Clinical trials: Hormone Replacement
Renal Replacement Therapy
A Clinical Study to Evaluate the Efficacy, Safety and PK/PD Profiles of Anticoagulation of HSK36273 for Injection in Continuous Renal Replacement Therapy Subjects
This study is divided into Stage I and Stage II. It is planned to include 156 subjects who need CRRT treatment for more than 48 hours.
- Trial name
- NCT05602129
- Testing
- HSK36273, Heparin sodium
- Where
- China
- Volunteers
- 156 people
- Sponsor
- Haisco Pharmaceutical Group Co., Ltd.
Spinal Muscular Atrophy
AveXis Managed Access Program Cohort for Access to AVXS-101
The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.
- Trial name
- NCT03955679
- Testing
- AVXS-101
- Where
- United States
- Sponsor
- United BioSource, LLC
Hunter Syndrome
An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV)…
- Trial name
- NCT01506141
- Testing
- Idursulfase-IT, Elaprase
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- Takeda
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Hypogonadism
Quality of Life With TESTIM®
The present non-interventional study is to document the testosterone levels in patients with clinical symptoms such as loss of sexual desire indicating hypogonadism (testosterone deficiency syndrome).
- Trial name
- LIFE (NCT01148433)
- Where
- 64 sites in Germany
- Volunteers
- 157 people
- Sponsor
- Ferring Pharmaceuticals
Fibrosing Colonopathy
Long-term Study in US Cystic Fibrosis Patients Receiving Digestive Enzyme Supplements to Assess Narrowing of the Large Intestine Causing Adverse Intestinal Symptoms (Fibrosing Colonopathy)
This is a long-term study in cystic fibrosis patients who are participating in the Cystic Fibrosis Patient Registry to assess the occurrence and risk factors for a rare bowel disorder called fibrosing colonopathy (narrowing of…
- Trial name
- NCT01652157
- Testing
- ULTRESA, PANCREAZE, CREON
- Where
- 123 sites in United States
- Volunteers
- 17 people
- Sponsor
- AbbVie
Mucopolysaccharidosis I
Immune Tolerance Study With Aldurazyme® (Laronidase)
The purpose of this study is to see if treatment with an antigen-specific immunosuppressive can decrease or stop an antibody response to laronidase (Aldurazyme®) during enzyme replacement therapy with laronidase in severe…
- Trial name
- NCT00741338
- Testing
- Laronidase, Cyclosporine A (CsA), Azathioprine (Aza)
- Where
- 2 sites in Brazil, Russia
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Anemia, Renal Failure, Chronic Renal Failure
Effects of Early Correction of Anemia in Patients With Chronic Renal Insufficiency
The purpose of the study is to assess the effect of an early and complete correction of anemia after treatment with epoetin alfa on the rate of progression of chronic renal failure (which involves improper functioning of the…
- Trial name
- NCT00312871
- Testing
- Epoetin alfa
- Volunteers
- 217 people
- Sponsor
- Janssen Cilag S.A.S.
Growth Hormone Disorders
A Post Marketing Surveillance Study for NutropinAq® in Adults With Growth Hormone Deficiency
This study is a multicenter, open-label, observational, post marketing surveillance program to collect information on the use of NutropinAq® in adults suffering from Growth Hormone Deficiency in Germany, Italy, and United Kingdom
- Trial name
- NCT00455884
- Where
- United Kingdom
- Volunteers
- 546 people
- Sponsor
- Ipsen
Hypophosphatasia
Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
This is an open-label, dose-escalating study to assess safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and immunogenicity of ALXN1850 when given intravenous (IV) and subcutaneous (SC) to adults with HPP.
