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Cystic FibrosisPhase 3NCT00297167

Study to Evaluate the Safety and Efficacy of EUR-1008 (APT-1008) Pancreatic Enzyme Product in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

A Phase 3 study testing EUR-1008 (APT-1008) in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Forest Laboratories at 1 site 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

The primary efficacy objective of this study is to compare the coefficient of fat absorption (CFA) following oral administration of Aptalis Pharma's (formerly Eurand Pharmaceuticals) pancreatic enzyme product (PEP) capsules and placebo in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency… Read more about this study

Who is it for?
Ages 7 years and older
What is being tested?
EUR-1008 (APT-1008)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
34 participants
Where
1 siteUnited States
Study dates
May 2006to Nov 2006
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 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Participants with age greater than or equal to (>=) 7 years at the time of enrollment
  • Participants with weight 70 kg or less and be in an adequate nutritional status as indicated by a body mass index (BMI) >=20 kg/m^2 for ages 18 and above, or a BMI above the twenty fifth percentile for participants aged 7 to 17 years
  • Participants with confirmed diagnosis of CF who have 2 clinical features consistent with CF, and have either a genotype with 2 identifiable mutations consistent with CF or a sweat chloride concentration that is more than 60 milliequivalent per liter by quantitative pilocarpine iontophoresis
  • Participants with confirmed diagnosis of EPI who are currently receiving treatment with a commercially available PEP and have documented with a fecal elastase of <100 microgram per gram stool (if no documentation was available, a stool sample was taken at Screening for determination of fecal elastase).
  • Clinically stable participants with no evidence of acute respiratory disease or any other acute condition
  • Participants who are willing and able to interrupt current CF treatment for CF-related malabsorption along with any medications that may affect gastric motility or stomach power of hydrogen (pH)
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  • Participants 18 years of age and older had to a) understand the requirements of the study, b) provide written informed consent, c) agree to abide by the study restrictions, and d) return for the required assessments
  • Participants 7 to 17 years of age must have a parent(s) or legal guardian who provides written informed consent, agree to abide by the study restrictions
  • Females participants of childbearing potential must have a negative serum pregnancy test at screening and must agree to use adequate birth control during the study

Who cannot join

  • Participants with fibrosing colonopathy, hyperuricemia or hyperuricosuria
  • Participants who are allergic to pork or other porcine PEPs
  • Participants with forced expiratory volume (FEV1) <30 percent of predicted FEV1 at screening
  • Participants with any acute systemic administration of an antibiotic for any reason in the previous 4 weeks; however, a low stable dose of an antibiotic or chronic treatment with an inhalatory antibiotic is allowed
  • Participants with hepatic insufficiency as defined by history or presence of ascites or serum albumin level of < 3.0 milligram/deciliter, or a coagulopathy with an international normalized ratio that is greater than 1.7
  • Participants who have used an acute dose of any steroid in the previous 2 weeks; however, low chronic doses of a steroid is allowed
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  • Participants with history of or current diagnosis of distal ileal obstruction syndrome (DIOS) as evidenced or suggested by constipation, abdominal pain, anorexia, early satiety, recurrent vomiting, and palpable fecal mass on physical examination (the absence of DIOS will be confirmed by an X-ray of the abdomen taken at screening)
  • Participants with any solid organ transplant or surgery affecting the bowel. Participants with a history of appendectomy and inguinal (non-incarcerated) hernioplasty or meconium ileus without the need for bowel resection could be enrolled. Gastrointestinal-tube-fed patients, in absence of dumping syndrome, were also eligible
  • Participants with history of or current screening evaluation of hyperglycemia as defined by an 8-hour fasting blood glucose (FBG) of >126 mg/dL, or of CF-related diabetes as determined according to the Cystic Fibrosis Foundation (CFF) Consensus Conference of January 1999 (Section IX Part II), that is: a) FBG >126 mg/dL (7.0 millimoles per liter [mmol/L]) on two or more occasions b)FBG >126 mg/dL (7.0 mmol/L) plus casual (without regard to time of day or last meal consumed) glucose level >200 mg/dL (11.1 mmol/L) c)Casual (previously called random) glucose levels >200 mg/dL (11.1 mmol/L) on two or more occasions with symptoms
  • Participants using an enzyme preparation in excess of 10,000-lipase units/kg/day
  • Participants using an immunosuppressive drug
  • Participants who are expecting an inability to tolerate the washout period and/or the placebo treatment
  • Participants participating in an investigational study of a drug, biologic, or device not currently approved for marketing, within 30 days of screening visit
  • Female participants who are pregnant or breastfeeding, or unwilling to use effective birth control during study
  • Participants with any condition that would, in the investigator's opinion, limit the participant's ability to complete the study

Trial locations and contacts

1 site in 1 country.

