Back to resultsAn Open-label Extension Trial of HZNP-HZN-825-301 in Adult Participants With Diffuse Cutaneous Systemic Sclerosis (Diffuse Cutaneous SSc)
Primary Purpose
Diffuse Cutaneous Systemic Sclerosis, Sclerosis, Systemic
Status
Enrolling by invitation
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
HZN-825
Sponsored by
About this trial
This is an interventional treatment trial for Diffuse Cutaneous Systemic Sclerosis focused on measuring Scleroderma, Forced vital capacity
Eligibility Criteria
Key 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: Anticipated use of another investigational agent for any condition during the course of the trial. 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). 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. 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. Pregnant or lactating women. 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.
Sites / Locations
- IRIS Research and Development LLC
- DelRicht Clinical Research, LLC
- Michigan Medicine University of Michigan
- Medical University of South Carolina - PPDS
- Metroplex Clinical Research Center
- UT Physicians Rheumatology
- Clínica Adventista Belgrano
- Framingham Centro Médico
- Aprillus Asistencia e Investigacion de Arcis Salud SRL
- Centro de Investigaciones Médicas Tucumán
- Centro de Investigaciones Reumatologicas
- Centre Hospitalier Universitaire de Bordeaux, Hopital Pellegrin
- Hopital Cochin
- Kianous Stavros
- Tel Aviv Sourasky Medical Center - PPDS
- Chonnam National University Hospital
- Hanyang University Medical Center
- Centro de Investigación y Tratamiento Reumatológico S.C
- Centro de Estudios de Investigacion Basica Y Clinica SC
- Centro Integral Reumatologia SA de CV
- Centro de Alta Especialidad En Reumatologia E Investigacion Del Potosi SC
- Clinica de Investigacion en Reumatologia y Obesidad
- Unidad de Atencion Medica e Investigacion en Salud
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HZN-825
Arm Description
HZN-825 will be administered by mouth (PO) twice daily (BID) for 52 weeks
Outcomes
Primary Outcome Measures
Change from trial baseline, defined as the latest measurement prior to the first dose of HZN-825 in FVC % predicted
As measured by a pulmonary function test called a spirometry.
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
As measured by a pulmonary function test called a spirometry.
Incidence of treatment emergent adverse events (TEAEs)
Incidence of adverse events of special interest (AESI) orthostatic hypotension
Incidence and frequency of use of concomitant medication
Change from trial baseline in vital signs as reported as TEAEs
Change from HZN-825 baseline in vital signs as reported as TEAEs
Change from trial baseline in abnormal and clinically significant 12-lead electrocardiogram (ECG) measurements.
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).
Change from HZN-825 trial baseline in abnormal and clinically significant 12-lead ECG measurements.
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).
Change from trial baseline in abnormal laboratory test results
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
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)
Secondary Outcome Measures
Full Information
NCT ID
NCT05626751
First Posted
November 1, 2022
Last Updated
September 13, 2023
Sponsor
Horizon Pharma Ireland, Ltd., Dublin Ireland
1. Study Identification
Unique Protocol Identification Number
NCT05626751
Brief Title
An Open-label Extension Trial of HZNP-HZN-825-301 in Adult Participants With Diffuse Cutaneous Systemic Sclerosis (Diffuse Cutaneous SSc)
Official Title
A Multicenter, Open-label Extension Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
November 4, 2022 (Actual)
Primary Completion Date
December 2025 (Anticipated)
Study Completion Date
December 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Horizon Pharma Ireland, Ltd., Dublin Ireland
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Primary Objectives:
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Diffuse Cutaneous Systemic Sclerosis, Sclerosis, Systemic
Keywords
Scleroderma, Forced vital capacity
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
246 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
HZN-825
Arm Type
Experimental
Arm Description
HZN-825 will be administered by mouth (PO) twice daily (BID) for 52 weeks
Intervention Type
Drug
Intervention Name(s)
HZN-825
Intervention Description
HZN-825 will be administered BID for 52 weeks
Primary Outcome Measure Information:
Title
Change from trial baseline, defined as the latest measurement prior to the first dose of HZN-825 in FVC % predicted
Description
As measured by a pulmonary function test called a spirometry.
Time Frame
Baseline to Week 52
Title
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
Description
As measured by a pulmonary function test called a spirometry.
