Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Conditions
Keywords
cystic fibrosis, pancreatic insufficiency, bucelipase alfa, bile salt stimulating lipase, BSSL, coefficient of fat absorption
Brief summary
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
Detailed description
In this open study, patients will enter a baseline period of 6 days where the pancreatic enzyme therapy will be discontinued and a standard diet given. After the baseline period, patients will enter a treatment period of 6 days where a fixed dose of BSSL will be administered. The primary efficacy measurements will be made by collecting stool during the last three days of each period.
Interventions
DRUGrhBSSL
oral suspension, 170 mg BSSL, 3 times daily for 5-6 days
Sponsors
Swedish Orphan Biovitrum
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients with cystic fibrosis and pancreatic insufficiency who are able to refrain from their ongoing pancreatic enzyme treatment for a period of 7 days and are able to produce stools \>= 5 times per week
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) | Final 3 days of baseline and treatment period | 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 % |
Countries
Netherlands, Poland
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| BSSL Bucelipase alfa (INN): oral suspension, 170 mg BSSL, 3 times daily for 5-6 days | 15 |
| Total | 15 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Baseline Period | Physician Decision | 1 |
| Treatment Period | Adverse Event | 1 |
Baseline characteristics
| Characteristic | BSSL |
|---|---|
| Age, Continuous | 24.4 years STANDARD_DEVIATION 10.45 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 15 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Region of Enrollment Netherlands | 3 participants |
| Region of Enrollment Poland | 12 participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 6 / 14 |
| serious Total, serious adverse events | 1 / 14 |
Outcome results
Primary
The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)
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 %
Time frame: Final 3 days of baseline and treatment period
Population: Per protocol
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| rhBSSL | The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) | 6.9 % CFA | Standard Deviation 15.56 |
p-value: 0.2179t-test, 2 sided