Clinical trials: Brain Developmental Diseases
- Condition Brain Developmental Diseases
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Growth Hormone Deficiency
Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)
This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD) and investigate a predictive enrichment marker (PEM) strategy to select subjects…
- Trial name
- OraGrowtH210 (NCT04614337)
- Testing
- LUM-201, rhGH Norditropin® pen (34 µg/kg)
- Where
- 43 sites in United States, Australia and 4 more
- Volunteers
- 104 people
- Sponsor
- Lumos Pharma
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
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
Growth Hormone Deficiency
Phase III Clinical Study of Pegylated Somatropin (PEG Somatropin) to Treat Growth Hormone Deficiency Children
This was a multicenter, randomized, open-label, parallel controlled phase III study, compared pegylated somatropin with Jintropin AQ (somatropin liquid injection, daily administration formulation).
- Trial name
- NCT01495468
- Testing
- PEG-somatropin
- Where
- 6 sites in China
- Volunteers
- 343 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Growth Hormone Deficiency
Clinical Study of Pegylated Somatropin to Treat Children Growth Hormone Deficiency
To evaluate the safety and efficacy of PEG Somatropin Injection (Jintrolong®) in the treatment of short stature due to endogenous growth hormone deficiency (GHD) in the broad of population of children.
- Trial name
- NCT02908958
- Testing
- PEG-somatropin
- Where
- 22 sites in China
- Volunteers
- 900 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Growth Hormone Deficiency, Growth Retardation
Treatment With Recombinant Human Growth Hormone Genotonorm (Registered) In Children With Short Stature Secondary
To assess the effect of long-term treatment by Genotonorm on linear growth
- Trial name
- NCT00174278
- Testing
- Somatropin
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Pfizer
Growth Hormone Deficiency
Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)
This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the…
- Trial name
- OraGrowtH213 (NCT05250063)
- Testing
- LUM-201
- Where
- 10 sites in United States, Poland
- Volunteers
- 15 people
- Sponsor
- Lumos Pharma
Dravet Syndrome
Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies
This is a multicenter, prospective, 2-year observational study in infants and children with developmental and epileptic encephalopathies (DEEs). The DEE currently being investigated is SCN1A-positive Dravet Syndrome.
- Trial name
- ENVISION (NCT04537832)
- Testing
- No Intervention
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 58 people
- Sponsor
- Encoded Therapeutics
Growth Hormone Deficiency
Easypod United States User Trial
This study will help to determine whether an injection log on a recombinant human growth hormone (rhGH) delivery device improves subjects' adherence with injection schedules.
- Trial name
- NCT00689260
- Testing
- easypod
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- EMD Serono
Dwarfism, Pituitary
Predictive Markers in Chinese Growth Hormone Deficiency (GHD) Children Treated With Saizen®
This is an open-label, prospective, multicentric, non-comparative, non-randomized Phase IV interventional study in which subjects pre-diagnosed with Growth Hormone Deficiency (GHD) were treated for 4 weeks with Saizen to compare…
- Trial name
- NCT01187550
- Testing
- Recombinant human growth hormone (r-hGH)
- Volunteers
- 214 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Pediatric Growth Hormone Deficiency
A Study to Compare the Experiences of Taking Daily Growth Hormone Injections to Weekly Ngenla in Children With Low Levels of Growth Hormone
The purpose of this study is to learn about: how children stick to taking their injections their experience with the study medicines (Ngenla and daily growth hormone) prescribed to children with low levels of growth hormone.
- Trial name
- NCT06113952
- Testing
- Sharps bin to collect used needles/injections
- Sponsor
- Pfizer
Adult Growth Hormone Deficiency
Post-Marketing Surveillance (Special Use-results Surveillance on Long-term Use) With Sogroya®
Participants are invited to take part in this study because they have AGHD (only severe case). The purpose of this study is to assess long term safety and effectiveness of Sogroya® in patients with AGHD (only severe case) under…
- Trial name
- NCT05230550
- Testing
- Somapacitan
- Where
- 47 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Novo Nordisk A/S
Brain Concussion, Mild Cognitive Impairment
Development of a Neurocognitive Screening Test
This research study will evaluate a standalone software application and is designed to standardize and validate new neurocognitive screening testing for children aged 6 through 11, and adolescents and adults ages 12 through 75.
- Trial name
- NCT02597504
- Testing
- Pen and paper neuropsychological test, Quick Test
- Where
- 4 sites in United States
- Volunteers
- 1,600 people, healthy volunteers welcome
- Sponsor
- ImPACT Applications, Inc.
Dwarfism, Growth Hormone Deficiency
Drug Use Investigation of Somatropin for GHD-ADULTS.
Post marketing drug use investigation of Genotropin for GHD-ADULTS.
- Trial name
- NCT00601419
- Testing
- Somatropin
- Volunteers
- 230 people
- Sponsor
- Pfizer
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
Growth Hormone Deficiency
Practicability and Acceptability of Stylomax® in Children
Primary objective: to evaluate the practicability and acceptability of STYLOMAX®, a new injection device for MAXOMAT®, in children, for 1 year.
- Trial name
- NCT00476385
- Testing
- somatropine
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Sanofi
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
Growth Hormone Deficiency
Patient Perception of Treatment Burden in Weekly Versus Daily Growth Hormone Injections in Children With GHD
This is an open label randomized 24 week crossover trial assessing the treatment burden of a weekly growth hormone injection regimen (somatrogon) compared to a daily growth hormone injection regimen (Genotropin).
- Trial name
- NCT03831880
- Testing
- Genotropin, somatrogon
- Where
- 32 sites in United States, Bulgaria and 3 more
- Volunteers
- 87 people
- Sponsor
- Pfizer
Pediatric Growth Hormone Deficiency
Versartis Trial in Children to Assess Long-Acting Growth Hormone
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317.
- Trial name
- VERTICAL (NCT01718041)
- Testing
- Experimental: VRS-317
- Where
- 23 sites in United States
- Volunteers
- 64 people
- Sponsor
- Versartis Inc.
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)
WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 to <84 months. The study follows an open-label, dose-escalation design.
- Trial name
- NCT06112275
- Testing
- ETX101
- Where
- Australia
- Volunteers
- 4 people
- Sponsor
- Encoded Therapeutics