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Trial Outcomes & Findings for Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency (NCT NCT00743483)

NCT ID: NCT00743483

Last Updated: 2014-11-18

Results Overview

The absolute difference between baseline and treatment CFA, i.e. the change from the baseline level. CFA was calculated as follows 100 x ((fat consumed - fat excreted)/fat consumed). Fat consumed was determined from the weight of fat of the dietary intake during a 72 hour period during the final 3 days of the baseline and treatment period. Fat excreted was determined from stool collected during the 72-hour periods and analyzed for fat using the Van de Kamer method. The unit of CFA is %

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

Final 3 days of baseline and treatment period

Results posted on

2014-11-18

Participant Flow

Participant milestones

Participant milestones
Measure
rhBSSL
Oral suspension, 170 mg rhBSSL, 3 times daily for 5-6 days
Baseline Period
STARTED
15
Baseline Period
COMPLETED
14
Baseline Period
NOT COMPLETED
1
Treatment Period
STARTED
14
Treatment Period
COMPLETED
13
Treatment Period
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
rhBSSL
Oral suspension, 170 mg rhBSSL, 3 times daily for 5-6 days
Baseline Period
Physician Decision
1
Treatment Period
Adverse Event
1

Baseline Characteristics

Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
BSSL
n=15 Participants
Bucelipase alfa (INN): oral suspension, 170 mg BSSL, 3 times daily for 5-6 days
Age, Continuous
24.4 years
STANDARD_DEVIATION 10.45 • n=5 Participants
Sex: Female, Male
Female
7 Participants
n=5 Participants
Sex: Female, Male
Male
8 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
15 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
Poland
12 participants
n=5 Participants
Region of Enrollment
Netherlands
3 participants
n=5 Participants

PRIMARY outcome

Timeframe: Final 3 days of baseline and treatment period

Population: Per protocol

The absolute difference between baseline and treatment CFA, i.e. the change from the baseline level. CFA was calculated as follows 100 x ((fat consumed - fat excreted)/fat consumed). Fat consumed was determined from the weight of fat of the dietary intake during a 72 hour period during the final 3 days of the baseline and treatment period. Fat excreted was determined from stool collected during the 72-hour periods and analyzed for fat using the Van de Kamer method. The unit of CFA is %

Outcome measures

Outcome measures
Measure
rhBSSL
n=9 Participants
oral suspension, 170 mg, 3 times daily for 5-6 days
The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)
6.9 % CFA
Standard Deviation 15.56

Adverse Events

BSSL

Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
BSSL
n=14 participants at risk
Oral suspension, 170 mg, 3 times daily for 5-6 days
Congenital, familial and genetic disorders
Cystic Fibrosis Exacerbation
7.1%
1/14 • Number of events 1 • Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is presented

Other adverse events

Other adverse events
Measure
BSSL
n=14 participants at risk
Oral suspension, 170 mg, 3 times daily for 5-6 days
Gastrointestinal disorders
Abdominal pain
28.6%
4/14 • Number of events 4 • Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is presented
Infections and infestations
Viral Infection
7.1%
1/14 • Number of events 1 • Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is presented
Investigations
C-reactive protein increased
7.1%
1/14 • Number of events 1 • Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is presented
Nervous system disorders
Headache
7.1%
1/14 • Number of events 2 • Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is presented
Psychiatric disorders
Insomnia
7.1%
1/14 • Number of events 1 • Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is presented
Congenital, familial and genetic disorders
Cystic fibrosis lung
7.1%
1/14 • Number of events 1 • Treatment period (5-6 days)
Only treatment emergent events (events that started post-rhBSSL administration) is presented

Additional Information

Maria Öhman, TA Clinical Head Neonatology

Swedish Orphan Biovitrum

Phone: +46 70 694 81 61

Results disclosure agreements

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

Restriction type: OTHER