Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Conditions
Keywords
Cystic Fibrosis, Pancreatic Exocrine Insufficiency
Brief summary
This study will assess the safety and tolerability of pancrelipase delayed release capsules in subjects up to 6 years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis.
Interventions
3,000, 6,000 and 12,000 unit Lipase Capsules
Sponsors
Solvay Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to 6 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed Cystic Fibrosis (CF) diagnosis by two positive chloride sweat tests or gene analysis * Current or historical human fecal elastase \< 50µg/gstool * Weight greater than 3.75 kg * Age 1 month to 6 years * 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE) | 10 Days | Treatment Emergent Adverse Events are defined as adverse events started at or after the first administration of study drug and include those events started prior to the first administration but which worsened after the first intake. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Stool Fat (% Fat) | Last 3 days in a 10-day treatment period | The stool fat content was calculated as percent fat of dry solid weight per bowel movement. Stool fat per patient was derived as mean over three bowel movements sampled (i.e. one sample per day). |
| Fat Intake (g) | Last 3 days in a 10-day treatment period | The mean daily fat intake was determined as the average of daily fat intake over a 3-day period. |
| Total Calorie Intake (kcal) | Last 3 days in a 10-day treatment period | The total calorie intake was determined as the average of total calorie of daily food intake intake over a 3-day period. |
Countries
United States
Participant flow
Recruitment details
Subjects were recruited in 9 centers in US between April 2009 and June 2009. During the period of screening (Days -21 to Days -7), subjects were evaluated for eligibility. They underwent a short period of up to 7 days on their usual pancreatic enzyme supplementation before receiving the study treatment.
Pre-assignment details
Nineteen subjects were consented and 18 subjects were allocated to pancrelipase delayed release capsule. One subject did not meet the fecal elastase requirement and did not receive study medication.
Participants by arm
| Arm | Count |
|---|---|
| Pancrelipase Delayed-Release Capsules Target dose of 8000 lipase units/kg/day. This dose was administered in divided doses with meals/snacks. | 18 |
| Total | 18 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol Violation | 1 |
Baseline characteristics
| Characteristic | Pancrelipase Delayed-Release Capsules |
|---|---|
| Age Continuous | 27.3 months STANDARD_DEVIATION 21.9 |
| Region of Enrollment United States | 18 participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 13 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 9 / 18 |
| serious Total, serious adverse events | 0 / 18 |
Outcome results
Primary
Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE)
Treatment Emergent Adverse Events are defined as adverse events started at or after the first administration of study drug and include those events started prior to the first administration but which worsened after the first intake.
Time frame: 10 Days
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Pancrelipase Delayed-Release Capsules | Number of Patients With at Least One Treatment Emergent Adverse Event (TEAE) | 9 Participants |
Secondary
Fat Intake (g)
The mean daily fat intake was determined as the average of daily fat intake over a 3-day period.
Time frame: Last 3 days in a 10-day treatment period
Population: The efficacy results are presented on the Full Analysis Set meaning patients having taken the study drug and with Post-baseline efficacy data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Pancrelipase Delayed-Release Capsules | Fat Intake (g) | 46.61 Grams | Standard Deviation 22.34 |
Secondary
Stool Fat (% Fat)
The stool fat content was calculated as percent fat of dry solid weight per bowel movement. Stool fat per patient was derived as mean over three bowel movements sampled (i.e. one sample per day).
Time frame: Last 3 days in a 10-day treatment period
Population: The efficacy results are presented on the Full Analysis Set meaning patients having taken the study drug and with Post-baseline efficacy data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Pancrelipase Delayed-Release Capsules | Stool Fat (% Fat) | 28.11 Percentage of fat | Standard Deviation 9.86 |
Secondary
Total Calorie Intake (kcal)
The total calorie intake was determined as the average of total calorie of daily food intake intake over a 3-day period.
Time frame: Last 3 days in a 10-day treatment period
Population: The efficacy results are presented on the Full Analysis Set meaning patients having taken the study drug and with Post-baseline efficacy data.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Pancrelipase Delayed-Release Capsules | Total Calorie Intake (kcal) | 1239.71 Kcal | Standard Deviation 605.78 |