Clinical trials: Brain Developmental Diseases
Growth Hormone Deficiency
Sustained Release Formulation of Somatropin (rDNA Origin)for Injection
Annualised height velocity after 12/24 months treatment and HV SDS height velocity after 12/24 months treatment expressed as number of standard deviations difference from the mean population height velocity for the appropriate…
- Trial name
- NCT00600808
- Testing
- BPLG-003
- Volunteers
- 51 people
- Sponsor
- BioPartners GmbH
Autism Spectrum Disorder
Brain Hero® Neurofeedback Training (With the Device Brainhero 2019, Type 201)
The study investigates the feasibility and the effects of training at home with personalised neurofeedback in children with an autism spectrum diagnosis and comorbidities.
- Trial name
- NCT05444816
- Testing
- Personalised Neurofeedback training at home
- Where
- 2 sites in Austria
- Volunteers
- 21 people
- Sponsor
- Brainhero GmbH
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- PACIFIC (NCT05364021)
- Testing
- LP352
- Where
- 34 sites in United States, Australia
- Volunteers
- 52 people
- Sponsor
- Longboard Pharmaceuticals
Glycogen Storage Disease Type II
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to ERT with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769…
- Trial name
- NCT05164055
- Testing
- Avalglucosidase alfa (GZ402666)
- Where
- 11 sites in France
- Volunteers
- 17 people
- Sponsor
- Genzyme, a Sanofi Company
Parkinson Disease
DTx Algorithms for Personalized Parkinson's Disease Treatment and Medication Plan Optimization
The study is aimed at developing Digital Therapeutics (DTx) algorithms for personalized PD treatment and medication plan optimization, based on Real World Data (RWD) collected from patients via digital mobile app and wearable…
- Trial name
- NCT05904288
- Testing
- Smartwatch Garmin Vivosmart [wearable sensor], Soturi™ [digital mobile app], Clinical Assessment
- Where
- 3 sites in Italy
- Volunteers
- 54 people
- Sponsor
- Newel Health SRL
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome
Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants < 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet…
- Conditions
- Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
- Trial name
- NCT04485104
- Testing
- GWP42003-P
- Where
- 11 sites in United States, Italy and 1 more
- Volunteers
- 3 people
- Sponsor
- Jazz Pharmaceuticals
Growth Hormone Deficiency
Safety and Efficacy of Y-shape Pegylated Somatropin in Growth Hormone Deficiency Children
This is a multicenter, randomized, open-labeled, positive controlled phase 2\&3 combined study to evaluate the safety and efficacy of weekly Y-shape pegylated somatropin, compared to daily somatropin (Norditropin®), in…
- Trial name
- NCT04513171
- Testing
- Y-shape pegylated somatropin, Norditropin®, Norditropin
- Where
- China
- Volunteers
- 434 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Alzheimer Disease, Alzheimer Dementia
Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
The purpose of the study is to evaluate the use of autologous Bone Marrow Derived Stem Cells (BMSC) as a means to improve cognitive impairment as occurs in Alzheimer's Disease and other dementias and to improve behavior and…
- Conditions
- Alzheimer Disease, Alzheimer Dementia, Vascular Dementia, Lewy Body Disease
- Trial name
- ACIST (NCT03724136)
- Testing
- Intravenous Bone Marrow Stem Cell (BMSC) Fraction, Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction, Near Infrared Light
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 100 people
- Sponsor
- MD Stem Cells
Growth Hormone Disorder, Adult Growth Hormone Deficiency
A Trial Investigating the Safety, Tolerability, Availability and Distribution in the Body of Once-weekly Long-acting Growth Hormone (Somapacitan) Compared to Once Daily Norditropin NordiFlex® in Adults With Growth Hormone Deficiency
This trial is conducted in Europe. The aim of the trial is to investigate the safety, tolerability, availability and distribution in the body of once-weekly long-acting growth hormone (NNC0195-0092, somapacitan) compared to once…
- Trial name
- NCT01706783
- Testing
- somapacitan, Norditropin NordiFlex®
- Where
- 4 sites in Denmark, Sweden
- Volunteers
- 35 people
- Sponsor
- Novo Nordisk A/S
Growth Hormone Deficiency
Online Surveillance of Treatment of Children With Growth Hormone Deficiency With ZOMACTON
The purpose of this study is to investigate long-term treatment with Zomacton® for pituitary short stature in children with insufficient growth hormone production and/or short stature caused by Turner syndrome.
