Trial Outcomes & Findings for Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies (NCT NCT01590628)

NCT ID: NCT01590628

Last Updated: 2022-05-20

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

16 participants

Primary outcome timeframe

24 hours post-infusion

Results posted on

2022-05-20

Participant Flow

The study enrolled and treated 16 patients in total. 13 patients were treated with omidubicel and an unmanipulated CBU and these served as the primary analysis population for all study endpoints. Three additional patients who were exposed to omidubicel as a single graft source, were included in the safety summaries in order to encompass the entire population exposed to omidubicel.

Participant milestones

Participant milestones
Measure	NiCord + Unmanipulated CBU NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 1 subjects, together with an unmanipulated CBU.	NiCord NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 2 subjects as a standalone graft.
Overall Study STARTED	13	3
Overall Study COMPLETED	11	3
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Reasons for withdrawal
Measure	NiCord + Unmanipulated CBU NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 1 subjects, together with an unmanipulated CBU.	NiCord NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. NiCord was transplanted in Part 2 subjects as a standalone graft.
Overall Study Death	2	0

Baseline Characteristics

Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	NiCord + Unmanipulated CBU n=13 Participants NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. The NiCord was transplanted together with an unmanipulated CBU graft.	NiCord n=3 Participants NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. The NiCord was transplanted as a standalone product.	Total n=16 Participants Total of all reporting groups
Age, Categorical <=18 years	13 Participants n=39 Participants	3 Participants n=41 Participants	16 Participants n=35 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Age, Categorical >=65 years	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Sex: Female, Male Female	9 Participants n=39 Participants	1 Participants n=41 Participants	10 Participants n=35 Participants
Sex: Female, Male Male	4 Participants n=39 Participants	2 Participants n=41 Participants	6 Participants n=35 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=39 Participants	3 Participants n=41 Participants	16 Participants n=35 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Asian	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Black or African American	13 Participants n=39 Participants	3 Participants n=41 Participants	16 Participants n=35 Participants
Race (NIH/OMB) White	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Region of Enrollment United States	13 Participants n=39 Participants	3 Participants n=41 Participants	16 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Hb SS	13 Participants n=39 Participants	2 Participants n=41 Participants	15 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Hb SC	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Hb SBeta0 Thal	0 Participants n=39 Participants	1 Participants n=41 Participants	1 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Hb SD	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Hb SOarab	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Other SCD Type	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Beta Thal Major	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Sickle Cell or Thalassemia Disease Type Alpha Thal Major	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
CMV Screen (IgG or Total) (Segment S) positive	7 Participants n=39 Participants	2 Participants n=41 Participants	9 Participants n=35 Participants
Performance Status 100	6 Participants n=39 Participants	2 Participants n=41 Participants	8 Participants n=35 Participants
Performance Status 90	3 Participants n=39 Participants	0 Participants n=41 Participants	3 Participants n=35 Participants
Performance Status 80	4 Participants n=39 Participants	0 Participants n=41 Participants	4 Participants n=35 Participants
Performance Status 70	0 Participants n=39 Participants	1 Participants n=41 Participants	1 Participants n=35 Participants
Disease characteristics Three or More Recurrent Painful Events in the Past 2 Years	6 participants n=39 Participants	2 participants n=41 Participants	8 participants n=35 Participants
Disease characteristics Acute Chest Syndrome (Two or More Episodes in the Past 2 Years)	2 participants n=39 Participants	0 participants n=41 Participants	2 participants n=35 Participants
Disease characteristics SCD-related & clinically significant neurologic event	4 participants n=39 Participants	1 participants n=41 Participants	5 participants n=35 Participants
Disease characteristics Abnormal MRI/MRA	5 participants n=39 Participants	1 participants n=41 Participants	6 participants n=35 Participants
Disease characteristics chronic PRBC transfusion therapy	0 participants n=39 Participants	0 participants n=41 Participants	0 participants n=35 Participants
Disease characteristics Patient has undergone TCD velocity tests	1 participants n=39 Participants	0 participants n=41 Participants	1 participants n=35 Participants
Hydroxyurea use prior to enrollment	10 Participants n=39 Participants	2 Participants n=41 Participants	12 Participants n=35 Participants

PRIMARY outcome

Timeframe: 24 hours post-infusion

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

Outcome measures

Outcome measures
Measure	NiCord + Unmanipulated CBU n=13 Participants Participants who received NiCord together with an unmanipulated CBU.	NiCord n=3 Participants Participants who received NiCord as a standalone graft.
Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. Grade 4-5 acute toxicity event	0 participants	0 participants
Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. Grade 3 acute toxicity event	3 participants	0 participants

PRIMARY outcome

Timeframe: By Day 42

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).

