Clinical trials: Gonadal Dysgenesis
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT07159841
- Testing
- Atumelnant
- Where
- 51 sites in United States, Argentina and 11 more
- Volunteers
- 153 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Hepatocellular Carcinoma (HCC), Cholangiocarcinoma
An Open-Label Safety and Tolerability Study of INCB062079 in Subjects With Advanced Hepatocellular Carcinoma and Other Malignancies
The purpose of this study is to evaluate the safety and tolerability, and determine the maximum tolerated dose of INCB062079 in subjects with advanced hepatocellular carcinoma and other malignancies.
- Conditions
- Hepatocellular Carcinoma (HCC), Cholangiocarcinoma, Esophageal Cancer, Nasopharyngeal Cancer
- Trial name
- NCT03144661
- Testing
- INCB062079
- Where
- 6 sites in United States, Belgium
- Volunteers
- 25 people
- Sponsor
- Incyte Corporation
Trisomy 13, Trisomy 18
High Risk Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our proprietary algorithm method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278874
- Where
- 4 sites in United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Congenital Adrenal Hyperplasia
Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult participants with classic CAH due to 21-hydroxylase deficiency.
- Trial name
- CAHtalyst (NCT04490915)
- Testing
- Crinecerfont
- Where
- 70 sites in United States, Austria and 16 more
- Volunteers
- 182 people
- Sponsor
- Neurocrine Biosciences
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
Congenital Adrenal Hyperplasia
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
This is a Phase 2 multicenter, single-blind, multiple dose study to evaluate the safety and efficacy of orally administered ATR-101 in subjects with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT02804178
- Testing
- ATR-101
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Millendo Therapeutics, Inc.
Growth Hormone Deficiency, Turner Syndrome
Evaluation of Convenience and Compliance of the Easypod™ Electronic Self-injector
This is an international, multicenter study involving children treated with Saizen®, a growth hormone, who will be trained to use easypod, a new electronic injector and will complete a questionnaire after 12 week of use.
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- NCT00738205
- Volunteers
- 834 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Advanced Cancer, Metastatic Cancer
A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors
This clinical trial is evaluating a drug called ART0380 in participants with advanced or metastatic solid tumors. The main goals of this study are to: Find the recommended dose of ART0380 that can be given safely to participants…
- Conditions
- Advanced Cancer, Metastatic Cancer, Ovarian Cancer, Primary Peritoneal Cancer
- Trial name
- NCT04657068
- Testing
- ART0380, Gemcitabine, Irinotecan
- Where
- 88 sites in United States, France and 2 more
- Volunteers
- 542 people
- Sponsor
- Artios Pharma Ltd
Hypothalamic Obesity
A Trial of Setmelanotide in Acquired Hypothalamic Obesity
The goal of this trial is to learn how well Setmelanotide works to improve weight reduction, hunger, and quality of life in patients 4 years of age and older with acquired Hypothalamic Obesity (HO).
- Trial name
- NCT05774756
- Testing
- Setmelanotide
- Where
- 28 sites in United States, Canada and 4 more
- Volunteers
- 143 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Safety and Efficacy of Long-term Somatropin Treatment in Children
This study is conducted in Europe. The aim of this study is to evaluate safety during the long-term use of somatropin (Norditropin®) in children as well as efficacy on change in height.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Foetal Growth Problem, Small for Gestational Age
- Trial name
- GrowthWIN II (NCT01543867)
- Testing
- somatropin
- Where
- Germany
- Volunteers
- 5,442 people
- Sponsor
- Novo Nordisk A/S
Trisomy 13, Trisomy 18
Multiple Gestation Study
The objectives of the clinical study are to demonstrate the accuracy of our new NATUS diagnostic method to determine the genetic health of the developing fetuses in a multiple gestation pregnancy from a maternal blood sample.
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Sex Chromosome Abnormalities
- Trial name
- NCT02278536
- Where
- 8 sites in United States, India and 1 more
- Volunteers
- 354 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia
This is a multicenter Phase 2, multiple dose, dose escalation study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SPR001 in adult patients with classic congenital adrenal hyperplasia (CAH).
