ANG003 for Cystic Fibrosis

Recruiting at 25 trial locations

Overseen By Evan Bailey, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Anagram Therapeutics, Inc.

Must be taking: Pancreatic enzymes

Disqualifiers: Fibrosing colonopathy, Lung transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 5

Duration: 3 weeks

113 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pancreatic insufficiency (EPI). People with EPI due to Cystic Fibrosis (CF) may be eligible to participate in this study. The primary objective of this study is to evaluate the safety of ANG003 and see if it works as well compared to Creon, an approved PERT.

Are You a Good Fit for This Trial?

Inclusion Criteria

I have severe EPI with a low fecal elastase test result.

I have controlled EPI and have been on the same PERT dose for at least 90 days.

My BMI is at or above the 25th percentile for my age (12-17 years).

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Acclimation

All subjects will be acclimated to Creon for 14 days and undergo a baseline CFA analysis

2 weeks

1 visit (in-person)

Treatment

Participants are randomized to ANG003 Dose A, ANG003 Dose B, Creon, or Off Enzyme for 21 days with subsequent CFA analysis

3 weeks

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ANG003

How Is the Trial Designed?

Group I: ANG003 Dose B Experimental Treatment 1 Intervention

All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is \>/= 80%, the subject may be randomized to ANG003 Dose B for a 21-day acclimation and subsequent CFA analysis.

Group II: ANG003 Dose A Experimental Treatment 1 Intervention

All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is \>/= 80%, the subject may be randomized to ANG003 Dose A for a 21-day acclimation and subsequent CFA analysis.

Group III: 60% to <80% CFA ANG003 Dose B Experimental Treatment 1 Intervention

All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. Participants with a Period A CFA result of 60% to \<80% will be assigned to ANG003 Dose B.

Group IV: Creon Active Control 1 Intervention

All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is \>/= 80%, the subject may be randomized to Creon for an additional 21-days and subsequent CFA analysis.

Group V: Off Enzyme Active Control 1 Intervention

All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is \>/= 80%, the subject may be randomized to the off enzyme arm. Subjects will receive Creon for an additional 21-day and the subsequent CFA analysis will be off enzyme. Total time off enzyme will be 4-7 days.

Who Is Running the Clinical Trial?

Anagram Therapeutics, Inc.

Lead Sponsor

Trials

1

Recruited

50+