An Open-label Extension Trial of HZNP-HZN-825-301 in Adult Participants With Diffuse Cutaneous Systemic Sclerosis (Diffuse Cutaneous SSc)

Phase 2

Terminated

• Conditions: Diffuse Cutaneous Systemic Sclerosis; Sclerosis, Systemic
• Interventions: Drug: HZN-825

• Registration Number: NCT05626751
• Lead Sponsor: Amgen

Brief Summary

Primary Objectives:

1. The primary efficacy objective is to assess the efficacy of 52 weeks of open-label treatment with HZN-825 in participants with diffuse cutaneous systemic sclerosis, as measured by change from both baselines in forced vital capacity percent (FVC %) predicted.

2. The primary safety objective is to examine the safety and tolerability of 52 weeks of open-label treatment with HZN-825, inclusive of, but not limited to, adverse events (AEs), serious AEs (SAEs) and the adverse event of special interest (AESI), from Day 1 to 4 weeks after last dose.

Detailed Description

This is an open-label, repeat-dose, multicenter extension trial of HZNP-HZN-825-301. Participants who complete the double-blind Treatment Period (Week 52) in Trial HZNP-HZN-825-301 will be eligible to enter this 52-week extension trial. Participants entering this extension trial will complete the Week 52 Visit activities in HZNP-HZN-825-301 and will not complete the Safety Follow-up Visit 4 weeks after the last dose of trial drug in HZNP-HZN-825-301.

Acquired from Horizon in 2024.

Study Timeline

• Study First Submitted: 2022-11-01
• Study Start: 2022-11-04
• Study First Submitted QC: 2022-11-15
• Study First Posted: 2022-11-25
• Primary Completion: 2025-02-24
• Study Completion: 2025-02-24
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-10
• Last Update Posted: 2025-03-12

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 174

Inclusion Criteria

1. Completed the double-blind Treatment Period (Week 52) in Trial HZNP-HZN-825-301; participants prematurely discontinued from trial drug in Trial HZNP-HZN-825-301 for reasons other than safety or toxicity can be included at the discretion of the Investigator after completing Trial HZNP-HZN-825-301 scheduled visits, including Week 52 assessments.

Key

Exclusion Criteria

1. Anticipated use of another investigational agent for any condition during the course of the trial.
2. New diagnosis of malignant condition after enrolling in Trial HZNP-HZN-825-301 (except successfully treated basal/squamous cell carcinoma of the skin or cervical cancer in situ).
3. Women of childbearing potential (WOCBP) or male participants not agreeing to use highly effective method(s) of birth control throughout the trial and for 4 weeks after last dose of trial drug as defined in the protocol.
4. Any new development with the participant's disease or condition or any significant laboratory test abnormality during the course of Trial HZNP-HZN-825-301 that, in the opinion of the Investigator, would potentially put the subject at unacceptable risk.
5. Pregnant or lactating women.
6. Participants will be ineligible if, in the opinion of the Investigator, they are unlikely to comply with the trial protocol or have a concomitant disease or condition that could interfere with the conduct of the trial.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HZN-825	HZN-825	HZN-825 will be administered by mouth (PO) twice daily (BID) for 52 weeks

Primary Outcome Measures

Name	Time	Method
Change from HZN-825 Baseline, defined as the latest measurement prior to the first dose of HZN-825 in either trial HZNP-HZN-825-301 or this extension trial in FVC % predicted	Baseline to Week 52	As measured by a pulmonary function test called a spirometry.
Incidence of adverse events of special interest (AESI) orthostatic hypotension	Day 1 to Week 52
Incidence and frequency of use of concomitant medication	Day 1 to Week 56
Change from trial baseline in vital signs as reported as TEAEs	Day 1 to Week 56
Change from trial baseline in abnormal and clinically significant 12-lead electrocardiogram (ECG) measurements.	Baseline to Week 52	Clinically significant changes in ECGs is defined as any clinical significant difference in heart rate, RR interval, PR interval, QRS, and QT interval corrected using Fridericia's formula (QTcF).
Incidence of treatment emergent adverse events (TEAEs)	Day 1 to Week 56
Change from trial baseline, defined as the latest measurement prior to the first dose of HZN-825 in FVC % predicted	Baseline to Week 52	As measured by a pulmonary function test called a spirometry.
Change from trial baseline in abnormal laboratory test results	Day 1 to Week 56	Clinically significant lab values in serum chemistry, hematology, lipids, coagulation tests and urinalyses will be assessed (including Grade 3 or higher per common terminology criteria for adverse events)
Change from HZN-825 baseline in abnormal laboratory test results	Day 1 to Week 56	Clinically significant lab values in serum chemistry, hematology, lipids, coagulation tests and urinalyses will be assessed (including Grade 3 or higher per common terminology criteria for adverse events)
Change from HZN-825 baseline in vital signs as reported as TEAEs	Day 1 to Week 56
Change from HZN-825 trial baseline in abnormal and clinically significant 12-lead ECG measurements.	Baseline to Week 52	Clinically significant changes in ECGs is defined as any clinical significant difference in heart rate, RR interval, PR interval, QRS, and QT interval corrected using Fridericia's formula (QTcF).

Trial Locations

Locations (65)

Arizona Arthritis and Rheumatology Associates -4550 E Bell Rd; 🇺🇸 Phoenix, Arizona, United States

UCLA Medical Center; 🇺🇸 Los Angeles, California, United States

University of Miami Miller School of Medicine; 🇺🇸 Miami, Florida, United States

IRIS Research and Development LLC; 🇺🇸 Plantation, Florida, United States

DelRicht Clinical Research, LLC - Internal - Covington - PPDS; 🇺🇸 New Orleans, Louisiana, United States

Boston University School Of Medicine; 🇺🇸 Boston, Massachusetts, United States

Michigan Medicine University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States

Mayo Clinic - Cancer Center - Rochester - PPDS; 🇺🇸 Rochester, Minnesota, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Medical University of South Carolina (MUSC) - PPDS; 🇺🇸 Charleston, South Carolina, United States

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