Clinical trials: Brain Developmental Diseases
Infant, Small for Gestational Age
Follow-up to Adult Height of a Cohort of Subjects Born Small for the Gestational Age and Treated With Growth Hormone
This non-interventional study for prospective follow up of a cohort of 220 subjects born small for gestational age (SGA) is planned for the purpose of finding out if normalisation of adult height is associated or not with…
- Trial name
- SGA (NCT01196156)
- Where
- 28 sites in Spain
- Volunteers
- 443 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Developmental and Epileptic Encephalopathy
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)
This (DEEp OCEAN Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DEE.
- Trial name
- NCT06719141
- Testing
- LP352
- Where
- 116 sites in United States, Australia and 15 more
- Volunteers
- 367 people
- Sponsor
- Longboard Pharmaceuticals
Developmental and Epileptic Encephalopathy, Dravet Syndrome
Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
- Conditions
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- NCT05626634
- Testing
- LP352, bexicaserin
- Where
- 28 sites in United States, Australia
- Volunteers
- 41 people
- Sponsor
- Longboard Pharmaceuticals
Growth Hormone Deficiency
A Study To Evaluate The Dose Response And Safety Of PHA-794428 In Adults With Growth Hormone Deficiency
The purpose of this study is to explore the safety, toleration and dose response of PHA-794428 after multiple weekly injections in male and female growth hormone deficient patients.
- Trial name
- NCT00308464
- Testing
- PHA-794428
- Where
- 35 sites in Belgium, Czechia and 10 more
- Volunteers
- 136 people
- Sponsor
- Pfizer
Pituitary Disorders, Adult Growth Hormone Deficiency
Treatment of Adults With Growth Hormone Deficiency
The purpose of this study is to evaluate efficacy and safety profile of a new weekly administered growth hormone preparation compared with placebo in adults with growth hormone deficiency.
- Trial name
- NCT00294619
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 147 people
- Sponsor
- LG Life Sciences
Short Stature, Idiopathic, Infant, Small for Gestational Age
Evaluation of the Ease of Use, Preference, and Safety of EutropinPen Inj.
The purpose of this study is to assess the ease of use, preference, and safety after 8 weeks subcutaneous administration of EutropinPen Inj. in patients pretreated with recombinant human growth hormone by reusable device.
- Conditions
- Short Stature, Idiopathic, Infant, Small for Gestational Age, Growth Hormone Deficiency, Chronic Renal Failure
- Trial name
- NCT03015909
- Testing
- Somatropin
- Where
- South Korea
- Volunteers
- 116 people
- Sponsor
- LG Life Sciences
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1A), aged ≥48 months to <18 years (Part 1B), and…
- Trial name
- ENDEAVOR (NCT05419492)
- Testing
- ETX101
- Where
- 14 sites in United States, Australia and 1 more
- Volunteers
- 113 people
- Sponsor
- Encoded Therapeutics
Growth Hormone Disorder, Adult Growth Hormone Deficiency
A Trial to Evaluate the Safety of Once Weekly Dosing of Somapacitan (NNC0195-0092) and Daily Norditropin® FlexPro® for 52 Weeks in Previously Human Growth Hormone Treated Japanese Adults With Growth Hormone Deficiency
This trial is conducted in Asia. The aim of this trial is to evaluate the safety of once weekly dosing of somapacitan (NNC0195-0092) and daily Norditropin® FlexPro® for 52 weeks in previously human growth hormone treated Japanese…
- Trial name
- NCT03075644
- Testing
- somapacitan, Norditropin
- Where
- 12 sites in Japan
- Volunteers
- 62 people
- Sponsor
- Novo Nordisk A/S
15q Duplication Syndrome, CDKL5 Deficiency Disease
A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)
The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
- Trial name
- NCT03694275
- Testing
- Soticlestat
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Takeda
Growth Hormone Deficiency, Turner's Syndrome
Evaluation of the Adherence and the Patient Acceptability of Zomacton® Treatment With the Zomajet® Vision X Device
The study is performed to collect long-term data on the treatment adherence and patient's acceptability when Zomacton®10 mg is administered with the Zomajet® Vision X device in patients with a growth hormone deficiency or…
- Trial name
- ZOMAXEPT (NCT02018172)
- Testing
- somatropin
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Ferring Pharmaceuticals
Growth Hormone Deficiency
Comparative Validation of the Growth Hormone Releasing Hormone and Arginine Test for the Diagnosis of Adult Growth Hormone Deficiency
The aim of the study is to determine the specificity and sensitivity of the combined growth hormone releasing hormone (GHRH) + Arginine test in healthy volunteers, subjects with highly probable adult growth hormone deficiency…
- Trial name
- NCT01060488
- Testing
- GHRH+Arg, GHRH+Arg, ITT, ITT, ITT, GHRH+Arg.
