NCT01131507

Brief Summary

A study to evaluate long term safety and effect on ability to thrive of EUR-1008 (APT-1008) 3,000 lipase units (Zenpep® \[pancrelipase\] delayed release capsules) in infants with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Jul 2010

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 25, 2010

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 27, 2010

Completed
1 month until next milestone

Study Start

First participant enrolled

July 1, 2010

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2011

Completed
2.3 years until next milestone

Results Posted

Study results publicly available

March 7, 2014

Completed
Last Updated

March 21, 2017

Status Verified

February 1, 2017

Enrollment Period

1.4 years

First QC Date

May 25, 2010

Results QC Date

January 27, 2014

Last Update Submit

February 14, 2017

Conditions

Keywords

Exocrine Pancreatic InsufficiencyInfants with CF

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    TEAE was any event not present prior to exposure to study drug or any event already present that worsened in either intensity or frequency following exposure to test drug. Serious AE (SAE) was any event that resulted in death, immediately life threatening, hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or a congenital anomaly/birth defect. Number of participants with TEAEs, SAEs, TEAE's relationship to study drug (unrelated, possible and probable) and on the basis of severity (mild \[minimal/no treatment and did not interfere with daily activities\], moderate \[resulted in a low level of inconvenience or concern with the therapeutic measures and may have caused some interference with functioning\] and severe \[interrupted participant's usual daily activity, may have required systemic drug therapy or other treatment and were usually incapacitating\]) with a frequency threshold of above 5% were reported.

    Up to Month 12 or early termination

Secondary Outcomes (1)

  • Change From Baseline in Growth Percentiles at Month 3, 6, 9 and 12

    Baseline, Month 3, 6, 9 and 12

Study Arms (1)

EUR-1008 (APT-1008)

EXPERIMENTAL
Drug: EUR-1008 (APT-1008)

Interventions

EUR-1008 (APT-1008) (Zenpep® \[pancrelipase\] 3,000 lipase units delayed release capsules) from open capsules mixed with a small amount of apple juice or apple sauce will be administered orally starting at the same dose as administered at the end of study PR-011 (NCT01100606), with dose increments of 3,000 lipase units. The dose will be adjusted based on participants' age and body weight. Total dose will not exceed 10,000 lipase units per kilogram (kg) of body weight per day unless clinically indicated. Total duration of study treatment will be up to 12 months.

Also known as: Zenpep® (pancrelipase) 3,000 lipase units delayed release capsules
EUR-1008 (APT-1008)

Eligibility Criteria

AgeUp to 12 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Participant's parent or guardian signed informed consent form (ICF)
  • Participants who have completed study PR-011 (NCT01100606)

You may not qualify if:

  • Participant having any condition that would, in the investigator's opinion, limit the participant's ability to complete the study or will result in excess risk to the participant that is above the standard of care

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Nemours Children's Clinic

Jacksonville, Florida, 32250, United States

Location

Children's Lung Specialists Ltd.

Las Vegas, Nevada, 89107, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Cystic Fibrosis Care Center

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Cystic FibrosisExocrine Pancreatic Insufficiency

Interventions

Pancrelipase

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

LipaseCarboxylic Ester HydrolasesEsterasesHydrolasesEnzymesEnzymes and CoenzymesPancreatic ExtractsTissue ExtractsComplex Mixtures

Results Point of Contact

Title
Robert Winkler, MD, VP, Clinical Development and Operations
Organization
Aptalis Pharma US, Inc.

Study Officials

  • Aptalis Medical Information

    Forest Laboratories

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 25, 2010

First Posted

May 27, 2010

Study Start

July 1, 2010

Primary Completion

December 1, 2011

Study Completion

December 1, 2011

Last Updated

March 21, 2017

Results First Posted

March 7, 2014

Record last verified: 2017-02

Locations