Phase 2 N=36 Treatment

Targeted Therapy of Bronchiolitis Obliterans Syndrome

Bronchiolitis Obliterans

Bottom Line

View on ClinicalTrials.gov: NCT01307462 ↗

Enrolled (actual)

Serious AEs

66.7%

Results posted

Oct 2017

Primary outcome: Primary: Number of Subjects Who Failed Treatment — 2 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: fluticasone propionate (Drug); montelukast sodium (Drug); azithromycin (Drug)
Age: Pediatric, Adult, Older Adult · 6+ yrs
Sex: All
Sponsor: Stephanie Lee
Primary completion: Sep 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Subjects Who Failed Treatment	2	—
SECONDARY Number of Subjects Who Experienced Grade 3-5 SAEs Attributable to FAM and Number of Subjects Who Stopped FAM as a Result	11; 1	—
SECONDARY Number of Subjects Who Experienced Statistically Significant Changes in FVC, TLC, RV, DLCO	—	—
SECONDARY Changes in Blood Molecular Markers: IL8 (Azithromycin), Cysteinyl and LTB4 (Monteleukast), and IL1B, TNF, and IL6, as Well as Neutrophil Count (Fluticasone)	—	—
SECONDARY Number of Subjects With Improvements in Other Chronic GVHD Characteristics	12	—
SECONDARY Number of Subjects Were Able to Reduce Their Systemic Steroid Exposure by >=50%	17	—
SECONDARY Changes in Symptoms as Measured by Patient Self-report--Short Form-36 (SF-36)	0.0; 0.0; 0.0; -2.38; 1.56; 5.45	.81
SECONDARY Changes in Symptoms as Measured by Patient Self-report--Functional Assessment of Chronic Illness Therapy (FACT)	0.5; -1; 0; 1; -0.78; 2	0.28
SECONDARY Changes in Symptoms as Measured by Patient Self-report--Human Activities Profile (HAP)	0.5; 4.5; 3.5	.37
SECONDARY Changes in Symptoms as Measured by Patient Self-report--Lee Chronic GVHD Symptom Scale	-5.63; -7.14; -5; -8.33; 0; 0	.11

Summary

This phase II trial studies how well giving fluticasone propionate, azithromycin, and montelukast sodium (FAM) together works in treating patients with bronchiolitis obliterans who previously underwent stem cell transplant. FAM may be an effective treatment for bronchiolitis obliterans

Eligibility Criteria

Inclusion Criteria

Diagnosis of BOS after HCT within the 6 months before study enrollment; for this study, BOS is defined as:
Forced expiratory volume in 1 second (FEV1) = 10% lower than the pre-transplant absolute FEV1 as defined by the pre-transplant FEV1 minus the baseline FEV1, both measured before administration of a bronchodilator
Participant (or parent/guardian) has the ability to understand and willingness to sign a written consent document

Exclusion Criteria

Recurrent or progressive malignancy requiring anticancer treatment
Known history of allergy to or intolerance of montelukast, zafirlukast, azithromycin, erythromycin, or clarithromycin
Pregnancy or nursing; all females of childbearing potential must have a negative serum or urine pregnancy test 5 X upper limit of normal (ULN)
Total bilirubin > 3 X ULN
Chronic treatment with any inhaled steroid for > 1 month in the past three months
Treatment with montelukast or zafirlukast for > 1 month during the past three months
Treatment with prednisone at > 1.2 mg/kg/day (or equivalent steroid)
Treatment with rifampin or phenobarbital, aspirin at doses > 325 mg/day, or ibuprofen at doses > 1200 mg/day
Treatment with any Food and Drug Administration (FDA) non approved study medication within the past 4 weeks; off-label treatment with an FDA-approved medication is allowed
Chronic oxygen therapy
Evidence of any viral, bacterial or fungal infection involving the lung and not responding to appropriate treatment
Clinical asthma (variable and recurring symptoms of airflow obstruction and bronchial hyper-responsiveness)
Any condition that, in the opinion of the enrolling investigator, would interfere with the subject's ability to comply with the study requirements
Uncontrolled substance abuse or psychiatric disorder
Inability to perform pulmonary function tests (PFT) reliably, as determined by the enrolling investigator or PFT lab
Life expectancy < 6 months at the time of enrollment as judged by the enrolling investigator
Baseline post-bronchodilator FEV1 < 20% of predicted normal before or after albuterol

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01307462). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.