Trial Outcomes & Findings for Efficacy and Safety of CUSA-081 in the Restoration of Central Venous Access Device (CVAD) Functionality (NCT NCT03594175)

Last Updated: 2024-08-23

Results Overview

CUSA-081 vs Placebo -- Single instillation of study drug -- Dwell Time Up To 90 Min -- Full Analysis Set (FAS) Treatment success was defined as the restoration of CVAD functionality, measured as the ability to withdraw 3 mL of blood and infuse 5 mL of saline. For this assessment, dwell time was up to 90 mins, after a single instillation of study drug. The percentage was calculated as the number of participants with treatment success divided by the total number of participants in the group, multiplied by 100%.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

462 participants

Primary outcome timeframe

Day 1 (up to 90 mins post dose)

Results posted on

2024-08-23

Participant Flow

Subjects who qualified for the study after completing of all screening assessments were randomized to treatment groups and received the randomized study drug.

Participant milestones

Participant milestones
Measure	CUSA-081 Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Overall Study STARTED	261	28	173
Overall Study COMPLETED	245	25	166
Overall Study NOT COMPLETED	16	3	7

Reasons for withdrawal

Reasons for withdrawal
Measure	CUSA-081 Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Overall Study Not treated	8	1	5
Overall Study Adverse Event	3	0	0
Overall Study Withdrawal by Subject	3	0	0
Overall Study Protocol Violation	2	2	2

Baseline Characteristics

In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CUSA-081 n=253 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose (if needed) at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen.	Placebo n=27 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants received the first dose at min 0, and the second dose (if needed) at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen.	Alteplase n=168 Participants Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose (if needed) at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen.	Total n=448 Participants Total of all reporting groups
Age, Categorical Age categorical · <=18 years	0 Participants n=253 Participants	0 Participants n=27 Participants	0 Participants n=168 Participants	0 Participants n=448 Participants
Age, Categorical Age categorical · Between 18 and 65 years	140 Participants n=253 Participants	15 Participants n=27 Participants	94 Participants n=168 Participants	249 Participants n=448 Participants
Age, Categorical Age categorical · >=65 years	113 Participants n=253 Participants	12 Participants n=27 Participants	74 Participants n=168 Participants	199 Participants n=448 Participants
Age, Continuous	60.6 years STANDARD_DEVIATION 13.5 • n=253 Participants	60.4 years STANDARD_DEVIATION 12.7 • n=27 Participants	60.9 years STANDARD_DEVIATION 14.3 • n=168 Participants	60.7 years STANDARD_DEVIATION 13.7 • n=448 Participants
Sex: Female, Male Female	150 Participants n=253 Participants	16 Participants n=27 Participants	103 Participants n=168 Participants	269 Participants n=448 Participants
Sex: Female, Male Male	103 Participants n=253 Participants	11 Participants n=27 Participants	65 Participants n=168 Participants	179 Participants n=448 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	44 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	7 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	24 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	75 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.
Ethnicity (NIH/OMB) Not Hispanic or Latino	132 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	9 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	101 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	242 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	2 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	1 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.	4 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on ethnicity.
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	1 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	1 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.
Race (NIH/OMB) Asian	3 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	1 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	1 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	5 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.
Race (NIH/OMB) Black or African American	7 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	5 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	12 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.
Race (NIH/OMB) White	166 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	17 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	119 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	302 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.
Race (NIH/OMB) More than one race	0 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=177 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=18 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	0 Participants n=126 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.	1 Participants n=321 Participants • In some countries (e.g. Belgium) it is against the national law to collect information on race.
Body mass index	28.05 kg/m^2 STANDARD_DEVIATION 6.49 • n=253 Participants	27.75 kg/m^2 STANDARD_DEVIATION 5.62 • n=27 Participants	28.01 kg/m^2 STANDARD_DEVIATION 6.11 • n=168 Participants	28.02 kg/m^2 STANDARD_DEVIATION 6.29 • n=448 Participants

PRIMARY outcome

Timeframe: Day 1 (up to 90 mins post dose)

Population: Full analysis set (FAS) was used for the analysis. FAS included all randomized subjects who received at least one dose of study drug, and with at least one available evaluation of efficacy after baseline (i.e., at least one CVAD assessment after study drug administration).

