Clinical trials: Newborn Diseases
Urea Cycle Disorders
Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
- Trial name
- NCT02489292
- Testing
- HepaStem
- Where
- 6 sites in Belgium, France and 2 more
- Volunteers
- 5 people
- Sponsor
- Cellaion SA
Down Syndrome-Associated Alzheimer's Disease (DS-AD)
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran.
- Trial name
- NCT07636811
- Testing
- Mivelsiran
- Where
- 10 sites in United States, Spain
- Volunteers
- 58 people
- Sponsor
- Alnylam Pharmaceuticals
Alpha1-Antitrypsin Deficiency
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran…
- Trial name
- NCT05677971
- Testing
- Fazirsiran Injection
- Where
- 89 sites in United States, Australia and 17 more
- Volunteers
- 160 people
- Sponsor
- Takeda
Moderate-to-severe Atopic Dermatitis
Subcutaneously CM310/Placebo in Patients With Moderate-to-Severe Atopic Dermatitis (AD)
This is a multi-center, randomized, double blind, placebo-controlled phase IIb study to evaluate the efficacy, safety, PK, PD and immunogenicity of CM310 in moderate-severe AD subjects.
- Trial name
- NCT04805411
- Testing
- CM310
- Where
- 25 sites in China
- Volunteers
- 120 people
- Sponsor
- Keymed Biosciences Co.Ltd
Acquired Bleeding Disorder, Acquired Haemophilia
Bioequivalence of NovoSeven® and a NovoSeven® Formulation Stable at Room Temperature in Healthy Male Subjects
This trial is conducted in Europe. The aim of this trial is to demonstrate bio-equivalence between the marketed activated recombinant human factor VII (NovoSeven®) (CP-rFVIIa) and a new formulation stable at 25°C (VII25).
- Conditions
- Acquired Bleeding Disorder, Acquired Haemophilia, Congenital Bleeding Disorder, Congenital FVII Deficiency
- Trial name
- NCT01561417
- Testing
- activated recombinant human factor VII
- Where
- France
- Volunteers
- 28 people
- Sponsor
- Novo Nordisk A/S
Dermatitis Atopic
Observational Study of Patients Receiving Dupixent® for Atopic Dermatitis (AD)
Primary Objective: To characterize the patients who receive Dupixent® (dupilumab) for AD in a real-world setting, with respect to their medical history, socio-demographic and disease characteristics, and prior and concomitant…
- Trial name
- GLOBOSTAD (NCT03992417)
- Testing
- Dupilumab SAR231893 (REGN668)
- Where
- 105 sites in Argentina, Australia and 20 more
- Volunteers
- 955 people
- Sponsor
- Sanofi
Hypothalamic Obesity
A Study of Octreotide Depot vs Saline Control in Pediatric Hypothalamic Obesity Patients
The extension protocol is designed to allow those patients randomized to placebo in the core portion of the protocol to receive a 6 month treatment of open label octreotide and allow those patients randomized to octreotide who…
- Trial name
- NCT00171613
- Testing
- Octreotide
- Volunteers
- 32 people
- Sponsor
- Novartis
Refractive Errors
Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact Lens
The objective of this clinical investigation is to collect valid scientific safety and effectiveness data on the Acuity 200 (fluoroxyfocon A) Rigid Gas Permeable Contact Lens, manufactured from a newly developed rigid gas…
- Trial name
- Acuity 200 (NCT03971227)
- Testing
- Acuity 200 (Fluoroxyfocon A) contact lens, Acuity 100 (Hexafocon A) contact lens
- Where
- 3 sites in United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- Acuity Polymers, Inc.
Atopic Dermatitis
Correlation Study Between PEST and SCORAD in Management of Atopic Dermatitis With Ceradan® Regimen
This is a prospective, open label, single arm, and observational and multicenter study to assess the correlation between PEST and SCORAD scores in the management of AD with the Ceradan® regimen.
- Trial name
- COPES (NCT02073591)
- Testing
- Ceradan Cream, Ceradan Wash
- Where
- 2 sites in Singapore
- Volunteers
- 58 people
- Sponsor
- Hyphens Pharma Pte Ltd
Atopic Dermatitis
Open-Label Study of RVT-501 Topical Ointment in Pediatric Patients With Atopic Dermatitis
This is a multicenter, open-label Phase 1b study in pediatric patients age 2-11 years old with extensive atopic dermatitis.
