Clinical trials: Hormone Replacement
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
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Chronic Kidney Disease - Stage V, Chronic Kidney Disease on Hemodialysis
Outcomes of Renal Replacement in Mexico
A descriptive, observational, prospective, multicenter study aimed at describing the main clinical outcomes of patients with chronic kidney disease receiving any form of renal replacement therapy in Mexico.
- Trial name
- ORReMex (NCT07474805)
- Volunteers
- 290 people
- Sponsor
- Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Sepsis
Cytokine Removal by CRRT in Pediatric Sepsis
This pilot study will compare the effect of diffusive versus convective Continuous Renal Replacement Therapy (CRRT) in children with sepsis who require CRRT.
- Trial name
- NCT00537693
- Testing
- Continuous Renal Replacement Therapy
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Sunnybrook Health Sciences Centre
Gaucher's Disease Type III
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or…
- Trial name
- LEAP2MONO (NCT05222906)
- Testing
- Venglustat, imiglucerase
- Where
- 22 sites in United States, Argentina and 9 more
- Volunteers
- 43 people
- Sponsor
- Sanofi
Fabry Disease
Safety Study of Replagal® Therapy in Children With Fabry Disease
The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).
- Trial name
- NCT01363492
- Testing
- Replagal (agalsidase alfa)
- Where
- 5 sites in United States
- Volunteers
- 15 people
- Sponsor
- Shire
Congenital Adrenal Hyperplasia
Open-label Comparison of Chronocort® Versus Standard Glucocorticoid Replacement Therapy
This study is an open-label, randomised, titration-blinded, parallel arm, multicenter study to compare twice daily Chronocort® with standard care in participants with Congenital Adrenal Hyperplasia (CAH).
- Trial name
- RESTORE (NCT03532022)
- Testing
- Chronocort®, Standard Care
- Where
- United States
- Sponsor
- Neurocrine UK Limited
Mucopolysaccharidosis Type 7
An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient
Emergency access granted to treat a single patient with advanced Mucopolysaccharidosis Type 7 with UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy via IV administration every other week (QOW) for up…
- Trial name
- NCT02097251
- Testing
- UX003
- Where
- United States
- Sponsor
- Joyce Fox
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
- Trial name
- PEDS (NCT04721366)
- Testing
- Standard of Care
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Takeda
Renal Insufficiency / Therapy
PD Catheter Data Collection
Peritoneal Dialysis (PD) is a Renal Replacement Therapy for patients with renal insufficiency that has been used more frequently in recent years.
- Trial name
- Catheter (NCT07156123)
- Where
- 2 sites in Germany
- Volunteers
- 300 people
- Sponsor
- Fresenius Medical Care Deutschland GmbH
Osteoporosis, Turner's Syndrome
Hormone Replacement Therapy to Treat Turner Syndrome
This study will evaluate the effects of hormone replacement therapy on patients with Turner syndrome (TS)-a genetic disorder in females in which part or all of one X chromosome is missing.
- Trial name
- NCT00013546
- Testing
- TMTDS
- Where
- United States
- Volunteers
- 92 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Pediatric Kidney Disease, Acute Kidney Injury
Registry for HF20 for Pediatric CRRT Under Emergency Use Authorization
Historically, innovations for acute kidney injury (AKI) and continuous renal replacement therapy (CRRT) have centered around an adult population.
- Trial name
- NCT04836026
- Testing
- HF20™
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Mucopolysaccharidosis
The Effect of Enzyme Replacement Therapy in Mucopolysaccharidosis
Mucopolysaccharidosis (MPS) causes chronic, progressive systemic disorders due to enzyme deficiency. Musculoskeletal manifestations of MPS include bone and vertebral deformities, restricted joint function and ROM (range of…
- Trial name
- NCT05006222
- Testing
- Enzyme Replacement Agent
- Where
- Turkey (Türkiye)
- Volunteers
- 12 people
- Sponsor
- Hasan Kalyoncu University
Endometrial Cancer
Estrogen Replacement Therapy in Treating Women With Early-Stage Endometrial Cancer
RATIONALE: Estrogen replacement therapy may improve quality-of-life in postmenopausal women with endometrial cancer. It is not yet known whether estrogen replacement therapy will affect cancer recurrence.
- Trial name
- NCT00002976
- Testing
- conjugated estrogens
- Where
- 149 sites in United States
- Volunteers
- 2,108 people
- Sponsor
- Gynecologic Oncology Group
Pineal Tumor
Melatonin Replacement Therapy in Pinealectomized Patients
This is an open-label, single-arm, single-center, proof-of-concept study to assess the effects of melatonin on cardiac autonomic activity in melatonin non-proficient pinealectomized patients.
- Trial name
- NCT03885258
- Testing
- Melatonin Replacement Therapy
- Where
- Brazil
- Volunteers
- 5 people
- Sponsor
- University of Sao Paulo
Cognitive Decline, Mucopolysaccharidosis I
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
This is a 24-month study of the use of laronidase administered into the spinal fluid to treat cognitive decline in mucopolysaccharidosis I (MPS I).
- Conditions
- Cognitive Decline, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00852358
- Testing
- laronidase
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Patricia I. Dickson, M.D.
Gaucher Disease
Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the…
- Trial name
- NCT01614574
- Testing
- velaglucerase alfa
- Where
- 3 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Shire
Fabry Disease
Replagal Enzyme Replacement Therapy for Adults With Fabry Disease
This study will determine the safety and effectiveness of the drug Replagal for treating people with Fabry disease, an inherited metabolic disorder.
- Trial name
- NCT00097890
- Testing
- Replagal (Agalsidase Alfa), Replagal
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Endocrine Disease, Infertility
Evaluation of Women With Endocrine and Reproductive-Related Conditions
This study was designed to allow inpatient and outpatient evaluation of women with a variety of reproductive and endocrine-related disorders for purposes of research and physician education.
- Conditions
- Endocrine Disease, Infertility, Leiomyoma, Endometriosis
- Trial name
- NCT00001850
- Where
- United States
- Volunteers
- 833 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Surfactant Deficiency Syndrome Neonatal, Respiratory Distress Syndrome in Premature Infant
Fast Assessment of Surfactant Deficiency in Preterm Infants to Speed up Treatment - Validation Study
The aim is to re-validate a FTIR spectroscopy test for measuring lung maturity/Respiratory Distress Syndrome (RDS) before conducting a RCT using this test to guide surfactant treatment of preterm infants.
- Trial name
- FAST2 (NCT05615428)
- Testing
- LS-ratio
- Where
- 3 sites in Denmark
- Volunteers
- 68 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet