Clinical trials: Development Disorders
Retinitis Pigmentosa
Development of Visual Function Evaluation Method
The objective of this study is to develop the comprehensive visual function evaluation method in severe visually impaired patient
- Trial name
- NCT03281005
- Where
- Japan
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Astellas Pharma Inc
Congenital Adrenal Hyperplasia
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due…
- Trial name
- NCT04806451
- Testing
- Crinecerfont
- Where
- 46 sites in United States, Belgium and 8 more
- Volunteers
- 103 people
- Sponsor
- Neurocrine Biosciences
Autism, Pediatric Autism
Open-label Study of Safety and Tolerability of Memantine in Children With Autism
The purpose of this study is to evaluate the long-term safety and tolerability of memantine in the treatment of autism in pediatric patients.
- Trial name
- NCT01999894
- Testing
- Memantine HCl
- Where
- 19 sites in United States
- Volunteers
- 102 people
- Sponsor
- Forest Laboratories
Autism
SynapDx Autism Gene Expression Analysis Study (STORY)
This study will prospectively enroll approximately 880 children, at least 18 months and less than 60 months of age, who have been referred to a pediatric developmental evaluation center.
- Trial name
- STORY (NCT01810341)
- Where
- 20 sites in United States, Canada
- Volunteers
- 880 people
- Sponsor
- SynapDx
Non-Small Cell Lung Cancer
Tissue Collection Study for Patients With Non-Small Cell Lung Cancer With Resectable Disease
A 15-gene lung signature was created to identify predictive and prognostic biomarkers for Non-Small Cell Lung Cancer (NSCLC) patients.
- Trial name
- NCT01450553
- Where
- 7 sites in United States
- Volunteers
- 450 people
- Sponsor
- Precision Therapeutics
Autism Spectrum Disorders
An Open Label Extension Study of STX209 in Subjects With Autism Spectrum Disorders
Study 22003, "An Open-Label, Flexible-Dose Evaluation of the Safety and Tolerability of STX209 for Treatment of Irritability in Subjects With Autism Spectrum Disorders(ASD)" currently is evaluating the efficacy of STX209…
- Trial name
- NCT01064973
- Testing
- arbaclofen
- Where
- 8 sites in United States
- Volunteers
- 32 people
- Sponsor
- Seaside Therapeutics, Inc.
Gorlin Syndrome
Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
- Trial name
- NCT06050122
- Testing
- Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib
- Where
- 40 sites in United States, France and 5 more
- Volunteers
- 113 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Multiple Sclerosis
Long Term Study of Fingolimod in MS Patients From the FTY Clinical Program
The purpose of this observational study is to collect long-term data on safety and effectiveness of fingolimod, particularly including clinical disease and patient outcome related parameters such as relapse and disability, in…
- Trial name
- NCT01281657
- Testing
- fingolimod
- Where
- 58 sites in United States, Canada
- Volunteers
- 64 people
- Sponsor
- Novartis
Axial Spondyloarthritis (AxSpA)
Retrospective Case-Control Study for Developing an Artificial Intelligence (AI) Tool for Lesion Detection Using Magnetic Resonance Imaging (MRI) and Clinical Variables for Early Diagnosis of Axial Spondyloarthritis (axSpA)
The goal of this observational study is to develop and validate an Artificial Intelligence (AI) tool that allows the lesion detection and early diagnosis of axial spondyloarthritis (axSpA) based on Magnetic Resonance Imaging…
- Trial name
- InnovaDetect (NCT06591481)
- Where
- 7 sites in Jordan, Saudi Arabia and 2 more
- Volunteers
- 925 people, healthy volunteers welcome
- Sponsor
- Ángel Alberich Bayarri
Autism Spectrum Disorder, Social Communication Disorder
To Evaluate the Efficacy and Safety of 'NDTx-01' in Patients With ASD or SCD
The purpose of this study is to demonstrate the superiority of the treatment of cognitive therapy software 'NDTx-01' compared to TAU (Treatment-As-Usual).
- Trial name
- NCT06446193
- Testing
- NDTx-01, TAU
- Where
- 5 sites in South Korea
- Volunteers
- 86 people
- Sponsor
- Neudive Inc.
Biliary Tract Cancer
Futibatinib (TAS-120) in Patients With Advanced Biliary Tract Cancer
To compare overall survival (OS) of patients in Futibatinib and Zimberelimab in Combination with Gemcitabine plus Cisplatin versus Durvalumab or Pembrolizumab in Combination with Gemcitabine plus Cisplatin for patients with…
- Trial name
- FOENIX-BTC (NCT07710885)
- Testing
- Futibatinib, Zimberelimab, Durvalumab
- Where
- 21 sites in Australia, Japan and 2 more
- Volunteers
- 784 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Turner's Syndrome
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years.
- Trial name
- NCT01066052
- Testing
- r-hGH
- Where
- France
- Volunteers
- 115 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Achondroplasia
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
- Trial name
- NCT04554940
- Testing
- vosoritide
- Where
- 3 sites in Australia, United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Healthy Volunteer, Autistic Disorder, Asperger's Syndrome
A Non-Drug Study on the Relationship Between Exploratory Biomarkers and Functional Dimensions in Individuals With Autistic Disorder or Asperger's Syndrome
This multi-center, non-drug study will explore the relationship between exploratory biomarkers and functional dimensions in male adult individuals with Autistic Disorder or Asperger's Syndrome and healthy volunteer controls.
- Trial name
- NCT01669889
- Where
- 3 sites in United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Intra-amniotic Infection
Non-invasive Test to Detect Intra-amniotic Infection in Women With Preterm Labor and Intact Amniotic Membranes
The purpose of this study is to collect clinical specimens and corresponding clinical data to develop a non-invasive test for detection of intra-amniotic infection and prediction of preterm birth in women and intact amniotic…
- Trial name
- IAI (NCT00700219)
- Where
- 21 sites in United States
- Volunteers
- 900 people
- Sponsor
- ProteoGenix, Inc.
Malignant PEComa (Perivascular Epithelioid Cell Tumors)
A Phase II Study of Orally Administered BEZ235 Monotherapy in Patients With Metastatic or Unresectable Malignant PEComa
Study objectives: The primary objective is to determine the efficacy of BEZ235 on Objective Response Rate (best response on study) according to RECIST 1.1 criteria The secondary objectives are: To determine the progression free…
- Trial name
- NCT01690871
- Testing
- BEZ235
- Where
- Spain
- Sponsor
- Novartis Pharmaceuticals
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
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.
Infertility (IVF Patients)
Study of Abnormally Fertilized Embryos
The goal of this observational study is to determine the diploidy rate of haploid and triploid embryos from in vitro fertilization (IVF) cycles.
- Trial name
- SAFE (NCT06940973)
- Testing
- Parental samples, PGT-A, TE rebiopsies and spent blastocyst media collection
- Where
- Spain
- Volunteers
- 300 people
- Sponsor
- Igenomix
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
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