Outcome measures

Outcome measures
Measure	NiCord + Unmanipulated CBU n=13 Participants Participants who received NiCord together with an unmanipulated CBU.	NiCord n=3 Participants Participants who received NiCord as a standalone graft.
Assessment of Cumulative Incidence of Donor-derived Neutrophil Engraftment.	13 Participants	3 Participants

SECONDARY outcome

Timeframe: at 100 days

Transplant-related mortality is defined as death not preceded by autologous recovery.

Outcome measures

Outcome measures
Measure	NiCord + Unmanipulated CBU n=13 Participants Participants who received NiCord together with an unmanipulated CBU.	NiCord n=3 Participants Participants who received NiCord as a standalone graft.
Proportion of Transplant-related Mortality.	0 percentage of participants	0 percentage of participants

SECONDARY outcome

Timeframe: 100 days post-transplant

Population: In the first group, 13 patients received NiCord together with a second unmanipulated CBU. In the 2nd group, 3 patients received NiCord as a standalone graft. No statistical analysis was performed for this outcome as this number of patients is too small for analysis.

Patients with event-free survival at 100 days post-transplant that did not have one of the following events: death, autologous recovery, primary or secondary graft failure.

Outcome measures

Outcome measures
Measure	NiCord + Unmanipulated CBU n=13 Participants Participants who received NiCord together with an unmanipulated CBU.	NiCord n=3 Participants Participants who received NiCord as a standalone graft.
Event-free Survival	92.3 percentage of participants Interval 56.6 to 98.9	66.6 percentage of participants No statistical analysis was performed for this outcome as this number of patients is too small for analysis.

SECONDARY outcome

Timeframe: 180 days

Population: In the first group, 13 patients received NiCord together with a second unmanipulated CBU. In the 2nd group, 3 patients received NiCord as a standalone graft.

Overall survival at 180 days

Outcome measures

Outcome measures
Measure	NiCord + Unmanipulated CBU n=13 Participants Participants who received NiCord together with an unmanipulated CBU.	NiCord n=3 Participants Participants who received NiCord as a standalone graft.
Overall Survival	92.3 percentage of participants Interval 56.6 to 98.9	100 percentage of participants Due to the insufficient number of patients who received a standalone NiCord transplant (3 in total), no statistical anlysis was performed.

Adverse Events

NiCord + Unmanipulated CBU

Serious events: 13 serious events

Other events: 0 other events

Deaths: 2 deaths

NiCord

Serious events: 3 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Adverse event data not reported

Additional Information

Kelly Myers, CRA

Gamida Cell

Phone: 026595631

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	NiCord + Unmanipulated CBU n=13 participants at risk NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. The primary safety objective was to assess the acute toxicity associated with the infusion of omidubicel within 24 hours post-infusion. Part 1: The primary efficacy objective of Part 1 was to assess the cumulative incidence of donor-derived neutrophil engraftment by Day 42 following co-transplantation of omidubicel and unmanipulated cord blood grafts.	NiCord n=3 participants at risk NiCord: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells. The primary safety objective was to assess the acute toxicity associated with the infusion of omidubicel within 24 hours post-infusion. Part 2: The primary efficacy objective of Part 2 was to assess the cumulative incidence of donor-derived neutrophil engraftment by Day 42 following transplantation of single unit omidubicel.
Gastrointestinal disorders Anal fissure	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Gastrointestinal disorders Nausea	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
General disorders Pyrexia	15.4% 2/13 • Number of events 2 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Immune system disorders Acute graft versus host disease	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Immune system disorders Anaphylactic reaction	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Immune system disorders Graft versus host disease	23.1% 3/13 • Number of events 3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Immune system disorders Transplant rejection	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	33.3% 1/3 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Infections and infestations Bacteraemia	23.1% 3/13 • Number of events 3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Infections and infestations Gastroenteritis	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Infections and infestations Gastroenteritis adenovirus	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Infections and infestations Infection	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Infections and infestations Pseudomonal bacteraemia	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Infections and infestations Sepsis	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Infections and infestations Sinusitis	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Injury, poisoning and procedural complications Subdural haematoma	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Metabolism and nutrition disorders Hyperglycaemia	23.1% 3/13 • Number of events 3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Musculoskeletal and connective tissue disorders Osteonecrosis	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Nervous system disorders Headache	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Nervous system disorders Posterior reversible encephalopathy syndrome	23.1% 3/13 • Number of events 3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Nervous system disorders Seizure	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Vascular disorders Hypovolaemic shock	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Vascular disorders Venous thrombosis	7.7% 1/13 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	0.00% 0/3 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Renal and urinary disorders Renal Failure	0.00% 0/13 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	33.3% 1/3 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/13 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	33.3% 1/3 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).
Vascular disorders Hypotension	0.00% 0/13 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).	33.3% 1/3 • Number of events 1 • adverse event data were collected throughout the post-transplant period (180 days) and 6 months post study completion (1 year post transplantation).