Trial Outcomes & Findings for Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis (NCT NCT05719311)

NCT ID: NCT05719311

Last Updated: 2024-09-19

Results Overview

Number of subjects reporting 1 or more adverse events.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

13 participants

Primary outcome timeframe

End of 3-week treatment period.

Results posted on

2024-09-19

Participant Flow

Participant milestones

Participant milestones
Measure	Adrulipase Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase. adrulipase: Enteric microgranule formulation of adrulipase.
Overall Study STARTED	13
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Adrulipase n=13 Participants Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase. adrulipase: Enteric microgranule formulation of adrulipase.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	33.2 years STANDARD_DEVIATION 9.86 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 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	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	12 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
Region of Enrollment United States	13 participants n=5 Participants

PRIMARY outcome

Timeframe: End of 3-week treatment period.

Number of subjects reporting 1 or more adverse events.

Outcome measures

Outcome measures
Measure	Adrulipase n=13 Participants Adrulipase
Safety of Adrulipase	2 Participants

PRIMARY outcome

Timeframe: End of 3-week treatment period.

Population: Number of participants that achieved a CFA \>= 80%

The primary efficacy endpoint is the CFA that will be assessed at the end of the 3-week treatment period. CFAs for adrulipase will be compared to the CFAs of PERT obtained at baseline/eligibility using descriptive methods.

Outcome measures

Outcome measures
Measure	Adrulipase n=13 Participants Adrulipase
Efficacy of Adrulipase: Coefficient of Fat Absorption (CFA)	2 Participants

SECONDARY outcome

Timeframe: Change between two time points: initial PERT confinement and end of 3-week treatment period.

Stool weights obtained during the supervised confinement visit at the end of the 3-week treatment period will be measured. Stool weights obtained during confinement on adrulipase will be compared to the stool weights during confinement on PERT obtained at baseline/eligibility using descriptive methods.

Outcome measures

Outcome measures
Measure	Adrulipase n=13 Participants Adrulipase
Stool Weight	647.1 grams Standard Deviation 496.41

SECONDARY outcome

Timeframe: Change between two time points: initial PERT confinement and end of 3-week treatment period

CNA that will be assessed at the end of the 3-week treatment period. CNAs for adrulipase will be compared to the CNAs of PERT obtained at baseline/eligibility using descriptive methods.

Outcome measures

Outcome measures
Measure	Adrulipase n=13 Participants Adrulipase
Coefficient of Nitrogen Absorption (CNA)	-27.8 Percentage of nitrogen absorbed Standard Deviation 14.47

Adverse Events

Low Dose

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Mid Dose

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

High Dose

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Low Dose n=1 participants at risk Adrulipase (1.95 g/day)	Mid Dose n=5 participants at risk Adrulipase (2.4 g/day)	High Dose n=7 participants at risk Adrulipase (3.3 g/day)
Gastrointestinal disorders Unclear etiology	0.00% 0/1 • 3-weeks	20.0% 1/5 • Number of events 1 • 3-weeks	0.00% 0/7 • 3-weeks
Infections and infestations Gastroenteritis	0.00% 0/1 • 3-weeks	20.0% 1/5 • Number of events 1 • 3-weeks	0.00% 0/7 • 3-weeks

Additional Information

VP, Regulatory, QA & Compliance

Entero Therapeutics

Phone: 9194491484

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER