Clinical trials: Patent
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
Pulmonary Infections, Pseudomonas Aeruginosa
Second Open Label Extension to Bridging Study CTBM100C2303
This was an open-label, single arm (uncontrolled) study in participants suffering from cystic fibrosis, who have completed their study participation in CTBM100C2303 and extension study one CTBM100C2303E1 (all visits), who were…
- Trial name
- NCT01069705
- Testing
- Tobramycin inhalation powder
- Where
- 17 sites in Bulgaria, Estonia and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Healthy
An Open-label, Single-dose, Non-randomized Study of IV 14C-labeled PER977 in Healthy Male Subjects
This is an open-label, single-dose, non-randomized study in healthy male subjects to characterize the mass balance, metabolic disposition, and identification of the metabolites and general metabolic pathways following…
- Trial name
- NCT02205905
- Testing
- 14C PER977
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Perosphere Pharmaceuticals Inc, a wholly owned subsidiary of AMAG Pharmaceuticals, Inc.
HIV Infections
A Study of Amprenavir in HIV-Infected Patients
The purpose of this study is to see if it is safe to give amprenavir (APV) to HIV-infected patients. This study also examines the effect APV has on the level of HIV in the blood.
- Trial name
- NCT00002405
- Testing
- Amprenavir
- Where
- United States
- Sponsor
- Glaxo Wellcome
Glycogen Storage Disease Type II Infantile Onset
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
- Trial name
- ROSSELLA (NCT04808505)
- Testing
- Cipaglucosidase alfa, Miglustat
- Where
- 14 sites in United States, Germany and 4 more
- Volunteers
- 36 people
- Sponsor
- Amicus Therapeutics
Gastroesophageal Reflux Disease
Safety and Efficacy of Dexlansoprazole Delayed-Release Capsules in Treating Symptomatic Non-Erosive Gastroesophageal Reflux Disease in Adolescents
The purpose of this study was to assess the safety and effectiveness of once daily oral administration of dexlansoprazole delayed-release capsules in adolescent participants with symptomatic non-erosive gastroesophageal reflux…
- Trial name
- NCT01642602
- Testing
- Dexlansoprazole
- Where
- 64 sites in United States, Belgium and 6 more
- Volunteers
- 104 people
- Sponsor
- Takeda
Spinal Muscular Atrophy, Neuromuscular Diseases
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec…
- Conditions
- Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
- Trial name
- RESILIENT (NCT05337553)
- Testing
- taldefgrobep alfa
- Where
- 53 sites in United States, Belgium and 7 more
- Volunteers
- 269 people
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Nephrotic Syndrome
The Efficacy of Huaiqihuang Granule in Children With Primary Nephrotic Syndrome
This is a multisite, open-label, prospective and registered study designed to evaluate the efficacy and safety of Huai-Qi-Huang granule in children with primary nephrotic syndrome.
- Trial name
- NCT03332420
- Where
- 17 sites in China
- Volunteers
- 1,507 people
- Sponsor
- Qidong Gaitianli Medicines Co., Ltd
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Congenital Bleeding Disorder, Haemophilia A
Comparison of the Action of Drugs in the Body and Safety of N8 and Advate® in Haemophilia A Subjects
This trial is conducted in Europe and Asia. The aim of this clinical trial is to compare two recombinant factor VIII drugs, turoctocog alfa (recombinant factor VIII (N8)) with Advate®, in haemophilia A subjects, investigating the…
- Trial name
- NCT00837356
- Testing
- Advate®, turoctocog alfa
- Where
- 7 sites in Germany, Israel and 3 more
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Epilepsy
Levetiracetam Versus Oxcarbazepine as Monotherapy to Evaluate Efficacy and Safety in Subjects With Newly or Recently Diagnosed Partial Epilepsy
To evaluate the long term effectiveness of Levetiracetam (LEV) monotherapy on Treatment Failure Rate in subjects with newly diagnosed partial onset seizures with or without secondary generalized seizures, compared to…
- Trial name
- OPTIMAL (NCT01498822)
- Testing
- Levetiracetam, Oxcarbazepine
- Where
- 23 sites in South Korea
- Volunteers
- 353 people
- Sponsor
- Korea UCB Co., Ltd.
