Trial Outcomes & Findings for PD and Safety of TG-0054 Combined With G-CSF in Multiple Myeloma, Non-Hodgkin Lymphoma and Hodgkin Disease Patients (NCT NCT02104427)

Last Updated: 2021-04-19

Results Overview

The primary efficacy endpoint was the proportion of patients from whom a total number of CD34+ cells ≥5.0 x 10\^6 cells/kg was collected within the first 4 leukapheresis sessions. For the primary efficacy endpoint, each patient's CD34+ cell number was calculated as the sum of CD34+ cell numbers collected from (up to) the first 4 leukapheresis sessions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Day 5 to Day 8

Results posted on

2021-04-19

Participant Flow

Participant milestones

Participant milestones
Measure	TG-0054 Combined With G-CSF 1\. G-CSF: 10 μg/kg/day, administrated via SC injections from Day 1 to Day 8; 2. TG-0054: 3.14 mg/kg, administrated via 15-min IV infusion from Day 5 to Day 9 as needed to reach the target collection goal.
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

PD and Safety of TG-0054 Combined With G-CSF in Multiple Myeloma, Non-Hodgkin Lymphoma and Hodgkin Disease Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	TG-0054 Combined With G-CSF n=12 Participants 1\. G-CSF: 10 μg/kg/day, administrated via SC injections from Day 1 to Day 8; 2. TG-0054: 3.14 mg/kg, administrated via 15-min IV infusion from Day 5 to Day 9 as needed to reach the target collection goal.
Age, Continuous	58 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Weight	82.4 kg n=5 Participants
BMI	29.3 kg/m^2 n=5 Participants

PRIMARY outcome

Timeframe: Day 5 to Day 8

Population: There were total 9 MM patients and 3 NHL or HD patients . A MM patient was treated with Revlimid, Following 2 leukapheresis sessions, a cumulative total of 0.18 x 10\^6 cells/kg were collected. PI and Medical Monitor didn't feel that further leukapheresis sessions would achieve more cells, so the patient didn't have additional sessions.

Outcome measures

Outcome measures
Measure	Within 1 Leukapheresis Session n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 1 leukapheresis session	Within 2 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 2 leukapheresis session	Within 3 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 3 leukapheresis session	Within 4 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 4 leukapheresis session	Within 5 Leukapheresis Sessions Cumulative CD34+ cell counts ≥6.0 x 10\^6 cells/kg within 5 leukapheresis session
Proportion of Patients From Whom a Total Number of CD34+ Cells ≥5.0 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions Overall	0.50000 Proportion of participants Interval 0.2109 to 0.7891	0.9167 Proportion of participants Interval 0.6152 to 0.9979	0.9167 Proportion of participants Interval 0.6152 to 0.9979	0.9167 Proportion of participants Interval 0.6152 to 0.9979	—
Proportion of Patients From Whom a Total Number of CD34+ Cells ≥5.0 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions Multiple Myeloma	0.6667 Proportion of participants Interval 0.2993 to 0.9251	0.8889 Proportion of participants Interval 0.5175 to 0.9972	0.8889 Proportion of participants Interval 0.5175 to 0.9972	0.8889 Proportion of participants Interval 0.5175 to 0.9972	—
Proportion of Patients From Whom a Total Number of CD34+ Cells ≥5.0 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions Non-Hodgkin Lymphoma or Hodgkin Disease	0.0000 Proportion of participants Interval 0.0 to 0.0	1.0000 Proportion of participants Interval 0.2924 to 1.0	1.0000 Proportion of participants Interval 0.2924 to 1.0	1.0000 Proportion of participants Interval 0.2924 to 1.0	—

SECONDARY outcome

Timeframe: Day 5 to Day 8

The secondary efficacy endpoint was the proportion of patients from whom a total number of CD34+ cells ≥2.5 x 10\^6 cells/kg was collected within the first 4 leukapheresis sessions. Each patient's CD34+ cell number was calculated as the sum of CD34+ cell numbers collected from (up to) the first 4 leukapheresis sessions.

