A Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravitreal Injections of RO7303359 in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration

Recruiting at 16 trial locations

Overseen By Reference Study ID Number: GR42163 https://forpatients.roche.com/

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Hoffmann-La Roche

Disqualifiers: Vitrectomy, Laser photocoagulation, Photodynamic therapy, others

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

Compensation: Varies

Number of Visits: Unknown

37 Participants Needed

Trial Summary

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It might be best to discuss this with the trial coordinators or your doctor.

What safety data exists for the treatment RO7303359?

There is no specific safety data available for RO7303359, but similar treatments targeting the same receptor have been tested. For example, IMU-935, a similar treatment, was generally safe in a study with no serious adverse events, although some mild to moderate side effects were reported. ^{1 2 3 4 5}

What makes the drug RO7303359 unique compared to other treatments?

RO7303359 is unique because it involves the use of oligonucleotide N3'-->P5' phosphoramidates, which are being explored as potential antisense drugs. These 'second generation' chimeric oligonucleotides have shown improved characteristics over traditional oligonucleotides, potentially offering more effective treatment options. ^{6 7 8 9 10}

What is the purpose of this trial?

This trial is testing a new drug called RO7303359, which is injected directly into the eye. It aims to help people with vision loss due to age-related macular degeneration. The study will check if the drug is safe and how it works in the body.

Research Team

Clinical Trials

Principal Investigator

Hoffmann-La Roche

Eligibility Criteria

Inclusion Criteria

You have GA secondary to AMD in the absence of CNV.

Ocular

You have a disc area of at least 1.25 mm2.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose Stage

Participants receive a single dose of RO7303359 in multiple escalating cohorts

4 weeks

Expansion Cohort Stage

Participants receive the maximum tolerated dose (MTD) or maximum tested dose (MTeD) of RO7303359

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• RO7303359

Participant Groups

Group I: Single Ascending Dose Stage Experimental Treatment 1 Intervention

Participants will receive a single dose of RO7303359, in multiple escalating cohorts (A-D).

Group II: Optional cohort F Experimental Treatment 1 Intervention

An optional additional cohort may be added with the dose not exceed the MTD or MTeD.

Group III: Optional Cohort E Experimental Treatment 1 Intervention

An optional additional cohort may be added with the dose not exceed the MTD or MTeD.

Group IV: Expansion Cohort Stage Experimental Treatment 1 Intervention

Participants will receive the maximum tolerated dose (MTD) or the maximum tested dose (MTeD) as determined in the single ascending dose stage.

Who Is Running the Clinical Trial?

Hoffmann-La Roche

Lead Sponsor

Trials

2,482

Recruited

1,107,000+

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Avastin, Herceptin, Rituxan, Accu-Chek

Dr. Levi Garraway

Hoffmann-La Roche

Chief Medical Officer since 2019

MD from the University of Basel

Dr. Thomas Schinecker

Hoffmann-La Roche

Chief Executive Officer since 2023

PhD in Molecular Biology from New York University

References

1. United States pubmed.ncbi.nlm.nih.gov

Safety, Tolerability, and Pharmacokinetics of IMU-935, a Novel Inverse Agonist of Retinoic Acid Receptor-Related Orphan Nuclear Receptor γt: Results From a Double-Blind, Placebo-Controlled, First-in-Human Phase 1 Study. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Structural determinant for inducing RORgamma specific inverse agonism triggered by a synthetic benzoxazinone ligand. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Identification of SR2211: a potent synthetic RORγ-selective modulator. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Retinoic acid-related orphan receptor gamma t (RORγt) inverse agonists/antagonists for the treatment of inflammatory diseases - where are we presently? [2022]

5. England pubmed.ncbi.nlm.nih.gov

Pharmacokinetics, pharmacodynamics and safety of the inverse retinoic acid-related orphan receptor γ agonist AZD0284. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Synthesis and purification of oligonucleotide N3'-->P5' phosphoramidates and their phosphodiester and phosphorothioate chimeras. [2016]

7. United States pubmed.ncbi.nlm.nih.gov

A picomole-scale method for rapid peptide sequencing through convenient and efficient N-terminal phosphorylation and electrospray ionization mass spectrometry. [2018]

8. Germany pubmed.ncbi.nlm.nih.gov

Unusual Oxaphosphoranes by Acyl Transfer from o-Acetoxy-o'-diphenylphosphanyltolane. [2019]

9. Germany pubmed.ncbi.nlm.nih.gov

Analysis of an oligonucleotide N3'-->P5' phosphoramidate/phosphorothioate chimera with capillary gel electrophoresis. [2000]

10. United States pubmed.ncbi.nlm.nih.gov

Synthesis of chimeric oligonucleotides containing phosphodiester, phosphorothioate, and phosphoramidate linkages. [2019]