Clinical trials: Development, Human, open to volunteers
Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children
GB06 Phase III Trial for Pediatric Growth Hormone Deficiency
This study aims to evaluate the efficacy and safety of GB06 (a biosimilar of Norditropin®FlexProTM from Novo Nordisk) for the treatment of growth disorders caused by growth hormone deficiency (GHD) in children.
- Conditions
- Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children, Growth Hormone Deficiency, Pediatric
- Trial name
- NCT07553351
- Testing
- GB06, Norditropin®FlexProTM
- Where
- China
- Volunteers
- 192 people
- Sponsor
- Kexing Biopharm Co., Ltd.
Autism Spectrum Disorder
A Study of Mesenchymal Stem Cell in the Treatment of Autism Spectrum Disorder
This clinical trial aims to evaluate the safety and preliminary efficacy of intravenous administration of human bone marrow-derived mesenchymal stem cells (hBMMSCs) in patients with autism spectrum disorder (ASD).
- Trial name
- ASD (NCT07177677)
- Testing
- hBMMSC
- Volunteers
- 42 people
- Sponsor
- Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.
Fetal Developmental Abnormality, Fetal Development
Scottish Advanced Fetal Research Study
In-utero exposure to drugs and chemicals through maternal smoking, alcohol use, drug abuse, prescription medicines and occupational/lifestyle exposures is widespread.
- Conditions
- Fetal Developmental Abnormality, Fetal Development, Human, Life Style
- Trial name
- SAFeR (NCT04613583)
- Testing
- SAFeR fetuses
- Where
- United Kingdom
- Volunteers
- 1,200 people, healthy volunteers welcome
- Sponsor
- University of Aberdeen
Charcot Marie Tooth Disease, DIFGAN
Genotype/Phenotype Correlation of MORC2 Mutations
The Microrchidia CW-type zinc finger 2 (MORC2) gene encodes a protein expressed in all tissues and enriched in the brain. It is involved in Charcot-Marie-Tooth disease, with mire than 30 families presenting MORC2 mutations.
- Conditions
- Charcot Marie Tooth Disease, DIFGAN, Developmental Delay (Disorder), Impaired Growth
- Trial name
- PhenoMORC2 (NCT07038239)
- Testing
- Skin biopsy, Blood sample
- Where
- 12 sites in France
- Volunteers
- 45 people
- Sponsor
- Hospices Civils de Lyon
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines.
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of
- Trial name
- NCT06092346
- Where
- United States
- Volunteers
- 999 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Human Body Composition, Human Body Function
Development and Validation of Multi-nuclear Magnetic Resonance Imaging and Spectroscopy for Whole Body Human Applications
The purpose of this study is to develop and refine the techniques for using magnetic resonance imaging and magnetic resonance spectroscopy to understand the composition and function of the human body
- Trial name
- MRS VALIDATION (NCT02663362)
- Testing
- To evaluate the reproducibility of acquiring multi-nuclear data on a new 3T Philips Magnet
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- AdventHealth Translational Research Institute
Pediatric
The Roles of Human Microbiome and Vitamin D in the Development of Childhood Allergic Diseases
A birth cohort study to evaluate the role of human microbiome and vitamin D in the development of allergic diseases in young child before one year of age.
- Trial name
- NCT05670262
- Where
- Taiwan
- Volunteers
- 6,000 people, healthy volunteers welcome
- Sponsor
- China Medical University Hospital
Leigh Syndrome (Maternally Inherited, MILS), Leigh Syndrome (AR, AD, XR)
Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
In this project, the investigators are using iPSC lines derived from patients with Leigh syndrome that carry mutations in the mitochondrial (mtDNA) and in the nuclear DNA (nDNA) to reprogram them into neural progenitor cells and…
- Trial name
- cureMILS (NCT06967831)
- Testing
- skin biopsy, generation of iPSCs, blood drawing
- Where
- 2 sites in Germany
- Volunteers
- 80 people
- Sponsor
- Charite University, Berlin, Germany
Very Low Birth Weight Baby, Early Nutrition and the Preterm Infant
Optimizing Nutrition and Milk (Opti-NuM) Project
Early nutrition critically influences growth, neurodevelopment and morbidity among infants born of very low birth weight (VLBW), but current one-size-fits-all feeding regimes do not optimally support these vulnerable infants.
- Conditions
- Very Low Birth Weight Baby, Early Nutrition and the Preterm Infant, Nutritional Requirements, Human Milk Fortification
- Trial name
- Opti-NuM (NCT06870981)
- Testing
- Opti-NuM is an observational secondary use of data/samples study, the investigators will analyze information and specimens from the MaxiMoM platform RCTs. No interventions form part of this study.
- Where
- 6 sites in United States, Canada
- Volunteers
- 1,100 people
- Sponsor
- The Hospital for Sick Children
Breastfeeding, Exclusive
Synergistic Activity of Human Milk Nutrients and Infant Cognition
Purpose: To establish a whole food, egg, as a viable study material to supplement mothers and infants with nutrients that support optimal brain development. There will be 84 breastfeeding dyads, 3 months postnatal.
