Cystic FibrosisPhase 3NCT00690820

Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency (PEI) Due to Cystic Fibrosis (CF)

A Phase 3 study testing Pancrelipase Delayed Release in people with Cystic Fibrosis and Pancreatic Exocrine Insufficiency, run by Solvay Pharmaceuticals at 10 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This study will assess the effect of pancrelipase delayed release 12,000 unit capsules on fat and nitrogen absorption in subjects 7 - 11 with pancreatic exocrine insufficiency due to Cystic Fibrosis. Read more about this study

Who is it for?: Ages 7 to 11 years
Conditions the study is for: Cystic Fibrosis, Pancreatic Exocrine Insufficiency
What is being tested?: Pancrelipase Delayed Release
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 17 participants
Where: 10 sitesUnited States
Study dates: Jun 2008to Dec 2008
Phase: Phase 3
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 7 to 11 years
All sexes
No healthy volunteers
Child

Who may be able to join

Confirmed CF diagnosis by two positive chloride sweat tests or gene analysis
Confirmed PEI by historical Coefficient of fat Absorption < 70% without supplementation or current or historical fecal elastase < 50µg/stool (within the last 12 months)
Currently receiving treatment with a commercially available pancreatic enzyme product on a stable dose for more than 3 months
Clinically stable condition without evidence of acute respiratory disease or any other acute condition
Stable body weight and agrees to abstain from sexual activity

Who cannot join

Ileus or acute abdomen
History of fibrosing colonopathy, celiac disease, gastrectomy, Crohn´s disease and small bowel surgery other than minor resection due to meconium ileus without resulting in malabsorption syndrome
History of distal ileal obstruction syndrome within 6 months of enrollment
Use of an immunosuppressive drug
Any type of malignancy involving the digestive tract in the last 5 years
Known infection with HIV

Trial locations and contacts

10 sites in 1 country.

City, postcode or hospital

10 sites in 1 country

United States10 sites

Site 8
Albuquerque, New Mexico
Site 6
Ann Arbor, Michigan
Site 9
Boston, Massachusetts
Site 1
Cincinnati, Ohio
Site 3
Hershey, Pennsylvania
Site 2
Iowa City, Iowa
Site 5
Louisville, Kentucky
Site 4
Minneapolis, Minnesota
Site 10
Oklahoma City, Oklahoma
Site 7
Oklahoma City, Oklahoma

Other trials in United States

About this study

Official title: A Double-blind, Randomized, Multi-center, Placebo-controlled, Cross-over Study to Assess the Efficacy and Safety of Pancrelipase Delayed Release 12,000 Unit Capsules in Subjects Aged 7 - 11 With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis

This study will assess the effect of pancrelipase delayed release 12,000 unit capsules on fat and nitrogen absorption in subjects 7 - 11 with pancreatic exocrine insufficiency due to Cystic Fibrosis.

Conditions: Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Being tested: Pancrelipase Delayed Release, Placebo Comparator
Keywords: Cystic Fibrosis, Pancreatic Exocrine Insufficiency
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.

Pancrelipase Delayed ReleaseDrug
12,000 unit Capsules, dosed individually based on fat intake.
Placebo ComparatorDrug
Placebo

Study groups in NCT00690820
Study group	Role	What they receive
A	Experimental	Not described Receives: Drug: Pancrelipase Delayed Release
B	Placebo comparator	Not described Receives: Drug: Placebo Comparator

What is being measured

The primary outcome is the main question the study is designed to answer.

Coefficient of Fat Absorption (%)
This coefficient is calculated from fat intake and fat excretion : 100*[fat intake-fat excretion]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.
Measured: 5 days

Secondary outcomes (7)

Coefficient of Nitrogen Absorption (%)
This coefficient is calculated from nitrogen intake and nitrogen excretion : 100*[nitrogen intake-nitrogen excretion]/nitrogen intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.
Measured: 5 days
Total Fat Excretion (Grams)
Total amount of fat excreted during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response.
Measured: 5 days
Total Stool Weight (Grams)
Total weight of the stools collected during the stool collection period. Stools were collected on 3 days during the 5 days treatment period. Lower values indicate a better response.
Measured: 5 days
Stool Frequency
Stool frequency is the average of the daily number of stools recorded during the treatment period. Lower values indicate a better response.
Measured: 5 days
Percentage of Days With no Flatulence.
The percentage of days with no flatulence is calculated from the diary during the treatment period: 100*(number of days with no flatulence/number of days recorded in diary). Higher values indicate a better response.
Measured: 5 days
Percentage of Days With Formed/Normal Stools.
The percentage of days with formed/normal stools is calculated from the diary during the treatment period: 100*(number of days with formed/normal stools/number of days with any stool). Higher values indicate a better response.
Measured: 5 days
Percentage of Days With no Abdominal Pain.
The percentage of days with no abdominal pain is calculated from the diary during the treatment period: 100*(number of days with no abdominal pain / number of days recorded in diary). Higher values indicate a better response.
Measured: 5 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2008Dec 2008

First posted5 Jun 2008 (estimated)
Study startJun 2008
Primary completionDec 2008
Study completionDec 2008

