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

Amgen

Status and phase: Active, not recruiting; Phase 2

Conditions: Diffuse Cutaneous Systemic Sclerosis; Sclerosis, Systemic

Treatments: Drug: Placebo; Drug: HZN-825 QD; Drug: HZN-825 BID

Study type: Interventional

Funder types: Industry

Identifiers

NCT04781543

HZNP-HZN-825-301

2020-005764-62 (EudraCT Number)

Details and patient eligibility

About

This is a randomized, double-blind, placebo-controlled, repeat-dose, multicenter trial. Participants will be screened within 6 weeks prior to the Baseline (Day 1) Visit. Approximately 300 participants who meet the trial eligibility criteria will be randomized on Day 1 in a 1:1:1 ratio to receive HZN-825 300 mg QD, HZN-825 300 mg BID or placebo for 52 weeks.

The trial will include up to a 42-day Screening Period and a 52-week Double-blind Treatment Period. Participants will take their first dose of trial drug at the clinic and will participate in trial visits at Week 4 and every 6 weeks thereafter until Week 52.

All participants who complete the Double-blind Treatment Period (Week 52) will be eligible to enter a 52-week extension trial (HZNP-HZN-825-302, NCT05626751). Participants not entering the extension trial will participate in a Safety Follow-up Visit 4 weeks after the last dose of trial drug.

Full description

Acquired from Horizon in 2024.

Enrollment: 301 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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 knee (diffuse cutaneous SSc subset by LeRoy and Medsger, 2001).
5. At the time of enrollment, less than or equal to 72 months (6 years) since the onset of the first SSc manifestation, other than Raynaud's phenomenon.
6. Skin thickening from SSc in the forearm suitable for repeat biopsy.
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.

Exclusion criteria

1. Positive for anti-centromere antibodies with the exception that subjects who are positive for both anti-centromere and anti-topoi

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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301 participants in 3 patient groups, including a placebo group

HZN-825 300 mg once daily (QD)

Experimental group

Description:

One set of 2 HZN-825 150mg tablets in the morning and one set of 2 placebo tablets in the evening.

Treatment:

Drug: HZN-825 QD

HZN-825 300 mg twice daily (BID)

Experimental group

Description:

One set of 2 HZN-825 150mg tablets in the morning and one set of 2 HZN-825 150mg tablets in the evening.

Treatment:

Drug: HZN-825 BID

Placebo

Placebo Comparator group

Description:

One set of 2 placebo tablets in the morning and one set of 2 placebo tablets in the evening.

Treatment:

Drug: Placebo