Clinical trials: Development Disorders
Infertility
An Efficacy and Safety Study of Corifollitropin Alfa (MK-8962) in Contrast to Recombinant FSH for Use in Controlled Ovarian Stimulation of Indian Women (P07056, Also Known as MK-8962-029)
This study, conducted in India, will be recruiting women participants who are between the ages of 18 and 42, and who have an indication for controlled ovarian stimulation (COS), but have not yet undergone in-vitro fertilization…
- Trial name
- NCT01599494
- Testing
- MK-8962, recFSH, Ganirelix
- Sponsor
- Organon and Co
Growth Hormone Deficiency
VRS-317 in Adult Subjects With Growth Hormone Deficiency
The purpose of this research study is to determine the safety and tolerability of up to five doses of VRS-317 in Adult Growth Hormone Deficient patients.
- Trial name
- NCT01359488
- Testing
- VRS-317
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Versartis Inc.
Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.
- Trial name
- NCT07536269
- Testing
- Crinecerfont
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Neurocrine Switzerland GmbH
Autistic Disorder
Study of Fluoxetine in Autism
The purpose of this study is to assess the effect of fluoxetine orally dissolving tablets (ODT) on the repetitive behaviors core domain in children and adolescents with Autistic Disorder.
- Trial name
- SOFIA (NCT00515320)
- Testing
- Fluoxetine
- Where
- 17 sites in United States
- Volunteers
- 158 people
- Sponsor
- Neuropharm
Colorectal Carcinoma, Gastric Carcinoma
A Study to Test Different Doses of BI 765049 in People With Advanced Cancer of the Colon, Rectum, Stomach, or Pancreas
This study is open to adults with advanced cancer of the colon, rectum, stomach, or pancreas, that is the cancer cannot be removed by surgery or has spread.
- Conditions
- Colorectal Carcinoma, Gastric Carcinoma, Pancreatic Ductal Adenocarcinoma
- Trial name
- NCT06882746
- Testing
- BI 765049
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 14 people
- Sponsor
- Boehringer Ingelheim
Amblyopia, Anisometropic, Amblyopia Strabismic
Validation of New Virtual Reality Treatment for Children With Lazy Eye Using the Vedea Amblyopia Therapy (VAT)
The purpose of this clinical study is to validate the effectiveness of the Vedea Amblyopia Therapy (VAT) as a treatment for children with lazy eye.
- Conditions
- Amblyopia, Anisometropic, Amblyopia Strabismic, Amblyopia
- Trial name
- VAT (NCT05620173)
- Testing
- Vedea Amblyopia Therapy (VAT), Occlusion therapy
- Where
- 2 sites in Netherlands
- Volunteers
- 74 people
- Sponsor
- Vedea Healthware BV
SCN8A Developmental and Epileptic Encephalopathy Syndrome
Study to Evaluate NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)
The objective of this study is to assess the efficacy, safety, and pharmacokinetics of NBI-921352 as adjunctive therapy for seizures in subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).
- Trial name
- NCT04873869
- Testing
- NBI-921352
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Neurocrine Biosciences
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Trial name
- NCT03687242
- Testing
- SPR001
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Spruce Biosciences
Pulmonary Nodule, Solitary, Whole-genome Methylation Sequencing
Benign/Malignant Pulmonary Nodule Classification Based on High-throughput Whole-genome Methylation Sequencing(GM-seq)
Lung cancer is the first cancer in China in terms of morbidity and mortality. The problem of early diagnosis/treatment has always been concerned.
- Trial name
- NCT05415670
- Testing
- Whole-genome Methylation Sequencing(GM-seq)
- Where
- 2 sites in China
- Volunteers
- 158 people, healthy volunteers welcome
- Sponsor
- Geneplus-Beijing Co. Ltd.
Congenital Adrenal Hyperplasia
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia
This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal…
- Trial name
- CONnECT (NCT05063994)
- Testing
- Chronocort, Cortef
- Where
- 21 sites in United States, France and 1 more
- Volunteers
- 55 people
- Sponsor
- Neurocrine UK Limited
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of…
- Trial name
- PROPEL3 (NCT06164951)
- Testing
- Infigratinib 0.25 mg/kg/day
- Where
- 27 sites in United States, Argentina and 8 more
- Volunteers
- 114 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Growth Hormone Deficiency
Crossover Study to Assess the Safety and Pharmacokinetic of Pegylated Somatropin(PEG Somatropin) in GHD Children
The purpose of the phase 1 study is to assess the safety and pharmacokinetics of PEG somatropin, which administered once per week, compared with the daily used somatropin, and to evaluate the safety and possibility to replace…
- Trial name
- Phase 1 (NCT01613573)
- Testing
- somatropin AQ, pegylated somatropin
- Volunteers
- 12 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Biomarker Development in LGMD2i
The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD2i to support therapeutic development.
- Trial name
- MLB-01-001 (NCT04202627)
- Where
- 11 sites in United States, Denmark
- Volunteers
- 101 people
- Sponsor
- ML Bio Solutions, Inc.
Amyotrophic Lateral Sclerosis
GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)
GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system.
- Trial name
- GALS (NCT01854294)
- Testing
- GM604
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genervon Biopharmaceuticals, LLC
Relapsed AL Amyloidosis, Refractory AL Amyloidosis
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.
- Conditions
- Relapsed AL Amyloidosis, Refractory AL Amyloidosis, Light Chain Amyloidosis, Amyloidosis
- Trial name
- ALACRITY (NCT07081646)
- Testing
- AZD0120
- Where
- 18 sites in United States, Canada and 1 more
- Volunteers
- 91 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cervical Radiculopathy, Myelopathy
Safety Study of Preservon-Treated Bone Implants for Cervical Fusion in the Treatment of Cervical Radiculopathy or Myelopathy
The purpose of this study is to compare the safety and efficacy of Preservon(TM) treated allograft bone dowels to traditional freeze-dried allograft bone dowels (control) for fusion of degenerated cervical intervertebral discs in…
- Trial name
- NCT00344890
- Testing
- Anterior Cervical Discectomy and Fusion
- Where
- United States
- Volunteers
- 106 people
- Sponsor
- LifeNet Health
Autism Spectrum Disorder
A Study to Investigate the Effect of Esomeprazole and the Effect of Food on the Pharmacokinetics of Balovaptan in Healthy Volunteers
This study will investigate the effect of food and the effect of esomeprazole on the pharmacokinetics (PK) of a single dose of balovaptan in healthy volunteers.
- Trial name
- NCT04156646
- Testing
- Balovaptan, Esomeprazole
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Creatine Deficiency, X-linked
Observational Study of Males With Creatine Transporter Deficiency
The objectives of this study are to illustrate the clinical, neuro/electrophysiologic, biochemical, and developmental status and progression of patients with Creatine Transporter Deficiency (CTD) and to evaluate the utility of…
- Trial name
- Vigilan (NCT02931682)
- Where
- 10 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Heart Failure
Study to Collect High-Resolution ECG Data and Patient Insights on Heart Failure Care
The purpose of this study is to establish an electrocardiogram (ECG) data library to facilitate future studies on heart disease.
- Trial name
- NCT06809686
- Where
- 2 sites in United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Richmond Research Institute
Alpha Mannosidosis
The Natural History of Alpha-Mannosidosis
The natural history study of the rare lysosomal disease alpha-mannosidosis will answer the question; why the rare disease develops as it does?
- Trial name
- HUE-MAN (NCT00498420)
- Where
- 4 sites in Czechia, Germany and 2 more
- Volunteers
- 45 people
- Sponsor
- Zymenex A/S
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