Clinical trials: Hormone Replacement
Fabry Disease
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.
- Trial name
- NCT00864851
- Testing
- Replagal
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Shire
Disorders Associated With Peritoneal Dialysis
Study to Evaluate the Effects Icodextrin Versus Dianeal on Insulin Resistance in Nondiabetic Automated Peritoneal Dialysis Patients
1. LOCATION OF STUDY: Multicentric study in Brazil. 2. PURPOSE OF THE STUDY: To measure changes in HOMA index when non-diabetic patients in APD were exposed to 7,5% Icodextrin for the long-dwell; and to compare such changes with…
- Trial name
- STARCH (NCT01021878)
- Testing
- icodextrin, Dianeal
- Where
- 8 sites in Brazil
- Volunteers
- 60 people
- Sponsor
- Pontifícia Universidade Católica do Paraná
Gaucher Disease
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00478647
- Testing
- GA-GCB (velaglucerase alfa)
- Where
- 15 sites in United States, Israel and 3 more
- Volunteers
- 40 people
- Sponsor
- Shire
Fabry Disease
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Primary Objective(s): To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease To assess the effect of Replagal on heart rate variability in patients 7 to 17…
- Trial name
- NCT00084084
- Testing
- Agalsidase alfa
- Where
- 14 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Congenital Bleeding Disorder, Congenital FXIII Deficiency
Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency
This trial will be conducted in Asia, Europe and the United States of America (USA). The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children…
- Trial name
- mentor™5 (NCT01253811)
- Testing
- catridecacog
- Where
- 4 sites in United States, Israel and 1 more
- Volunteers
- 6 people
- Sponsor
- Novo Nordisk A/S
Hypoleptinemia, Generalized Lipodystrophy
Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Lipodystrophies represent a therapeutic challenge with regards to insulin resistance, hypertriglyceridemia and fatty liver which often is coupled with significant adipose tissue loss.
- Conditions
- Hypoleptinemia, Generalized Lipodystrophy, Partial Lipodystrophy, Insulin Resistance
- Trial name
- NCT00896298
- Testing
- Leptin
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Texas Southwestern Medical Center
Panhypopituitarism
Testosterone Gel Applied to Women With Pituitary Gland Problems
The principal aim of this study is to evaluate if application of testosterone gel leads to improvement in measures of sexual function, thinking ability and quality of life in women with dysfunction of the pituitary gland.
- Trial name
- NCT00144391
- Testing
- Transdermal Testosterone gel
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Charles Drew University of Medicine and Science
Smoking, Nicotine Addiction
The END Perioperative Smoking Pilot Study
The purpose of this pilot randomized trial is to determine the feasibility of e-cigarettes and telephone counselling (compared to transdermal nicotine replacement and telephone counselling) as a harm-reduction tool that may lead…
- Conditions
- Smoking, Nicotine Addiction, Surgery
- Trial name
- NCT02482233
- Testing
- ENDD (NJOY), NRT (NicoDerm CQ), telephone counseling
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Iron Deficiency Anemia, Iron Deficiency Anaemia
An Extension Trial to Assess the Safety of Re-dosing of Iron Isomaltoside/Ferric Derisomaltose (Monofer®/Monoferric®)
Evaluate safety and efficacy of intravenous (IV) iron isomaltoside/ferric derisomaltose re-dosing, in subjects who were previously treated with iron isomaltoside/ferric derisomaltose.
- Trial name
- FERWON-EXT (NCT02962648)
- Testing
- Iron isomaltoside/ferric derisomaltose
- Where
- 22 sites in United States
- Volunteers
- 103 people
- Sponsor
- Pharmacosmos A/S
Chronic Kidney Disease
Empowering Patients On Choices for Renal Replacement Therapy (Aim 3) (EPOCH-RRT)
Empowering Patients On Choices for Renal Replacement Therapy (EPOCH-RRT) study seeks to identify factors that matter the most to patients with kidney disease and study how they are impacted by different types of dialysis.
- Trial name
- EPOCH-RRT (NCT02488317)
- Testing
- Decision Aid
- Where
- United States
- Volunteers
- 234 people
- Sponsor
- Arbor Research Collaborative for Health
SMA - Spinal Muscular Atrophy, Gene Therapy
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
- Trial name
- STR1VE (NCT03306277)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- 16 sites in United States
- Volunteers
- 22 people
- Sponsor
- Novartis Gene Therapies
Sly Syndrome, MPS VII
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.
- Conditions
- Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
- Trial name
- NCT02432144
- Testing
- UX003
- Where
- 7 sites in United States, Brazil and 2 more
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Mild Cognitive Impairment, Alzheimer's Disease
Hormone and Information Processing Study
The purpose of this study is to examine the effects of testosterone (T) replacement on changes in thinking and memory, as well as mood in older men with mild cognitive impairment (MCI) and low T levels.
- Trial name
- HIP (NCT00539305)
- Testing
- testosterone gel
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- University of Washington
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
The primary objectives of this study are to assess the safety and efficacy of MS1819-SD vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).
- Trial name
- OPTION (NCT03746483)
- Testing
- MS1819, Porcine PERT
- Where
- 14 sites in United States, Poland
- Volunteers
- 41 people
- Sponsor
- Entero Therapeutics
Nicotine Addiction, Smoking Cessation
e-Cigarettes Versus NRT Gum for Smoking Cessation
This is a randomized trial comparing electronic cigarettes (e-Cigarettes) to nicotine gum for smoking cessation. Participants will be randomly assigned to either e-cigarette use or nicotine gum use during a quit attempt.
- Trial name
- NCT01925781
- Testing
- Nicotine polacrilex, STAM 1100mAh CE4 eGo Clearomizer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Creighton University
Gaucher Disease Type 1
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy…
- Trial name
- NCT00319046
- Testing
- Miglustat
- Volunteers
- 42 people
- Sponsor
- Actelion
Critically Ill Renal Failure Requiring CVVHD and Oseltamivir, Critically Ill Requiring ECMO and Oseltamivir
Pharmacokinetics of Tamiflu® (Oseltamivir) in Patients Receiving Extracorporeal Membrane Oxygenation (ECMO)and or Continuous Venovenous Hemodialysis (CVVHD)
Critically ill patients with flu may receive a drug called oseltamivir. They may also receive medical therapies to support their lung function (extracorporeal membrane oxygenation; ECMO) and kidney function (continuous venovenous…
- Trial name
- NCT01048879
- Testing
- pharmacokinetic blood sampling, pharmacokinetic blood and dialysate sampling
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Michigan
Immunologic Deficiency Syndromes
Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases
Octagam is a human normal immunoglobulin (IGIV) solution for intravenous administration. Octagam 5% is currently registered in more than 60 countries.
- Trial name
- NCT00811174
- Testing
- Octagam 10%
- Where
- Austria
- Volunteers
- 5 people
- Sponsor
- Octapharma
Tobacco Dependence
Bioequivalence Between Two New Oral Nicotine Replacement Therapy Products and Nicorette® Microtab
The purpose of this study is to assess the bioequivalence between two new oral nicotine replacement therapy products and Nicorette® microtab.
- Trial name
- NCT01238640
- Testing
- Code STD, Code STE, Nicorette Microtab
- Where
- Sweden
- Volunteers
- 84 people
- Sponsor
- McNeil AB
Pompe Disease
Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
In this study the study team proposes to investigate the efficacy of albuterol on motor function of individuals with Late Onset Pompe Disease (LOPD) who are receiving enzyme replacement therapy, given albuterol was well-tolerated…
- Trial name
- NCT01885936
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Duke University