- Trial name
- NCT03415282
- Testing
- RVT-501 0.5% topical ointment
- Where
- 12 sites in United States
- Volunteers
- 24 people
- Sponsor
- Dermavant Sciences GmbH
Osteogenesis Imperfecta
Bisphosphonate Treatment of Osteogenesis Imperfecta
The primary purpose of this trial is to evaluate whether the investigational medication is safe, effective and has the ability to increase spine bone density in osteogenesis imperfecta (OI) patients.
- Trial name
- NCT00063479
- Testing
- Zoledronic Acid
- Where
- 10 sites in United States
- Volunteers
- 158 people
- Sponsor
- Novartis Pharmaceuticals
Osteoarthritis, Hip, Rheumatoid Arthritis
Exception Cementless Hip Stem
This study is a multicenter, retrospective and prospective, non-controlled, non-randomized post market clinical follow-up study.
- Conditions
- Osteoarthritis, Hip, Rheumatoid Arthritis, Inflammatory Arthritis, Congenital Hip Dysplasia
- Trial name
- Exception (NCT04349046)
- Testing
- Total hip arthoplasty (THA)
- Where
- 6 sites in Belgium, France
- Volunteers
- 332 people
- Sponsor
- Zimmer Biomet
Hemophilia A
Relative Bioavailability Of Two Formulations Of Moroctocog Alfa (AF-CC)
This study is being conducted to compare how moroctocog alfa (AF-CC) acts in the body when administered as 2 different dose presentations.
- Trial name
- NCT01579903
- Testing
- moroctocog alfa (AF-CC)
- Where
- 3 sites in Bulgaria, Hungary and 1 more
- Volunteers
- 16 people
- Sponsor
- Pfizer
Hemophilia B
A Safety and Efficacy Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
This study will examine the safety, pharmacokinetics and efficacy of rIX-FP for the control and prevention of bleeding episodes in subjects who have previously received factor replacement therapy for hemophilia B.
- Trial name
- NCT01496274
- Testing
- rIX-FP
- Where
- 30 sites in United States, Austria and 8 more
- Volunteers
- 63 people
- Sponsor
- CSL Behring
Cystic Fibrosis
Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
The purpose of this study was to evaluate the safety and efficacy of aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).
- Trial name
- AIR-CF2 (NCT00104520)
- Testing
- AZLI 75 mg two times a day (BID)/three times a day (TID)
- Where
- 56 sites in United States
- Volunteers
- 211 people
- Sponsor
- Gilead Sciences
Hypothalamic Obesity, Multiple Pituitary Hormone Deficiency Genetic Form
A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)
This is a sub-study of Study RM-493-040 (NCT05774756). The goal of this sub-study is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with…
- Conditions
- Hypothalamic Obesity, Multiple Pituitary Hormone Deficiency Genetic Form, Septo-Optic Dysplasia, Optic Nerve Hypoplasia
- Trial name
- NCT06760546
- Testing
- Setmelanotide
- Where
- 11 sites in United States, United Kingdom
- Volunteers
- 39 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Dystrophic Epidermolysis Bullosa
Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa
This is a phase I/II open-label study to evaluate the efficacy and safety of ALLO-ASC-DFU in patients with Dystrophic Epidermolysis Bullosa.
- Trial name
- NCT02579369
- Testing
- Allogeneic mesenchymal stem cells, Polyurethene Film
- Where
- South Korea
- Volunteers
- 2 people
- Sponsor
- Anterogen Co., Ltd.
Gout
The Drug-Drug Interaction of SHR4640, Febuxostat and Colchicine in Patients With Gout
The objective of the study is to assess the pharmacodynamic properties of drug-drug of SHR4640, Febuxostat and Colchicine interaction in patients with gout.
- Trial name
- NCT03131583
- Testing
- Colchicine, Febuxostat, SHR4640
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Hunter Syndrome
Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®
This study is being conducted to identify pediatric patients with Hunter syndrome who have neurodevelopmental disease characteristics, who are currently receiving treatment with Elaprase, and who may be suitable to participate in…
- Trial name
- NCT00937794
- Testing
- Neurobehavioral testing, Visual and auditory assessments
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Shire
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD).
- Trial name
- NCT05154851
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
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