A Study to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 300

Inclusion Criteria

1.Written informed consent.
2.Male or female between the ages of 18 and 75 years, inclusive, at
Screening.
3.Meets the 2013 American College of Rheumatology/European League
Against Rheumatism classification criteria for SSc with a total score of =9
(Van den Hoogen et al., 2013).
4.Classified as having skin involvement proximal to the elbow and/or knee
(diffuse cutaneous SSc subset by LeRoy and Medsger, 2001).
5.At the time of enrollment, less than 36 months since the onset of the
first SSc manifestation, other than Raynaud's phenomenon.
6.Skin in the forearm suitable for repeat biopsy (only applicable to the first 110 subjects for whom biopsy will be performed).
7.mRSS units =15 at Screening.
8.FVC =45% predicted at Screening, as determined by spirometry.
9.Willing and able to comply with the prescribed treatment protocol
and evaluations for the duration of the trial.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 285
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15

Exclusion Criteria

1.Positive for anti-centromere antibodies.
2.Diagnosed w/sine scleroderma or limited cutaneous SSc.
3.Diagnosed w/other autoimmune connective tissue diseases ,except for
fibromyalgia, scleroderma-associated myopathy & secondary
Sjogren's syndrome.
4.Scleroderma renal crisis diagnosed within6months of the Screening
Visit.
5.Any of the following cardiovascular diseases: a. uncontrolled, severe
hypertension(=160/100mmHg)or persistent low blood pressure
(systolic blood pressure<90 mmHg)within6months of Screening, b.
myocardial infarction within6months of Screening, c. unstable cardiac
angina within6months of Screening.
6.DLCO<40%predicted(corrected for hemoglobin).If severe acute
respiratory syndrome coronavirus2 (SARS-CoV-2) exposure is of clinical
concern for any subject, consider using a DLCO up to6months before the
Screening Visit.
7.Pulmonary arterial hypertension(PAH)by right heart catheterization
requiring treatment w/more than1oral PAH-approved therapy or any
parenteral therapy. Treatment is allowed for erectile dysfunction and/or
Raynaud's phenomenon/digital ulcers.
8.Corticosteroid use for conditions other than SSc within4weeks prior to
Screening(topical steroids for dermatological conditions&
inhaled/intranasal/intra-articular steroids are allowed).
9.Use of any other non-steroid immunosuppressive agent, small biologic
molecule, cytotoxic or antifibrotic drug within4weeks prior to Screening,
including cyclophosphamide, azathioprine(Imuran®)or other
immunosuppressive or cytotoxic medication. Exceptions include
mycophenolate mofetil (CellCept®),mycophenolic acid
(Myfortic®),methotrexate and low-dose prednisone, as follows: use of
CellCept =3g/day, Myfortic =2.14g/day, methotrexate =15 mg/week
and prednisone =10 mg/day (or equivalent dosing of glucocorticoids) is
allowed. See Table 9.1 for full details. Subjects taking CellCept, Myfortic
or methotrexate must have been doing so for=6months and the dose
must have been stable for =16 weeks prior to the Day 1 Visit.
Prednisone must have been at a stable dose for =8 weeks prior to the
Day1Visit. It is acceptable to be on background low-dose prednisone
&anti-malarial drug along with CellCept, Myfortic or methotrexate.
Rituximab must not have been used within 6 months of the
Day1Visit.Subjects must not be withdrawn from any standard-of-care
treatment that is considered necessary for the clinical management of
the subject in order to fulfill the trial eligibility requirements.
10.Known active bacterial, viral, fungal, mycobacterial or other infection,
including tuberculosis or atypical mycobacterial disease(fungal
infections of nail beds are allowed).
11.Use of a United States Food and Drug Administration-approved agent
for SSc or an investigational agent for any condition within 90days or
5half-lives,whichever is longer, prior to Screening or anticipated use
during the course of the trial.
12.Malignant condition in the past5years(except successfully treated
basal/squamous cell carcinoma of the skin or cervical cancer in situ).
13.Women of childbearing potential (WOCBP) or male subjects not
agreeing to use highly effective method(s)of birth control throughout
the trial and for 4 weeks after last dose of trial drug. Male subjects must
refrain from sperm donation and females from egg/ova donation for this
same time period. Women are considered of childbearing potential if
they are not postmenopausal and not surgically sterile(documented
bilateral salpingectomy, bilateral oophorectomy,

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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