- Trial name
- NCT03257462
- Testing
- SPR001
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Spruce Biosciences
Breast Cancer, Solid Tumors, Adult
First-in-Human Study of Tersolisib (STX-478) as Monotherapy and in Combination With Other Antineoplastic Agents in Participants With Advanced Solid Tumors
Study STX-478-101 (LY4064809) is a multipart, open-label, phase 1/2 study evaluating the safety, tolerability, pharmacokinetics (PK), and preliminary antitumor activity of STX-478 (LY4064809) in participants with advanced solid…
- Trial name
- PIKALO-1 (NCT05768139)
- Testing
- STX-478, Fulvestrant, Ribociclib
- Where
- 65 sites in United States, Belgium and 7 more
- Volunteers
- 880 people
- Sponsor
- Eli Lilly and Company
Turner's Syndrome, Human Growth Hormone Deficiency
Evaluation of Overall Compliance and Duration of Zomacton® Treatment With the Zomajet® Needle-free Device
The purpose of this strictly observational, prospective, longitudinal study is to evaluate with sufficient precision the rate of overall treatment compliance from one year to 3 years of follow-up of the patients.
- Trial name
- ZOMAJET (NCT01306357)
- Testing
- Somatropin
- Where
- 17 sites in France
- Volunteers
- 87 people
- Sponsor
- Ferring Pharmaceuticals
Colorectal Cancer, Gastric Cancer
A Phase 1 in Patients With HLA-A*0201+ and WT1+ Recurrent/Metastatic Cancers
This is a Phase 1, open-label, 2-part, multi-center study evaluating the safety, tolerability, PK, pharmacodynamics (PD), immunogenicity, and antitumor activity of CUE-102 intravenous (IV) monotherapy in HLA-A*0201 positive…
- Conditions
- Colorectal Cancer, Gastric Cancer, Pancreatic Cancer, Ovarian Cancer
- Trial name
- NCT05360680
- Testing
- CUE-102
- Where
- 15 sites in United States
- Volunteers
- 42 people
- Sponsor
- Cue Biopharma
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)
The purpose of this Phase 2, open-label, sequential dose cohort study is to evaluate the safety, efficacy, and pharmacokinetics (PK) of atumelnant (CRN04894) in participants with classic congenital adrenal hyperplasia (CAH)…
- Trial name
- NCT05907291
- Testing
- atumelnant (CRN04894)
- Where
- 27 sites in United States, Argentina and 5 more
- Volunteers
- 38 people
- Sponsor
- Crinetics Pharmaceuticals Inc.
Congenital Adrenal Hyperplasia
Comparison of Chronocort® With Standard Glucocorticoid Therapy in Patients With Congenital Adrenal Hyperplasia
This study is a parallel arm, randomised, open-label study, including dose titration and admissions for four overnight stays for 24-hour endocrine profiles.
- Trial name
- NCT02716818
- Testing
- Chronocort®, standard glucocorticoid therapy
- Where
- United States
- Volunteers
- 122 people
- Sponsor
- Neurocrine UK Limited
Trisomy 13, Trisomy 18
Prenatal Non-invasive Aneuploidy Test Utilizing SNPs Trial
This prospective blinded study will assess the diagnostic capability of an informatics enhanced SNP based technology (Parental Support) to identify pregnant women who are carrying a fetus with an aneuploidy from fee floating DNA…
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Aneuploidy
- Trial name
- PreNATUS (NCT01545674)
- Testing
- Blood Draw
- Where
- 29 sites in United States, Canada and 4 more
- Volunteers
- 937 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Hypothalamic Obesity, Prader-Willi Syndrome
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
The purpose of this study is to evaluate the safety, tolerability, and PK of RM-718 in healthy subjects with obesity and in patients with MC4R Pathway Impairment
- Conditions
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- Trial name
- NCT06239116
- Testing
- Part C: RM-718, Part D: RM-718
- Where
- 7 sites in United States
- Volunteers
- 111 people, healthy volunteers welcome
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Endocrine Disease, Adrenal Insufficiency
Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH
The purpose of this study is to gather safety and effectiveness information about a new formulation of Hydrocortisone (Chronocort®) used to treat patients with a disease called congenital adrenal hyperplasia (CAH).
- Conditions
- Endocrine Disease, Adrenal Insufficiency, Congenital Adrenal Hyperplasia
- Trial name
- NCT01735617
- Testing
- Hydrocortisone Modified Release Capsules
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Neurocrine UK Limited