Clinical trials: Development Disorders
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia.
- Trial name
- teACH (NCT06732895)
- Testing
- Navepegritide
- Where
- 5 sites in Canada, Denmark and 3 more
- Volunteers
- 25 people
- Sponsor
- Ascendis Pharma A/S
Autoimmune Diseases, Inflammation
Prospective Study of White Blood Cells
The purpose of this study is to investigate white blood cells from volunteering donors to support the development of engineered regulatory T cell therapies.
- Conditions
- Autoimmune Diseases, Inflammation, Rejection; Transplant, Liver
- Trial name
- NCT04838171
- Testing
- White blood cell and blood collection
- Where
- United Kingdom
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Quell Therapeutics Limited
Basal Cell Carcinomas
Clinical Trial of Patidegib Gel 2%, 4%, and Vehicle Applied Once or Twice Daily to Decrease the GLI1 Biomarker in Sporadic Nodular Basal Cell Carcinomas
This is a double-blind, dose escalating, randomized, vehicle-controlled study designed to compare the efficacy and safety of patidegib gel 2% and 4% applied once or twice daily in comparison with that of vehicle in patients with…
- Trial name
- BCC (NCT02828111)
- Testing
- Patidegib, Vehicle gel
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- PellePharm, Inc.
Schizophrenia, Bipolar I Disorder
Open-Label, Flexible-dose Study to Evaluate the Long-Term Safety and Tolerability of Cariprazine in the Treatment of Pediatric Participants With Schizophrenia, Bipolar I Disorder, or Autism Spectrum Disorder
The purpose of this study is to evaluate the long-term safety and tolerability of cariprazine in the treatment of pediatric participants with schizophrenia, bipolar I disorder, or autism spectrum disorder (ASD) and to establish…
- Conditions
- Schizophrenia, Bipolar I Disorder, Autism Spectrum Disorder (ASD)
- Trial name
- 3070 Ped OLE (NCT04578756)
- Testing
- Cariprazine Flexible Dose
- Where
- 42 sites in United States, Puerto Rico
- Volunteers
- 310 people
- Sponsor
- AbbVie
Neurodevelopmental Disorders, Autism Spectrum Disorder
A Game-based Neurodevelopmental Assessment for Young Children
The aim of this study is to evaluate a novel tablet game-based neurodevelopmental assessment tool for young children aged 3 to 8 years old.
- Conditions
- Neurodevelopmental Disorders, Autism Spectrum Disorder, Attention Deficit Hyperactivity Disorder, Dyslexia
- Trial name
- BRIGHTEN (NCT05780853)
- Testing
- Tablet Game-based Assessment - YARN assessment service, Adaptive Behavior Assessment System, Third Edition (ABAS-3), Demographic questionnaire
- Where
- United Kingdom
- Volunteers
- 590 people, healthy volunteers welcome
- Sponsor
- Brightlobe Limited
Achondroplasia
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in future studies sponsored by BioMarin.
- Trial name
- NCT01603095
- Where
- 20 sites in United States, Australia and 4 more
- Volunteers
- 363 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
VIrtual STudy in Achondroplasia for the US (VISTA)
This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™.
- Trial name
- VISTA (NCT06168201)
- Where
- 8 sites in United States
- Volunteers
- 170 people
- Sponsor
- BioMarin Pharmaceutical
Cataract
Randomized Contralateral Clinical Trial With Single-piece (SN60WF) vs. Three Piece (MA60AC) AcrySof Intraocular Lenses (IOLs) on Development of Posterior Chamber Opacification (PCO).
Randomized contralateral clinical trial with single piece (Model SN60WF) vs. three piece (Model MA60AC) AcrySof Intraocular Lenses (IOLs) on development of Posterior Chamber Opacifiation (PCO).
- Trial name
- NCT00758745
- Testing
- Model SN60WF, Model MA60AC
- Where
- United States
- Volunteers
- 81 people
- Sponsor
- Alcon Research
Dwarfism
Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS
Phase 1: To evaluate the safety and efficacy of 0.05mg/kg/d of rhGH (Jintropin®) in the treatment of children with idiopathic short stature (ISS) in 52 weeks.
- Trial name
- NCT03635580
- Testing
- Negative control
- Where
- 9 sites in China
- Volunteers
- 480 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Non Small Cell Lung Cancer, Microbiome
BioForte Technology for in Silico Identification of Candidates for a New Microbiome-based Therapeutics and Diagnostics
The goal of the project is to develop and validate the BioForte technology. Its main functionality should be to in silico determine candidates for novel microbiome-based therapeutics and diagnostics.