United States1 site
  • University of Texas Health Center at Tyler

    Tyler, Texas, 75708

Other trials in United States

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, Two-Treatment, Crossover Study to Evaluate the Safety and Efficacy of Eurand Pancreatic Enzyme Product (PEP) in Patients With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

The primary efficacy objective of this study is to compare the coefficient of fat absorption (CFA) following oral administration of Aptalis Pharma's (formerly Eurand Pharmaceuticals) pancreatic enzyme product (PEP) capsules and placebo in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).

Read the detailed description

This is a randomized, double-blind, placebo-controlled, 2-treatment, crossover, multicenter trial in participants with CF and EPI. The study consists of a screening period (1 to 14 days), a washout period (2 days), a dose titration/stabilization period (6 to 9 days), a blinded randomized treatment period (6 to 7 days), an open-label normalization period 1 (5 to 14 days), a blinded crossover treatment period (6 to 7 days), followed by an open-label normalization period 2 (7 days). The order of treatments (placebo followed by EUR-1008 [APT-1008] or EUR-1008 [APT-1008] followed by placebo) will be determined by randomization at the beginning of randomization treatment period only and will be carried through the crossover treatment period. The starting dose will be 1,000 lipase units per kilogram per meal (lipase units/kg/meal), which will be titrated to control symptoms of EPI, with the total dose not exceeding 10,000 lipase units/kg/day.

Being tested
EUR-1008 (APT-1008), Placebo
Keywords
CF, Cystic Fibrosis, EPI, Exocrine Pancreatic Insufficiency, Pancreatic, Enzyme, PEP
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.

  • EUR-1008 (APT-1008)Drug

    EUR-1008 (APT-1008) microtablets (5000 lipase units) or minitablets (10000, 15000 or 20000 lipase units) contained in a capsule will be given orally daily in first double-blind intervention period; at a dose based on investigator's discretion as achieved during dose titration and stabilization period (6 to 9 days). The capsules could be opened and sprinkled on acidic food and half of the established dose was used with snacks. Treatment duration for first double-blind intervention period will be 2 days home treatment and 3 to 5 days hospital treatment.

  • PlaceboDrug

    Placebo matched to EUR-1008 (APT-1008) microtablets or minitablets contained in a capsule will be given orally daily in second double-blind intervention period; at a dose based on investigator's discretion as achieved during dose titration and stabilization period (6 to 9 days). Treatment duration for second double-blind intervention period will be 2 days home treatment and 3 to 5 days hospital treatment.

  • PlaceboDrug

    Placebo matched to EUR-1008 (APT-1008) microtablets or minitablets contained in a capsule will be given orally daily in first double-blind intervention period; at a dose based on investigator's discretion as achieved during dose titration and stabilization period (6 to 9 days). Treatment duration for first double-blind intervention period will be 2 days home treatment and 3 to 5 days hospital treatment.

  • EUR-1008 (APT-1008)Drug

    EUR-1008 (APT-1008) microtablets (5000 lipase units) or minitablets (10000, 15000 or 20000 lipase units) contained in a capsule will be given orally daily in second double-blind intervention period; at a dose based on investigator's discretion as achieved during dose titration and stabilization period (6 to 9 days). Treatment duration for second double-blind intervention period will be 2 days home treatment and 3 to 5 days hospital treatment.

Study groups in NCT00297167
Study groupRoleWhat they receive
EUR-1008 (APT-1008) First, Then PlaceboExperimentalNot described
Receives: Drug: EUR-1008 (APT-1008); Drug: Placebo
Placebo First, Then EUR-1008 (APT-1008)ExperimentalNot described
Receives: Drug: Placebo; Drug: EUR-1008 (APT-1008)

What is being measured

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

  1. Percent Coefficient of Fat Absorption (CFA%)

    Percent CFA was calculated as ([fat intake - fat excretion]/fat intake)multiplied by 100, determined in the stools collected during the 72-hour hospitalization period. Mean percent CFA was calculated for Day 3 to Day 6 during hospital treatment in first and second double-blind (DB) intervention periods.