Time Frame
Baseline to Week 52
Title
Incidence of treatment emergent adverse events (TEAEs)
Time Frame
Day 1 to Week 56
Title
Incidence of adverse events of special interest (AESI) orthostatic hypotension
Time Frame
Day 1 to Week 52
Title
Incidence and frequency of use of concomitant medication
Time Frame
Day 1 to Week 56
Title
Change from trial baseline in vital signs as reported as TEAEs
Time Frame
Day 1 to Week 56
Title
Change from HZN-825 baseline in vital signs as reported as TEAEs
Time Frame
Day 1 to Week 56
Title
Change from trial baseline in abnormal and clinically significant 12-lead electrocardiogram (ECG) measurements.
Description
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).
Time Frame
Baseline to Week 52
Title
Change from HZN-825 trial baseline in abnormal and clinically significant 12-lead ECG measurements.
Description
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).
Time Frame
Baseline to Week 52
Title
Change from trial baseline in abnormal laboratory test results
Description
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)
Time Frame
Day 1 to Week 56
Title
Change from HZN-825 baseline in abnormal laboratory test results
Description
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)
Time Frame
Day 1 to Week 56
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key 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:
Anticipated use of another investigational agent for any condition during the course of the trial.
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).
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.
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.
Pregnant or lactating women.
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Horizon Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
IRIS Research and Development LLC
City
Plantation
State/Province
Florida
ZIP/Postal Code
33324
Country
United States
Facility Name
DelRicht Clinical Research, LLC
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70115
Country
United States
Facility Name
Michigan Medicine University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-5000
Country
United States
Facility Name
Medical University of South Carolina - PPDS
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425-8900
Country
United States
Facility Name
Metroplex Clinical Research Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231-4345
Country
United States
Facility Name
UT Physicians Rheumatology
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Clínica Adventista Belgrano
City
Cuiudad Autónoma De
State/Province
Buenos Aires
ZIP/Postal Code
C1430EGF
Country
Argentina
Facility Name
Framingham Centro Médico
City
La Plata
State/Province
Buenos Aires
ZIP/Postal Code
B1900
Country
Argentina
Facility Name
Aprillus Asistencia e Investigacion de Arcis Salud SRL
City
Ciudad Autónoma de Buenos Aires
State/Province
Ciudad Autónoma De BuenosAires
ZIP/Postal Code
C1406AGA
Country
Argentina
Facility Name
Centro de Investigaciones Médicas Tucumán
City
San Miguel De Tucumán
State/Province
Tucumán
ZIP/Postal Code
T4000AXL
Country
Argentina
Facility Name
Centro de Investigaciones Reumatologicas
City
San Miguel De Tucumán
State/Province
Tucumán
ZIP/Postal Code
T4000AXL
Country
Argentina
Facility Name
Centre Hospitalier Universitaire de Bordeaux, Hopital Pellegrin
City
Bordeaux
State/Province
Gironde
ZIP/Postal Code
33000
Country
France
Facility Name
Hopital Cochin
City
Paris
ZIP/Postal Code
75014
Country
France
Facility Name
Kianous Stavros
City
Thessaloniki
ZIP/Postal Code
546 36
Country
Greece
Facility Name
Tel Aviv Sourasky Medical Center - PPDS
City
Tel Aviv-Yafo
State/Province
Tel-Aviv
ZIP/Postal Code
64239
Country
Israel
Facility Name
Chonnam National University Hospital
City
Gwangju
ZIP/Postal Code
61469
Country
Korea, Republic of
Facility Name
Hanyang University Medical Center
City
Seoul
ZIP/Postal Code
4763
Country
Korea, Republic of
Facility Name
Centro de Investigación y Tratamiento Reumatológico S.C
City
San Miguel Chapultepec
State/Province
Distrito Federal
ZIP/Postal Code
11850
Country
Mexico
Facility Name
Centro de Estudios de Investigacion Basica Y Clinica SC
City
Guadalajara
State/Province
Jalisco
ZIP/Postal Code
44690
Country
Mexico
Facility Name
Centro Integral Reumatologia SA de CV
City
Guadalajara
State/Province
Jalisco
ZIP/Postal Code
ZC 44160
Country
Mexico
Facility Name
Centro de Alta Especialidad En Reumatologia E Investigacion Del Potosi SC
City
Burocratas Del Estado
State/Province
San Luis Potosí
ZIP/Postal Code
78213
Country
Mexico
Facility Name
Clinica de Investigacion en Reumatologia y Obesidad
City
Guadalajara
ZIP/Postal Code
44600
Country
Mexico
Facility Name
Unidad de Atencion Medica e Investigacion en Salud
City
Merida
ZIP/Postal Code
97000
Country
Mexico
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
An Open-label Extension Trial of HZNP-HZN-825-301 in Adult Participants With Diffuse Cutaneous Systemic Sclerosis (Diffuse Cutaneous SSc)
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