Orally inhaled heparin in patients with cystic fibrosis (CF)

A phase I/II randomised, placebo-controlled, double blind trial to assess the safety, tolerability, pharmacodynamics and exploratory efficacy of heparin inhalation in patients with cystic fibrosis (CF)

Status

Recruiting

Phases

Phase 1

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12610000328077

Enrollment

Registered

2010-04-23

Start date

2009-01-26

Completion date

Unknown

Last updated

2020-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

The clinical trial is to assess the safety and tolerability and to explore the efficacy of orally inhaled heparin in patients with cystic fibrosis (CF). Heparin is expected to provide advantages over currently available treatments for CF in a convenient delivery system.

Interventions

Patients will be randomised to receive one of three daily dose levels of heparin treatment or matching placebo; to be self-administered by inhalation by the patient twice daily for four consecutive weeks. Nominal Daily Doses to be studied are: 11400 International Units (IU), 22800 IU and 45600 IU. For each patient there will be a screening period of 4 weeks, a treatment period of 4 weeks with a follow-up period of 2 weeks.

Study design

Allocation

Randomised controlled trial

Intervention model

Parallel

Primary purpose

Treatment

Masking

Blinded (masking used)

Eligibility

Sex/Gender

All

Age

16 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Male or female, aged 16 years or older 2. Non-smoker 3. Written informed consent obtained prior to any trial specific procedures 4. Confirmed diagnosis of CF lung disease (i.e., respiratory clinical symptoms and positive sweat test or disease inducing mutations) by CF expert/investigator 5. Forced expiratory volume in one second (FEV1) at 40 - 90% of predicted value for age, sex and height at screening and baseline 6. FEV1 value at Baseline is within +/-15% of value at screening 7. Regular mucus production due to CF 8. Ease of sputum expectoration as defined by VAS score equal to or less than 80 mm 9. Inflammatory markers above upper limit of normal range. 10. Adequate contraceptive measures. 11. Able to comply with all protocol requirements 12. Able to use inhalation device.

Exclusion criteria

To be eligible for inclusion into this trial, each patient must not violate any one of the following exclusion criteria at the time of screening, at the time of assessment or as specifically described below: 1. Any contraindication to Monoparin( Registered Trademark) considered clinically relevant 2. Increased bleeding risk 3. History of heparin-induced thrombocytopaenia 4. Patients with bleeding diathesis 5. Evidence of portal hypertension (e.g., hypersplenism or known grade III/IV oesophageal varices) 6. Clinically significant liver disease 7. Pregnancy at screening, or lactation 8. Previous thoracic or scheduled major surgery during trial 9. Any regular anticoagulant therapy (e.g., warfarin, aspirin) in the two weeks prior to screening 10. Modification of medication to treat respiratory disease between screening and baseline (Day 1) 11. Diagnosis or history of aspergilloma 12. Clinically significant serious disease or organ system disease not currently controlled / stable on present therapy 13. Planned hospitalisations which could interfere with trial compliance 14. Unable for any other reason to satisfactorily comply with the protocol (e.g., attendance for trial visits, treatment or assessments)

Outcome results