Clinical trials: Newborn Complication
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.
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH
- Trial name
- NCT04842032
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 28 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Small for Gestational Age Infant, Fetal Growth Restriction
Developing and Testing AI Models for Fetal Biometry and Amniotic Volume Assessment in Fetal Ultrasound Scans.
Routine fetal ultrasound scan during the second trimester of the pregnancy is a low-cost, noninvasive screening modality that has been proven to lower fetal mortality by up to 20%.
- Conditions
- Small for Gestational Age Infant, Fetal Growth Restriction, Oligohydramnios, Polyhydramnios
- Trial name
- NCT05059093
- Testing
- Classification and segmentation deep learning models
- Where
- 6 sites in Morocco
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- Deepecho
SGA, Turner Syndrome
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
The purpose of this study is to find out if somapacitan is safe and how well somapacitan works in children either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature.
- Conditions
- SGA, Turner Syndrome, Noonan Syndrome, ISS
- Trial name
- REAL 9 (NCT05723835)
- Testing
- Somapacitan
- Where
- 18 sites in United States, Malaysia and 4 more
- Volunteers
- 47 people
- Sponsor
- Novo Nordisk A/S
End Stage Renal Disease, AV Fistula
Percutaneous Less Invasive AV Fistula for Vascular Access in ESRD
A prospective single-arm well-controlled study to evaluate the safety and effectiveness of a less invasive means of establishing vascular access to facilitate dialysis in patients with end stage renal disease.
- Conditions
- End Stage Renal Disease, AV Fistula, Fistula, End Stage Kidney Disease
- Trial name
- NCT02363972
- Testing
- Ellipsys Vascular Access Catheter
- Where
- 5 sites in United States
- Volunteers
- 103 people
- Sponsor
- Medtronic Endovascular
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetic parameters of VX-407 in healthy participants.
- Trial name
- NCT06345755
- Testing
- VX-407, Midazolam
- Where
- 2 sites in United States, Canada
- Volunteers
- 159 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Usability and Tolerability of the Norditropin NordiFlex® Injection Device in Children Never Previously Treated With Growth Hormone
This study is conducted in Europe. The purpose of this study is to assess the impact on daily life for children new to using a growth hormone injection device.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Genetic Disorder, Turner Syndrome
- Trial name
- NCT01327924
- Testing
- Norditropin NordiFlex®
- Where
- France
- Volunteers
- 77 people
- Sponsor
- Novo Nordisk A/S
Turner Syndrome
A Study of PEG-somatropin Injection to Treat Children of Turner Syndrome
This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children of Turner syndrome (TS), preliminarily evaluate its safety and efficacy and provide scientific and…
- Trial name
- NCT03189160
- Testing
- PEG-rhGH low dose, PEG-rhGH high dose, Non-treatment control group
- Where
- 9 sites in China
- Volunteers
- 180 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Nephritis, Hereditary, Hematuria-Nephropathy-Deafness Syndrome
Biomarker for Alport Syndrome (BioAlport)
International, multicenter, observational, longitudinal monitoring study to identify biomarker/s for Alport syndrome and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s
- Trial name
- BioAlport (NCT02718027)
- Where
- 7 sites in Albania, Georgia and 5 more
- Volunteers
- 12 people
- Sponsor
- CENTOGENE GmbH Rostock
Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency
Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia
This study will test a new, extended release form of hydrocortisone called Chronocort in patients with congenital adrenal hyperplasia (CAH).
- Conditions
- Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency, Adrenogenital Syndrome
- Trial name
- NCT00519818
- Testing
- Chronocort, Cortef
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Neurocrine UK Limited
Primary Hyperoxaluria
Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. Pilot clinical study, open, prospective and multicenter.
- Trial name
- NCT03819647
- Testing
- stiripentol (Diacomit)
- Where
- 3 sites in France
- Volunteers
- 15 people
- Sponsor
- Biocodex
Congenital Anomalies, Neonatal Complications
AI-Powered Neonatal Risk Assessment for Improved Perinatal Outcomes
This study aims to develop advanced artificial intelligence (AI) models that predict neonatal risks and complications based on historical multimodal health data, including ultrasound and MRI scans.
- Conditions
- Congenital Anomalies, Neonatal Complications, Perinatal Outcomes, Perinatal Outcomes of the Mother and Fetus
- Trial name
- NCT07064356
- Volunteers
- 50,000 people, healthy volunteers welcome
- Sponsor
- FetalFirst Limited
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
Mass Balance and Absolute Bioavailability Study of JMKX003142 In Healthy Volunteers
A phase 1 healthy volunteer study to assess the excretion and metabolism as well as the absolute bioavailability of oral JMKX003142.
- Trial name
- NCT07463053
- Testing
- [14C]JMKX003142, JMKX003142 tablets, [14C]JMKX003142 solution for infusion
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Jemincare
Multiple Myeloma
WT1 Vaccine Treatment of Patients With Multiple Myeloma After Autologous Stem Cell Transplantation
The purpose of this trial is to assess the immune response after vaccination with a peptide vaccine called galinpepimut-S (or GPS) in a type of blood cancer called multiple myeloma.
- Trial name
- NCT01827137
- Testing
- Galinpepimut-S, GM-CSF, Montanide
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sellas Life Sciences Group
Endometrial Cancer
Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel and a Substudy With an Insulin-Suppressing Diet in Patients With Advanced/Recurrent Endometrial Cancer
This is a Phase 2, multicenter, open-label, single-arm study to evaluate the efficacy and safety of sapanisertib and serabelisib (PIKTOR) with paclitaxel in participants with advanced or recurrent endometrial cancer.
- Trial name
- NCT06463028
- Testing
- Sapanisertib, Serabelisib, Paclitaxel
- Where
- 18 sites in United States
- Volunteers
- 40 people
- Sponsor
- Faeth Therapeutics
Congenital Adrenal Hyperplasia
Nevanimibe HCl for the Treatment of Classic CAH
This is a multicenter, intra-subject dose-titration open-label study of nevanimibe hydrochloride (HCl) for the treatment of classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT03669549
- Testing
- Nevanimibe hydrochloride
- Where
- 11 sites in Brazil, Czechia and 3 more
- Volunteers
- 15 people
- Sponsor
- Millendo Therapeutics US, Inc.
Autosomal Dominant Hypocalcemia (ADH)
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
- Trial name
- CALIBRATE (NCT05680818)
- Testing
- Encaleret, Standard of Care
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 67 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Congenital Adrenal Hyperplasia
A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
This trial will evaluate the effects of different doses of Lu AG13909 in adult participants with congenital adrenal hyperplasia, also called CAH.
- Trial name
- NCT05669950
- Testing
- Lu AG13909
- Where
- 17 sites in United States, Denmark and 7 more
- Volunteers
- 42 people
- Sponsor
- H. Lundbeck A/S
Cancer
Fentanyl Sublingual Spray in Treating Patients With Breakthrough Cancer Pain
This is a phase III, randomized, double-blind, placebo-controlled, multicenter study of the clinical response to fentanyl sublingual spray as a treatment for breakthrough cancer pain.
- Trial name
- NCT00538850
- Testing
- Fentanyl sublingual spray
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- INSYS Therapeutics Inc