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.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 17 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00690820
Sponsor's protocol ID: S245.3.127
Status last verified: Feb 2010
First submitted: 3 Jun 2008
Last updated: 2 Jun 2010
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Graff GR, Maguiness K, McNamara J, Morton R, Boyd D, Beckmann K, Bennett D. Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: a multicenter, randomized, double-blind, placebo-controlled, two-period crossover, superiority study. Clin Ther. 2010 Jan;32(1):89-103. doi: 10.1016/j.clinthera.2010.01.012.
PubMed 20171415
Caras S, Boyd D, Zipfel L, Sander-Struckmeier S. Evaluation of stool collections to measure efficacy of PERT in subjects with exocrine pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2011 Dec;53(6):634-40. doi: 10.1097/MPG.0b013e3182281c38.
PubMed 21681115

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Jun 2010. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Double-blind, Randomized, Multi-center, Placebo-controlled, Cross-over Study to Assess the Efficacy and Safety of Pancrelipase Delayed Release 12,000 Unit Capsules in Subjects Aged 7 - 11 With Pancreatic Exocrine Insufficiency Due to Cystic Fibrosis
ClinicalTrials.gov ID: NCT00690820
Acronym / sponsor ID: S245.3.127
Conditions: Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Keywords: Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 17 participants (actual)
Lead sponsor: Solvay PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Jun 2008
Primary completion: Dec 2008 (actual)
Study completion: Dec 2008 (actual)
First posted: 5 Jun 2008 (estimated)
Results: Posted 10 Mar 2010

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This study will assess the effect of pancrelipase delayed release 12,000 unit capsules on fat and nitrogen absorption in subjects 7 - 11 with pancreatic exocrine insufficiency due to Cystic Fibrosis.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
A	Experimental	Drug: Pancrelipase Delayed Release
B	Placebo comparator	Drug: Placebo Comparator

Who may qualify

Ages: 7 to 11 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

Confirmed CF diagnosis by two positive chloride sweat tests or gene analysis
Confirmed PEI by historical Coefficient of fat Absorption < 70% without supplementation or current or historical fecal elastase < 50µg/stool (within the last 12 months)
Currently receiving treatment with a commercially available pancreatic enzyme product on a stable dose for more than 3 months
Clinically stable condition without evidence of acute respiratory disease or any other acute condition
Stable body weight and agrees to abstain from sexual activity

Key exclusion criteria (6 listed)

Ileus or acute abdomen
History of fibrosing colonopathy, celiac disease, gastrectomy, Crohn´s disease and small bowel surgery other than minor resection due to meconium ileus without resulting in malabsorption syndrome
History of distal ileal obstruction syndrome within 6 months of enrollment
Use of an immunosuppressive drug
Any type of malignancy involving the digestive tract in the last 5 years

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
Pancrelipase Delayed Release12,000 unit Capsules, dosed individually based on fat intake.	Drug	A
Placebo ComparatorPlacebo	Drug	B

Primary outcome

Coefficient of Fat Absorption (%)
Time frame: 5 days
This coefficient is calculated from fat intake and fat excretion : 100*[fat intake-fat excretion]/fat intake. Stools were collected on 3 days during the 5 days treatment period. Higher values indicate a better response.

7 secondary outcomes

Coefficient of Nitrogen Absorption (%)
Time frame: 5 days
Total Fat Excretion (Grams)
Time frame: 5 days
Total Stool Weight (Grams)
Time frame: 5 days
Stool Frequency
Time frame: 5 days
Percentage of Days With no Flatulence.
Time frame: 5 days
Percentage of Days With Formed/Normal Stools.
Time frame: 5 days
Percentage of Days With no Abdominal Pain.
Time frame: 5 days

Results and safety

Results posted to the registry on 10 Mar 2010, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo/Pancrelipase	8	8
Pancrelipase/Placebo	9	8

Primary outcome: Coefficient of Fat Absorption (%)

Time frame: 5 days. Mean (Standard Deviation). Unit: Percentage

Measure	Placebo n = 16	Pancrelipase n = 16
Value	47.41 (16.84)	82.81 (8.29)

Placebo vs Pancrelipase: p <0.001, ANOVA

Population analysed: The analysis was done on the Full Analysis Sample defined as the randomized subjects with at least one post-baseline efficacy measurement.

7 secondary outcomes are reported in the full results.

Adverse events

Time frame: The adverse events were collected from Screening to the end of the second period of the cross-over trial. A safety follow-up call was performed 5-7 days after the last visit to the clinic.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	0 of 16 (0%)	9 of 16 (56.3%)	Not reported
Pancrelipase	0 of 17 (0%)	5 of 17 (29.4%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2010.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 20171415Graff GR, Maguiness K, McNamara J, Morton R, Boyd D, Beckmann K, Bennett D. Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: a multicenter, randomized, double-blind, placebo-controlled, two-period crossover, superiority study. Clin Ther. 2010 Jan;32(1):89-103. doi: 10.1016/j.clinthera.2010.01.012.
Derived from this study: PubMed 21681115Caras S, Boyd D, Zipfel L, Sander-Struckmeier S. Evaluation of stool collections to measure efficacy of PERT in subjects with exocrine pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2011 Dec;53(6):634-40. doi: 10.1097/MPG.0b013e3182281c38.

Related trials for Cystic Fibrosis

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CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
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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.

Global Clinical Director Solvay study director

Solvay Pharmaceuticals

10 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Pancrelipase Delayed Release in people with Cystic Fibrosis and Pancreatic Exocrine Insufficiency, run by Solvay Pharmaceuticals at 10 sites in United States.

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 5 inclusion and 6 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 2 Jun 2010. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in United States. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 10 Mar 2010). 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?

Coefficient of Fat Absorption (%) (time frame: 5 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00690820, which also lists 2 linked publications.

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 2 Jun 2010.