- Where
- France
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Merck KGaA, Darmstadt, Germany
Carotid Artery Plaque, Arteriosclerosis
Atherosclerosis Underlying Development Assessed by Intima-Media Thickness in Patients on Rimonabant
Objectives: Primary: To evaluate the effect of rimonabant 20-mg once daily in comparison with placebo, on the quantitative progression of atherosclerosis as assessed by carotid artery intima-media thickness (CIMT) Secondary: To…
- Conditions
- Carotid Artery Plaque, Arteriosclerosis, Obesity, Metabolic Syndrome X
- Trial name
- AUDITOR (NCT00228176)
- Testing
- Rimonabant
- Where
- 6 sites in United States, Canada and 4 more
- Volunteers
- 661 people
- Sponsor
- Sanofi
Dravet Syndrome
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
This (DEEp SEA Study) is a double-blind, randomized, placebo-controlled, multicenter study to investigate the efficacy, safety, and tolerability of LP352 in the treatment of seizures in children and adults with DS.
- Trial name
- NCT06660394
- Testing
- LP352
- Where
- 105 sites in United States, Australia and 16 more
- Volunteers
- 108 people
- Sponsor
- Longboard Pharmaceuticals
Growth Hormone Deficiency Without Epiphyseal Closure
Ngenla Subcutaneous Injection Special Investigation
The purpose of this study is to learn about the long-term safety and effects of Ngenla. Ngenla is approved for treatment of GHD (Growth hormone deficiency) without epiphyseal closure under daily medical practice.
- Trial name
- NCT05602766
- Testing
- NGENLA (Somatrogon)
- Where
- Japan
- Volunteers
- 1 people
- Sponsor
- Pfizer
Pediatric Growth Hormone Deficiency
Versartis Long-Term Safety Study of Somavaratan
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration. Patients will be monitored for safety throughout their participation in the study.
- Trial name
- VISTA (NCT02068521)
- Testing
- somavaratan
- Volunteers
- 385 people
- Sponsor
- Versartis Inc.
Epilepsy, Dravet Syndrome (DS)
A Study of Soticlestat in Adults and Children With Rare Epilepsies
The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment. Participants will receive soticlestat twice a day.
- Conditions
- Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
- Trial name
- Endymion 1 (NCT03635073)
- Testing
- Soticlestat
- Where
- 59 sites in United States, Australia and 6 more
- Volunteers
- 156 people
- Sponsor
- Takeda
Lennox-Gastaut Syndrome
Study of Rufinamide in Pediatric Subjects 1 to Less Than 4 Years of Age With Lennox-Gastaut Syndrome Inadequately Controlled With Other Anti-epileptic Drugs
This study was designed to evaluate the cognitive effect, safety, and pharmacokinetics (PK) of rufinamide on Lennox-Gastaut Syndrome (LGS) inadequately controlled in pediatric participants already taking other anti-epileptic…
- Trial name
- NCT01405053
- Testing
- Rufinamide, Any other approved Antiepileptic Drug
- Where
- 47 sites in United States, Canada and 5 more
- Volunteers
- 37 people
- Sponsor
- Eisai Inc.
Growth Hormone Deficiency
A US Non-interventional, Effectiveness and Safety Study of Patients Treated With SKYTROFA
The goal of this study is to genrate evidence on long-term effectiveness and safety of SKYTROFA (lonapegsomatropin) in patients with growth hormone deficiency under routine clinical care
- Trial name
- SkybriGHt (NCT05820672)
- Testing
- No intervention
- Where
- 27 sites in United States
- Volunteers
- 900 people
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
Idiopathic Growth Hormone Deficiency, Turner Syndrome
First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study
PREDICT Validation is a validation pharmacogenetic trial. The purpose of this study is to confirm that some genes can be used to predict how well a subject diagnosed with idiopathic growth hormone deficiency (IGHD) or turner…
- Trial name
- NCT01419249
- Testing
- Blood sampling
- Where
- 31 sites in Argentina, Canada and 7 more
- Volunteers
- 458 people
- Sponsor
- Merck KGaA, Darmstadt, Germany