A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7303509 in Participants With Systemic Sclerosis

A Phase Ib, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Multiple-Ascending Doses of RO7303509 in Participants With Systemic Sclerosis

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of RO7303509 treatment in participants with systemic sclerosis (SSc) during a multiple-ascending-dose (MAD) portion of the trial. In the MAD phase, increasing doses of study drug will be tested sequentially. For each dose tested, the MAD stage will consist of a treatment period of 12 weeks followed by either a safety follow-up period of 13 weeks or continued treatment in an optional open-label safety extension (OSE) stage of 52 weeks to assess the long-term safety. All patients in the OSE stage will receive RO7303509 and no patient will receive placebo.

Study Overview

• Status: Completed
• Conditions: Systemic Sclerosis
• Intervention / Treatment: Drug: RO7303509; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 56
• Phase: Phase 1

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina, B1888AAE
    • Hospital de Alta Complejidad en Red ?El Cruce? Dr. Néstor Kirchner ? S.A.M.I.C.
  • San Miguel de Tucumán, Argentina, T4000IHE
    • Clínica Mayo de U.M.C.B. S.R.L
• Belgium
  • Leuven, Belgium, 3000
    • UZ Leuven
• France
  • La Tronche, France, 38700
    • Centre Hospitalier Universitaire de Grenoble - Albert Michallon
  • Paris, France, 75014
    • Hôpital Cochin
  • Pessac, France, 33600
    • CHU de Bordeaux
  • Rennes, France, 35000
    • CHRU Rennes
  • Strasbourg, France, 67200
    • Hôpitaux Universitaires
  • Toulouse, France, 31000
    • Hopital Purpan
• Israel
  • Haifa, Israel, 3109601
    • Rambam Medical Center - PPDS
  • Kfar Saba, Israel, 4428164
    • Meir Medical Center
• Poland
  • Bydgoszcz, Poland, 85-168
    • Szpital Uniwersytecki nr 2 im. dr Jana Biziela w Bydgoszczy
  • Lublin, Poland, 20-607
    • Zespol Poradni Specjalistycznych REUMED
• Portugal
  • Almada, Portugal, 2801-915
    • Hospital Garcia de Orta
  • Coimbra, Portugal, 3000-075
    • Centro Hospitalar E Universitario de Coimbra EPE
• Puerto Rico
  • San Juan, Puerto Rico, 00935-0001
    • The Alliance Medical Sciences Campus
• Serbia
  • Belgrade, Serbia, 11040
    • Military Medical Academy
  • Belgrade, Serbia, 11000
    • Institute of Rheumatology Belgrade - PPDS
• Spain
  • Barcelona, Spain, 08025
    • Hospital De La Santa Creu I Sant Pau
  • Barcelona, Spain, 08035
    • Hospital Universitario Vall d Hebron
  • Sevilla, Spain, 41010
    • Hospital Quironsalud Infanta Luisa
• United Kingdom
  • London, United Kingdom, NW3 2QG
    • Royal Free Hospital
• United States
  • California
    • Palo Alto, California, United States, 94304
      • Stanford University
  • New York
    • New York, New York, United States, 10021-4823
      • Hospital For Special Surgery
  • Texas
    • Dallas, Texas, United States, 75235-6262
      • Metroplex Clinical Research Centre

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Inclusion Criteria for the MAD Stage:

• Weight of 45-150 kg at screening
• Diagnosis of SSc, as defined by 2013 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) criteria and ≤ 10 years disease duration from first non-Raynaud's symptom
• Agreement to remain abstinent or use an effective contraceptive method among males and females with childbearing potential for 4 months after last dose of study drug

Inclusion Criteria for the OSE Stage:

• No clinically significant change in eligibility status
• Completion of the MAD and ability to roll over into the OSE within 5 days

Exclusion Criteria:

• Active rheumatic autoimmune disease other than SSc requiring treatment with disease-modifying therapy
• Pulmonary disease with forced vital capacity (FVC) ≤ 50% of predicted
• History or clinical manifestations of significant metabolic, hepatic, renal, pulmonary, cardiovascular, hematologic, gastrointestinal, urologic, neurologic, or psychiatric disorders
• History of severe allergic or anaphylactic reactions to human, humanized, or murine monoclonal antibodies
• Pregnant or breastfeeding, or intending to become pregnant during the study or within 4 months after the final dose of study drug
• Major surgery within 8 weeks prior to screening, or major planned surgery during the study or within 3 months after the final dose
• Positive hepatitis C virus (HCV) antibody, hepatitis B surface antigen (HBsAg), or human immunodeficiency virus (HIV) antibody test at screening
• Any serious medical condition or abnormality in clinical laboratory tests

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MAD Stage; Participants will be randomized in a ratio of 4:1 to receive RO7303509 or placebo, as subcutaneous (SC) injection, one time per month for 3 months. You have a 20% chance of getting placebo.	Drug: RO7303509; RO7303509 will be administered as SC injection monthly, as specified in each treatment group.; Drug: Placebo; RO7303509 matching placebo will be administered as SC injection monthly, during the MAD stage.
Experimental: OSE Stage; Every participant in the OSE stage will receive study drug and no participant will receive placebo. Participants will receive RO7303509 as SC injection at the same dose as that administered during the MAD stage, one time per month for up to a year.	Drug: RO7303509; RO7303509 will be administered as SC injection monthly, as specified in each treatment group.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	Up to approximately 17 months
Number of Participants With Clinically Significant Change From Baseline in Vital Signs	Up to approximately 17 months
Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Test Results	Up to approximately 17 months
Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Parameters	Up to approximately 17 months

Secondary Outcome Measures

Outcome Measure	Time Frame
MAD Stage: Maximum Serum Concentration (Cmax) of RO7303509	Predose on Day 1 and at multiple timepoints up to Day 113 or early termination (ET) visit
MAD Stage: Area Under the Concentration vs Time Curve (AUC) of RO7303509	Predose on Day 1 and at multiple timepoints up to Day 113 or ET visit
MAD Stage: Time to Maximum Concentration (Tmax) of RO7303509	Predose on Day 1 and at multiple timepoints up to Day 113 or ET visit
MAD Stage: Total Clearance (CL) of RO7303509	Predose on Day 1 and at multiple timepoints up to Day 113 or ET visit
MAD Stage: Volume of Distribution (V) of RO7303509	Predose on Day 1 and at multiple timepoints up to Day 113 or ET visit
MAD Stage: Half-Life (t1/2) of RO7303509	Predose on Day 1 and at multiple timepoints up to Day 113 or ET visit
MAD and OSE Stage: Percentage of Participants With Anti-Drug Antibodies (ADAs) Against RO7303509	MAD Stage: Predose on Day 1 and at multiple timepoints up to Day 113 (Week 16) or ET visit; OSE Stage: Predose and multiple timepoints up to Week 52; (up to approximately 1.3 years)

Collaborators and Investigators

• Sponsor: Genentech, Inc.
• Investigators: Study Director: Clinical Trials, Hoffmann-La Roche

Study record dates

Study Major Dates

• Study Start (Actual): January 16, 2023
• Primary Completion (Actual): July 11, 2025
• Study Completion (Actual): July 11, 2025

Study Registration Dates

• First Submitted: July 14, 2022
• First Submitted That Met QC Criteria: July 14, 2022
• First Posted (Actual): July 18, 2022

Study Record Updates

• Last Update Posted (Actual): July 20, 2025
• Last Update Submitted That Met QC Criteria: July 16, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Connective Tissue Diseases; Skin Diseases; Sclerosis; Scleroderma, Systemic; Scleroderma, Diffuse
• Other Study ID Numbers: GA43360; 2021-004578-68 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No