Clinical trials: Development Disorders
Idiopathic Short Stature, Small for Gestational Age
A Trial of YPEG-rhGH in Children With Short Stature
To explore the dose-response relationship between pharmacokinetics and pharmacodynamics of Y- Shaped Pegylated growth hormone injection (YPEG-GH) in children with short stature (idiopathic short stature (ISS), small for…
- Conditions
- Idiopathic Short Stature, Small for Gestational Age, Turner Syndrome
- Trial name
- NCT05838885
- Testing
- YPEG-rhGH, rhGH
- Where
- 19 sites in China
- Volunteers
- 78 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Heart Failure
CardioMEMS HF System Coverage With Evidence Development Study
The purpose of the CardioMEMS CED Study is to determine if pulmonary artery (PA) pressure-guided heart failure management using the CardioMEMS™ HF System improves long-term health outcomes in heart failure (HF) patients in the…
- Trial name
- NCT06779552
- Testing
- CardioMEMS HF System
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Abbott Medical Devices
Multiple Myeloma
A Study Comparing JNJ-79635322 and an Anti-B-cell Maturation Antigen (BCMA)xCD3 Bispecific Antibody in Participants With Relapsed or Refractory Multiple Myeloma
The purpose of this study is to evaluate how well JNJ-79635322 works when compared with an anti-B-cell maturation antigen (BCMA)xCD3 bispecific antibody.
- Trial name
- TRIlogy-4 (NCT07258511)
- Testing
- JNJ-79635322, Teclistamab
- Where
- 157 sites in United States, Australia and 13 more
- Volunteers
- 400 people
- Sponsor
- Janssen Research & Development, LLC
Absence Epilepsy, Jeavons Syndrome
BMB-101 in Absence Epilepsy and DEE
The study is a pilot, open-label, study to test whether BMB-101 is safe and effective in reducing the frequency of seizures in subjects with Absence Epilepsy including Epilepsy with Eyelid Myoclonia (also called Jeavons Syndrome)…
- Conditions
- Absence Epilepsy, Jeavons Syndrome, Dravet Syndrome, Lennox Gastaut Syndrome
- Trial name
- NCT06401538
- Testing
- BMB-101
- Where
- 5 sites in Australia
- Volunteers
- 20 people
- Sponsor
- Bright Minds Biosciences Pty Ltd
Galactose Single Point (GSP), Residual Liver Function
Clinical Study for Development of Oral Galactose Single Point (OGSP) Solution (G.S.P. Oral Solution®)
The primary objective is to determine the oral galactose single point (OGSP) cutoff values to discriminate subjects with different hepatic function.
- Trial name
- NCT03457311
- Testing
- G.S.P. Oral Solution 400 mg/ml
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Richever Enterprise Co., Ltd.
Rheumatoid Arthritis, Osteoarthritis
Multi-Centre Study to Assess the Long-term Performance of the DePuy ASR™ System in Resurfacing and Primary Total Hip Replacement
The purpose of this study is to monitor the performance of a large metal-on-metal bearing from the DePuy ASR™ System in the treatment of patients with hip joint disease requiring hip resurfacing surgery or a total hip…
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00872547
- Testing
- DePuy ASR™ Hip System, DePuy ASR™ XL Head / ASR™ Acetabular Cup System
- Where
- 8 sites in Austria, Czechia and 6 more
- Volunteers
- 329 people
- Sponsor
- DePuy International
Growth Hormone Deficiency
Evolution Of Growth Rate In Children With Growth Retardation Due to Glucocorticosteroid Therapy And Treated By Genotonorm
To estimate the evolution of height and growth rate over 5 years of growth hormone (GH) treatment To estimate the prognostic factors of growth rate with GH treatment To confirm the good clinical and biological safety of GH…
- Trial name
- NCT00163189
- Testing
- Somatropin
- Where
- 19 sites in France
- Volunteers
- 98 people
- Sponsor
- Pfizer
Hypothalamic Obesity
Open-Label Study of Setmelanotide in Hypothalamic Obesity
Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in participants with hypothalamic obesity (HO).
- Trial name
- NCT04725240
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 18 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Fragile X Syndrome
A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Fragile X Syndrome in adolescent and adult males.
- Trial name
- NCT01894958
- Testing
- NNZ-2566
- Where
- 17 sites in United States
- Volunteers
- 72 people
- Sponsor
- Neuren Pharmaceuticals Limited
Puberty, Precocious, McCune-Albright Syndrome
Faslodex in McCune-Albright Syndrome
The purpose of this study is to evaluate the safety, effectiveness and pharmacokinetics of a study drug called Faslodex (fulvestrant) in the treatment of progressive precocious puberty (PPP) (early puberty) in girls with…
- Trial name
- FMAS (NCT00278915)
- Testing
- Fulvestrant
- Where
- 16 sites in United States, France and 4 more
- Volunteers
- 30 people
- Sponsor
- AstraZeneca
Murmur, Heart, Aortic Valve Stenosis
Development of an Algorithm to Differentiate Heart Murmurs Using Electronic Stethoscopes
The Eko CORE and DUO stethoscopes are FDA-approved electronic devices that have the capacity to record heart sounds. The study seeks to expand murmur detection to include VHD classification through the development of novel ML…
- Conditions
- Murmur, Heart, Aortic Valve Stenosis, Tricuspid Regurgitation, Mitral Regurgitation
- Trial name
- NCT04400513
- Testing
- Use of Eko CORE and Eko DUO electronic stethoscopes
- Where
- United States
- Volunteers
- 1,145 people
- Sponsor
- Eko Devices, Inc.