Attention Deficit Disorder With Hyperactivity
An Efficacy and Safety Study of Osmotic Release Oral System (OROS) Methylphenidate in Participants With Attention Deficit Hyperactivity Disorder (ADHD)
The purpose of this study is to evaluate the safety and efficacy of Osmotic Release Oral System (OROS) methylphenidate in participants with Attention Deficit Hyperactivity Disorder (ADHD).
- Trial name
- NCT01012622
- Testing
- Osmotic Release Oral System (OROS) Methylphenidate Hydrochloride
- Volunteers
- 142 people
- Sponsor
- Janssen Korea, Ltd., Korea
Meningoccocal Disease, Meningococcal Meningitis
Antibody Persistence, and Safety and Tolerability of a Booster Dose of the Meningococcal B Vaccine After the Completion of the Vaccination Course in Study V72_28
The aim of this extension study is to explore the antibody persistence 24 to 36 months after the last dose of vaccine, in infants that received a two or three dose primary series plus a booster dose at 11 months of age, of the…
- Trial name
- NCT01894919
- Testing
- Bexsero® vaccine (1 dose at study month zero), Bexsero® vaccine (2 doses 1 month apart)
- Where
- 17 sites in Hungary, Spain
- Volunteers
- 851 people, healthy volunteers welcome
- Sponsor
- Novartis Vaccines
Lupus Erythematosus, Systemic, Lupus Nephritis
A Study of CC-97540 (BMS-986353 or Zola-cel), CD19-Targeted NEX-T CAR T Cells, in Participants With Active SLE Despite Immunosuppressants (Breakfree-SLE)
The purpose of this study is to evaluate the efficacy, safety and drug levels of CC-97540 in participants with active systemic lupus erythematosus (SLE) including lupus nephritis with inadequate response to glucocorticoids and at…
- Trial name
- NCT07015983
- Testing
- CC-97540, Fludarabine, Cyclophosphamide
- Where
- 97 sites in United States, Argentina and 14 more
- Volunteers
- 89 people
- Sponsor
- Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Chronic Pain
Open-Label Safety and PK Study of ER Hydromorphone Tablets in Opioid-Tolerant Pediatric Chronic Pain Patients
This pediatric study is designed to provide safety information, dosing guidelines, and a pharmacokinetic (PK) evaluation of once-daily hydromorphone hydrochloride (HCl) extended-release (ER) tablets in children with chronic…
- Trial name
- NCT02321319
- Testing
- Hydromorphone HCl ER Tablets
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Mallinckrodt
Hepatitis A Virus Infection
A Study of 2 Doses of VAQTA™ in Healthy Children 12 to 23 Months of Age (V251-069)
This study will demonstrate the immunogenicity and evaluate the safety/tolerability of the vaccine in Chinese children between 12 and 23 months of age.
- Trial name
- NCT00835380
- Testing
- Hepatitis A Vaccine, Purified Inactivated (VAQTA™)
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Merck Sharp & Dohme LLC
Chronic Graft Versus Host Disease
A Study to Investigate the Efficacy and Safety of Belumosudil Compared With Best Available Therapy in Participants Aged 12 Years or Older With Chronic Graft-versus-host Disease
Participants will be randomized 1:1 to receive either belumosudil or Best Available Therapy (BAT), with stratification based on baseline cGVHD severity as defined by the 2014◦NIH consensus criteria (moderate versus severe), use…
- Trial name
- NCT07771439
- Testing
- Belumosudil, Best available therapy (BAT)
- Volunteers
- 356 people
- Sponsor
- Sanofi
Cystic Fibrosis
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
- Trial name
- NCT05444257
- Testing
- VX-121/TEZ/D-IVA
- Where
- 195 sites in United States, Australia and 21 more
- Volunteers
- 822 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Recessive Dystrophic Epidermolysis Bullosa
A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.
- Trial name
- NCT04599881
- Testing
- PTR-01
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Phoenix Tissue Repair, Inc.
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