Roche Holding AG / Genentech

This study will evaluate the success and safety of subretinal surgical delivery as well as the preliminary activity of OpRegen in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). All endpoints are assessed for the study eye unless otherwise indicated. The substudy will evaluate the operational feasibility and scientific interpretability of incorporating AO retinal imaging using the EarlySight Cellularis® Discovery device. Participants who have fulfilled the eligibility requirements for the parent study and meet the substudy's eligibility criteria will have the option to participate in the substudy. The EarlySight Cellularis® Discovery device will be used only as an assessment tool and data obtained from this device will not be used to guide clinical care or influence clinical outcomes for participants.Show more

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.Show more

The main purpose of the study is to evaluate participant satisfaction after administration of ocrelizumab subcutaneously (SC) after 12 months using the therapy administration satisfaction questionnaire subcutaneous (TASQ-SC).

This study will evaluate safety, pharmacokinetics (PK), clinical activity and pharmacodynamics (PD) of RO7851624 in participants with RRMM who are triple-class exposed (treated with proteasome inhibitors \[PIs\], immunomodulators \[IMiDs\], and anti-cluster of differentiation 38 \[anti-CD38\] monoclonal antibodies), and have limited remaining standard treatment options due to refractoriness, intolerance, or multiple prior therapies.Show more

The purpose of the study is to evaluate safety and tolerability of the combination of cevostamab plus elranatamab and also determine the recommended Phase II regimen (RP2R) for the study treatment. The study consists of a safety lead-in stage, and an expansion stage.

This phase II, randomized, double-blind, placebo-controlled study is designed to evaluate the safety, efficacy and pharmacokinetics (PK) of obinutuzumab in adolescent participants (AP) aged 12 to less than 18 with biopsy-confirmed proliferative lupus nephritis (LN). It will also evaluate open label safety and PK of obinutuzumab in pediatric participants (PP), aged 5 to \<12 with LN.Show more

The study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of cevostamab in participants with systemic lupus erythematosus (SLE) with or without active lupus nephritis (LN).

Roche, through its subsidiary Genentech, develops therapies across oncology, immunology, neurology, and rare diseases. The company applies its expertise in biotechnology and precision medicine to address rare genetic and complex diseases with limited treatment options.

Roche Holding AG / Genentech has active programs across 65 rare diseases: Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Angelman Syndrome, Atypical Hemolytic Uremic Syndrome, Autoimmune Hepatitis, Bullous Pemphigoid, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, Cystic Fibrosis, Dermatomyositis, Desmoplastic Small Round Cell Tumor, Duchenne Muscular Dystrophy, Eosinophilic Esophagitis, Eosinophilic Granulomatosis with Polyangiitis, Essential Thrombocythemia, Familial Medullary Thyroid Carcinoma, Fragile X Syndrome, Facioscapulohumeral Muscular Dystrophy, Gastrointestinal Stromal Tumor, Geographic Atrophy, Giant Cell Arteritis, Granulomatosis with Polyangiitis, Guillain-Barré Syndrome, Hemophilia A, Hemophilia B, Hereditary Diffuse Gastric Cancer, Hereditary Hemorrhagic Telangiectasia, Hodgkin Lymphoma, Huntington Disease, Idiopathic Pulmonary Fibrosis, IgA Nephropathy, Immune Thrombocytopenia, Light Chain Amyloidosis, Medullary Thyroid Cancer, Membranous Nephropathy, Mesothelioma, MOG Antibody-Associated Disease, Multiple Myeloma, Multiple Sclerosis, Myasthenia Gravis, Myelodysplastic Syndromes, Myelofibrosis, Neurofibromatosis Type 1, Neuromyelitis Optica, Paraganglioma, Paroxysmal Nocturnal Hemoglobinuria, Pemphigus Vulgaris, Pheochromocytoma, Polycythemia Vera, Primary Biliary Cholangitis, Primary Sclerosing Cholangitis, Progressive Supranuclear Palsy, Pulmonary Arterial Hypertension, RET Fusion Cancer, Sarcoidosis, Sickle Cell Disease, Sjögren's Syndrome, Spinal Muscular Atrophy, Systemic Juvenile Idiopathic Arthritis, Systemic Sclerosis, Thalassemia, Thyroid Eye Disease, Turner Syndrome, Von Willebrand Disease, Waldenström Macroglobulinemia. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Roche Holding AG / Genentech is a diversified pharmaceutical company headquartered in Basel, Switzerland, founded in 1896. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 65 active rare disease programs including Adrenocortical Carcinoma, Amyotrophic Lateral Sclerosis, Angelman Syndrome. Many of its rare disease treatments hold orphan drug designations from the FDA.

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Roche Holding AG / Genentech, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

Roche Holding AG / Genentech was founded in 1896 and is headquartered in Basel, Switzerland. The company has been active for 130 years. It trades publicly under the ticker symbol RHHBY.