Clinical trials: Hormone Replacement Therapy
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
Primary Immune Deficiency Disorder
Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)
The objective of this study is to assess the long-term safety, tolerability, and efficacy of IgPro20 in subjects with primary immunodeficiency (PID) as a follow-up to the pivotal study ZLB06_002CR (NCT01199705).
- Trial name
- NCT01458171
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 23 people
- Sponsor
- CSL Behring
Mucopolysaccharidosis IVA, Morquio A Syndrome
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01609062
- Testing
- BMN 110
- Where
- 11 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- BioMarin Pharmaceutical
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
Glaucoma
Assessing the Efficacy and Tolerability of AZARGA® (Brinzolamide 1%/Timolol 0.5% Fixed Combination) as Replacement Therapy in Patients on Brimonidine 0.2%/Timolol 0.5% Fixed Combination Therapy (COMBIGAN®) in Latin America
The purpose of this study was to assess the efficacy and tolerability of changing to AZARGA® from prior COMBIGAN® pharmacotherapy in participants with open-angle glaucoma or ocular hypertension.
- Trial name
- NCT01518244
- Testing
- Brinzolamide/timolol maleate fixed combination
- Volunteers
- 50 people
- Sponsor
- Alcon Research
Physical Frailty, Hip Fracture
Exercise and Testosterone Therapy in Elderly Men With Physical Frailty
The primary aim of this study is to determine, in hypogonadal older men with physical frailty, whether exercise training combined with testosterone replacement therapy can improve skeletal muscle strength, and lean mass, to a…
- Conditions
- Physical Frailty, Hip Fracture, Elective Hip Replacement, Hypogonadism
- Trial name
- NCT00345969
- Testing
- Transdermal Testosterone gel (1%), Supervised exercise training
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Washington University School of Medicine
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency
Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
This was the first clinical study of SBC-102 (sebelipase alfa) for the treatment of Lysosomal Acid Lipase (LAL) Deficiency. It was an open-label dose escalation study in adult participants with liver dysfunction due to LAL…
- Conditions
- Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Trial name
- NCT01307098
- Testing
- Sebelipase alfa 0.35 mg/kg, Sebelipase alfa 1 mg/kg, Sebelipase alfa 3 mg/kg
- Where
- 7 sites in United States, Czechia and 2 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pompe Disease
Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
Funding Source- FDA OOPD The purpose of this study is to investigate the safety and efficacy of clenbuterol on motor function in individuals with late-onset Pompe disease (LOPD) who are treated with enzyme replacement therapy…
- Trial name
- NCT01942590
- Testing
- Clenbuterol
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Dwight Koeberl, M.D., Ph.D.
Acute Kidney Injury
Safety and Efficacy of a Selective Cytopheretic Device (SCD) in Pediatric Patients With Acute Kidney Injury (AKI).
The SCD (Selective Cytopheretic Device) is an extracorporeal device used as an adjunct to renal replacement therapy (RRT) to improve the outcomes of pediatric patients with acute kidney injury (AKI).
- Trial name
- SCD-PED-01 (NCT02820350)
- Testing
- SCD-F40
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- CytoPherx, Inc
Bladder Cancer, Cervical Cancer
Counseling and Nicotine Replacement Therapy in Helping Adult Smokers Quit Smoking
RATIONALE: Stop-smoking plans, including counseling and nicotine replacement therapy, may help smokers quit smoking. It is not yet known whether counseling and the nicotine lozenge is more effective than counseling and the…
- Conditions
- Bladder Cancer, Cervical Cancer, Esophageal Cancer, Gastric Cancer
- Trial name
- NCT00365508
- Testing
- nicotine lozenge, nicotine patch
- Where
- 10 sites in United States
- Volunteers
- 642 people
- Sponsor
- Fox Chase Cancer Center
Septic Shock
Albumin Replacement Therapy in Septic Shock
Albumin is a key regulator of fluid distribution within the extracellular space and possesses several properties beyond its oncotic activity, including binding and transport of several endogenous molecules, anti-inflammatory and…
- Trial name
- ARISS (NCT03869385)
- Testing
- Albutein® 200 g/L or Plasbumin® 20
- Where
- 27 sites in Germany
- Volunteers
- 440 people
- Sponsor
- Jena University Hospital
Growth Hormone Deficiency, Endocrine System Diseases
A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency
A 38-week dosing trial of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus placebo-control.
- Conditions
- Growth Hormone Deficiency, Endocrine System Diseases, Hormone Deficiency
- Trial name
- foresiGHt (NCT04615273)
- Testing
- Lonapegsomatropin, Somatropin
- Where
- 113 sites in United States, Armenia and 22 more
- Volunteers
- 264 people
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
HIV Prevention, Opioid-Related Disorders
Effectiveness of Opiate Replacement Therapy Administered Prior to Release From a Correctional Facility - 1
Much of the HIV/AIDS epidemic is driven by transmission from or to persons addicted to opiates. Many of these individuals pass through a correctional setting each year, creating an opportunity for linkage to substance abuse…
- Trial name
- NCT00142935
- Testing
- Pre-release Initiation of MMT, Post Release Initiation of MMT., Standard of Care Plus
- Where
- United States
- Volunteers
- 87 people
- Sponsor
- The Miriam Hospital
MPS IV A
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
- Trial name
- NCT01275066
- Testing
- BMN 110 Weekly, BMN 110 Every Other Week
- Where
- 28 sites in United States, Argentina and 15 more
- Volunteers
- 177 people
- Sponsor
- BioMarin Pharmaceutical
Open-Angle Glaucoma, Ocular Hypertension
Efficacy of Changing to DUOTRAV® From Prior Therapy
The purpose of this study was to assess the efficacy and tolerability of changing to DUOTRAV® from prior bimatoprost 0.03%/timolol 0.5% pharmacotherapy in subjects with open-angle glaucoma or ocular hypertension having…
- Conditions
- Open-Angle Glaucoma, Ocular Hypertension, Pigment Dispersion Glaucoma
- Trial name
- NCT01327599
- Testing
- Travoprost 0.004%+Timolol 0.5% ophthalmic solution
- Volunteers
- 60 people
- Sponsor
- Alcon Research
Replacement Therapy, Renal
Pharmacokinetics of Selected Antiinfectives During Sustained Low-efficiency Dialysis (SLED)
In a prospective, non-interventional, monocentric observational study the pharmacokinetic properties of selected antiinfective drugs during sustained low-efficiency dialysis (SLED) will be analyzed.
- Trial name
- PhA-SLED (NCT02287493)
- Testing
- Pharmacokinetic Analysis
- Where
- Germany
- Volunteers
- 35 people
- Sponsor
- Universitätsklinikum Hamburg-Eppendorf
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
Sarcopenia, Hypogonadism
TOM: Testosterone in Older Men With Sarcopenia
The purpose of this study is to determine whether testosterone replacement in older men with low testosterone levels will increase muscle strength, improve physical performance and overall sense of well being, and reduce fatigue.
- Conditions
- Sarcopenia, Hypogonadism, Muscular Diseases
- Trial name
- NCT00240981
- Testing
- Topical testosterone gel 1% (active formulation)
- Where
- 2 sites in United States
- Volunteers
- 209 people
- Sponsor
- Boston Medical Center
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
Hyperuricemia
Lowering Serum Uric Acid to Prevent Acute Kidney Injury
Acute kidney injury is associated with a rise in serum uric acid during cardiovascular surgery and can cause poor blood flow to the kidneys making them vulnerable to kidney injury.
- Trial name
- RasbAKI (NCT00756964)
- Testing
- Rasburicase
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- University of Florida