Outcome measures

Outcome measures
Measure	CUSA-081 n=253 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=27 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Percentage Of Participants With Treatment Success Following A Single Instillation Of Study Drug With A Dwell Time Up To 90 Min -- CUSA-081 vs Placebo -- Full Analysis Set (FAS)	159 Participants	9 Participants	—

SECONDARY outcome

Timeframe: Day 1 (up to 90 min post dose)

Population: Per protocol (PP) set included all subjects from the SAF without any important protocol deviations.

CUSA-081 vs Alteplase -- Single instillation of study drug -- Dwell Time Up To 90 Min -- Per Protocol set (PP) Treatment success was defined as the restoration of CVAD functionality, measured as the ability to withdraw 3 mL of blood and infuse 5 mL of saline. For this assessment, dwell time was up to 90 mins, after a single instillation of study drug. The percentage was calculated as the number of participants with treatment success divided by the total number of participants in the group, multiplied by 100%. The PP set was used for sensitivity analysis when testing for non-inferiority.

Outcome measures

Outcome measures
Measure	CUSA-081 n=228 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=152 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Percentage Of Participants With Treatment Success Following A Single Instillation Of Study Drug With A Dwell Time Up To 90 Min -- CUSA-081 vs Alteplase -- Per Protocol Set (PP)	152 Participants	117 Participants	—

SECONDARY outcome

Timeframe: Day 1 (up to 60 min post dose)

CUSA-081 vs Placebo -- Single instillation of study drug -- Dwell Time Up To 60 Min -- Full Analysis set (FAS) Treatment success was defined as the restoration of CVAD functionality, measured as the ability to withdraw 3 mL of blood and infuse 5 mL of saline. For this assessment, dwell time was up to 60 mins, after a single instillation of study drug. The percentage was calculated as the number of participants with treatment success divided by the total number of participants in the group, multiplied by 100%.

Outcome measures

Outcome measures
Measure	CUSA-081 n=253 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=27 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Percentage Of Participants With Treatment Success Following A Single Instillation Of Study Drug With A Dwell Time Up To 60 Min -- CUSA-081 vs Placebo -- Full Analysis Set (FAS)	136 Participants	8 Participants	—

SECONDARY outcome

Timeframe: Day 1 (up to 180 min post dose)

CUSA-081 vs Placebo -- 2 Instillations of study drug -- Dwell Time Up To 180 Min -- Full Analysis set (FAS) Treatment success was defined as the restoration of CVAD functionality, measured as the ability to withdraw 3 mL of blood and infuse 5 mL of saline. For this assessment, dwell time was up to 180 mins, following 2 installations of the study drug. The percentage was calculated as the number of participants with treatment success divided by the total number of participants in the group, multiplied by 100%. Subjects received the first instillation of study drug (CUSA-081, placebo, or alteplase). If patency was not restored after 90 minutes following the first instillation, a second dose of study drug (the same drug as at first instillation) was administered.

Outcome measures

Outcome measures
Measure	CUSA-081 n=253 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=27 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Percentage Of Participants With Treatment Success Following 2 Instillations Of Study Drug With A Dwell Time Up To 180 Min -- CUSA-081 vs Placebo -- Full Analysis Set (FAS)	207 Participants	11 Participants	—

SECONDARY outcome

Timeframe: Day 1 (up to 90 min post dose)

CUSA-081 vs Alteplase -- Single instillation of study drug -- Dwell Time Up To 90 Min -- Full Analysis set (FAS) Treatment success was defined as the restoration of CVAD functionality, measured as the ability to withdraw 3 mL of blood and infuse 5 mL of saline. For this assessment, dwell time is up to 90 mins, after a single instillation of study drug. The percentage was calculated as the number of participants with treatment success divided by the total number of participants in the group, multiplied by 100%.

Outcome measures

Outcome measures
Measure	CUSA-081 n=253 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=168 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Percentage Of Participants With Treatment Success Following A Single Instillation Of Study Drug With A Dwell Time Up To 90 Min -- CUSA-081 vs Alteplase -- Full Analysis Set (FAS)	159 Participants	124 Participants	—

SECONDARY outcome

Timeframe: Day 1 (post dose) up to Day 30

The rate of recurrent catheter dysfunction is defined as re-occlusion. The rate of recurrent catheter dysfunction within 30 days following treatment and the results of the Kaplan-Meier and Cox proportional hazards analyses of the time to first re-occlusion are presented in the FAS. This analysis is based on all participants with treatment success following up to 2 administrations of study drug with a total dwell time up to 180 min. Subjects received the first instillation of study drug (CUSA-081, placebo, or alteplase). If patency was not restored after 90 minutes following the first instillation, a second dose of study drug (the same drug as at first instillation) was administered.

