Cystic FibrosisPhase 4MBTNCT00572975
Malabsorption Blood Test:Toward a Novel Approach to Quantify Steatorrhea
A Phase 4 study testing Pentadecanoic acid (PA) and Triheptadecanoin (THA) in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial summary
*The purpose of this study is to develop a more accurate, reliable, specific and more acceptable alternative clinical test to the 72-hour stool and diet collection for quantifying fat malabsorption in people with CF and pancreatic insufficiency. Read more about this study
- Who is it for?
- Ages 8 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Pancreatic Insufficiency
- What is being tested?
- Pentadecanoic acid (PA) and Triheptadecanoin (THA)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2006to Mar 2008
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 8 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Subjects with cystic fibrosis and pancreatic insufficiency (n=9), 8 years of age or older, in usual state of good health will be eligible to participate in this Aim.
- Subjects will be screened for genotype and a history of pancreatic insufficiency (PI). PI will be confirmed by measurement of fecal elastase 1< 200 mg/g stool.
Who cannot join
- Subjects will be ineligible if they have an FEV1 % predicted of < 40%, a history of fibrosing colonopathy or any significant bowel resection (>10 cm) with the exception of a routine, uncomplicated appendectomy.
- Subjects with diabetes mellitus or disorders associated with altered energy metabolism (i.e. hypothyroidism), or any major illnesses that affect the gastrointestinal tract, will not be eligible for the study.
- Subjects with significant liver disease, or significant developmental delay will also be excluded.
- Subjects will be excluded if they have a food allergy related to any components of the experimental shake (i.e., soy or chocolate) or the stool marker, carmine red.
- Subjects who are pregnant are not eligible. Subjects with a history of intolerance or allergy to Creon 20 will be excluded from the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
About this study
Official title: MALABSORPTION BLOOD TEST: Toward a Novel Approach to Quantify Steatorrhea
*The purpose of this study is to develop a more accurate, reliable, specific and more acceptable alternative clinical test to the 72-hour stool and diet collection for quantifying fat malabsorption in people with CF and pancreatic insufficiency.
Read the detailed description
- Nine subjects with CF will be recruited to complete the MBT on two separate occasions, with different doses of pancreatic enzymes
- At each MBT visit, subjects will receive a specific enzyme dose just before they drink the test meal (shake)
- Fat absorption is maximized when the enzymes are taken with the shake.
- If subjects usually receive brands of pancreatic enzymes different from Creon, their dose will be converted to an equivalent dose of Creon 20 for the study.
Prior to each study visit all subjects will be required to:
- Eat their usual diet for 2 days prior to MBT
- Refrain from consuming alcohol or dairy products for 24 hours prior to MBT
- Refrain from non-routine physical exercise for 20 hours prior to MBT
- Adhere to a 12-hour fast prior to MBT (from 8 PM previous night)
- Can drink water from 8:00 PM on the night before the test to 2:00 AM on the day of the test
- At visits 1 and 2, blood samples will be obtained at hourly intervals over a period of eight hours.
- Samples will be analyzed for PA, HA and Triglyceride concentrations.
- Patients will be permitted to ingest non-caloric and non-caffeinated beverages.
- At hour 6, patients will be provided 1000 Kcal, low fat (12 grams of fat) lunch meal.
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Being tested
- Pentadecanoic acid (PA) and Triheptadecanoin (THA)
- Keywords
- New diagnostic test for Cystic Fibrosis.
- 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.
Pentadecanoic acid (PA) and Triheptadecanoin (THA)Other
Two fats (Pentadecanoic acid and Triheptadecanoin) are administered in a breakfast shake for the purposes of determining malabsorption.
in breakfast shake x1
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Other: Pentadecanoic acid (PA) and Triheptadecanoin (THA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Aim 1:Comparing Gastric Emptying and Small Bowel Transit Protocol in Healthy Controls
Measured: 72 hours
Secondary outcomes (3)
Aim 2:The results will establish which pattern of timing of enzyme ingestion results in optimal fat absorption of PA and HA. The results will also elucidate the role of gastric emptying and small bowel transit in subjects in CF.
Measured: 12-hour fast on 4 separate occasions, each at least 5 days apart
Aim 3: The results will establish the reproducibility of MBT as well as estimate the fecal loss of odd chain fatty acids (PA & THA)
Measured: 12-hour fast on three separate occasions, each at least 5 days apart
Aim 4: Dosage Titration Protocol is being tested in order to see changes in absorption with MBT
Measured: 12-hour fast on two separate occasions
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2007 (estimated)
- Study startAug 2006
- Primary completionMar 2008
- Study completionMar 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe 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 4
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- Solvay Pharmaceuticals
- Investigators
- Maria Mascarenhas, M.D., Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00572975
- Sponsor's protocol ID
- 2004-5-3733
- Acronym
- MBT
- Status last verified
- Sep 2008
- First submitted
- 11 Dec 2007
- Last updated
- 30 Sep 2008
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MALABSORPTION BLOOD TEST: Toward a Novel Approach to Quantify Steatorrhea
- ClinicalTrials.gov ID
- NCT00572975
- Acronym / sponsor ID
- MBT / 2004-5-3733
- Conditions
- Cystic Fibrosis, Pancreatic Insufficiency
- Keywords
- New diagnostic test for Cystic Fibrosis.
