Trial Outcomes & Findings for A Phase 3, Randomized, Double-blind, Placebo-controlled Study For Subjects With Locally-advanced Unresectable or Metastatic Synovial Sarcoma (V943-003, IMDZ-04-1702) (NCT NCT03520959)

Results Overview

PFS is defined as the time from randomization to the investigator-determined date of disease progression or death, whichever comes first, using Response Evaluation Criteria in Solid Tumors (RECIST v1.1).

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

1 participants

Primary outcome timeframe

From randomization to investigator-determined date of disease progression or death, assessed up to 24 months.

Results posted on

2020-04-16

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo CMB305 placebo control	CMB305 CMB305: Sequentially administered LV305 \[lentiviral vector encoding New York esophogeal squamous cell carcinoma-1 {NY-ESO-1} gene\] and G305 \[NY-ESO-1 recombinant protein plus glucopyranosyl lipid A stable emulsion {GLA-SE}\]
Overall Study STARTED	0	1
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo CMB305 placebo control	CMB305 CMB305: Sequentially administered LV305 \[lentiviral vector encoding New York esophogeal squamous cell carcinoma-1 {NY-ESO-1} gene\] and G305 \[NY-ESO-1 recombinant protein plus glucopyranosyl lipid A stable emulsion {GLA-SE}\]
Overall Study Study terminated by Sponsor	0	1

Baseline Characteristics

A Phase 3, Randomized, Double-blind, Placebo-controlled Study For Subjects With Locally-advanced Unresectable or Metastatic Synovial Sarcoma (V943-003, IMDZ-04-1702)

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: From randomization to investigator-determined date of disease progression or death, assessed up to 24 months.

Population: No data were collected or analyzed for this outcome measure due to early termination of the study.

PFS is defined as the time from randomization to the investigator-determined date of disease progression or death, whichever comes first, using Response Evaluation Criteria in Solid Tumors (RECIST v1.1).

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: From randomization to date of death, assessed up to 66 months.

Population: No data were collected or analyzed for this outcome measure due to early termination of the study.

OS is defined as the time from randomization to the date of death.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From last dose of CMB305 to initiation of new therapy, assessed up to 24 months.

Population: No data were collected or analyzed for this outcome measure due to early termination of the study.

TTNT is defined as the time from randomization to the start of post-study treatment subsequent intervention: \[TTNT = start date of subsequent intervention - randomization date + 1\]. Subsequent intervention includes anticancer therapy, cancer-related surgery and local regional therapy. Participants who do not start any post-study treatment intervention will be censored at their last known date of being alive.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From randomization to investigator-determined date of disease progression or death, assessed up to 24 months.

Population: No data were collected or analyzed for this outcome measure due to early termination of the study.

DMFS is defined as the time from randomization to evidence of a new distant metastasis not documented at time of randomization: \[DMFS = a new distant metastasis documented date - randomization date + 1\]. Participants who do not have any new distant metastasis will be censored at their last tumor assessment.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From randomization to investigator-determined date of disease progression, assessed up to 24 months.

Population: No data were collected or analyzed for this outcome measure due to early termination of the study.

ORR defined by RECIST v1.1 will be summarized by the number and percent of subjects who achieve a complete response (CR) or partial response (PR) based on the investigator's assessment. ORR will be compared between treatment arms using a logistic regression.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From randomization to investigator-determined date of disease progression or death, assessed up to approximately 2 months.

Population: All participants taking any amount of study drug.

Safety will be assessed primarily based on reported adverse events (AEs), Medical Events of Interest (MEOIs), laboratory values, and concomitant medications reported from initiation of treatment with CMB305 or placebo.

Outcome measures

Outcome measures
Measure	Placebo CMB305 placebo control	CMB305 n=1 Participants CMB305: Sequentially administered LV305 \[lentiviral vector encoding New York esophogeal squamous cell carcinoma-1 {NY-ESO-1} gene\] and G305 \[NY-ESO-1 recombinant protein plus glucopyranosyl lipid A stable emulsion {GLA-SE}\]
Number of Participants Who Experienced a Treatment-Emergent Adverse Event (TEAE)	—	1 Participants

SECONDARY outcome

Timeframe: From Day 1 up to 12 months

Population: No data were collected or analyzed for this outcome measure due to early termination of the study.

QoL evaluated using the EQ-5D-5L for participants ≥18 years of age or using the EQ-5D-Y for participants 12 to \<18 years of age. EQ-5D-5L descriptive system is comprised of 5 dimensions-mobility, self-care, usual activities, pain/discomfort \& anxiety/depression. Each dimension has 5 levels: not at all (level 1), mild (level 2), moderate (level 3), severe (level 4), extreme/leading to incapacity (level 5), with highest level corresponding to worst outcome. Participants indicated their health state by choosing the appropriate level from each dimension. The 5 digit health states thus obtained for each dimension were then converted into a single median index value using the EQ-5D-5L crosswalk index value calculator as recommended by EuroQol group. In the EQ-VAS, participants recorded their health state on a scale ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to approximately 2 months

Population: All participants taking any amount of study drug.

The number of all participants who discontinued study treatment due to an AE is presented.

Outcome measures

Outcome measures
Measure	Placebo CMB305 placebo control	CMB305 n=1 Participants CMB305: Sequentially administered LV305 \[lentiviral vector encoding New York esophogeal squamous cell carcinoma-1 {NY-ESO-1} gene\] and G305 \[NY-ESO-1 recombinant protein plus glucopyranosyl lipid A stable emulsion {GLA-SE}\]
Number of Participants Who Discontinued Study Treatment Due to an AE	—	0 Participants

Adverse Events

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

CMB305

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Senior Vice President, Global Clinical Development

Merck Sharp & Dohme Corp.

Phone: 1-800-672-6372

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60