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Cyclophosphamide for Acute Exacerbation of Idiopathic Pulmonary Fibrosis

Phase 3
Completed
Conditions
Idiopathic Pulmonary Fibrosis
Interventions
Drug: Placebo
Drug: Corticosteroid (prednisolone)
Registration Number
NCT02460588
Lead Sponsor
Assistance Publique - Hôpitaux de Paris
Brief Summary

Acute exacerbation of idiopathic pulmonary fibrosis (AE-IPF) is a major event of IPF with an annual incidence between 5 and 10% and is responsible for the death of one third of IPF patients. When AE-IPF occurs, it is associated with poor survival with an overall mortality at 3 months upper of 50%. To date, no treatment has been proved to be effective in AE-IPF but the efficacy of cyclophosphamide (CYC) on survival has been suggested, mainly by retrospective series and needs to be confirmed. This confirmation is mandatory to improve prognosis of AE-IPF but also to avoid unsuspected deleterious effect as it as been shown with immunosuppressor in stable IPF.

Detailed Description

Acute exacerbation of idiopathic pulmonary fibrosis (AE-IPF) is a major event of IPF with an annual incidence between 5 and 10% and is responsible for the death of one third of IPF patients. When AE-IPF occurs, it is associated with poor survival with an overall mortality at 3 months upper of 50%. To date, no treatment has been proved to be effective in AE-IPF but the efficacy of CYC on survival has been suggested, mainly by retrospective series and needs to be confirmed. This confirmation is mandatory to improve prognosis of AE-IPF but also to avoid unsuspected deleterious effect as it as been shown with immunosuppressor in stable IPF.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
120
Inclusion Criteria

Not provided

Exclusion Criteria
  • Identified etiology for acute worsening (i.e. infectious disease)
  • Known hypersensitivity or contra-indication to CYC or to any component of the study treatment
  • Patient on mechanical ventilation
  • Active bacterial, viral, fungal or parasitic infection
  • Active cancer
  • Patient on a lung transplantation waiting list
  • Treatment with CYC in the last 12 months
  • Patient participating to another clinical trial
  • Pregnancy or lactation

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Corticosteroid with placeboCorticosteroid (prednisolone)Population is IPF patients with an AE who meet the inclusion and exclusion criteria defined below. All patients will receive non experimental medication with high dose of corticosteroid.
Corticosteroid with placeboPlaceboPopulation is IPF patients with an AE who meet the inclusion and exclusion criteria defined below. All patients will receive non experimental medication with high dose of corticosteroid.
Corticosteroid associated with CyclophosphamideCorticosteroid (prednisolone)Population is IPF patients with an AE who meet the inclusion and exclusion criteria defined below. All patients will receive non experimental medication with high dose of corticosteroid. Intravenous Cyclophosphamide (CYC), 600 mg/m² (adapted to age and renal function, maximal dose of 1.2 g) at Day 0, Day 15, M1, M2
Corticosteroid associated with CyclophosphamideCyclophosphamidePopulation is IPF patients with an AE who meet the inclusion and exclusion criteria defined below. All patients will receive non experimental medication with high dose of corticosteroid. Intravenous Cyclophosphamide (CYC), 600 mg/m² (adapted to age and renal function, maximal dose of 1.2 g) at Day 0, Day 15, M1, M2
Primary Outcome Measures
NameTimeMethod
"Early" survival3 months

All cause of mortality at 3 months

Secondary Outcome Measures
NameTimeMethod
Diabetes mellitus (capillary blood glucose monitoring and fasting plasma glucose > 1.26 g/l)6 months
Respiratory disease-specific mortality6 months

Respiratory disease-specific mortality at M3 and M6

Prognosis factors of AE-IPF3 months

Chest HRCT classification before AE-IPF (definite UIP, probable UIP, indeterminate), if available

Clinical laboratory evaluation (blood count, serum creatinin measurement composite) according to Common Terminology Criteria for Adverse Event (CTCAE).6 months
Overall Survival6 months and 12 montns

Overall Survival at M6 and M12

Respiratory Morbidity6 months

\\Worsening dyspnea (0-100-mm visual analogue (VAS) scale anchored with 0 ''no breathlessness'' and 10 or 100 ''worst imaginable breathlessness". Worsening is defined an absolute decrease of 10 mm)

* Or Increase need of supplemental oxygen of more than 3l/min to obtained a SaO2 \> 90% or decrease of PaO2 of more than 10 mmHg with the same rate of flow supplemental oxygen

* Or Decrease FVC of more than 10% of predicted value

* Or Decrease diffuse capacity for carbon monoxide (DLCO) of more than 15% prednisolone

Time to visit after clinical worsening3 months
Hemorrhagic cystitis (occurence of hematuria on urine dipstick and pelvic pain and/or dysuria should lead to cystoscopy)6 months
Chest HRCT features (HRCT images will be scored at 5 levels)6 months

Chest HRCT features at M3 and M6 compared to inclusion

Time to dispense treatment of AE-IPF3 months
Hypertension (Blood pressure > 160/100 mmHg)6 months
Laboratory evaluation (LDH, CRP) at AE diagnosis (composite)3 months
Number of Infectious disease6 months

Trial Locations

Locations (1)

Hôpital Tenon

🇫🇷

Paris, France

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