Clinical trials: Hormone Replacement
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study.
- Trial name
- NCT00630747
- Testing
- Idursulfase
- Where
- 52 sites in United States, Brazil and 8 more
- Volunteers
- 94 people
- Sponsor
- Shire
Smoking Cessation
Smoking Reduction or Cessation With Nicotine Replacement Therapy
The purpose of this study is to test the success rate of smoking reduction or cessation with different nicotine products.
- Trial name
- NCT00840242
- Testing
- Nicotine gum, Nicotine inhaler
- Where
- 2 sites in Czechia
- Volunteers
- 314 people
- Sponsor
- McNeil AB
Lysosomal Acid Lipase Deficiency
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).
- Trial name
- NCT02112994
- Testing
- Sebelipase Alfa
- Where
- 19 sites in United States, Australia and 13 more
- Volunteers
- 31 people
- Sponsor
- Alexion 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
Gaucher Disease, Type 1
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to this deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00430625
- Testing
- VPRIV ®,
- Where
- 5 sites in Argentina, Israel and 3 more
- Volunteers
- 25 people
- Sponsor
- Shire
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725
This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency.
- Trial name
- mentor™2 (NCT00978380)
- Testing
- catridecacog
- Where
- 34 sites in United States, Austria and 10 more
- Volunteers
- 63 people
- Sponsor
- Novo Nordisk A/S
Growth Hormone Deficiency
A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
- Trial name
- SkyPASS (NCT05775523)
- Testing
- No intervention
- Where
- 27 sites in United States
- Volunteers
- 500 people
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
Hypogonadism, Hypogonadism, Male
A Study of the Effect of Testosterone Replacement Therapy on Blood Pressure in Adult Male Participants With Hypogonadism
Testosterone is the principal androgen produced by the male testes. Male Hypogonadism is the result of inadequate production of testosterone by the Leydig cells of the testes and is reflected by total serum concentrations of…
- Conditions
- Hypogonadism, Hypogonadism, Male, Hypogonadotropic Hypogonadism
- Trial name
- NCT04456296
- Testing
- Aveed Injectable Product, Fortesta, Testim
- Where
- 35 sites in United States
- Volunteers
- 673 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Acute Kidney Injury
Safety and Performance Evaluation of multiPlus Dialysate During CRRT
Assessment of the performance of multiPlus dialysate based on the serum creatinine removal 6 hours (360 min) after start of continuous veno-venous haemodialysis/ haemodiafiltration [CVVHD(F)].
- Trial name
- multiPlus (NCT05315817)
- Testing
- multiPlus dialysate
- Where
- Germany
- Volunteers
- 1 people
- Sponsor
- Fresenius Medical Care Deutschland GmbH
End Stage Renal Failure on Dialysis, End Stage Renal Disease on Dialysis
Human Factors Validation Testing for the Peripal System; a Manual Connection Assist Device for Peritoneal Dialysis Devices
It is estimated that there are currently over 3 million patients receiving dialysis treatment worldwide. With effective pre-dialysis counselling, a majority of patients choose the home-based therapy peritoneal dialysis (PD) but…
- Conditions
- End Stage Renal Failure on Dialysis, End Stage Renal Disease on Dialysis, Renal Failure, Peritoneal Dialysis
- Trial name
- NCT04292210
- Testing
- Perisafe
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Peripal AG
Growth Hormone Deficiency
Efficacy of Somatropin in Adult Patients With Isolated Growth Hormone Deficiency
The study will investigate the effect on growth hormone replacement in patients with isolated growth hormone deficiency on body composition, especially visceral fat mass.
- Trial name
- IGHD (NCT00630487)
- Testing
- Somatropin
- Where
- 2 sites in Germany
- Volunteers
- 9 people
- Sponsor
- Pfizer
Male Hypogonadism
Safety and Efficacy of Weekly Testosterone Cypionate Auto-Injector in Men With Hypogonadism
The goal of this Phase 3 clinical trial is to evaluate the safety, efficacy, and usability of a weekly subcutaneous testosterone cypionate auto-injector in adult males with hypogonadism.
- Trial name
- NCT07898072
- Testing
- Testosterone Cypionate 75 mg, Testosterone cypionate 50 mg, Testosterone cypionate 100 mg
- Where
- 10 sites in United States
- Volunteers
- 190 people
- Sponsor
- Amneal Pharmaceuticals, LLC
Hunter Syndrome
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter…
- Trial name
- AIM-IT (NCT02055118)
- Testing
- idursulfase-IT, No IT treatment
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 58 people
- Sponsor
- Shire
Mucopolysaccharidosis Type 7
An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
UX003-CL201 is an open-label Phase 1/2 study to assess the safety, efficacy, and dose of UX003 in MPS 7 patients via intravenous (IV) administration every other week (QOW) for 36 weeks with up to an additional 36 weeks from the…
- Trial name
- NCT01856218
- Testing
- UX003
- Where
- United Kingdom
- Volunteers
- 3 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Acquired Bleeding Disorder, Cardiac Surgery Requiring Cardiopulmonary Bypass
Activated Recombinant Human Factor VII Following Cardiac Bypass Surgery for Paediatric Congenital Heart Disease
This trial is conducted in Oceania. The aim of this trial is to investigate the efficacy of activated recombinant human factor VII and standard treatment compared with standard haemostatic replacement therapy in cardiopulmonary…
- Trial name
- NCT01562574
- Testing
- activated recombinant human factor VII
- Where
- Australia
- Volunteers
- 82 people
- Sponsor
- Novo Nordisk A/S
Smoking Cessation
Pilot Study of a New Nicotine Replacement Therapy
Pilot study of a new nicotine replacement therapy for smoking cessation.
- Trial name
- NCT00764439
- Testing
- Nicotine replacement therapy
- Where
- 2 sites in United Kingdom
- Volunteers
- 258 people
- Sponsor
- McNeil AB
MPS 7, Sly Syndrome
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003.
- Conditions
- MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
- Trial name
- NCT02230566
- Testing
- UX003
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy
A Phase 1/2 Study of High-dose Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure
The purpose of this trial is to characterize the safety profile and preliminary activity of high-dose MYDICAR® in persons with advanced heart failure when added to their maximal and optimized therapy.
- Conditions
- Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy, Heart Failure, Cardiomyopathies
- Trial name
- NCT02346422
- Testing
- MYDICAR Phase 1, MYDICAR Phase 2
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- Celladon Corporation
Glycogen Storage Disease Type II, Pompe Disease
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
GSD-II (also known as Pompe disease) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Glycogen Storage Disease Type II, Pompe Disease, Acid Maltase Deficiency Disease, Glycogenosis 2
- Trial name
- NCT00051935
- Testing
- Alglucosidase alfa
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Genzyme, a Sanofi Company
Food-effect
Effect of Food on the Bioavailability of 30 mg Estetrol (E4) Tablet in Healthy Postmenopausal Female Volunteers
Assessment of the effect of a high fat meal on the quantity in blood of a female sex hormone called estetrol (E4). The study also aims at determining how subject tolerate the study drug and how safe it is for them.
- Trial name
- NCT03798197
- Testing
- 30 mg estetrol (E4)
- Where
- Bulgaria
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Donesta Bioscience