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Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis (SPAN)

Primary Purpose

Exocrine Pancreatic Insufficiency, Cystic Fibrosis

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
adrulipase
Sponsored by
First Wave BioPharma, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Exocrine Pancreatic Insufficiency

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: A confirmed diagnosis of cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride >60 mmol/L by quantitative pilocarpine iontophoresis (measured while not on a CFTR modulator) or genotype. On stable dose of porcine PERT ≥1 month (30 days) prior to screening; stable dose is defined as dose of medication not changed during this time period, and the medication must be commercially available and be administered in the recommended dose range. CFA = or > 80% at screening while on stable PERT A fair or better nutritional status as defined by: BMI ≥16.0 kg/m2 for female patients ≥18 years of age, or BMI ≥16.5 kg/m2 for male patients ≥18 years of age Fecal elastase <100 µg/g of stool at screening Standard-of-Care medications including CFTR modulators are allowed Exclusion Criteria: History or diagnosis of fibrosing colonopathy Any chronic diarrheal illness unrelated to pancreatic insufficiency Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level 5 ×upper limit of normal (ULN), or total bilirubin level ≥1.5 ×ULN at Screening Feeding via an enteral tube during 6 months before screening Forced expiratory volume ≤30% at the Screening visit Changes in gastric acid suppressant therapy during the one month prior to screening for patients already on suppressant therapy.

Sites / Locations

  • Central Florida Pulmonary GroupRecruiting
  • The Cystic Fibrosis InstituteRecruiting
  • Childrens Lung SpecialistsRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Adrulipase

Arm Description

Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase.

Outcomes

Primary Outcome Measures

Safety of adrulipase
Number of subjects reporting 1 or more adverse events.
Efficacy of adrulipase: Coefficient of fat absorption (CFA)
The primary efficacy endpoint is the CFA that will be assessed at the end of the 3-week treatment period. CFAs for adrulipase will be compared to the CFAs of PERT obtained at baseline/eligibility using descriptive methods.

Secondary Outcome Measures

Stool weight
Stool weights obtained during the supervised confinement visit at the end of the 3-week treatment period will be measured. Stool weights obtained during confinement on adrulipase will be compared to the stool weights during confinement on PERT obtained at baseline/eligibility using descriptive methods.
Coefficient of Nitrogen absorption (CNA)
CNA that will be assessed at the end of the 3-week treatment period. CNAs for adrulipase will be compared to the CNAs of PERT obtained at baseline/eligibility using descriptive methods.

