A Phase 2 Study to Evaluate Safety and Efficacy of AMB-05X in Subjects With Idiopathic Pulmonary Fibrosis

Study Overview
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Brief Summary
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AMB-053-01 is a randomized, placebo controlled, multicenter study which will enroll approximately 36 subjects ages 40 and older with IPF for 6 doses over a 24-week dosing period.

Official Title
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A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of AMB-05X in Subjects With Idiopathic Pulmonary Fibrosis

Conditions
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IPF

Intervention / Treatment
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* Biological: AMB-05X
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects ≥40 years old. History of confirmed diagnosis of IPF Chest HRCT at Screening Subjects who are either: Not being treated with approved IPF therapies (i.e., nintedanib or pirfenidone) or Being treated with approved IPF therapies (i.e., nintedanib or pirfenidone) Has FVC ≥45% predicted of normal AND DLCO ≥25% and ≤90% predicted Has a FEV1/FVC ratio (Tiffeneau-Pinelli Index) ≥ 0.70 Has adequate hematologic, hepatic, and renal function Exclusion Criteria: Prior investigational drug use within 30 days or 5 half-lives Presence of emphysema exceeding the extent of fibrosis Active or anticipated need for lung transplant Treatment with prednisone Active cancer Active or chronic infection with HCV, HBV, or HIV Known active tuberculosis History of or current immunosuppressive condition IPF exacerbation within 12 weeks Lower respiratory-tract infection requiring antibiotic therapy Smoking Other forms of interstitial lung disease History of lung volume reduction surgery or lung transplant Contraindications for forced expiratory maneuvers during spirometry Unstable cardiac or pulmonary disease (other than IPF) Fridericia-corrected QT interval (QTcF) ≥ 450 ms (men) or ≥ 470 ms (women) History of drug or alcohol abuse -

Ages Eligible for Study
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Minimum Age: 40 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: 2:1 randomization of active to placebo
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: AMB-05X<br>AMB-05X will be administered every 4 weeks for 24 weeks (for 6 treatments in total). | Biological: AMB-05X<br>* A fully human monoclonal immunoglobulin (IgG2) directed against c-fms<br>|
| Placebo Comparator: Placebo<br>Placebo will be administered every 4 weeks for 24 weeks (for 6 treatments in total). | Drug: Placebo<br>* Saline/D5W<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment-emergent adverse events | Frequency and severity of reported treatment-emergent adverse events will be graded per CTCAE 5-point scale | Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pulmonary Function | Change in forced vital (FVC) capacity in liters | Week 28 | 
| Pharmacodynamics via CSF1 | Plasma CSF1 assessment and other exploratory biomarkers | Week 28 | 
| ST GEORGE'S RESPIRATORY QUESTIONNAIRE IDIOPATHIC PULMONARY FIBROSIS (SGRQ-I) | Change in health-related quality of life, as measured by St. George's Respiratory Questionnaire (SGRQ-I) | Week 28 | 
| Cmax | Peak Plasma Concentration (Cmax) measurement | Week 24 | 
| AUC | Area under the plasma concentration versus time curve (AUC) measurement | Week 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Idiopathic Pulmonary Fibrosis, IPF