 (50.00%)  7
Hypotension   1/12 (8.33%)  1
Tachycardia   3/12 (25.00%)  3
Sinus bradycardia   1/12 (8.33%)  1
Arrhythmia   1/12 (8.33%)  1
Gastrointestinal disorders   
Nausea   4/12 (33.33%)  4
Elevated LFTs   1/12 (8.33%)  1
CMV gastritis   1/12 (8.33%)  1
Immune system disorders   
aGvHD   7/12 (58.33%)  10
Infections and infestations   
CMV reactivation   6/12 (50.00%)  6
HHV-6 Reactivation   6/12 (50.00%)  6
Staph Coagulase Infection   2/12 (16.67%)  3
BK virus infection   2/12 (16.67%)  2
E coli infection   1/12 (8.33%)  1
RSV infection   1/12 (8.33%)  1
Pseudomonas infection   1/12 (8.33%)  1
Enterococcus infection   1/12 (8.33%)  1
Investigations   
Hypomagnesemia   4/12 (33.33%)  4
Hypocalcemia   3/12 (25.00%)  3
Hypoalbuminemia   1/12 (8.33%)  1
Renal and urinary disorders   
Hematuria   2/12 (16.67%)  2
Proteinuria   3/12 (25.00%)  3
Cystitis   1/12 (8.33%)  1
Respiratory, thoracic and mediastinal disorders   
Hypoxia   1/12 (8.33%)  1
Dyspnea   1/12 (8.33%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Kelly Myers
Organization: Gamida Cell
Phone: +972-2-659-5631
Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT01221857     History of Changes
Other Study ID Numbers: GC P#01.01.020
First Submitted: October 14, 2010
First Posted: October 15, 2010
Results First Submitted: May 24, 2017
Results First Posted: February 26, 2019
Last Update Posted: April 23, 2019