Clinical trials: Genetics Syndrome
MPS IIIA, Sanfilippo Syndrome
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Trial name
- NCT02716246
- Testing
- UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 36 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Severe Hemophilia A
A Study of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary…
- Trial name
- NCT05265286
- Testing
- FRSW117
- Where
- 5 sites in China
- Volunteers
- 15 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
Dermatitis, Atopic
Study Of Single And Ten Day Repeat Atopical Applications Of GW842470X Cream On The Skin Of Patients With Atopic Dermatitis
The purpose of this study of this study is to investigate the safety, tolerability and pharmacokinetics of GW842470X in patients with atopic dermatitis. GW842470X is an inhibitor of phosphodiesterase 4 (PDE4).
- Trial name
- NCT00356642
- Testing
- GW842470X
- Where
- Germany
- Volunteers
- 45 people
- Sponsor
- GlaxoSmithKline
Genetic Disorder, Noonan Syndrome
Somatropin Effect on Linear Growth and Final Height in Subjects With Noonan Syndrome
This trial is conducted in Europe. The aim of this trial is to evaluate the effect of somatropin (Norditropin®) on final height in children with Noonan syndrome having being treated for up to 10 years with somatropin…
- Trial name
- NCT01529840
- Testing
- somatropin
- Where
- Sweden
- Volunteers
- 24 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A (Moderate or Severe), Hemophilia, Classic
A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of…
- Conditions
- Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia
- Trial name
- NCT06752850
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Italy, Norway and 2 more
- Volunteers
- 37 people
- Sponsor
- Swedish Orphan Biovitrum
Skin Laxity
Safety and Effectiveness Evaluation of the Device in Achieving Submental Lift
A prospective evaluation of the ThermiRF device in treating skin laxity under the chin and neck. Seventy male and female subjects between the age of 35 and 65 (inclusive) will be enrolled in this trial.
- Trial name
- NCT02832674
- Testing
- Percutaneous Radiofrequency single treatment
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- ThermiGen, LLC
Atopic Dermatitis
A Study of Two Different Formulations of 611 in Healthy Adult Subjects in China
The main purpose of this study is to compare two different formulations (mixtures) of 611 in healthy participants. This study will compare how much of each formulation gets into the blood stream.
- Trial name
- 611 (NCT07042113)
- Testing
- new formulations of 611, existing formulations of 611
- Where
- 2 sites in China
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd.
Atopic Dermatitis
A Dose-ranging Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Galvokimig in Adult Study Participants With Atopic Dermatitis
The purpose of the study is to evaluate the dose-response relationship of galvokimig compared with placebo in study participants with moderate-to-severe atopic dermatitis (AtD).
- Trial name
- PossibILity (NCT07277660)
- Testing
- Galvokimig
- Where
- 59 sites in United States, Bulgaria and 7 more
- Volunteers
- 165 people
- Sponsor
- UCB Biopharma SRL
Duchenne Muscular Dystrophy (DMD)
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level.
- Trial name
- DELPHI (NCT00654784)
- Testing
- idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Dermatitis, Atopic
A Study of BMS-986166 or Branebrutinib for the Treatment of Participants With Atopic Dermatitis
The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986166 and of branebrutinib, each versus placebo, for the treatment of participants with moderate to severe atopic dermatitis.
