Trial Outcomes & Findings for Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies (NCT NCT01221857)

Last Updated: 2021-08-03

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

12 participants

Primary outcome timeframe

180 days post-transplant

Results posted on

2021-08-03

Participant Flow

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.

Participant milestones

Participant milestones
Measure	NiCord NiCord: NiCord is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	NiCord n=12 Participants NiCord®: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Age, Continuous	45 years n=93 Participants
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	12 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	6 Participants n=93 Participants
Sex: Female, Male Male	6 Participants n=93 Participants
Region of Enrollment United States	12 Participants n=93 Participants

PRIMARY outcome

Timeframe: 180 days post-transplant

Population: Patients transplanted with NiCord

Outcome measures

Outcome measures
Measure	NiCord n=11 Participants Analysis population is patients transplanted with NiCord plus unmanipulated cord blood unit.	Grade III-IV GvHD All patients transplanted were assessed for acute GvHD grade III-IV.
Acute Toxicity Associated With the Infusion of NiCord	0 Participants	—

PRIMARY outcome

Timeframe: 42 days

Population: Patients transplanted with NiCord

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.

Outcome measures

Outcome measures
Measure	NiCord n=11 Participants Analysis population is patients transplanted with NiCord plus unmanipulated cord blood unit.	Grade III-IV GvHD All patients transplanted were assessed for acute GvHD grade III-IV.
Proportion of Patients With Neutrophil Engraftment	0.91 proportion of patients	—

SECONDARY outcome

Timeframe: 180 days

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.

Outcome measures

Outcome measures
Measure	NiCord n=11 Participants Analysis population is patients transplanted with NiCord plus unmanipulated cord blood unit.	Grade III-IV GvHD n=11 Participants All patients transplanted were assessed for acute GvHD grade III-IV.
Proportion of Patients Who Developed Acute GvHD Grade II-IV and III-IV	0.45 proportion of patients	0 proportion of patients

SECONDARY outcome

Timeframe: 100 days

Proportion of patients who had non-relapse mortality at 100 days.

Outcome measures

Outcome measures
Measure	NiCord n=11 Participants Analysis population is patients transplanted with NiCord plus unmanipulated cord blood unit.	Grade III-IV GvHD All patients transplanted were assessed for acute GvHD grade III-IV.
Non-relapse Mortality	0.09 proportion of patients	—

Adverse Events

NiCord

Serious events: 8 serious events

Other events: 11 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	NiCord n=12 participants at risk NiCord®: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Infections and infestations Pneumonia	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Gastrointestinal Disorder	16.7% 2/12 • Number of events 2
Psychiatric disorders Depression	8.3% 1/12 • Number of events 1
Cardiac disorders Hypertension	16.7% 2/12 • Number of events 2
Psychiatric disorders Altered mental status	8.3% 1/12 • Number of events 1
Respiratory, thoracic and mediastinal disorders Hemoptysis	8.3% 1/12 • Number of events 1
Blood and lymphatic system disorders Graft rejection	8.3% 1/12 • Number of events 1
Cardiac disorders Supraventricular tachycardia	8.3% 1/12 • Number of events 1
Renal and urinary disorders Hematuria	8.3% 1/12 • Number of events 1
Renal and urinary disorders Acute renal failure	8.3% 1/12 • Number of events 1
Investigations Disease relapse	8.3% 1/12 • Number of events 1

Other adverse events

Other adverse events
Measure	NiCord n=12 participants at risk NiCord®: NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.
Cardiac disorders Hypertension	50.0% 6/12 • Number of events 7
Investigations Hypomagnesemia	33.3% 4/12 • Number of events 4
Immune system disorders aGvHD	58.3% 7/12 • Number of events 10
Infections and infestations CMV reactivation	50.0% 6/12 • Number of events 6
Gastrointestinal disorders Nausea	33.3% 4/12 • Number of events 4
Cardiac disorders Hypotension	8.3% 1/12 • Number of events 1
Cardiac disorders Tachycardia	25.0% 3/12 • Number of events 3
Infections and infestations HHV-6 Reactivation	50.0% 6/12 • Number of events 6
Infections and infestations Staph Coagulase Infection	16.7% 2/12 • Number of events 3
Investigations Hypocalcemia	25.0% 3/12 • Number of events 3
Infections and infestations BK virus infection	16.7% 2/12 • Number of events 2
Gastrointestinal disorders Elevated LFTs	8.3% 1/12 • Number of events 1
Infections and infestations E coli infection	8.3% 1/12 • Number of events 1
Renal and urinary disorders Hematuria	16.7% 2/12 • Number of events 2
Cardiac disorders Sinus bradycardia	8.3% 1/12 • Number of events 1
Renal and urinary disorders Proteinuria	25.0% 3/12 • Number of events 3
Cardiac disorders Arrhythmia	8.3% 1/12 • Number of events 1
Infections and infestations RSV infection	8.3% 1/12 • Number of events 1
Infections and infestations Pseudomonas infection	8.3% 1/12 • Number of events 1
Infections and infestations Enterococcus infection	8.3% 1/12 • Number of events 1
Gastrointestinal disorders CMV gastritis	8.3% 1/12 • Number of events 1
Investigations Hypoalbuminemia	8.3% 1/12 • Number of events 1
Renal and urinary disorders Cystitis	8.3% 1/12 • Number of events 1
Respiratory, thoracic and mediastinal disorders Hypoxia	8.3% 1/12 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	8.3% 1/12 • Number of events 1

Additional Information

Kelly Myers

Gamida Cell

Phone: +972-2-659-5631

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place