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
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Iron Deficiency Anaemia, Iron Deficiency Anemia
Incidence of Hypophosphatemia After Treatment With Iron Isomaltoside/Ferric Derisomaltose vs Ferric Carboxymaltose in Subjects With Iron Deficiency Anaemia
The trial was designed to evaluate the incidence of unintended hypophosphatemia (low level of phosphate in the blood) in subjects with iron deficiency anaemia (IDA).
- Trial name
- NCT03238911
- Testing
- Iron isomaltoside/ferric derisomaltose, Ferric carboxymaltose
- Where
- 16 sites in United States
- Volunteers
- 123 people
- Sponsor
- Pharmacosmos A/S
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
Smoking Cessation
Provoked Craving Relief Study by NRT
This study is designed to assess the ability of the mint nicotine mouth strip to relieve provoked cigarette craving in light smokers compared to nicotine lozenge and nicotine gum.
- Trial name
- NRT (NCT01476202)
- Testing
- nicotine
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Hypoparathyroidism, Endocrine System Diseases
A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Daily in Adults With Hypoparathyroidism
During the first 26 weeks of the trial, participants were randomly assigned to one of two groups: one group received TransCon PTH and one group received placebo.
- Conditions
- Hypoparathyroidism, Endocrine System Diseases, Parathyroid Diseases
- Trial name
- PaTHway (NCT04701203)
- Testing
- TransCon PTH
- Where
- 21 sites in United States, Canada and 5 more
- Volunteers
- 84 people
- Sponsor
- Ascendis Pharma Bone Diseases A/S
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
Hypogonadism
A Study of the Effect of Topical Testosterone Replacement Therapy on Blood Pressure in Adult Male Participants With Hypogonadism
Hypogonadism is an endocrine disorder characterized by absent or deficient testosterone levels along with signs and symptoms of androgen deficiency, including delayed development or regression of sexual characteristics, impaired…
- Trial name
- NCT04274894
- Testing
- AndroGel 1.62%
- Where
- 45 sites in United States
- Volunteers
- 246 people
- Sponsor
- AbbVie
Hormone Replacement Therapy
Hormone Replacement Therapy for Use in Postmenopausal Women for Relief of Hot Flushes and Urogenital Symptoms.
Multicenter, double-blind, controlled, parallel group, randomized study to compare the clinical benefit of Estradiol acetate tablets, estradiol tablets and conjugated equine estrogen tablets, each administered orally, once daily…
- Trial name
- NCT01070979
- Testing
- Estradiol acetate, Estradiol, Conjugated equine estrogens
- Where
- 32 sites in United States
- Volunteers
- 249 people
- Sponsor
- Warner Chilcott
Glaucoma
AZARGA Transition Study in Taiwan for Patients With Uncontrolled Intraocular Pressure
The purpose of this study was to assess the safety and efficacy of switching to Azarga from prior pharmacotherapy in patients with open-angle glaucoma.
- Trial name
- NCT01484951
- Testing
- Brinzolamide 1% and timolol 0.5% fixed combination eye drops
- Where
- United States
- Volunteers
- 74 people
- Sponsor
- Alcon Research
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
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
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
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
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
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
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