Trial Outcomes & Findings for SIMPLICITY: Studying Impacts on Malabsorption With Liprotamase in Cystic Fibrosis (NCT NCT02734810)

NCT ID: NCT02734810

Last Updated: 2018-05-11

Results Overview

Number of subjects reporting 1 or more adverse events

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 15 participants
• Primary outcome timeframe: 1 week
• Results posted on: 2018-05-11

Participant Flow

Participant milestones

Measure	Part A Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy	Part B Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy
Overall Study STARTED	15	0
Overall Study COMPLETED	13	0
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	Part A Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy	Part B Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy
Overall Study Adverse Event	1	0
Overall Study Lack of Efficacy	1	0

Baseline Characteristics

SIMPLICITY: Studying Impacts on Malabsorption With Liprotamase in Cystic Fibrosis

Baseline characteristics by cohort

Measure	Part A n=15 Participants Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy	Part B Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy	Total n=15 Participants Total of all reporting groups
Age, Continuous	24.1 years n=5 Participants	—	24.1 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants	0 Participants n=7 Participants	12 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 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	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants	0 Participants n=7 Participants	15 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	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants	—	15 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 week

Number of subjects reporting 1 or more adverse events

Outcome measures

Measure	Part A n=15 Participants Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy	Part B Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy
Safety	3 Participants	0 Participants

Adverse Events

Part A

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Part B

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Part A n=15 participants at risk Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy	Part B Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy
Gastrointestinal disorders Gastroduodenitis	6.7% 1/15 • Number of events 1 • 1 week	— 0/0 • 1 week

Other adverse events

Measure	Part A n=15 participants at risk Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy	Part B Liprotamase Powder for Oral Solution in Subjects aged 28 days to \<7 years Liprotamase Powder for Oral Solution: Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy
Infections and infestations Infective exacerbation of cystic fibrosis	20.0% 3/15 • Number of events 3 • 1 week	— 0/0 • 1 week
Investigations Aspartate aminotransferase increased	6.7% 1/15 • Number of events 15 • 1 week	— 0/0 • 1 week

Additional Information

Monica Gangal

Anthera Pharmaceuticals

Phone: 510-856-5600

Email: info@anthera.com

Results disclosure agreements

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

Restriction type: OTHER