Genu Varum, Genu Valgum
Retrospective Evaluation of Guided Growth Plate System Plus's Safety and Clinical Performance for the Treatment of Lower Limbs Deformities.
Orthofix is conducting this retrospective Post-market clinical follow up (PMCF) study to assess the safety and clinical performance of the Guided Growth Plate System Plus (GGPSP) device, which is a new version of the Guided…
- Conditions
- Genu Varum, Genu Valgum, Joint Diseases, Knee Deformity
- Trial name
- NCT05241691
- Testing
- Guided Growth Plate System Plus
- Where
- Italy
- Volunteers
- 69 people
- Sponsor
- Orthofix s.r.l.
Osteogenesis Imperfecta
Zoledronic Acid in Children (1 -17 Years) With Severe Osteogenesis Imperfecta
This is an extension study to examine the long-term safety and efficacy of zoledronic acid in patients who have completed the core CZOL446H2202 study.
- Trial name
- NCT00131118
- Testing
- Zoledronic Acid
- Where
- 10 sites in United States
- Volunteers
- 127 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
A Non-Drug Study Detecting And Quantifying Nocturnal Scratch Behaviors From Wrist Actigraphy Data In Adult Healthy Volunteers And Participants With Atopic Dermatitis (AD)
Sleep disturbance is very common in patients with atopic dermatitis (AD) and is a major factor leading to impaired quality of life. Nocturnal scratching is one of the factors causing sleep disturbance in AD patients.
- Trial name
- NCT04262791
- Testing
- Wrist Actigraphy Device, Sleep Headband
- Where
- 3 sites in United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- AbbVie
Autism
Deep rTMS (Repetitive Transcranial Magnetic Stimulation)for Treatment of Autism Symptoms in Children.
Autism is a developmental disorder characterized by abnormalities in speech and communication, impaired social functioning, and repetitive behaviors and interests.
- Trial name
- NCT01388179
- Testing
- Transcranial Magnetic Stimulation
- Where
- Israel
- Volunteers
- 20 people
- Sponsor
- Brainsway
Autism Spectrum Disorder
Efficacy and Safety of Slenyto for Insomnia in Children With ASD
This is a randomized placebo controlled study in children diagnosed with autism spectrum disorder (ASD), to Investigate the Efficacy and Safety of Slenyto® to alleviate Sleep Disturbances in these children. .
- Trial name
- ASD (NCT04233502)
- Testing
- Melatonin
- Sponsor
- Neurim Pharmaceuticals Ltd.
Chronic Adrenal Insufficiency
A European Post-Authorisation Observational Study (Registry) of Patients With Chronic Adrenal Insufficiency (AI)
As a post-approval requirement of the European Medicines Agency, this European patient post authorization safety study is an observational study being conducted to monitor the safety of long-term treatment with Plenadren and…
- Trial name
- NCT01661387
- Where
- 24 sites in Germany, Italy and 3 more
- Volunteers
- 3,258 people
- Sponsor
- Shire
GHD
A Study to Investigate the Safety and Efficacy of Once-weekly PEG-somatropin (GenSci004) in Children With Growth Hormone Deficiency
The purpose of this Phase 3 study is to evaluate the safety and efficacy of once-weekly GenSci004 over 108 weeks in children with GHD aged from 6 months to 17 years old, inclusive.
- Trial name
- ESCALATE (NCT06024967)
- Testing
- PEG-somatropin
- Volunteers
- 180 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
SCN2A-DEE, Epilepsy
A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
The goal of this trial is to learn about the effect of PRAX-222 in pediatric participants with early onset SCN2A developmental and epileptic encephalopathy (DEE), aged 2 to 18 years.
- Trial name
- NCT05737784
- Testing
- PRAX-222 - Initial Dose, PRAX-222 - Initial Ascending Doses, PRAX-222 - Optional Ascending Doses
- Where
- 3 sites in United States, Brazil
- Volunteers
- 60 people
- Sponsor
- Praxis Precision Medicines
Non-Small Cell Lung Cancer (NSCLC)
Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Relapsed or Refractory Advanced Non-Small Cell Lung Cancer With an FGFR Alteration
This is an open-label, single arm study to study the safety, efficacy and tolerability of Pemigatinib when used on participants with squamous or nonsquamous NSCLC with a documented FGFR1-3 mutations or fusions/rearrangement who…
- Trial name
- FIGHT-210 (NCT05253807)
- Testing
- Pemigatinib
- Where
- 36 sites in United States, France and 3 more
- Volunteers
- 8 people
- Sponsor
- Incyte Corporation
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