Outcome measures

Outcome measures
Measure	CUSA-081 n=207 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=11 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase n=148 Participants Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Rate Of Recurrent Catheter Dysfunction Within 30 Days Following Treatment With Study Drug	17 Participants	1 Participants	18 Participants

SECONDARY outcome

Timeframe: Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose).

Population: The safety set (SAF) included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF.

Treatment-emergent AEs leading to study discontinuation were evaluated and the percentage of participants with at least one event reported. For all subjects who discontinued the study, the AE was 'Device breakage'.

Outcome measures

Outcome measures
Measure	CUSA-081 n=253 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=27 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase n=168 Participants Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Percentage of Participants With Treatment-emergent Adverse Events (AEs) Leading to Study Discontinuation	1.2 % of subjects with at least one event	0 % of subjects with at least one event	0 % of subjects with at least one event

SECONDARY outcome

Timeframe: Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose).

Population: The safety set (SAF) included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF.

Treatment-emergent AEs of special interest (AESI) were monitored. These included major bleeding (defined as severe blood loss \[\>5 mL/kg\] or blood loss requiring transfusion or causing hypotension requiring use of inotropic agents), embolism, thrombosis, and catheter-related blood stream infection. An adverse event was considered as treatment-emergent if it started on or after the first dose of study drug intake up to the end of the 180-minute treatment period.

Outcome measures

Outcome measures
Measure	CUSA-081 n=253 Participants Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=27 Participants Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase n=168 Participants Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
Percentage of Participants With Treatment-emergent Adverse Events (AEs) of Special Interest (AESI)	0 % of subjects with at least one event	0 % of subjects with at least one event	0 % of subjects with at least one event

Adverse Events

CUSA-081

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Alteplase

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	CUSA-081 n=253 participants at risk Participants received 1 or 2 doses of CUSA-081, 0.7 milligrams (mg) (0.4 units) per 2 milliliter (mL) directly into the catheter lumen. Participants received the first dose at minute (min) 0, and the second dose, if needed, at min 90. CUSA-081: Participants received 1 or 2 doses of CUSA-081 0.7 mg/2 mL directly into the catheter lumen	Placebo n=27 participants at risk Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen. Participants will receive the first dose at min 0, and the second dose, if needed, at min 90. Placebo: Participants received 1 or 2 doses of placebo (normal saline) directly into the catheter lumen	Alteplase n=168 participants at risk Participants received 1 or 2 doses of alteplase, 2 mg/mL, directly into the catheter lumen. Participants received the first dose at min 0, and the second dose, if needed, at min 90. Alteplase: Participants received 1 or 2 doses of alteplase, 2 mg/2 mL, directly into the catheter lumen
General disorders Fatigue	0.40% 1/253 • Number of events 1 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/168 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).
Infections and infestations Clostridium difficile infection	0.40% 1/253 • Number of events 1 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/168 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).
Infections and infestations Sinusitis	0.40% 1/253 • Number of events 1 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/168 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).
Metabolism and nutrition disorders Decreased appetite	0.79% 2/253 • Number of events 2 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/168 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).
Musculoskeletal and connective tissue disorders Back pain	0.40% 1/253 • Number of events 1 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/168 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).
Nervous system disorders Polyneuropathy	0.00% 0/253 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.60% 1/168 • Number of events 1 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).
Product Issues Device breakage	1.2% 3/253 • Number of events 3 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/168 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).
Vascular disorders Hypotension	0.40% 1/253 • Number of events 1 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/27 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).	0.00% 0/168 • Day 1 (start of treatment) and until the end of the treatment period (up to 180 min post dose). Safety set (SAF) was used for the evaluation of AEs. SAF included all randomized subjects who received at least one dose of study drug. Subjects discontinued after dosing were included in the SAF. Reported are AEs that started on or after the first dose of study drug intake and up to the end of the 180-minute treatment period (treatment-emergent AE).

Additional Information

Clinical Trial Transparency

Chiesi Farmaceutici S.p.A.

Phone: + 39 0521 2791

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Chiesi can publish and/or present any results of this study at scientific meetings, and to submit the clinical trial data to national and international Regulatory Authorities. Chiesi reserves the right to use such data for industrial purposes. Investigators will inform Chiesi before using the results of the study for publication or presentation, and agree to provide the Sponsor with a copy of the proposed presentation. Data from individual study sites must not be published separately.
Publication restrictions are in place

Restriction type: OTHER