- Trial name
- NCT05014438
- Testing
- BMS-986166, Branebrutinib
- Where
- 42 sites in United States, Australia and 5 more
- Volunteers
- 17 people
- Sponsor
- Bristol-Myers Squibb
Hemophilia A
EFFEKT - Efficacy and Safety of Long-term Treatment With KOGENATE Bayer/FS
The aim of this international prospective, non-interventional post-marketing surveillance study is to obtain data on treatment procedures, long-term safety and efficacy and patient acceptance of KOGENATE Bayer in treatment of…
- Trial name
- NCT00874926
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 17 sites in Bahrain, Bosnia and Herzegovina and 15 more
- Volunteers
- 405 people
- Sponsor
- Bayer
RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer
Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors
This is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667)…
- Conditions
- RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer, RET-altered Papillary Thyroid Cancer, RET-altered Colon Cancer
- Trial name
- ARROW (NCT03037385)
- Testing
- pralsetinib (BLU-667)
- Where
- 71 sites in United States, Belgium and 11 more
- Volunteers
- 590 people
- Sponsor
- Hoffmann-La Roche
DCild Development Disorders, Pervasive, Autistic Disorder
A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD)
The purpose of the study is to evaluate the effectiveness of an oral solution of risperidone (an antipsychotic medication) versus placebo in the treatment of behavioral symptoms in children with Pervasive Developmental Disorders…
- Conditions
- DCild Development Disorders, Pervasive, Autistic Disorder, Developmental Disabilities, Asperger Syndrome
- Trial name
- NCT00261508
- Testing
- risperidone
- Volunteers
- 80 people
- Sponsor
- Janssen-Ortho Inc., Canada
Cystic Fibrosis
Early Signs of Efficacy Study With Riociguat in Adult Homozygous Delta F508 Cystic Fibrosis Patients
Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi
- Trial name
- NCT02170025
- Testing
- Riociguat (Adempas, BAY63-2521)
- Where
- 10 sites in United States, Belgium and 5 more
- Volunteers
- 21 people
- Sponsor
- Bayer
Graft vs Host Disease, Congenital Aniridia
Safety and Performance Study of the ARGOS-IO System in Patients Undergoing Boston Keratoprosthesis Implantation
The ARGOS-IO pressure sensor is intended to be implanted in the human eye in combination with Boston Keratoprosthesis (BKPro) surgery and to remain in place indefinitely.
- Conditions
- Graft vs Host Disease, Congenital Aniridia, Chemical Burns, Stevens-Johnson Syndrome
- Trial name
- NCT02945176
- Testing
- ARGOS-IO system
- Where
- 2 sites in Germany
- Volunteers
- 13 people
- Sponsor
- Implandata Ophthalmic Products GmbH
Hemophilia A, Prophylaxis of Bleeding
An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A.
This is an observational study in which data from people with hemophilia A who decide on their own or by recommendation of their doctors to take Jivi are collected and studied.
- Trial name
- JOIHA (NCT05643560)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- Italy
- Volunteers
- 33 people
- Sponsor
- Bayer
Cystic Fibrosis
A Study in Pediatric Patients With Cystic Fibrosis Lung Disease
The purpose of this study is to investigate the safety and effectiveness of two dose strengths of study drug compared to placebo in pediatric patients with cystic fibrosis (CF).
- Trial name
- NCT00130182
- Testing
- denufosol tetrasodium (INS37217)
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Merck Sharp & Dohme LLC
Retinitis Pigmentosa, Leber Congenital Amaurosis
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101).
- Trial name
- NCT05203939
- Testing
- OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose
- Where
- 7 sites in United States
- Volunteers
- 22 people
- Sponsor
- Ocugen
Atopic Dermatitis
A Study of Baricitinib (LY3009104) in Combination With Topical Corticosteroids in Adults With Moderate to Severe Atopic Dermatitis
The purpose of this study is to evaluate the efficacy and safety of baricitinib in combination with topical corticosteroids (TCS) in participants with moderate to severe atopic dermatitis.
- Trial name
- BREEZE-AD7 (NCT03733301)
- Testing
- Baricitinib, Topical corticosteroid
- Where
- 68 sites in Argentina, Australia and 8 more
- Volunteers
- 329 people
- Sponsor
- Eli Lilly and Company
Moderate-to-Severe Atopic Dermatitis, Dermatitis, Dermatitis Atopic
Efficacy and Safety of Dupilumab in Participants ≥12 to <18 Years of Age, With Moderate-to-severe Atopic Dermatitis
The primary objective of the study was to demonstrate the efficacy of dupilumab as a monotherapy in participants ≥12 years to <18 years of age with moderate-to-severe atopic dermatitis (AD).
- Conditions
- Moderate-to-Severe Atopic Dermatitis, Dermatitis, Dermatitis Atopic, Eczema, Skin Diseases, Skin, Diseases Genetic, Genetic
- Trial name
- NCT03054428
- Testing
- Dupilumab
- Where
- 45 sites in United States, Canada
- Volunteers
- 251 people
- Sponsor
- Regeneron Pharmaceuticals