Outcome measures

Outcome measures
Measure	Within 1 Leukapheresis Session n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 1 leukapheresis session	Within 2 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 2 leukapheresis session	Within 3 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 3 leukapheresis session	Within 4 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 4 leukapheresis session	Within 5 Leukapheresis Sessions Cumulative CD34+ cell counts ≥6.0 x 10\^6 cells/kg within 5 leukapheresis session
Proportion of Patients From Whom a Total Number of CD34+ Cells ≥2.5 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions Overall	0.9167 Proportion of patients Interval 0.6152 to 0.9979	0.9167 Proportion of patients Interval 0.6152 to 0.9979	0.9167 Proportion of patients Interval 0.6152 to 0.9979	0.9167 Proportion of patients Interval 0.6152 to 0.9979	—
Proportion of Patients From Whom a Total Number of CD34+ Cells ≥2.5 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions Multiple Myeloma	0.8889 Proportion of patients Interval 0.5175 to 0.9972	0.8889 Proportion of patients Interval 0.5175 to 0.9972	0.8889 Proportion of patients Interval 0.5175 to 0.9972	0.8889 Proportion of patients Interval 0.5175 to 0.9972	—
Proportion of Patients From Whom a Total Number of CD34+ Cells ≥2.5 x 10^6 Cells/kg Was Collected Within the First 4 Leukapheresis Sessions Non-Hodgkin Lymphoma or Hodgkin Disease	1.0000 Proportion of patients Interval 0.2924 to 1.0	1.0000 Proportion of patients Interval 0.2924 to 1.0	1.0000 Proportion of patients Interval 0.2924 to 1.0	1.0000 Proportion of patients Interval 0.2924 to 1.0	—

SECONDARY outcome

Timeframe: Day 5 to Day 9

The secondary efficacy endpoint was the proportion of patients from whom a total number of CD34+ cells ≥6.0 x 10\^6 cells/kg was collected within 5 leukapheresis sessions. Each patient's CD34+ cell number was calculated as the sum of CD34+ cell numbers collected from (up to) 5 leukapheresis sessions.

Outcome measures

Outcome measures
Measure	Within 1 Leukapheresis Session n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 1 leukapheresis session	Within 2 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 2 leukapheresis session	Within 3 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 3 leukapheresis session	Within 4 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 4 leukapheresis session	Within 5 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥6.0 x 10\^6 cells/kg within 5 leukapheresis session
Proportion of Patients Who Mobilized the Targeted Total Number of CD34+ Cells (≥6.0 x 10^6 Cells/kg) Within 5 Leukapheresis Sessions Multiple myeloma	0.6667 Proportion of patients Interval 0.2993 to 0.9251	0.8889 Proportion of patients Interval 0.5175 to 0.9972	0.8889 Proportion of patients Interval 0.5175 to 0.9972	0.8889 Proportion of patients Interval 0.5175 to 0.9972	0.8889 Proportion of patients Interval 0.5175 to 0.9972
Proportion of Patients Who Mobilized the Targeted Total Number of CD34+ Cells (≥6.0 x 10^6 Cells/kg) Within 5 Leukapheresis Sessions Overall	0.5000 Proportion of patients Interval 0.2109 to 0.7891	0.6667 Proportion of patients Interval 0.3489 to 0.9008	0.6667 Proportion of patients Interval 0.3489 to 0.9008	0.6667 Proportion of patients Interval 0.3489 to 0.9008	0.6667 Proportion of patients Interval 0.3489 to 0.9008
Proportion of Patients Who Mobilized the Targeted Total Number of CD34+ Cells (≥6.0 x 10^6 Cells/kg) Within 5 Leukapheresis Sessions Non-Hodgkin Lymphoma or Hodgkin Disease	0.0000 Proportion of patients Interval 0.0 to 0.0	0.0000 Proportion of patients Interval 0.0 to 0.0	0.0000 Proportion of patients Interval 0.0 to 0.0	0.0000 Proportion of patients Interval 0.0 to 0.0	0.0000 Proportion of patients Interval 0.0 to 0.0