    Measured: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods

Secondary outcomes (7)
  1. Percent Coefficient of Nitrogen Absorption (CNA%)

    Percent CNA was calculated as ([nitrogen intake-nitrogen excretion]/nitrogen intake)*100, determined in the stools collected during the 72-hour hospitalization period. Nitrogen intake was calculated as protein intake/6.2. Nitrogen excretion was measured as total fecal nitrogen. Mean percent CNA was calculated for Day 3 to Day 6 during hospital treatment in first and second double-blind intervention periods.

    Measured: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods

  2. Lipid Levels

    Lipid levels were reported for total cholesterol (TC) and high-density lipoprotein cholesterol (HDL-C) from fasted blood and urine samples. Mean lipid levels for Day 6 during first and second double-blind intervention periods were calculated.

    Measured: End of treatment (Day 6 during first and second double-blind intervention periods)

  3. Vitamin A Levels

    Mean Vitamin A levels for Day 6 during first and second double-blind intervention periods were calculated.

    Measured: End of treatment (Day 6 during first and second double-blind intervention periods)

  4. Vitamin E Levels

    Mean Vitamin E levels for Day 6 during first and second double-blind intervention periods were calculated.

    Measured: End of treatment (Day 6 during first and second double-blind intervention periods)

  5. Mean Daily Number of Stools

    Mean daily number of stools of each participant was calculated from frequency of stools by the participant per day. Mean daily number of stools during the collection period (Day 3 to Day 6 in first and second double-blind intervention periods) for total participants was summarized.

    Measured: Day 3 up to Day 6 during first and second double-blind intervention periods

  6. Percentage of Stool Categorized as Per Consistency

    Stool consistency was categorized as hard, formed/normal, soft, watery, or overt diarrhea. Percentage of stools of a specific consistency for each participant at first and second double-blind intervention periods was calculated. Mean percentage of stool consistency during the collection period (Day 3 to Day 6 in first and second intervention periods) for total participants was summarized.

    Measured: Day 3 up to Day 6 during first and second double-blind intervention periods

  7. Mean Number of Abdominal Symptoms

    Abdominal symptoms included abdominal pain, flatulence and bloating. Symptoms were classified by severity as mild (no impairment of daily activities), moderate (slight impairment of daily activities), or severe (unable to perform daily activities). Mean number of symptom of specific severity per day for each participant was calculated. Mean number of symptoms per day was calculated for Day 3 to Day 6 in first and second double-blind intervention periods for total participants.

    Measured: Day 3 up to Day 6 during first and second double-blind intervention periods

Timeline

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

  1. First posted28 Feb 2006 (estimated)
  2. Study startMay 2006
  3. Primary completionNov 2006
  4. Study completionNov 2006

Resources

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
Enrolment
34 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00297167
Sponsor's protocol ID
EUR-1008-M
Status last verified
Feb 2017
First submitted
27 Feb 2006
Last updated
16 Mar 2017

Publications

  • Wooldridge JL, Heubi JE, Amaro-Galvez R, Boas SR, Blake KV, Nasr SZ, Chatfield B, McColley SA, Woo MS, Hardy KA, Kravitz RM, Straforini C, Anelli M, Lee C. EUR-1008 pancreatic enzyme replacement is safe and effective in patients with cystic fibrosis and pancreatic insufficiency. J Cyst Fibros. 2009 Dec;8(6):405-17. doi: 10.1016/j.jcf.2009.07.006. Epub 2009 Aug 15.

    PubMed 19683970

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing EUR-1008 (APT-1008) in people with Cystic Fibrosis and Exocrine Pancreatic Insufficiency, run by Forest Laboratories 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 9 inclusion and 15 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 16 Mar 2017. 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 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 4 Apr 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?

Percent Coefficient of Fat Absorption (CFA%) (time frame: Day 3 up to Day 6 of hospital treatment in first and second double-blind intervention periods). 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 NCT00297167, which also lists 1 linked publication.

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 16 Mar 2017.

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