- Trial name
- NCT03838536
- Testing
- Whole Egg Powder, Egg White Powder
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Autism Spectrum Disorder (ASD, Prospective Study
Prospective Clinical Study on Human Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes for the Treatment of Childhood Autism
This clinical study aims to evaluate whether a nasal spray containing exosomes derived from human umbilical cord mesenchymal stem cells (hUC-MSC-EXOs) can safely and effectively improve core symptoms in children aged 3-7 years…
- Trial name
- EXO-ASD (NCT07243561)
- Testing
- hUC-MSC-Exos Nasal Spray
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Dongfang People's Hospital
Growth Hormone Deficiency
A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
- Trial name
- SkyPASS (NCT05775523)
- Testing
- No intervention
- Where
- 27 sites in United States
- Volunteers
- 500 people
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
Craniosynostoses
Burosumab and 1-25 (OH) Vitamin D on Human Osteoblasts
FGF23 is the cornerstone of phosphate / calcium / vitamin D metabolism: it is synthesized mainly by osteocytes and acts as a phosphaturizing agent, inhibitor of dihydroxyvitamin D, and inhibitor of synthesis and secretion of PTH…
- Trial name
- HYPO-BLASTES (NCT04159675)
- Testing
- osteoblast biology study
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hospices Civils de Lyon
Human Development, Genetic Disease
Sanger Human Cell Atlasing Project
"Cell Atlasing" refers to a novel strategy to characterise cells in tissues at the molecular level in a quantitative manner. The international Human Cell Atlas consortium brings together a community of biologists, clinicians…
- Trial name
- HCA (NCT06497673)
- Testing
- sample collection
- Where
- United Kingdom
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- The Wellcome Sanger Institute
Neurodevelopmental Disorders, Developmental Delay
Development of Motility and Cognition in Infants
PILKE study uses wearables for assessing motor development in infants in order to define functional growth trajectories in the normal infants and infants at risk of neurological compromise.
- Conditions
- Neurodevelopmental Disorders, Developmental Delay, Development, Infant, Child Development
- Trial name
- PILKE (NCT05527080)
- Testing
- physiotherapy, brace
- Where
- Finland
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Helsinki University Central Hospital
Atopic Dermatitis (AD), Autoantibody
Birth Cohort: Development of IgE Autoantibodies in Newborns With (High Risk of) Atopic Dermatitis
Previous research has shown that some patients with atopic eczema have specific self-reactive antibodies, known as IgE autoantibodies, that react to their own skin cells, referred to as "self-reactive antibodies" or…
- Conditions
- Atopic Dermatitis (AD), Autoantibody, Auto-Immunity, Microbiome, Human
- Trial name
- DIANA (NCT07316465)
- Testing
- Development of atopic dermatitis with/without IgE autoantibodies
- Where
- Belgium
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Universitair Ziekenhuis Brussel
HIV, HIV - Human Immunodeficiency Virus
Intervention to Reduce HIV Related Stigma Among Pregnant and Postpartum Women
Purpose of this Study: The main purpose of this study is to adapt an existing evidence-based intervention, Project Accept Post-Test Support Services (PTSS) Module 3, specifically for pregnant and postpartum women living with HIV…
- Conditions
- HIV, HIV - Human Immunodeficiency Virus, Postpartum, Pregnancy
- Trial name
- WAT (NCT07064928)
- Testing
- Adapted Project Accept's Post-Test Support Services (PTSS) Module 3
- Where
- Ghana
- Volunteers
- 163 people
- Sponsor
- University of California, San Francisco
Epileptic Encephalopathy
A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy
Study CL1-230815-001 (KANDLE) is a Phase Ib/II, First In Human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamic (PD) effect of S230815 in…
- Trial name
- KANDLE (NCT07227857)
- Testing
- S230815- Starting dose A, S230815- Dose B, S230815- Dose C
- Where
- 15 sites in United States, France and 3 more
- Volunteers
- 20 people
- Sponsor
- Institut de Recherches Internationales Servier
Brain Malformation, Neuronal Migration Disorder
Human Epilepsy Genetics--Neuronal Migration Disorders Study
The purpose of this study is to identify genes responsible for epilepsy, brain malformations and disorders of human cognition.
- Conditions
- Brain Malformation, Neuronal Migration Disorder, Cognition Disorder, Epilepsy
- Trial name
- NCT00041600
- Where
- United States
- Volunteers
- 3,500 people
- Sponsor
- Harvard University Faculty of Medicine
Spasticity, Muscle, Microcephaly
Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)
MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss.
- Conditions
- Spasticity, Muscle, Microcephaly, Intellectual Deficiency, Growth Retardation
- Trial name
- NCT05518188
- Testing
- MELPIDA
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Elpida Therapeutics SPC