A Study of OligoG in Cystic Fibrosis Subjects With Burkholderia Spp. Infection

NCT02453789 | Completed Phase 2 DMC

• 1 other identifier: SMR-2591
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of the study is to assess the efficacy of Alginate oligosaccharide (OligoG) dry powder for inhalation in cystic fibrosis (CF) patients with a Burkholderia spp. infection.

Conditions

Cystic Fibrosis; Burkholderia Infection

Keywords

alginate oligosaccharide; cystic fibrosis; Burkholderia; aztreonam

Outcome Measures

Primary Outcomes (1)

• Changes in Burkholderia spp. density in expectorated sputum and/or induced sputum.

  28 days, i.e. at start and end of treatment

Secondary Outcomes (4)

• Clinical safety as measured by vital signs

  Time Frame: Screening, day 0, 14, 28, 56, 70, 84 and follow up at day 112

• Clinical safety as measured by ECG

  Time Frame: Screening, day 0, 14, 28, 56, 70, 84 and follow upat day 112

• Clinical safety as measured by blood oxygen saturation

  Time Frame: Screening, day 0, 14, 28, 56, 70, 84 and follow upat day 112

• Clinical safety as measured by FEV1 (Forced Expiratory Volume in 1 second)

  Time Frame: Screening, day 0, 14, 28, 56, 70, 84 and follow up at day 112

Study Arms (2)

Alginate oligosaccharide

ACTIVE COMPARATOR

Inhalation of a dry powder OligoG in the first treatment period, and placebo in the second period

Drug: Alginate oligosaccharide

Placebo

PLACEBO COMPARATOR

Inhalation of placebo dry powder in the first treatment period, and OligoG in the second period

Drug: Placebo

Interventions

Alginate oligosaccharide DRUG

Inhalation of dry powder for inhalation (DPI)

Also known as: OligoG

Alginate oligosaccharide

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female with a confirmed diagnosis of cystic fibrosis defined by:
• Clinical features consistent with the diagnosis of CF; AND
• Sweat chloride ≥60 mmol/L by pilocarpine iontophoresis; OR
• Genotypic confirmation of CFTR mutation
• Aged 18 years or older
• Ability to provide sputum samples for microbiological evaluation throughout the study either spontaneously or induced.
• Chronic colonization with Burkholderia spp. defined as at least two positive microbiological cultures in expectorated sputum within the last 12 months from Visit 1.
• Use of inhaled aztreonam three times daily in a 4 weeks on/off cycle treatment regimen or a continuous intake regimen for at least 4 weeks before screening visit. For on/off cycles, screening visit should take place in the off phase. Randomization visit should take place the first day "on" to harmonize the aztreonam inhalation period with the IMP intake period.
• Willingness to stop treatment with other inhaled antibiotics.
• At Screening no clinical or laboratory findings suggestive of significant pulmonary illness, other than CF, which in the opinion of the investigator would preclude participation in the study.
• FEV1 greater than 25% of the predicted normal value following adjustment for age, gender, and height according to the Global Lung Initiative
• Female subjects of child bearing potential and male subjects participating in the study who are sexually active must use acceptable contraception. Female subjects documented as being of non-child-bearing potential are exempt from the contraceptive requirements.
• Provision of written informed consent.

You may not qualify if:

• Changes in underlying therapy within the 14 days prior to Day 0. Subjects must be willing to remain on the same underlying stable therapy regimens for the duration of the study until the final follow-up visit at Day 98.
• Changes in physiotherapy technique or schedule within 14 days prior to Day 0.
• Prohibited medications within 7 days prior to Day 0. Concomitant administration of inhaled mannitol or hypertonic saline within 7 days prior to Day 0.
• Concomitant use of inhaled antibiotics other than aztreonam.
• Pulmonary exacerbation within 28 days of Screening.
• Lactose intolerance/milk allergy.
• On-going acute illness. Subjects must not have needed an outpatient visit, hospitalization or required any change in therapy for other pulmonary disease between Screening and Day 0.
• History of, or planned organ transplantation.
• Active allergic bronchopulmonary aspergillosis (ABPA) in the last 12 months prior to Screening, defined as having received treatment for ABPA.
• Inability or unwillingness to provide sputum samples for microbiological evaluation throughout the study either spontaneously or induced by means of using inhaled hypertonic saline.
• Clinically significant abnormal findings on haematology or clinical chemistry. In addition, any value ≥ 3 x the upper limit of normal will exclude the subject from participating in the study.
• Subjects unable to perform pulmonary function tests according to the ATS/ERS criteria.
• Pregnant or breast-feeding women. A negative urine pregnancy test must be demonstrated in females of child-bearing potential at Screening.
• Subjects who have participated in any interventional clinical trial within the 28 days prior to Day 0.
• Subjects with documented or suspected, clinically significant, alcohol or drug abuse, as determined by the Investigator.

Sponsors & Collaborators

• AlgiPharma AS: lead

Study Sites (2)

Pneumologische Praxis Pasing

Münich, Münich-Pasing, 81241, Germany

Location

Charité Universitätsmedizin Berlin

Berlin, 13353, Germany

Location

Related Publications (2)

• Fischer R, Schwarz C, Weiser R, Mahenthiralingam E, Smerud K, Meland N, Flaten H, Rye PD. Evaluating the alginate oligosaccharide (OligoG) as a therapy for Burkholderia cepacia complex cystic fibrosis lung infection. J Cyst Fibros. 2022 Sep;21(5):821-829. doi: 10.1016/j.jcf.2022.01.003. Epub 2022 Jan 24.

  PMID: 35086790 DERIVED

• Hurley MN, Smith S, Forrester DL, Smyth AR. Antibiotic adjuvant therapy for pulmonary infection in cystic fibrosis. Cochrane Database Syst Rev. 2020 Jul 16;7(7):CD008037. doi: 10.1002/14651858.CD008037.pub4.

  PMID: 32671834 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis; Burkholderia Infections

Interventions

oligoG CF-5-20

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Gram-Negative Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Study Officials

• Rainald Fischer, MD, PD

  Pneumologische Praxis Pasing

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 14, 2015
• First Posted: May 27, 2015
• Study Start: February 1, 2015
• Primary Completion: March 1, 2017
• Study Completion: December 1, 2017
• Last Updated: April 19, 2018

Record last verified: 2018-04

Locations

DE (2)