- Conditions
- Non Small Cell Lung Cancer, Microbiome, Metagenome, Immunotherapy
- Trial name
- NCT04136470
- Testing
- Collection of stool, blood (PBMC) and biopsy (FFPE)
- Where
- 10 sites in Poland
- Volunteers
- 130 people
- Sponsor
- Ardigen
Myasthenia Gravis, Generalized
Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)
This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis
- Trial name
- NCT04146051
- Testing
- Descartes-08
- Where
- 15 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Cartesian Therapeutics
Dwarfism, Growth Hormone Deficiency
A Multicentre, Randomised, Open-label, Controlled Study to Evaluate the Effects of Saizen® on Cardiac Function in Growth Hormone Deficient(GHD) Subjects During the Transition Phase From Childhood to Adulthood
This was a 48-week, open-label, prospective, multicentric, randomised, comparative with parallel control, Phase 4 study to evaluate the effects of Saizen on cardiac function in GHD subjects during the transition phase from…
- Trial name
- NCT01157793
- Testing
- r-hGH
- Volunteers
- 34 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Macular Degeneration, Diabetic Retinopathy (DR)
Artificial Intelligence-assisted Diagnosis in Ophthalmology
This is a retrospective, multicenter, observational study designed to develop and validate an artificial intelligence (AI) system capable of detecting and classifying major ophthalmic diseases (glaucoma, cataract, diabetic…
- Conditions
- Macular Degeneration, Diabetic Retinopathy (DR), Glaucoma, Keratoconus
- Trial name
- AI-OPHTH-CR (NCT07497815)
- Testing
- No interventions
- Where
- 2 sites in Costa Rica
- Volunteers
- 15,000 people, healthy volunteers welcome
- Sponsor
- Marisse Masis-Solano
Diabetes Mellitus, Type II
Development and Exploration of the Effectiveness and Feasbility of a Digital Intervention for Type 2 Diabetes Mellitus
The study is conducted to assess potential effectiveness and feasibility of a comprehensive digital intervention for people with poorly-controlled Type 2 Diabetes Mellitus (T2DM), to explore the intervention effects of a combined…
- Trial name
- DEsireD (NCT05364476)
- Testing
- 16 weeks digital intervention
- Where
- Singapore
- Volunteers
- 122 people
- Sponsor
- EVYD Technology
Pneumococcal Disease, Invasive, Pneumococcal Disease
Serum Collection Study for Assay Development
The study will collect serum from 12 participants vaccinated with Gamma-PN to assist with assay development and validation
- Trial name
- NCT07205289
- Testing
- Gamma-PN
- Where
- Australia
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- GPN Vaccines
GHD
A Study to Investigate Efficacy and Safety of Weekly PEG-somatropin (GenSci004) in Treatment Naive Children With Growth Hormone Deficiency
The purpose of this study is to evaluate the efficacy and safety of weekly GenSci004 compared with daily Genotropin in treatment-naive children with growth failure due to GHD.
- Trial name
- ELEVATE (NCT06007417)
- Testing
- GenSci004, Genotropin
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Achondroplasia
Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to…
- Trial name
- NCT05116046
- Testing
- Recifercept
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Pfizer
ADHD, Autism
Acceptability, Feasibility, and Development of an AI Driven, Multi-sensor, Non-invasive Digital Mental Health Platform in Children With ADHD and Emotional Symptoms
This is an observational study collecting data using a wearable to measure the biometric features in youth with Attention deficit hyperactivity disorder(ADHD) and Autism.
- Trial name
- NCT06311370
- Testing
- Observation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- MaxisHealth
Birth Asphyxia
Asphyxia Associated Metabolite Biomarker Investigation 2
Follow-up of participants of AAMBI1 study at age of at least 2 years. AAMBI1(ClinicalTrials.gov ID: NCT03354208): Verification of biomarkers in a human population for their ability to diagnose the severity of neonatal asphyxia.
- Trial name
- AAMBI2 (NCT04714502)
- Where
- 4 sites in Turkey (Türkiye)
- Volunteers
- 144 people
- Sponsor
- InfanDx AG
Achondroplasia
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
- Trial name
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 people
- Sponsor
- BioMarin Pharmaceutical
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