- Trial name
- NCT01365351
- Testing
- Growth hormone
- Where
- 59 sites in Germany
- Volunteers
- 1,034 people
- Sponsor
- Ferring Pharmaceuticals
Dwarfism, Pituitary
A Study to Learn About Somatrogon in Patients With Pediatric Growth Hormone Deficiency (p GHD) in India.
The purpose of this study is to learn about the safety of Somatrogon for the treatment of pediatric growth hormone deficiency (p GHD) in India.
- Trial name
- NCT06587035
- Testing
- Somatrogon
- Where
- 4 sites in India
- Volunteers
- 50 people
- Sponsor
- Pfizer
Alzheimer s Disease, MCI Conversion to Dementia
Development of a Home Test for Measuring Blood P-tau217 in Alzheimer's Disease Using the Tasso Lancet Device
This study compares p-tau217 concentrations in blood collected via the Tasso device versus standard venipuncture in subjects with and without Alzheimer's disease, including a subset with confirmed amyloid pathology.
- Conditions
- Alzheimer s Disease, MCI Conversion to Dementia, pTau217, Biomarkers
- Trial name
- NCT07701161
- Where
- 5 sites in United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Neurogen Biomarking LLC
Growth Hormone Deficiency
Surveillance of Treatment of Children With Growth Hormone Deficiency With Zomacton®
The purpose of this study is to investigate the long-term treatment with Zomacton® for pituitary short stature in children with insufficient growth hormone production and/or short stature caused by Turner's syndrome.
- Trial name
- NCT01731028
- Testing
- Somatropin
- Sponsor
- Ferring Pharmaceuticals
SCN8A Developmental and Epileptic Encephalopathy Syndrome
Extension Study to Evaluate How Safe and Tolerable NBI-921352 is as an Adjunctive Therapy for Participants With SCN8A-DEE
Extension study to evaluate how safe and tolerable the drug NBI-921352 is when used as adjunctive therapy in participants with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE).
- Trial name
- NCT05226780
- Testing
- NBI-921352
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Neurocrine Biosciences
Child Development, Child Development Disorder
Digital Epidemiology Study on Brain Functioning
Neurobiology has improved the integrative understanding of brain development. With digital technologies, digital (bio)markers would be relevant variables for a better understanding of the heterogeneity of cognitive and behavioral…
- Trial name
- EPIDIA4Kids (NCT05577533)
- Testing
- O-Kidia game battery, O-Brain
- Where
- 2 sites in France
- Volunteers
- 736 people, healthy volunteers welcome
- Sponsor
- O-Kidia
Alzheimer Disease, Early Onset
Ryan Companion Robot for Assisting Elderly People With Alzheimer's Disease
The objective of this project is to conduct research and a pilot study to demonstrate the potential clinical impact and technical feasibility of a socially-assistive robot, called Ryan Companionbot (hereafter Ryan), for life…
- Trial name
- NCT05052866
- Testing
- Social Robot
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- DreamFace Technologies, LLC
Growth Hormone Deficiency
Pegylated Somatropin (PEG Somatropin) in the Treatment of Children With Growth Hormone Deficiency
To evaluate the safety and efficacy of PEG Somatropin Injection in the treatment of children with growth hormone deficiency, as well as to study the feasibility of extending the dosing intervals (once per two weeks) of PEG…
- Trial name
- NCT02976675
- Testing
- PEG-somatropin
- Where
- 24 sites in China
- Volunteers
- 600 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Adult Growth Hormone Deficiency
Extension Study on Safety of Long-Term Growth Hormone Replacement in Adult Patients With Growth Hormone Deficiency
To evaluate long-term safety of growth hormone replacement in adult patients with growth hormone deficiency
- Trial name
- NCT00191360
- Testing
- Somatropin
- Where
- 14 sites in Japan
- Volunteers
- 68 people
- Sponsor
- Eli Lilly and Company
Wilson Disease
Establishment of Human Cellular Disease Models for Wilson Disease
Establishment of human cellular disease models for Wilson disease for an individualized therapy develop-ment having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSWILSON (NCT03867526)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Adult Growth Hormone Deficiency
A Study to Follow Patients With Adult Growth Hormone Deficiency (AGHD) Treated With Sogroya® for Long Term Safety Information
In this study, the general long-term safety and effectiveness of Sogroya (somapacitan) in adults with growth hormone deficiency (AGHD) being treated per normal clinical practice is looked into.
- Trial name
- NCT05718570
- Testing
- Somapacitan
- Where
- 55 sites in United States, France and 4 more
- Volunteers
- 400 people
- Sponsor
- Novo Nordisk A/S