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

Phase 1

Active, not recruiting

• Conditions: Systemic Sclerosis
• Interventions: Drug: RO7303509; Drug: Placebo

• Registration Number: NCT05462522
• Lead Sponsor: Genentech, Inc.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-07-14
• Study First Submitted QC: 2022-07-14
• Study First Posted: 2022-07-18
• Study Start: 2023-01-16
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-18
• Last Update Posted: 2025-04-20
• Primary Completion: 2025-07-08
• Study Completion: 2025-07-08

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 100

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
MAD Stage	Placebo	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.
MAD Stage	RO7303509	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.
OSE Stage	RO7303509	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.

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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: 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)
MAD Stage: Volume of Distribution (V) of RO7303509	Predose on Day 1 and at multiple timepoints up to Day 113 or ET visit

Trial Locations

Locations (24)

Centre Hospitalier Universitaire de Grenoble - Albert Michallon; 🇫🇷 La Tronche, France

Hopital Cochin; 🇫🇷 Paris, France

CHU de Bordeaux; 🇫🇷 Pessac, France

CHRU Rennes; 🇫🇷 Rennes, France

Hopitaux Universitaires; 🇫🇷 Strasbourg, France

Hopital Purpan; 🇫🇷 Toulouse, France

Rambam Medical Center - PPDS; 🇮🇱 Haifa, Israel

Meir Medical Center; 🇮🇱 Kfar Saba, Israel

Zespol Poradni Specjalistycznych REUMED; 🇵🇱 Lublin, Poland

Hospital Garcia de Orta; 🇵🇹 Almada, Portugal

Centro Hospitalar E Universitário de Coimbra EPE; 🇵🇹 Coimbra, Portugal

The Alliance Medical Sciences Campus; 🇵🇷 San Juan, Puerto Rico

Institute of Rheumatology Belgrade - PPDS; 🇷🇸 Belgrade, Serbia

Military Medical Academy; 🇷🇸 Belgrade, Serbia

Hospital de la Santa Creu i Sant Pau; 🇪🇸 Barcelona, Spain

Hospital Universitario Vall d Hebron; 🇪🇸 Barcelona, Spain

Hospital Quironsalud Infanta Luisa; 🇪🇸 Sevilla, Spain

Royal Free Hospital; 🇬🇧 London, United Kingdom

Stanford University; 🇺🇸 Palo Alto, California, United States

Hospital For Special Surgery; 🇺🇸 New York, New York, United States

Metroplex Clinical Research Centre; 🇺🇸 Dallas, Texas, United States

Hospital de Alta Complejidad en Red ?El Cruce? Dr. Néstor Kirchner ? S.A.M.I.C.; 🇦🇷 Buenos Aires, Argentina

Clinica Mayo de U.M.C.B. S.R.L; 🇦🇷 San Miguel de Tucumán, Argentina

UZ Leuven; 🇧🇪 Leuven, Belgium