- Trial name
- NCT04980248
- Testing
- ALXN1850
- Where
- 4 sites in United States
- Volunteers
- 15 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Prevention of Thromboembolic Events
Study to Assess the Safety, Tolerability, Effects on the Body, Absorption, Distribution and Elimination of 25 mg BAY2433334 in Renal Impairment Including Renal Replacement Therapy ("Dialysis")
BAY2433334 is under clinical development for prevention of complications in diseases such as heart attack, irregular heart beat or stroke which can arise by formation of blood clots elsewhere in the body and travels through the…
- Trial name
- NCT04510987
- Testing
- BAY2433334 single dose in treatment groups 1-4 and 6 as well as on the dialysis free day of treatment 5, BAY2433334 on dialysis treatment day
- Where
- Germany
- Volunteers
- 48 people
- Sponsor
- Bayer
Gaucher Disease, Type 3
An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
The purpose of this study is to evaluate the efficacy and safety of autologous hemotopoietic stem cell (HSC) gene therapy, AVR-RD-02, compared to enzyme replacement therapy, for the treatment of Gaucher disease Type 3 in male and…
- Trial name
- NCT05815004
- Testing
- Gene therapy, Enzyme Replacement Agent
- Sponsor
- AVROBIO
Hunter Syndrome
Safety and Efficacy of Hunterase
The objective of this study is to determine the safety and efficacy of once weekly dosing of idursulfase-beta 0.5mg/kg administered by intravenous(IV) infusion for Hunter syndrome patients < 6 years old.
- Trial name
- GC1111 (NCT01645189)
- Testing
- Hunterase
- Where
- South Korea
- Volunteers
- 6 people
- Sponsor
- Green Cross Corporation
Adult Growth Hormone Deficiency
Versartis International Trial in Adults With Long-Acting Growth Hormone
A Phase 2, open-label dose-finding safety study of individualized monthly VRS-317 dosing for five months in adults with GHD.
- Trial name
- VITAL (NCT02526420)
- Testing
- somavaratan
- Where
- 18 sites in United States, Australia and 2 more
- Volunteers
- 36 people
- Sponsor
- Versartis Inc.
Hypogonadism, Paraplegia
Efficacy of Nebido on Bone Mineral Density (BMD) in Hypogonadal Paraplegic Patients With Confirmed Osteoporosis
Both conditions hypogonadism and immobilisation (paraplegia) may contribute or lead to decreased bone mineral density resp osteoporosis.
- Conditions
- Hypogonadism, Paraplegia, Bone Density, Osteoporosis
- Trial name
- NCT00838838
- Testing
- Testosterone Undeconate (Nebido-R, BAY86-5037)
- Where
- Germany
- Volunteers
- 26 people
- Sponsor
- Bayer
MPS IV A
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed…
- Trial name
- NCT00884949
- Testing
- BMN 110
- Where
- 3 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Late-onset Pompe Disease
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
The purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) subjects receiving standard-of-care enzyme replacement…
- Trial name
- NCT03347253
- Where
- 21 sites in United States, Australia and 2 more
- Volunteers
- 12 people
- Sponsor
- Amicus Therapeutics
Major Bleeding
A Study to Describe the Characteristics, Health Care Interventions and Outcomes of Patients With Major Bleedings in the Presence of Factor Xa Inhibitor Treatment
REVERXaL study aims to increase the understanding of the patient characteristics, bleeding presentation, health care interventions provided, and the clinical as well as self-reported health outcomes of patients with major…
- Trial name
- REVERXaL (NCT06147830)
- Testing
- None (Observational study)
- Where
- 75 sites in United States, Germany and 2 more
- Volunteers
- 2,202 people
- Sponsor
- AstraZeneca
Growth Hormone Disorder, Adult Growth Hormone Deficiency
Cardiovascular Effects on Growth Hormone Therapy in Adults With Growth Hormone Deficiency
This trial is conducted in Europe. The aim of this trial is to investigate the effect of growth hormone on left ventricle morphology and function (systolic and diastolic).
- Trial name
- NCT01698944
- Testing
- somatropin
- Where
- Greece
- Volunteers
- 7 people
- Sponsor
- Novo Nordisk A/S
Acute Kidney Injury, Renal Failure
Retrospective Data Analysis of CKRT (Continuous Kidney Replacement Therapy) Treatments in Adult Mode With multiFiltratePRO
The multiFiltratePRO is a device for extracorporeal blood purification treatments. In this retrospective analysis, the treatments of patients who received at least one treatment with the investigational device as mentioned…
- Trial name
- CKRT-AKI (NCT06236984)
- Testing
- multiFiltratePRO with all CKRT treatment modes
- Where
- 3 sites in Germany
- Volunteers
- 80 people
- Sponsor
- Fresenius Medical Care Deutschland GmbH