Clinical trials: Birth Defect
Atopic Dermatitis
Long-term Trial of OPA-15406 Ointment in Adult and Pediatric Patients With Atopic Dermatitis
To demonstrate the safety of 1% OPA-15406 ointment in adult patients with AD and of 0.3% and 1% OPA-15406 ointments in pediatric patients with AD administered twice daily for 52 weeks.
- Trial name
- NCT03961529
- Testing
- 0.3% OPA-15406 ointment, 1% OPA-15406 ointment
- Where
- Japan
- Volunteers
- 366 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Atopic Dermatitis
Comparison of the Efficacy and Safety of Two Topical Creams for Pediatric Atopic Dermatitis
This study compares the effectiveness of two topical creams for atopic dermatitis in pediatric subjects. Subjects will be randomly assigned to use one of the two creams twice daily for 6 weeks or until clear.
- Trial name
- NCT00828412
- Testing
- EpiCeram Skin Barrier Emulsion, Desonide Cream 0.05%
- Where
- 5 sites in United States
- Volunteers
- 100 people
- Sponsor
- Promius Pharma, LLC
Hemophilia A
Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A
SIG-001-121 is a first-in-human (FIH), phase 1/2, multi-centre, open-label, dose escalation study to assess the safety, tolerability, and preliminary efficacy of SIG-001 in adults with severe or moderately severe haemophilia A…
- Trial name
- NCT04541628
- Testing
- SIG-001
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Eli Lilly and Company
Haemophilia A
Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A
Primary objective: To assess post-marketing immunogenicity of Optivate® by monitoring plasma inhibitor levels for at least 100 Exposure Days (EDs) for each subject.
- Trial name
- NCT01811875
- Testing
- Optivate 500IU
- Where
- 4 sites in Colombia, Germany and 1 more
- Volunteers
- 7 people
- Sponsor
- Bio Products Laboratory
Hereditary Angioedema
A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)
This is a multicenter, randomized, placebo-controlled, parallel-arm, phase 2 study to investigate the clinical efficacy, pharmacokinetics, and safety of CSL312 as prophylaxis to prevent attacks in subjects with HAE.
- Trial name
- NCT03712228
- Testing
- Factor XIIa antagonist monoclonal antibody
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 44 people
- Sponsor
- CSL Behring
Nervous System Malformations, Arthropathy, Neurogenic
Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease
This study will evaluate the safety and effectiveness of anakinra (Kineret) for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurological, cutaneous and…
- Conditions
- Nervous System Malformations, Arthropathy, Neurogenic, Urticaria, Papilledema
- Trial name
- NCT00069329
- Testing
- anakinra
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Cystic Fibrosis
Rollover Study of VX-770 in Cystic Fibrosis Subjects
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF).
- Trial name
- NCT01117012
- Testing
- Ivacaftor
- Where
- 58 sites in United States, Australia and 6 more
- Volunteers
- 192 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Achondroplasia
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
Approximately 63 participants will be randomized to one of three doses to receive Recifercept either Low Dose Medium Dose High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2…
- Trial name
- NCT04638153
- Testing
- Recifercept
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 60 people
- Sponsor
- Pfizer
Hypercholesterolemia, Familial Hypercholesterolemia
A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-013) CORALreef Lipids
The goal of this study is to evaluate the efficacy, safety, and tolerability of enlicitide decanoate in adult participants with hypercholesterolemia.
- Trial name
- NCT05952856
- Testing
- Enlicitide Decanoate
- Where
- 168 sites in United States, Argentina and 12 more
- Volunteers
- 2,912 people
- Sponsor
- Merck Sharp & Dohme LLC
Dilated Cardiomyopathy, Ischemic Cardiomyopathy
Optimized Biventricular Pacing Allograft Recipients
This study tests optimization of biventricular pacing (BiVP) in patients with dilated cardiomyopathy (DCM) or ischemic cardiomyopathy (ICM) during cardiac transplantation in patients with advanced cardiac failure.
