Trial Outcomes & Findings for Topical Raplixa for Surgical Bleeding in Children (NCT NCT02117349)

NCT ID: NCT02117349

Last Updated: 2020-02-05

Results Overview

Count of Participants who Reached Hemostasis at the TBS within 4 minutes of the first study drug application

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 55 participants
• Primary outcome timeframe: within 4 minutes
• Results posted on: 2020-02-05

Participant Flow

Of 87 planned participants, only 55 were randomized before the trial was terminated early due to device related (non-safety) issues

Participant milestones

Measure	Raplixa Plus Gelfoam Participants treated with Raplixa in addition to Gelfoam	Gelfoam Alone Participants treated only with Gelfoam
Overall Study STARTED	37	18
Overall Study Intent-to-treat Population	37	18
Overall Study Safety Population	36	18
Overall Study Modified Intent-to-treat (mITT)	36	18
Overall Study COMPLETED	34	17
Overall Study NOT COMPLETED	3	1

Reasons for withdrawal

Measure	Raplixa Plus Gelfoam Participants treated with Raplixa in addition to Gelfoam	Gelfoam Alone Participants treated only with Gelfoam
Overall Study Withdrawal by Subject	1	0
Overall Study Lost to Follow-up	1	1
Overall Study Death	1	0

Baseline Characteristics

Topical Raplixa for Surgical Bleeding in Children

Baseline characteristics by cohort

Measure	Raplixa Plus Gelfoam n=36 Participants Participants treated with Raplixa in addition to Gelfoam	Gelfoam Alone n=18 Participants Participants treated only with Gelfoam	Total n=54 Participants Total of all reporting groups
Age, Continuous	6.6 years n=5 Participants	9.7 years n=7 Participants	6.9 years n=5 Participants
Age, Customized Age Cohorts · Cohort 1 = 11 to < 18 years	13 Participants n=5 Participants	9 Participants n=7 Participants	22 Participants n=5 Participants
Age, Customized Age Cohorts · Cohort 2 = 2 to < 11 years	19 Participants n=5 Participants	9 Participants n=7 Participants	28 Participants n=5 Participants
Age, Customized Age Cohorts · Cohort 3 = 6 months to < 2 years	4 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants
Age, Customized Age Cohorts · Cohort 4 = Birth to < 6 months	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants	9 Participants n=7 Participants	24 Participants n=5 Participants
Sex: Female, Male Male	21 Participants n=5 Participants	9 Participants n=7 Participants	30 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	13 Participants n=5 Participants	8 Participants n=7 Participants	21 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	22 Participants n=5 Participants	10 Participants n=7 Participants	32 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Race (NIH/OMB) White	26 Participants n=5 Participants	9 Participants n=7 Participants	35 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	6 Participants n=5 Participants	3 Participants n=7 Participants	9 Participants n=5 Participants

PRIMARY outcome

Timeframe: within 4 minutes

Population: modified Intent to Treat (mITT)

Count of Participants who Reached Hemostasis at the TBS within 4 minutes of the first study drug application

Outcome measures

Measure	Raplixa Plus Gelfoam n=36 Participants Participants treated with Raplixa in addition to Gelfoam	Gelfoam Alone n=18 Participants Participants treated only with Gelfoam
Number of Participants Who Reached Hemostasis at the Target Bleeding Site (TBS) Within 4 Minutes	30 Participants	5 Participants

SECONDARY outcome

Timeframe: within 5 minutes

Population: mITT

Count of Participants who Reached Hemostasis at the TBS within 5 minutes of the first study drug application

Outcome measures

Measure	Raplixa Plus Gelfoam n=36 Participants Participants treated with Raplixa in addition to Gelfoam	Gelfoam Alone n=18 Participants Participants treated only with Gelfoam
Number of Participants Who Reached Hemostasis at the TBS Within 5 Minutes	33 Participants	7 Participants

SECONDARY outcome

Timeframe: within 97 days

Population: Safety Population

Clinically significant changes in safety measures are recorded as adverse events. Participants with TEAEs that are deemed by the principal investigator or the independent data monitoring committee as possibly or definitely treatment-related (including device-related) are included in the count for this secondary outcome measure.

Outcome measures

Measure	Raplixa Plus Gelfoam n=36 Participants Participants treated with Raplixa in addition to Gelfoam	Gelfoam Alone n=18 Participants Participants treated only with Gelfoam
Number of Participants With Treatment-Related Treatment-Emergent Adverse Events (TEAEs)	0 Participants	0 Participants

Adverse Events

Raplixa Plus Gelfoam

Serious events: 7 serious events

Other events: 0 other events

Deaths: 1 deaths

Gelfoam Alone

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Raplixa Plus Gelfoam n=36 participants at risk Participants treated with Raplixa in addition to Gelfoam	Gelfoam Alone n=18 participants at risk Participants treated only with Gelfoam
Gastrointestinal disorders Ascites	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Gastrointestinal disorders Intestinal perforation	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Gastrointestinal disorders Intussusception	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Infections and infestations Gastrointestinal norovirus	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	0.00% 0/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Infections and infestations Incision site cellulitis	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Infections and infestations Stoma site cellulitis	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Injury, poisoning and procedural complications Post procedural bile leak	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Injury, poisoning and procedural complications Procedural haemorrhage	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Investigations Gastrointestinal stoma output abnormal	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).
Metabolism and nutrition disorders Dehydration	2.8% 1/36 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).	5.6% 1/18 • within 97 days All-cause mortality is reported in the Intent-to-treat population (37/18) to include one who was randomized, but died before treatment. Serious and Other Adverse events are reported in the Safety population (36/18), defined as randomized participants who were exposed to study drug (group based on actual treatment received).

Other adverse events

Adverse event data not reported

Additional Information

Medical Information Call Center

Mallinckrodt Pharmaceuticals

Phone: 800-556-3314

Email: clinicaltrials@mnk.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place