RO7303509 for Systemic Sclerosis

No longer recruiting at 47 trial locations

Overseen By Reference Study ID Number: GA43360 https://forpatients.roche.com/

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Genentech, Inc.

Must not be taking: Disease-modifying therapy

Disqualifiers: Pulmonary disease, Metabolic disorders, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effects of a new treatment called RO7303509, an experimental therapy for systemic sclerosis, a condition that causes hardening and tightening of the skin and connective tissues. Participants receive the treatment through a series of increasing doses to assess tolerance. Individuals diagnosed with systemic sclerosis who first noticed symptoms other than Raynaud's within the last 10 years might find this trial suitable. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you have an active rheumatic autoimmune disease other than systemic sclerosis that requires treatment with disease-modifying therapy, you may be excluded from participating.

Is there any evidence suggesting that RO7303509 is likely to be safe for humans?

Research has shown that RO7303509 has been well tolerated in humans so far. In earlier studies with healthy volunteers, researchers administered single doses up to 1200 mg. The treatment showed good results, meaning it didn't cause serious problems and was absorbed by the body as expected. These findings are promising for its safety in people with systemic sclerosis. The current study takes extra precautions by starting with lower doses and gradually increasing them to ensure safety for participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for systemic sclerosis?

Researchers are excited about RO7303509 for systemic sclerosis because it offers a novel approach by targeting specific pathways involved in the disease. Unlike current treatments that primarily focus on managing symptoms and slowing disease progression, RO7303509 is designed to directly modulate the underlying disease mechanisms. It is administered as a subcutaneous injection, which might offer greater convenience and potentially improved compliance compared to more invasive treatments. By addressing the root causes rather than just the symptoms, RO7303509 has the potential to provide significant benefits for patients with systemic sclerosis.

What evidence suggests that RO7303509 might be an effective treatment for systemic sclerosis?

Research has shown that RO7303509 could be a promising treatment for systemic sclerosis, a condition that causes thickening and scarring of connective tissue. This treatment targets a protein called TGFβ3, which plays a role in the scarring process. Studies have found that RO7303509 is safe for healthy individuals and is processed well by the body. Early results suggest it might help reduce scarring, making it a potential therapy for systemic sclerosis. However, more research is needed to confirm its effectiveness for this condition. Participants in this trial will receive RO7303509, with some in the MAD stage randomized to receive either the study drug or a placebo. ^{1 2 4 6 7}

Who Is on the Research Team?

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Are You a Good Fit for This Trial?

This trial is for adults weighing 45-150 kg with systemic sclerosis diagnosed within the last 10 years. They must not have severe lung, liver, kidney, heart or other major health issues and agree to use effective contraception. Excluded are those with significant pulmonary disease, positive tests for certain viruses like HIV or hepatitis B/C, recent or upcoming major surgery, pregnancy/breastfeeding women, and those with other active autoimmune diseases.

Inclusion Criteria

I completed the initial study phase and can start the next phase within 5 days.

No clinically significant change in eligibility status

My weight is between 45 and 150 kg.

Exclusion Criteria

I am being treated for an autoimmune disease other than systemic sclerosis.

I do not have any serious medical conditions or abnormal lab test results.

My lung function is less than half of what it should be.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive RO7303509 or placebo as subcutaneous injection once a month for 12 weeks in the MAD phase

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

13 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment with RO7303509 for long-term safety assessment

52 weeks

What Are the Treatments Tested in This Trial?

Interventions

• RO7303509

Trial Overview The study is testing RO7303509's safety and how the body processes it in people with systemic sclerosis over a period of up to 65 weeks. Participants will receive increasing doses of RO7303509 or placebo during a first phase (12 weeks), followed by an optional long-term safety extension where everyone gets RO7303509.

How Is the Trial Designed?

Group I: OSE Stage Experimental Treatment 1 Intervention

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.

Group II: MAD Stage Experimental Treatment 2 Interventions

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.

Who Is Running the Clinical Trial?

Genentech, Inc.

Lead Sponsor

Trials

1,578

Recruited

569,000+

Ashley Magargee

Genentech, Inc.

Chief Executive Officer since 2024

MBA from Harvard University, BA from Princeton University

Levi Garraway

Genentech, Inc.

Chief Medical Officer since 2021

MD, PhD

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11111615/

A Phase 1a Study to Evaluate Safety, Tolerability ... RO7303509 was well tolerated at single SC doses up to 1200 mg in HVs with favorable pharmacokinetic data that appeared to increase dose-proportionally.

2. genentech-clinicaltrials.com genentech-clinicaltrials.com/en/trials/autoimmune-disorder/systemic-sclerosis/a-study-to-evaluate-the-safety--tolerability--and-pharm-79881.html

Clinical trial for Systemic Sclerosis-Genentech A Member ... The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of RO7303509 treatment in participants with systemic sclerosis ...

3. ard.bmj.com ard.bmj.com/content/82/Suppl_1/976.2

POS1266 A PHASE IA, RANDOMIZED STUDY TO ... RO7303509, a high-affinity, TGFβ3-specific, humanized immunoglobulin G1 (IgG1) monoclonal antibody, is being developed as a potential anti-fibrotic therapy.

4. withpower.com withpower.com/trial/phase-1-scleroderma-systemic-6-2022-05312

RO7303509 for Systemic Sclerosis · Info for Participants Trial Overview The study is testing RO7303509's safety and how the body processes it in people with systemic sclerosis over a period of up to 65 weeks.

5. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38662148/

A Phase 1a Study to Evaluate Safety, Tolerability ... RO7303509 was well tolerated at single SC doses up to 1200 mg in HVs with favorable pharmacokinetic data that appeared to increase dose-proportionally.

6. forpatients.roche.com forpatients.roche.com/en/trials/healthy-volunteers/a-study-to-look-at-how-safe-different-doses-of-a-new-study-medic.html

Clinical trial - systemic sclerosis – RO7303509 - ForPatients Safety study of RO7303509, a new drug for systemic sclerosis, tested in healthy volunteers who received a single treatment in six different treatment dose ...

7. mayo.edu mayo.edu/research/clinical-trials/diseases-conditions/scleroderma

Scleroderma Clinical Trials The long-term safety and tolerability of up to 52 weeks of RO7303509 treatment will also be evaluated in an optional open-label safety extension (OSE).