Study Evaluating Efficacy and Safety of Froniglutide (PF1801) in Patients With Idiopathic Inflammatory Myopathy

Study Overview
=================
Brief Summary
-----------------
This is a Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Froniglutide in Patients With Idiopathic Inflammatory Myopathy (FROniGlutide Study)

Official Title
-----------------
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Froniglutide (PF1801) in Patients With Idiopathic Inflammatory Myopathy (IIM)

Conditions
-----------------
Dermatomyositis, Polymyositis, Idiopathic Inflammatory Myopathies

Intervention / Treatment
-----------------
* Drug: Froniglutide
* Drug: Placebo


Participation Criteria
=================
Eligibility Criteria
-----------------
Key Inclusion Criteria: Diagnosis of IIM (DM or polymyositis [PM]) as per Bohan and Peter classification criteria MMT-8 ≤125 units and two out of the following CSM items, or MMT-8 >125 units and three out of the following CSM items, together with verifiable muscular weakness A. PGA VAS ≥2 cm B. SGA VAS ≥2 cm C. HAQ-DI ≥0.25 D. Extramuscular activity (MDAAT) ≥2 cm E. Increase in one or more muscle enzyme (CK, LDH, AST, ALT, aldolase) values (must be ≥1.3 × ULN) On treatment with standard of care (immunosuppressants and/or corticosteroids) for >12 weeks and on stable therapy for at least 4 weeks Key Exclusion Criteria: Inclusion body myositis (IBM) or amyopathic DM Severe muscle damage (myositis damage index [MDI] >7/10 cm). Permanent deterioration caused by reasons other than PM/DM, or myositis with cardiac involvement Clinically significant renal/hepatic impairment Severe interstitial lung disease requiring supportive oxygen therapy

Ages Eligible for Study
-----------------
Minimum Age: 19 Years
Maximum Age: 80 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Froniglutide 50 mg<br>Froniglutide (PF1801) 50 mg will be administered subcutaneously once a week. | Drug: Froniglutide<br>* SC Weekly Injection<br>* Other names: PF1801;|
| Experimental: Froniglutide 70 mg<br>Froniglutide (PF1801) 70 mg will be administered subcutaneously once a week. | Drug: Froniglutide<br>* SC Weekly Injection<br>* Other names: PF1801;|
| Placebo Comparator: Placebo<br>Matching placebo will be administered subcutaneously once a week. | Drug: Placebo<br>* SC Weekly Injection<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| IMACS-TIS Moderate Improvement at Week 24 | Proportion of subjects who achieve moderate improvement (≥40) from baseline in IMACS TIS at Week 24 | Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| IMACS-TIS Minimal Improvement at Week 4, 8, 12, 16, 24 | Proportion of subjects who achieve minimal improvement (≥20) from baseline in IMACS TIS at Week 4, 8, 12, 16, and 24 | Week 4, 8, 12, 16, 24 |