A1334-02 - Phase 1b Study to Evaluate ACE-1334 in Systemic Sclerosis
Research type
Research Study
Full title
Phase 1b Randomized, Double-Blind, PlaceboControlled, Multiple-Ascending Dose Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of ACE-1334 Plus Standard of Care in Participants with Systemic Sclerosis
IRAS ID
1004117
Contact name
Danielle Littee
Contact email
Sponsor organisation
Acceleron Pharma Inc.
Eudract number
2021-001004-15
Clinicaltrials.gov Identifier
Research summary
Systemic sclerosis (SSc) is a rare connective tissue disorder characterised by fibrosis, inflammation, and injury to the small blood vessels of the skin. Interstitial lung disease (ILD) is common and tends to occur early in the course of the disease. There remains a need for new, safe, and effective therapies for patients with SSc ILD.
The experimental drug ACE-1334 is being studied as a potential treatment for reducing lung function loss, skin thickness, and other symptoms in patients with SSc. ACE-1334 is given by an injection under the skin. ACE-1334 is a protein that potentially inhibits TGF-β1 and TGF-β3, which are believed to contribute to the underlying fibrosis observed in patients with SSc-ILD. Nonclinical studies suggest that ACE-1334 represents a promising treatment to disrupt the underlying fibrogenic processes responsible for the lung function decline in SSc-ILD.
There are 2 parts to this study:
• Part 1 is to learn more about the safety of taking multiple doses of ACE 1334 with current medications. Participants will be randomly assigned to receive ACE-1334 every 2 weeks or ACE-1334 every 4 weeks at different dose levels. Part 1 will enrol approximately 36 participants and will have a 4 week screening period, 12-week treatment period, potential further 40-week treatment period, and 16 week follow-up period.
• Part 2 is to assess the efficacy and safety of ACE-1334 plus standard of care (SOC) versus placebo plus SOC in participants with SSc ILD. Part 2 will enrol approximately 174 participants and will have a 28-day screening period, 52-week double-blind treatment period, and 16-week follow-up period. Dose levels and frequency of administration will be determined from the Phase 1b study.
Assessments will include physical examination, lung function tests, ECG, CT scans, blood, urine, stool and skin biopsy sampling, and health related quality of life questionnaires.
This study is sponsored by Acceleron Pharma Inc.REC name
London - Hampstead Research Ethics Committee
REC reference
21/LO/0881
Date of REC Opinion
7 Feb 2022
REC opinion
Further Information Favourable Opinion