SECONDARY outcome

Timeframe: Day 5 (1st leukapheresis session) to Day 6 (2nd leukapheresis session)

Population: There were 5 patients who achived target number of CD34+ cells (\>=5.0\*10\^6 cells/kg) within two leukapheresis sessions and therefore didn't undergo addtional sessions. Among 7 patients who undergone 6 sessions, there were 1 patient didn't collect blood sample at 4h, 6h post-infusion and post-leukapheresis, and 1 patient 6h post-infusion not done.

determine circulating CD34+ cell counts in peripheral blood

Outcome measures

Outcome measures
Measure	Within 1 Leukapheresis Session n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 1 leukapheresis session	Within 2 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 2 leukapheresis session	Within 3 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 3 leukapheresis session	Within 4 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥5.0 x 10\^6 cells/kg within 4 leukapheresis session	Within 5 Leukapheresis Sessions n=12 Participants Cumulative CD34+ cell counts ≥6.0 x 10\^6 cells/kg within 5 leukapheresis session
the Pharmacodynamics (PD) Following Treatment With TG-0054 When Combined With G-CSF Day 5 (1st leukapheresis session)	32.50 cells/µL Interval 1.0 to 123.0	43.50 cells/µL Interval 0.0 to 141.0	42.00 cells/µL Interval 1.0 to 122.0	60.50 cells/µL Interval 1.0 to 206.0	35.50 cells/µL Interval 3.0 to 119.0
the Pharmacodynamics (PD) Following Treatment With TG-0054 When Combined With G-CSF Day 6 (2nd leukapheresis session)	16.00 cells/µL Interval 1.0 to 59.5	28.00 cells/µL Interval 14.0 to 45.0	20.00 cells/µL Interval 0.0 to 45.0	34.00 cells/µL Interval 2.0 to 70.0	21.00 cells/µL Interval 16.0 to 45.0

Adverse Events

10 µg/kg/Day G-CSF Alone

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

10 µg/kg/Day G-CSF + 3.14 mg/kg TG-0054

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Overall

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	10 µg/kg/Day G-CSF Alone n=12 participants at risk Days 1 through 4	10 µg/kg/Day G-CSF + 3.14 mg/kg TG-0054 n=12 participants at risk Day 5 until end of study	Overall n=12 participants at risk
Metabolism and nutrition disorders Hypocalcaemia	8.3% 1/12 • Number of events 1	0.00% 0/12	8.3% 1/12 • Number of events 1
Musculoskeletal and connective tissue disorders Bone pain	16.7% 2/12 • Number of events 2	0.00% 0/12	16.7% 2/12 • Number of events 2
Gastrointestinal disorders Diarrhoea	0.00% 0/12	8.3% 1/12 • Number of events 1	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Dry mouth	0.00% 0/12	8.3% 1/12 • Number of events 1	8.3% 1/12 • Number of events 1
Gastrointestinal disorders Nausea	0.00% 0/12	8.3% 1/12 • Number of events 1	8.3% 1/12 • Number of events 1
Metabolism and nutrition disorders Hypokalaemia	0.00% 0/12	8.3% 1/12 • Number of events 1	8.3% 1/12 • Number of events 1
General disorders Oedema peripheral	0.00% 0/12	8.3% 1/12 • Number of events 1	8.3% 1/12 • Number of events 1
Investigations Platelet count decreased	0.00% 0/12	8.3% 1/12 • Number of events 1	8.3% 1/12 • Number of events 1
Nervous system disorders Paraesthesia	0.00% 0/12	8.3% 1/12 • Number of events 1	8.3% 1/12 • Number of events 1

Additional Information

Li-Wen Chang

TaiGen Biotechnology Co., Ltd.

Phone: +886-2-8177-7072

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60