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- Solvay Pharmaceuticals
- Study start
- Aug 2006
- Primary completion
- Mar 2008 (actual)
- Study completion
- Mar 2008 (actual)
- First posted
- 13 Dec 2007 (estimated)
- Results
- Not posted in the source record
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 develop a more accurate, reliable, specific and more acceptable alternative clinical test to the 72-hour stool and diet collection for quantifying fat malabsorption in people with CF and pancreatic insufficiency.
Detailed description
- Nine subjects with CF will be recruited to complete the MBT on two separate occasions, with different doses of pancreatic enzymes
- At each MBT visit, subjects will receive a specific enzyme dose just before they drink the test meal (shake)
- Fat absorption is maximized when the enzymes are taken with the shake.
- If subjects usually receive brands of pancreatic enzymes different from Creon, their dose will be converted to an equivalent dose of Creon 20 for the study.
Prior to each study visit all subjects will be required to:
- Eat their usual diet for 2 days prior to MBT
- Refrain from consuming alcohol or dairy products for 24 hours prior to MBT
- Refrain from non-routine physical exercise for 20 hours prior to MBT
- Adhere to a 12-hour fast prior to MBT (from 8 PM previous night)
- Can drink water from 8:00 PM on the night before the test to 2:00 AM on the day of the test
- At visits 1 and 2, blood samples will be obtained at hourly intervals over a period of eight hours.
- Samples will be analyzed for PA, HA and Triglyceride concentrations.
- Patients will be permitted to ingest non-caloric and non-caffeinated beverages.
- At hour 6, patients will be provided 1000 Kcal, low fat (12 grams of fat) lunch meal.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Other: Pentadecanoic acid (PA) and Triheptadecanoin (THA) |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Subjects with cystic fibrosis and pancreatic insufficiency (n=9), 8 years of age or older, in usual state of good health will be eligible to participate in this Aim.
- Subjects will be screened for genotype and a history of pancreatic insufficiency (PI). PI will be confirmed by measurement of fecal elastase 1< 200 mg/g stool.
Key exclusion criteria (5 listed)
- Subjects will be ineligible if they have an FEV1 % predicted of < 40%, a history of fibrosing colonopathy or any significant bowel resection (>10 cm) with the exception of a routine, uncomplicated appendectomy.
- Subjects with diabetes mellitus or disorders associated with altered energy metabolism (i.e. hypothyroidism), or any major illnesses that affect the gastrointestinal tract, will not be eligible for the study.
- Subjects with significant liver disease, or significant developmental delay will also be excluded.
- Subjects will be excluded if they have a food allergy related to any components of the experimental shake (i.e., soy or chocolate) or the stool marker, carmine red.
- Subjects who are pregnant are not eligible. Subjects with a history of intolerance or allergy to Creon 20 will be excluded from the study.
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 |
|---|---|---|
| Pentadecanoic acid (PA) and Triheptadecanoin (THA)Two fats (Pentadecanoic acid and Triheptadecanoin) are administered in a breakfast shake for the purposes of determining malabsorption. in breakfast shake x1 | Other | 1 |
Primary outcome
Aim 1:Comparing Gastric Emptying and Small Bowel Transit Protocol in Healthy Controls
Time frame: 72 hours
3 secondary outcomes
Aim 2:The results will establish which pattern of timing of enzyme ingestion results in optimal fat absorption of PA and HA. The results will also elucidate the role of gastric emptying and small bowel transit in subjects in CF.
Time frame: 12-hour fast on 4 separate occasions, each at least 5 days apart
Aim 3: The results will establish the reproducibility of MBT as well as estimate the fecal loss of odd chain fatty acids (PA & THA)
Time frame: 12-hour fast on three separate occasions, each at least 5 days apart
Aim 4: Dosage Titration Protocol is being tested in order to see changes in absorption with MBT
Time frame: 12-hour fast on two separate occasions
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2008.
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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.
Maria Mascarenhas, M.D. principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Pentadecanoic acid (PA) and Triheptadecanoin (THA) in people with Cystic Fibrosis and Pancreatic Insufficiency, run by Children's Hospital of Philadelphia at 1 site 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 2 inclusion and 5 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 30 Sep 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Aim 1:Comparing Gastric Emptying and Small Bowel Transit Protocol in Healthy Controls (time frame: 72 hours). 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 30 Sep 2008.