Clinical trials: Congenital Deformity
Heart Defects, Congenital
Xeltis Pulmonary Valved Conduit Safety and Performance Study
This is a multi-center prospective, single-arm, non-randomized, open label study to assess safety and performance of the Xeltis Pulmonary Valved Conduit in subjects requiring Right Ventricular Outflow Tract correction or…
- Trial name
- NCT03405636
- Testing
- Xeltis Pulmonary Valved Conduit
- Where
- 4 sites in Hungary, Malaysia and 2 more
- Sponsor
- Xeltis
Cystic Fibrosis
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
- Trial name
- NCT05844449
- Testing
- VNZ/TEZ/D-IVA
- Where
- 37 sites in United States, Australia and 8 more
- Volunteers
- 174 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR)…
- Trial name
- NCT05274269
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 90 sites in Austria, Belgium and 13 more
- Volunteers
- 307 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Retinopathy of Prematurity (ROP)
Safety, PK and PD of FLQ-101 in Premature Neonates
The purpose of this study is to evaluate safety and efficacy outcomes following exposure to FLQ-101.
- Trial name
- tROPhy-1 (NCT07093255)
- Testing
- FLQ-101
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- FELIQS INC.
Carcinoma, Renal Cell
DFF332 as a Single Agent and in Combination With Everolimus & Immuno-Oncology Agents in Advanced/Relapsed Renal Cancer & Other Malignancies
This was a first in human study of DFF332, a small molecule that targets a protein called HIF2α. By acting on HIF2α, DFF332 may be able to stop the growth of certain types of cancer.
- Trial name
- NCT04895748
- Testing
- DFF332, RAD001, PDR001
- Where
- 11 sites in United States, Czechia and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
- Trial name
- NCT04956289
- Testing
- Viltolarsen
- Where
- 8 sites in United States, China and 4 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Atopic Dermatitis
In-home Use Test of the New Modified Diprobase Formulation to Assess the Safety and Tolerability in Infants and Children Under Physician's Control
To investigate the safety and tolerability of the modified Diprobase formulation over 14 days in infants and children with a history of Atopic Dermatitis (AD).
- Trial name
- NCT03441568
- Testing
- Modified Diprobase formulation (BAY987534)
- Where
- United Kingdom
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Bayer
Spinal and Bulbar Muscular Atrophy
Specified Drug-Use Survey of Leuprorelin Acetate Injection Kit 11.25 mg "All-Case Investigation: Spinal and Bulbar Muscular Atrophy (SBMA)"
The purpose of this survey is to evaluate the long-term safety and efficacy of leuprorelin acetate injection kit 11.25 mg in patients with spinal and bulbar muscular atrophy (SBMA) in the routine clinical setting.
- Trial name
- NCT03555578
- Testing
- Leuprorelin Acetate
- Where
- Japan
- Volunteers
- 1,905 people
- Sponsor
- Takeda
Atopic Dermatitis
Targeting Staphylococci in Atopic Dermatitis
The aim of the study is to deliver a better understanding of the molecular mechanisms used by S. epidermidis strains in their adhesion and colonization on the stratum corneum across a broad spectrum of atopic dermatitis in mild…
- Trial name
- NCT05502185
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- L'Oreal
Dermatitis, Atopic, Hay Fever
Safety, Tolerability and Immune Response of IMVAMUNE (MVA-BN)Smallpox Vaccine in Patients With Atopic Disorders
The purpose of this study is to gather information on the safety and immunogenicity of an investigational smallpox vaccine in populations with atopic disorders.
- Trial name
- NCT00189917
- Testing
- IMVAMUNE (MVA-BN)
- Where
- Germany
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Bavarian Nordic
Congenital Hemangioma, Hemangioendothelioma
A Study to Evaluate How Safe and Effective is the Mixture of Lipiodol® Ultra Fluid and Glue When Used for Embolization Procedures
This was a multicenter phase IV Post-Marketing Study. The study is designed to investigate the safety of Lipiodol® Ultra Fluid in association with surgical glues used according to each site medical practice of vascular…
- Conditions
- Congenital Hemangioma, Hemangioendothelioma, Angiosarcoma, Arteriovenous Malformations
- Trial name
- NCT02625389
- Testing
- Lipiodol Ultra Fluid with surgical glues
- Where
- 16 sites in India
- Volunteers
- 132 people
- Sponsor
- Guerbet
Primary Hyperoxaluria Type 1
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
- Trial name
- NCT02795325
- Testing
- DCR-PH1
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 41 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Mucopolysaccharidosis I, Hurlers Syndrome
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
- Conditions
- Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT00912925
- Testing
- rhIDU (recombinant human-Alpha-L-Iduronidase)
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 45 people
- Sponsor
- Genzyme, a Sanofi Company
Gouty Arthritis (GA)
A Bioequivalence Study of Two Different Dosage Form of Genakumab
The purpose of this study is to investigate whether Genakumab Injection is bioequivalent to Genakumab for Injection.
- Trial name
- NCT06451848
- Testing
- Genakumab Injection (T, Solution for injection), Genakumab for Injection (R, Powder for solution for injection)
- Where
- China
- Volunteers
- 178 people, healthy volunteers welcome
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Pediatric Oncology
A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy
Dose escalation phase of the study : To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in…
- Trial name
- NCT02085148
- Testing
- Regorafenib (BAY73-4506), Vincristine (Cellcristin®), Irinotecan (Irinotecan Cell pharm®)
- Where
- 12 sites in France, Italy and 2 more
- Volunteers
- 62 people
- Sponsor
- Bayer
Duchenne Muscular Dystrophy
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
- Trial name
- NCT02752048
- Testing
- TAS-205
- Where
- 11 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Hemophilia A
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
- Trial name
- NCT07226206
- Testing
- SPK-8011QQ
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Hoffmann-La Roche
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants.
- Trial name
- PROTECT-ALT (NCT06684314)
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Taiwan
- Volunteers
- 79 people
- Sponsor
- Sanofi
Down Syndrome, Edwards Syndrome
Whole Blood Specimen Collection From Pregnant Subjects
To obtain whole blood specimens from pregnant subjects to be used for research and development and clinical validation studies of prenatal assays.
- Conditions
- Down Syndrome, Edwards Syndrome, Patau Syndrome, Klinefelter Syndrome
- Trial name
- NCT02430584
- Testing
- Observational - no intervention
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Progenity, Inc.
Missing Teeth
Post Market Clinical FoIIow up (PMCF) Medical Device (MD) of Z-Systems Monotype Zirconia Dental Product: Z5m Dental Implants Registry
Commercially pure (CP) titanium is the gold standard to produce dental implants because it has a huge volume of scientific publications over a period of more than 50 years, it is cheap and simple to produce (i.e., making dental…
- Trial name
- NCT06007365
- Testing
- Z5m Dental Implants
- Where
- Switzerland
- Volunteers
- 70 people
- Sponsor
- Z-Systems
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