R 723 / Rigel - LARVOL DELTA

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2/3; N=166; Completed; Sponsor: Hoffmann-La Roche; Terminated ➔ Completed

Clinical • Trial completion • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: Hoffmann-La Roche; Trial completion date: Feb 2021 ➔ Dec 2021; Trial primary completion date: Feb 2021 ➔ Dec 2021

Clinical • Trial completion date • Trial primary completion date • Genetic Disorders • Huntington's Disease • Movement Disorders

A Study of RO7239958 to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Healthy Volunteers and Participants With Chronic Hepatitis B Virus Infection (clinicaltrials.gov) - P1; N=55; Terminated; Sponsor: Hoffmann-La Roche; Trial completion date: Mar 2021 ➔ Apr 2020; Active, not recruiting ➔ Terminated; Trial primary completion date: Mar 2021 ➔ Apr 2020; The study was terminated due to program discontinuation.

Clinical • Trial completion date • Trial primary completion date • Trial termination • Hepatitis B • Infectious Disease

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD (clinicaltrials.gov) - P1/2; N=43; Terminated; Sponsor: Hoffmann-La Roche; Completed ➔ Terminated; A pre-planned futility analysis indicated lack of efficacy.

Clinical • Trial termination • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Patients Who Participated in Prior Roche and Genentech Sponsored Studies (clinicaltrials.gov) - P3; N=1050; Recruiting; Sponsor: Hoffmann-La Roche; Trial completion date: Jul 2023 ➔ Jun 2024; Trial primary completion date: Jul 2023 ➔ Jun 2024

Clinical • Trial completion date • Trial primary completion date • Genetic Disorders • Huntington's Disease • Movement Disorders

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD (clinicaltrials.gov) - P1/2; N=43; Completed; Sponsor: Hoffmann-La Roche; Active, not recruiting ➔ Completed

Clinical • Trial completion • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2/3; N=166; Terminated; Sponsor: Hoffmann-La Roche; Trial completion date: Dec 2024 ➔ Apr 2020; Active, not recruiting ➔ Terminated; Trial primary completion date: Dec 2020 ➔ Apr 2020; A pre-planned futility analysis indicated lack of efficacy

Clinical • Trial completion date • Trial primary completion date • Trial termination • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

A Study of RO7239958 to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Healthy Volunteers and Participants With Chronic Hepatitis B Virus Infection (clinicaltrials.gov) - P1; N=75; Active, not recruiting; Sponsor: Hoffmann-La Roche; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Hepatitis B • Infectious Disease

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD (clinicaltrials.gov) - P1/2; N=43; Active, not recruiting; Sponsor: Hoffmann-La Roche; Trial completion date: Aug 2020 ➔ Jun 2022

Trial completion date • Biosimilar • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: Hoffmann-La Roche; Suspended ➔ Recruiting

Clinical • Enrollment open • Genetic Disorders • Huntington's Disease • Movement Disorders

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease (clinicaltrials.gov) - P1; N=20; Suspended; Sponsor: Hoffmann-La Roche; Recruiting ➔ Suspended

Clinical • Trial suspension

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: Hoffmann-La Roche; Trial completion date: Sep 2020 ➔ Jan 2021; Trial primary completion date: Sep 2020 ➔ Jan 2021

Clinical • Trial completion date • Trial primary completion date

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139) (clinicaltrials.gov) - P2; N=46; Completed; Sponsor: Hoffmann-La Roche; Active, not recruiting ➔ Completed

Clinical • Trial completion

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy (clinicaltrials.gov) - P2/3; N=166; Active, not recruiting; Sponsor: Hoffmann-La Roche; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

A Study of RO7239958 to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Healthy Volunteers and Participants With Chronic Hepatitis B Virus Infection (clinicaltrials.gov) - P1; N=82; Recruiting; Sponsor: Hoffmann-La Roche; Trial completion date: Nov 2021 ➔ Mar 2021; Trial primary completion date: Nov 2021 ➔ Mar 2021

Clinical • Trial completion date • Trial primary completion date

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease (clinicaltrials.gov) - P1; N=20; Recruiting; Sponsor: Hoffmann-La Roche; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease (clinicaltrials.gov) - P1; N=20; Not yet recruiting; Sponsor: Hoffmann-La Roche

Clinical • New P1 trial

Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD (clinicaltrials.gov) - P1/2; N=43; Active, not recruiting; Sponsor: Hoffmann-La Roche; Trial completion date: Jun 2022 ➔ Aug 2020

Clinical • Trial completion date

An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Patients Who Participated in Prior Roche and Genentech Sponsored Studies (clinicaltrials.gov) - P3; N=900; Recruiting; Sponsor: Hoffmann-La Roche; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of RO7234292 (RG6042) in Patients With Huntington's Disease (clinicaltrials.gov) - P3; N=900; Not yet recruiting; Sponsor: Hoffmann-La Roche

Clinical • New P3 trial

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139) (clinicaltrials.gov) - P2; N=46; Active, not recruiting; Sponsor: Hoffmann-La Roche; Phase classification: P1 ➔ P2

Clinical • Phase classification

A Study of RO7239958 to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Healthy Volunteers and Participants With Chronic Hepatitis B Virus Infection (clinicaltrials.gov) - P1; N=75; Recruiting; Sponsor: Hoffmann-La Roche; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139) (clinicaltrials.gov) - P1; N=46; Active, not recruiting; Sponsor: Hoffmann-La Roche; Phase classification: P2 ➔ P1

Clinical • Phase classification