A Phase 2 interventional study of rhBSSL in Cystic Fibrosis and Exocrine Pancreatic Insufficiency, sponsored by Swedish Orphan Biovitrum. Completed at 4 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2014-11-18.
Sponsored by Swedish Orphan Biovitrum · Phase 2, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
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.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 15 is below the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Swedish Orphan Biovitrum is the lead sponsor of 78 studies on the registry; 4 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 15 (88%) have results posted.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria:
170 mg rhBSSL three times daily for 5 to 6 consecutive days
Drug: rhBSSL
oral suspension, 170 mg BSSL, 3 times daily for 5-6 days
Also known as: BSSL, Bucelipase alfa, rhBSSL
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
| Milestone | rhBSSL |
|---|---|
| Started | 15 |
| Completed | 14 |
| Not completed | 1 |
| Withdrew: Physician decision | 1 |
| Milestone | rhBSSL |
|---|---|
| Started | 14 |
| Completed | 13 |
| Not completed | 1 |
| Withdrew: Adverse event | 1 |
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 %
| % CFA | rhBSSL |
|---|---|
| The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) | 6.9 ± 15.56 |
Collected over Treatment period (5-6 days). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BSSL | — | 1/14 (7.1%) | 6/14 (42.9%) |
| Event | BSSL |
|---|---|
| Cystic Fibrosis ExacerbationCongenital, familial and genetic disorders | 1/14 |
| Event | BSSL |
|---|---|
| Abdominal painGastrointestinal disorders | 4/14 |
| Viral InfectionInfections and infestations | 1/14 |
| C-reactive protein increasedInvestigations | 1/14 |
| HeadacheNervous system disorders | 1/14 |
| InsomniaPsychiatric disorders | 1/14 |
| Cystic fibrosis lungCongenital, familial and genetic disorders | 1/14 |
| Age, Continuous(years) | BSSL |
|---|---|
| Mean | 24.4 ± 10.45 |
| Sex: Female, Male(Participants) | BSSL |
|---|---|
| Female | 7 |
| Male | 8 |
| Ethnicity (NIH/OMB)(Participants) | BSSL |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 15 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | BSSL |
|---|---|
| Poland | 12 |
| Netherlands | 3 |
This study is completed, as verified in Nov 2014. You cannot join it, but the record below documents what was studied.
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Swedish Orphan Biovitrum