Long-term Safety Follow-up Study in the Prevention of Bronchiolitis Obliterans Syndrome (BOS)

A Long-term Safety Follow-up Study of L-CsA Therapy for Patients Who Participated in Study 12011.201 and Volunteered to Continue or to Cross-over to L-CsA Inhalation Therapy

The purpose of this study is to evaluate long-term safety of L-CsA in prevention of bronchiolitis obliterans syndrome (BOS) following lung transplantation (LTx) in patients previously enrolled in phase II/III L-CsA clinical trial 12011.201.

Study Overview

• Status: Terminated
• Conditions: Bronchiolitis Obliterans
• Intervention / Treatment: Drug: Inhalation

Detailed Description

This is an uncontrolled extension study open only to patients who completed the Phase II/III core study 12011.201 and volunteered to continue or to cross-over to L-CsA inhalation therapy.

Determination of long-term safety and efficacy outcome over a maximum of three years will be assessed.

• Study Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 3

Contacts and Locations

Study Locations

• Germany
  • Graefelfing, Germany, 82166
    • PARI Pharma GmbH

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Patient has completed the L-CsA clinical trial 12011.201
2. Patient is capable of understanding the purpose and risks of the follow-up study, has been fully informed and has given written informed consent to participate in the study
3. Female patients of child bearing potential must test negative on standard urine pregnancy test prior to continuation and must agree to practice effective birth control during the study
4. Estimated life expectancy > 6 months
5. Capable of self-administration of medications
6. Patient has stable creatinine levels

Exclusion Criteria:

1. Patients with ongoing irreversible L-CsA related serious adverse events
2. Patients with known hypersensitivity for ciclosporin A
3. Patient intends to participate in another IMP clinical trial other than listed in the inclusion criteria
4. Patient receives mechanical ventilation
5. Patients underwent pulmonary re-transplantation
6. Patient is a pregnant or breast-feeding woman
7. Patient is unlikely to comply with visits, inhalation procedures or spirometric measurements scheduled in the protocol
8. Patient receives any systemic or topical Rosuvastatin
9. Patient has been previously enrolled in this study
10. Patient with malignancy diagnosed during study 12011.201 (with the exception of skin cancer)
11. Documented respiratory infections unless on appropriate antimicrobial therapy with evidence of clinical response
12. Patient is not eligible to continue IMP inhalation according to the Investigator's discretion

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: L-CsA; Twice daily inhalation of L-CsA	Drug: Inhalation; Twice daily inhalation for a maximum of three years.; Other Names: aerosolized liposomal CsA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long-term safety of L-CsA in prevention of bronchiolitis obliterans syndrome (BOS)	Safety will be assesses by the numbers of treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) and overall rate of mortality.	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long-term efficacy outcome over a maximum of three years of patients previously enrolled in phase II/III L-CsA clinical trial 12011.201	Efficacy will be assessed mainly for the following efficacy endpoints: BOS-free survival; Incidence of BOS; Pulmonary function	3 years

Collaborators and Investigators

• Sponsor: Pari Pharma GmbH
• Investigators: Principal Investigator: Claus Neurohr, MD, Ludwig-Maximilians - University of Munich

Study record dates

Study Major Dates

• Study Start: February 1, 2012
• Primary Completion (Actual): July 1, 2013
• Study Completion (Actual): December 1, 2014

Study Registration Dates

• First Submitted: September 22, 2011
• First Submitted That Met QC Criteria: September 22, 2011
• First Posted (Estimate): September 23, 2011

Study Record Updates

• Last Update Posted (Estimate): April 14, 2015
• Last Update Submitted That Met QC Criteria: April 13, 2015
• Last Verified: April 1, 2015

More Information

Terms related to this study

• Keywords: Inhalation; Cyclosporine; Lung Transplantation
• Additional Relevant MeSH Terms: Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Bronchitis; Bronchiolitis; Bronchiolitis Obliterans
• Other Study ID Numbers: 12011.203; 2011-004304-38 (EudraCT Number)