Full Information

First Posted
January 31, 2023
Last Updated
August 28, 2023
Sponsor
First Wave BioPharma, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05719311
Brief Title
Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis
Acronym
SPAN
Official Title
SPAN: A Phase 2, Open Label, Multicenter, Pilot Study to Assess Safety and Efficacy of an Enteric Microgranule Formulation of Adrulipase in Patients With Exocrine Pancreatic Insufficiency (EPI) Due to Cystic Fibrosis (CF)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 1, 2023 (Actual)
Primary Completion Date
September 2023 (Anticipated)
Study Completion Date
October 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
First Wave BioPharma, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI). Currently, these patients take pancreatic enzymes that are obtained from pig pancreas to aid the digestion of food. The goals of this clinical study are to evaluate the safety and efficacy of a novel formulation of a non-porcine lipase, called adrulipase, in patients with EPI due to cystic fibrosis. The main question[s] the study aims to answer are: Is the novel formulation of adrulipase safe to use at the doses being evaluated in the clinical study. Is adrulipase as effective, or more effective, compared to the pig enzymes the patients currently use. Researchers will compare the results obtained with adrulipase to how the patients typically respond to their pig enzymes to see if adrulipase helps patients digest fats adequately and if their stomach feels good (signs and symptoms of malabsorption).
Detailed Description
This is an Phase 2, open label, single arm pilot study assessing the safety and efficacy of adrulipase in an enteric microgranule formulation. Patients with a confirmed diagnosis of cystic fibrosis who are 18 years of age or greater will be screened for eligibility if they have been clinically controlled on a stable dose of commercial pancreatic enzyme replacement therapy (PERT) for at least one month. Patients on cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies must have been on a stable dose for at least 3 months prior to study entry, and no dose changes will be made during the study. Patients receiving gastric acid suppressants must have been on a stable dose for at least one month prior to study entry and no dose changes will be made during the study. Upon obtaining an informed consent, potentially eligible patients will receive dietary counselling during the week prior to the scheduled date of confinement for collecting stool samples for calculation of baseline coefficient of fat absorption (CFA). This counselling will emphasize the importance of dietary stability during the study. Patients found to have a CFA of 80% or greater while receiving their commercial PERT and meeting the other eligibility criteria will be enrolled into the study. Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. The patient will remain on study for approximately three weeks, after which a repeat CFA will be obtained. A dose titration scheme will be used for determining whether a low, medium, or high dose of adrulipase may succeed in controlling signs and symptoms of exocrine pancreatic insufficiency (EPI) and provide a CFA of 80% or greater. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase. After patients reach 3 weeks of study and complete their end of study CFA, they will be returned to their pre-study commercial PERT. An end of study safety visit will be scheduled for one week after finishing adrulipase therapy. Safety assessments will be made by collecting adverse events, safety lab assessments, and immunologic assays to assess drug induced immune responses.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Exocrine Pancreatic Insufficiency, Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Adrulipase
Arm Type
Experimental
Arm Description
Upon study enrolment, the patient will be switched from their commercial PERT to receive adrulipase. Patients will initially receive a low dose of adrulipase. Upon the appearance of EPI symptoms, lasting at least three days, and upon discussion with the investigator, the patient will be switched to the medium dose of adrulipase. If signs and symptoms of EPI persist for three or more days, the patient will be switched to the high dose of adrulipase.
Intervention Type
Drug
Intervention Name(s)
adrulipase
Other Intervention Name(s)
MS1819
Intervention Description
Enteric microgranule formulation of adrulipase.
Primary Outcome Measure Information:
Title
Safety of adrulipase
Description
Number of subjects reporting 1 or more adverse events.
Time Frame
End of 3-week treatment period.
Title
Efficacy of adrulipase: Coefficient of fat absorption (CFA)
Description
The primary efficacy endpoint is the CFA that will be assessed at the end of the 3-week treatment period. CFAs for adrulipase will be compared to the CFAs of PERT obtained at baseline/eligibility using descriptive methods.
Time Frame
End of 3-week treatment period.
Secondary Outcome Measure Information:
Title
Stool weight
Description
Stool weights obtained during the supervised confinement visit at the end of the 3-week treatment period will be measured. Stool weights obtained during confinement on adrulipase will be compared to the stool weights during confinement on PERT obtained at baseline/eligibility using descriptive methods.
Time Frame
End of 3-week treatment period.
Title
Coefficient of Nitrogen absorption (CNA)
Description
CNA that will be assessed at the end of the 3-week treatment period. CNAs for adrulipase will be compared to the CNAs of PERT obtained at baseline/eligibility using descriptive methods.
Time Frame
End of 3-week treatment period.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: A confirmed diagnosis of cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride >60 mmol/L by quantitative pilocarpine iontophoresis (measured while not on a CFTR modulator) or genotype. On stable dose of porcine PERT ≥1 month (30 days) prior to screening; stable dose is defined as dose of medication not changed during this time period, and the medication must be commercially available and be administered in the recommended dose range. CFA = or > 80% at screening while on stable PERT A fair or better nutritional status as defined by: BMI ≥16.0 kg/m2 for female patients ≥18 years of age, or BMI ≥16.5 kg/m2 for male patients ≥18 years of age Fecal elastase <100 µg/g of stool at screening Standard-of-Care medications including CFTR modulators are allowed Exclusion Criteria: History or diagnosis of fibrosing colonopathy Any chronic diarrheal illness unrelated to pancreatic insufficiency Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level 5 ×upper limit of normal (ULN), or total bilirubin level ≥1.5 ×ULN at Screening Feeding via an enteral tube during 6 months before screening Forced expiratory volume ≤30% at the Screening visit Changes in gastric acid suppressant therapy during the one month prior to screening for patients already on suppressant therapy.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Rey Driz
Phone
561-589-7020
Email
rdriz@firstwavebio.com
First Name & Middle Initial & Last Name or Official Title & Degree
James Pennington, MD
Phone
561-589-7020
Email
jpennington@firstwavebio.com
Facility Information:
Facility Name
Central Florida Pulmonary Group
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kathleen Summo
Phone
407-841-1100
Ext
209
First Name & Middle Initial & Last Name & Degree
Daniel Layish, MD
Facility Name
The Cystic Fibrosis Institute
City
Northfield
State/Province
Illinois
ZIP/Postal Code
60093
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Steven Boas, MD
Phone
847-998-3434
Email
sboas@wecare4lungs.com
First Name & Middle Initial & Last Name & Degree
Steven Boas, MD
Facility Name
Childrens Lung Specialists
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89109
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Barbara Glover
Phone
702-598-4411
Email
cfcentersn@gmail.com
First Name & Middle Initial & Last Name & Degree
Craig Nakamura, MD

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis

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