A Dose-finding Study of Inhaled OligoG vs Placebo in Patients With Cystic Fibrosis (SMR3372)

A Randomized, Double Blind, Dose-finding Study of Inhaled Oligosaccharide (OligoG) vs Placebo in Patients With Cystic Fibrosis

Randomized, double blind, placebo controlled study. The study has two parts:

Dose-finding part, followed by longer term follow-up (6 months)

Study Overview

• Status: Withdrawn
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: OligoG Dry powder for inhalation

Detailed Description

Part 1: Randomized, double blind, placebo controlled dose-finding. Patients will be assigned to 1 of 3 doses OligoG, or to placebo, on top of Standard of Care. Patients will be treated for 12 weeks, followed by 4 weeks washout.The primary endpoint is relative change in % predicted FEV1. Secondary endpoints include additional spirometry parameters, exacerbation rate, Quality of Life, sputum rheology and microbiology, safety laboratory tests and adverse event reporting.

Part 2: Randomized double-blind 6 -month study, for longer term follow-up of the dose identified in Part 1. New patients will be recruited in part 2, in addition to patients who received placebo in Part 1. In addition to the endpoints studied in Part 1, Part 2 will include Lung Clearance Index (LCI), chest imaging by MRI or CT, and pharmaco-economic parameters.

• Study Type: Interventional
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Germany
  • Cologne, Germany, 50924
    • CF Zentrum Köln, Universitätskrankenhaus Köln

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Confirmed diagnosis of CF
• FEV1 at screening >40 and < 90% of predicted normal
• History of PA infection in last 12 m
• History of antibiotic treatment due tp PA infection (not for eradication) during last 12 m
• Concomitant inhaled tobramycin, colistin, aztreonam, or levoflaxin (cycled or continuous)
• Stable CF disease
• Willing to remain on stable CF medication (Standard of care)
• Women of child-bearing potential must habe negative urine pregnany test
• Males & females must use acceptable contraception
• Capable of inhaling dry powder
• willing to sign informed consent
• willing and able to follow study procedures

Exclusion Criteria:

• Use of hypertonic saline > twice daily
• Clinically significant abnormal lab findings, except CRP. In case of high GGT values, case will be discussed by experts before deciding
• History of comorbidity that may distort results or cause additional risk
• pulmonary exacerbation within 28 days prior to randomisation
• Change in CF therapy within 28 days prior to randomisation
• Burkholderia spp. finding within 12 m prior to screening
• pregnant or breast feeding females
• History of allergic reactions to IMP ingredients, incl milk protein
• Inability to perform lung function tests according to ATS/ ERS criteria
• Uncontrolled or unstable diseases that might limit compliance
• History of, or planned organ transplantation
• Allergic ABPA in the last 12 months prior to the screening visit
• Requirement for continuous oxygen supplementation
• Current participation in another clinical study
• medical condition, other than CFwhich exposes the patient to an unacceptably high risk
• Concurrent mlignant disease, except BCC and cervical neoplasia
• Clinically significant alcohol or drug abuse

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo DPI; Matching placebo dry powder for inhalation. OligoG is replaced by lactose. 10 capsules, BID	Drug: OligoG Dry powder for inhalation; 10 capsules Dry Powder for Inhalation, BID; Other Names: Placebo dry powder for inhalation
Active Comparator: Low dose OligoG DPI; 17.5 mg OligoG dry powder for inhalation. 10 capsules, BID	Drug: OligoG Dry powder for inhalation; 10 capsules Dry Powder for Inhalation, BID; Other Names: Placebo dry powder for inhalation
Active Comparator: medium dose OligoG DPI; 27.5 mg OligoG dry powder for inhalation. 10 capsules, BID	Drug: OligoG Dry powder for inhalation; 10 capsules Dry Powder for Inhalation, BID; Other Names: Placebo dry powder for inhalation
Active Comparator: High dose OligoG DPI; 37.5 mg OligoG dry powder for inhalation. 10 capsules, BID	Drug: OligoG Dry powder for inhalation; 10 capsules Dry Powder for Inhalation, BID; Other Names: Placebo dry powder for inhalation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Forced Expiratory Volume in 1 second (FEV1)	the amount of air a person can forcefully exhale in one second	Change from before to after 12 weeks treatment (Part 1) and 26 weeks (Part 2)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lung Clearance Index (LCI) (Part two only)	Lung clearance index (LCI) measured by multiple breath washout is a sensitive measure of ventilation inhomogeneity.	Change from before to after 26 weeks treatment
Sputum culture microbiology	Reduction in Pseudomonas Aeruginosa CFU Counts and total counts	Change from before to after 12 weeks treatment (Part 1) and 26 weeks (Part 2)
Pulmonary Exacerbations	Acute pulmonary exacerbations	Change from before to after 12 weeks treatment (Part 1) and 26 weeks (Part 2)

Collaborators and Investigators

• Sponsor: AlgiPharma AS
• Collaborators: Imperial College London; Cystic Fibrosis Foundation; University Hospital of Cologne; European Cystic Fibrosis Society; Cystic Fibrosis Europe
• Investigators: Principal Investigator: Silke van Koningsbruggen-Rietschel, MD, PhD, University Hospital Cologne

Study record dates

Study Major Dates

• Study Start (Anticipated): September 1, 2021
• Primary Completion (Anticipated): October 1, 2022
• Study Completion (Anticipated): November 1, 2022

Study Registration Dates

• First Submitted: October 3, 2018
• First Submitted That Met QC Criteria: October 5, 2018
• First Posted (Actual): October 9, 2018

Study Record Updates

• Last Update Posted (Actual): June 30, 2022
• Last Update Submitted That Met QC Criteria: June 27, 2022
• Last Verified: June 1, 2022

More Information

Terms related to this study

• Keywords: Oligosaccharide
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: 3372; 2018-000378-30 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No