Cystic FibrosisPhase 2NCT00743483
Efficacy of Bucelipase Alfa (BSSL) in Patients With Cystic Fibrosis and Pancreatic Insufficiency
A Phase 2 study testing rhBSSL in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Swedish Orphan Biovitrum at 4 sites in 2 countries.
Trial summary
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- What is being tested?
- rhBSSL
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 4 sitesin 2 countries
- Study dates
- Aug 2008to Jun 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
None listed.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Poland3 sites
Specjalistyczny Zespól Opieki Zdrowotnej nad Matka i Dzieckiem w Gdansku
Gdansk
Uniwersytet Medyczny im. K. Marcinkowskiego
Poznan
Oddzialu Terenowego Instytutu, Gruzlicy i Chorob Pluc w Rabce-Zdroju
Rabka-Zdrój
About this study
Official title: An Open Label, Exploratory Study on the Effect of rhBSSL on the Fat Absorption in Patients With Cystic Fibrosis and Pancreatic Insufficiency
The purpose of this study is to evaluate the efficacy in patients with cystic fibrosis and pancreatic insufficiency following treatment with BSSL
Read the 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.
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Being tested
- rhBSSL
- Keywords
- cystic fibrosis, pancreatic insufficiency, bucelipase alfa, bile salt stimulating lipase, BSSL, coefficient of fat absorption
- Medical subject headings
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhBSSLDrug
Also called BSSL, Bucelipase alfa, rhBSSL
oral suspension, 170 mg BSSL, 3 times daily for 5-6 days
| Study group | Role | What they receive |
|---|---|---|
| rhBSSL | Experimental | 170 mg rhBSSL three times daily for 5 to 6 consecutive days Receives: Drug: rhBSSL |
What is being measured
The primary outcome is the main question the study is designed to answer.
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 %
Measured: Final 3 days of baseline and treatment period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2008 (estimated)
- Study startAug 2008
- Primary completionJun 2009
- Study completionJun 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jean-Pierre Isal, MD, PhD, Study director, Swedish Orphan BiovitrumJanneke Langendonk, MD, PhD, Principal investigator, Erasmus Medical Center, Rotterdam, Netherlands
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00743483
- Sponsor's protocol ID
- BVT.BSSL-002
- Other IDs
- 2007-004063-21
- Status last verified
- Nov 2014
- First submitted
- 26 Aug 2008
- Last updated
- 18 Nov 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Exploratory Study on the Effect of rhBSSL on the Fat Absorption in Patients With Cystic Fibrosis and Pancreatic Insufficiency
- ClinicalTrials.gov ID
- NCT00743483
- Acronym / sponsor ID
- BVT.BSSL-002
- Conditions
- Cystic Fibrosis, Exocrine Pancreatic Insufficiency
- Keywords
- cystic fibrosis, pancreatic insufficiency, bucelipase alfa, bile salt stimulating lipase, BSSL, coefficient of fat absorption
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- Aug 2008
- Primary completion
- Jun 2009 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 28 Aug 2008 (estimated)
- Results
- Posted 18 Nov 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rhBSSL | Experimental | Drug: rhBSSL170 mg rhBSSL three times daily for 5 to 6 consecutive days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rhBSSLAlso: BSSL, Bucelipase alfa, rhBSSLoral suspension, 170 mg BSSL, 3 times daily for 5-6 days | Drug | rhBSSL |
Primary outcome
The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA)
Time frame: 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 %
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jean-Pierre Isal, MD, PhD study director
Swedish Orphan Biovitrum
Janneke Langendonk, MD, PhD principal investigator
Erasmus Medical Center, Rotterdam, Netherlands
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rhBSSL in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Swedish Orphan Biovitrum at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Poland, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Nov 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Absolute Difference Between Baseline and Treatment Coefficient of Fat Absorption (CFA) (time frame: Final 3 days of baseline and treatment period). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2014.