Clinical trials: sponsored by Hoffmann-La Roche
Duchenne Muscular Dystrophy
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD)
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks.
- Trial name
- ENVOL (NCT06128564)
- Testing
- delandistrogene moxeparvovec
- Where
- 7 sites in Belgium, France and 4 more
- Volunteers
- 13 people
- Sponsor
- Hoffmann-La Roche
Solid Tumors
Tumor-agnostic Precision Immuno-oncology and Somatic Targeting Rational for You (TAPISTRY) Platform Study
TAPISTRY is a Phase II, global, multicenter, open-label, multi-cohort study designed to evaluate the safety and efficacy of targeted therapies or immunotherapy as single agents or in rational, specified combinations in…
- Trial name
- NCT04589845
- Testing
- Entrectinib, Alectinib, Atezolizumab
- Where
- 112 sites in United States, Australia and 21 more
- Volunteers
- 920 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory…
- Trial name
- SHIELD DMD (NCT06450639)
- Testing
- Satralizumab
- Where
- 18 sites in United States, Denmark and 4 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors
This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against…
- Conditions
- Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A
- Trial name
- HAVEN 6 (NCT04158648)
- Testing
- Emicizumab
- Where
- 22 sites in United States, Belgium and 8 more
- Volunteers
- 73 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 3 (NCT04654468)
- Testing
- Crovalimab
- Where
- 5 sites in China
- Volunteers
- 51 people
- Sponsor
- Hoffmann-La Roche
Dup15q Syndrome
A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome
This study consists of two parts. Part 1 will evaluate the safety, efficacy, and pharmacodynamics of 52-weeks of basmisanil treatment in children and adolescents (aged 2-14 years) with Dup15q syndrome.
- Trial name
- NCT05307679
- Testing
- Basmisanil
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 7 people
- Sponsor
- Hoffmann-La Roche
ALK Fusion-positive Solid or CNS Tumors
A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors
This study will evaluate the safety, pharmacokinetics, and efficacy of alectinib in children and adolescents with ALK fusion-positive solid or CNS tumors for whom prior treatment has proven to be ineffective or for whom there is…
- Trial name
- NCT04774718
- Testing
- Alectinib
- Where
- 31 sites in United States, Australia and 9 more
- Volunteers
- 42 people
- Sponsor
- Hoffmann-La Roche
Carcinoma, Non-Small-Cell Lung
A Study of Participants in China With Non-Small-Cell Lung Cancer That is Unable to be Treated With Surgery
This is a multicenter, observational cohort study in China with both primary prospective data collection and retrospective collection of prior treatment information from medical records, which enrolls and follows patients who are…
- Trial name
- NCT05872763
- Where
- 12 sites in China
- Volunteers
- 901 people
- Sponsor
- Hoffmann-La Roche
Relapsing-remitting Multiple Sclerosis
A Study to Evaluate Safety and Efficacy of Ocrelizumab in Comparison With Fingolimod in Children and Adolescents With Relapsing-remitting Multiple Sclerosis (RRMS)
This double-blind, double-dummy study will evaluate the safety and efficacy of ocrelizumab compared with fingolimod in children and adolescents with RRMS aged between 10 and < 18 years over a flexible duration.
- Trial name
- Operetta 2 (NCT05123703)
- Testing
- Ocrelizumab, Fingolimod
- Where
- 71 sites in United States, Argentina and 23 more
- Volunteers
- 188 people
- Sponsor
- Hoffmann-La Roche
Asthma
A Study of Lebrikizumab in Adolescent Participants With Uncontrolled Asthma Who Are on Inhaled Corticosteroids and a Second Controller Medication
This randomized, multicenter, double-blind, placebo-controlled, parallel-group study will evaluate the efficacy, safety, and tolerability of lebrikizumab in adolescent participants with asthma whose disease remains uncontrolled…
- Trial name
- NCT01875003
- Testing
- Lebrikizumab, Standard of Care
- Where
- 81 sites in United States, Argentina and 19 more
- Volunteers
- 346 people
- Sponsor
- Hoffmann-La Roche
Muscular Atrophy, Spinal
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
- Trial name
- PUPFISH (NCT05808764)
- Testing
- Risdiplam
- Where
- 13 sites in United States, Belgium and 6 more
- Volunteers
- 11 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Disease
A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD)
This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with SCD.
- Trial name
- CROSSWALK-c (NCT05075824)
- Testing
- Crovalimab
- Where
- 27 sites in United States, Brazil and 9 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
Influenza
Study to Assess the Safety, Pharmacokinetics, and Efficacy of Baloxavir Marboxil in Chinese Pediatric Participants 1 to <12 Years of Age With Influenza Symptoms
The purpose of this study is to evaluate the safety of a single dose baloxavir marboxil compared with 5 days of oseltamivir administered twice a day (BID) in Chinese pediatric participants aged 1 to < 12 years with influenza…
- Trial name
- NCT06774859
- Testing
- Baloxavir Marboxil, Oseltamivir
- Where
- 16 sites in China
- Volunteers
- 100 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A
Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants
This multicenter, open-label, Phase 3 study with randomized and non-randomized arms is designed to investigate the efficacy, safety, and pharmacokinetics of emicizumab in participants with hemophilia A regardless of factor VIII…
- Trial name
- HAVEN 5 (NCT03315455)
- Testing
- Emicizumab
- Where
- 13 sites in China, Hong Kong and 2 more
- Volunteers
- 85 people
- Sponsor
- Hoffmann-La Roche
Angelman Syndrome
A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.
This is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion AS aged 5-17 years (inclusive) will be enrolled in the study.
- Trial name
- Aldebaran (NCT05630066)
- Testing
- Alogabat
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- Hoffmann-La Roche
Spinal Muscular Atrophy
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA)…
- Trial name
- Jewelfish (NCT03032172)
- Testing
- Risdiplam
- Where
- 24 sites in United States, Belgium and 7 more
- Volunteers
- 174 people
- Sponsor
- Hoffmann-La Roche
Juvenile Idiopathic Arthritis
A Long-Term Extension Study of RoActemra/Actemra (Tocilizumab) in Patients With Juvenile Idiopathic Arthritis From France Who Completed WA19977 Core Study
This long-term, open-label extension study will evaluate the safety of RoActemra/Actemra (tocilizumab) in patients with polyarticular-course juvenile idiopathic arthritis who completed the WA19977 core study.
- Trial name
- NCT01673919
- Testing
- tocilizumab [RoActemra/Actemra]
- Where
- 4 sites in France
- Volunteers
- 7 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Disease
A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).
The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD.
- Trial name
- CROSSWALK-a (NCT04912869)
- Testing
- Crovalimab
- Where
- 16 sites in United States, Brazil and 8 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Angelman Syndrome
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
This is a phase I, multicenter, non-randomized, adaptive, open-label, multiple ascending, intra-participant, dose-escalation study with a long-term extension (LTE) part and an optional open-label extension (OOE) part.
- Trial name
- NCT04428281
- Testing
- RO7248824
- Where
- 12 sites in United States, Italy and 2 more
- Volunteers
- 74 people
- Sponsor
- Hoffmann-La Roche
Influenza
Study to Assess the Efficacy of Baloxavir Marboxil Versus Placebo to Reduce Onward Transmission of Influenza A or B in Households
Otherwise healthy index patients (IP) are randomized to either baloxavir marboxil or placebo if their influenza symptoms onset was within 48 hours of screening.
- Trial name
- NCT03969212
- Testing
- Baloxavir Marboxil
- Where
- 142 sites in United States, Bulgaria and 14 more
- Volunteers
- 4,138 people
- Sponsor
- Hoffmann-La Roche