- Trial name
- BiBET (NCT01290822)
- Testing
- BiVP, AAI Pacing
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Henry M. Spotnitz
Hemophilia B
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor…
- Trial name
- NCT01361126
- Testing
- Recombinant Coagulation Factor IX Albumin Fusion Protein
- Where
- 2 sites in Bulgaria, Israel
- Volunteers
- 17 people
- Sponsor
- CSL Behring
Dermatitis, Atopic
Study of Dupilumab Monotherapy Administered to Adult Patients With Moderate-to-Severe Atopic Dermatitis
This is a randomized, double-blind, placebo-controlled, parallel group study to confirm the efficacy and safety of Dupilumab monotherapy in adults with moderate-to-severe atopic dermatitis (AD).
- Trial name
- SOLO 1 (NCT02277743)
- Testing
- Dupilumab
- Where
- 101 sites in United States, Bulgaria and 8 more
- Volunteers
- 671 people
- Sponsor
- Regeneron Pharmaceuticals
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory).
- Trial name
- NCT03190915
- Testing
- Bone Marrow Aspiration and Biopsy, Trametinib
- Where
- 57 sites in United States
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
Surgical Complication, Paresthesia
Trial of Bilateral Sagittal Split Osteotomy Induced Paresthesia Using Ultrasonic vs. Reciprocating Saw Instrumentation
The aim of this prospective study is to analyze the postoperative paresthesias experienced in patients who undergo bilateral sagittal split osteotomies (BSSO) using an ultrasonic saw, versus a reciprocating saw.
- Conditions
- Surgical Complication, Paresthesia, Fracture, Nerve Injury
- Trial name
- NCT05205616
- Testing
- Sonopet ultrasonic saw, Reciprocating saw
- Where
- 2 sites in United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Gout
Phase 3, Febuxostat, Allopurinol and Placebo-Controlled Study in Gout Subjects.
The purpose of this study is to compare febuxostat, allopurinol and placebo, once daily (QD), in subjects with gout.
- Trial name
- APEX (NCT00174915)
- Testing
- Febuxostat, Allopurinol
- Volunteers
- 1,072 people
- Sponsor
- Takeda
Cystic Fibrosis
A Pharmacokinetic and Safety Study of IV Gallium Nitrate (Ganite) in Cystic Fibrosis Patients
The purpose of this research study is to test the pharmacokinetics, safety, and tolerability of an intravenous infusion of a drug called Ganite (gallium nitrate) in patients with cystic fibrosis.
- Trial name
- NCT01093521
- Testing
- 100 mg/m2 dose, 200 mg/m2 dose
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- University of Washington
Atopic Eczema/Dermatitis (Non-Specific)
Skin bioMARkers for Atopic Eczema Therapy Evaluation
The study aims to investigate two new non-invasive technologies for assessing skin properties to identify and validate a range of safety biomarkers that may be considered useful as primary outcome measures for evaluating the…
- Trial name
- SMART (NCT04194814)
- Testing
- crisaborole (2%) ointment, betamethasone valerate 0.1% cream
- Where
- United Kingdom
- Volunteers
- 37 people
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
Skin Laxity
Lifting and Tightening of the Face and Neck Following an Increased Density Treatment
The purpose of this study is to evaluate the Ulthera® System for obtaining lift and tightening of the cheek tissue, reduction of jowling, and improving jawline definition and/or submental skin laxity when increased density…
- Trial name
- NCT01519206
- Testing
- Ulthera System Treatment
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
Clubfoot
Clubfoot Tenotomy Trial
Infants undergoing Ponseti treatment for idiopathic clubfoot often require percutaneous tendoachilles lengthening (TAL) after serial casting.
- Trial name
- ClubTT (NCT04766684)
- Testing
- Jet injection of 1% lidocaine, 4% liposomal lidocaine cream
- Where
- United States
- Volunteers
- 107 people
- Sponsor
- Vanderbilt University Medical Center
Angiofibroma of Face, Tuberous Sclerosis
Topical Sirolimus Ointment for Cutaneous Angiofibromas in Subjects With Tuberous Sclerosis Complex
The objective of this study is to evaluate the safety and efficacy of sirolimus (0.2% and 0.4% formulations) and its vehicle when applied topically once daily for 12 weeks for the treatment of cutaneous angiofibromas in pediatric…
- Trial name
- NCT03363763
- Testing
- Sirolimus 0.2%, Sirolimus 0.4%
- Where
- 8 sites in United States, China
- Volunteers
- 24 people
- Sponsor
- Aucta Pharmaceuticals, Inc