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

NCT05462522 | Completed Phase 1 DMC FDA Drug

• 2 other identifiers: GA433602021-004578-68
• Study Type: interventional
• Target: 56
• Locations: 11 countries
• Sites: 25

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.

Conditions

Systemic Sclerosis

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (7)

• 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

Study Arms (2)

MAD Stage

EXPERIMENTAL

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; Drug: Placebo

OSE Stage

EXPERIMENTAL

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

Interventions

RO7303509 DRUG

RO7303509 will be administered as SC injection monthly, as specified in each treatment group.

MAD Stage; OSE Stage

Placebo DRUG

RO7303509 matching placebo will be administered as SC injection monthly, during the MAD stage.

MAD Stage

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Genentech, Inc.: lead

Study Sites (25)

Stanford University

Palo Alto, California, 94304, United States

Location

Hospital For Special Surgery

New York, New York, 10021-4823, United States

Location

Metroplex Clinical Research Centre

Dallas, Texas, 75235-6262, United States

Location

Hospital de Alta Complejidad en Red ?El Cruce? Dr. Néstor Kirchner ? S.A.M.I.C.

Buenos Aires, B1888AAE, Argentina

Location

Clinica Mayo de U.M.C.B. S.R.L

San Miguel de Tucumán, T4000IHE, Argentina

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Centre Hospitalier Universitaire de Grenoble - Albert Michallon

La Tronche, 38700, France

Location

Hopital Cochin

Paris, 75014, France

Location

CHU de Bordeaux

Pessac, 33600, France

Location

CHRU Rennes

Rennes, 35000, France

Location

Hopitaux Universitaires

Strasbourg, 67200, France

Location

Hopital Purpan

Toulouse, 31000, France

Location

Rambam Medical Center - PPDS

Haifa, 3109601, Israel

Location

Meir Medical Center

Kfar Saba, 4428164, Israel

Location

Szpital Uniwersytecki Nr 2 im. Dr Jana Biziela w Bydgoszczy

Bydgoszcz, 85-168, Poland

Location

Zespol Poradni Specjalistycznych REUMED

Lublin, 20-607, Poland

Location

Hospital Garcia de Orta

Almada, 2801-915, Portugal

Location

Centro Hospitalar E Universitário de Coimbra EPE

Coimbra, 3000-075, Portugal

Location

The Alliance Medical Sciences Campus

San Juan, 00935-0001, Puerto Rico

Location

Institute of Rheumatology Belgrade - PPDS

Belgrade, 11000, Serbia

Location

Military Medical Academy

Belgrade, 11040, Serbia

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, 08025, Spain

Location

Hospital Universitario Vall d Hebron

Barcelona, 08035, Spain

Location

Hospital Quironsalud Infanta Luisa

Seville, 41010, Spain

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

MeSH Terms

Conditions

Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 14, 2022
• First Posted: July 18, 2022
• Study Start: January 16, 2023
• Primary Completion: July 11, 2025
• Study Completion: July 11, 2025
• Last Updated: July 20, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

IL (2); PL (2); PT (2); SE (2); BE (1); US (3); FR (6); GB (1); ES (3); AR (2); PR (1)