Clinical trials: Hormone Replacement
Acute Kidney Injury, Renal Failure
Analysis of Continuous Kidney Replacement Therapy (CKRT) Treatments With multiFlux Hemofilters Using Regional Citrate Anticoagulation (RCA) or Systemic Heparin Anticoagulation
The goal of this observational study is to assess the performance of the multiFlux hemofilterwith regional or systemic anticoagulation during CKRT treatments.
- Trial name
- RCA-Flux (NCT07679568)
- Testing
- multiFlux 160 hemofilter with regional heparin anticoagulation, multiFlux 160 hemofilter with RCA with regional citrate anticoagulation, multiFlux 130 hemofilter with regional heparin anticoagulation
- Where
- Germany
- Volunteers
- 62 people
- Sponsor
- Fresenius Medical Care Deutschland GmbH
Mucopolysaccharidosis I
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I. Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate…
- Trial name
- NCT05134571
- Testing
- Laronidase
- Where
- 5 sites in China
- Volunteers
- 12 people
- Sponsor
- Genzyme, a Sanofi Company
Gaucher Disease, Type 3
Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Gaucher disease has been classified into 3 clinical subtypes based on the presence or absence of neurological…
- Trial name
- NCT01685216
- Testing
- velaglucerase alfa
- Where
- 5 sites in Egypt, India and 1 more
- Volunteers
- 7 people
- Sponsor
- Shire
Smoking
Efficacy and Safety Study of Nicotine Mint Lozenge (2mg and 4mg) in Smoking Cessation
A multi-center, randomized, double-blind, placebo-controlled, 4-arm clinical study to evaluate efficacy and safety of nicotine lozenge (2mg and 4mg) in smoking cessation in adult cigarette smokers who are motivated to quit…
- Trial name
- NCT00985985
- Testing
- Nicotine
- Volunteers
- 723 people
- Sponsor
- GlaxoSmithKline
Renal Failure, CVVHD
Analysis of Paediatric Treatments With multiFiltratePRO
This clinical study evaluates the performance of the MultifiltratePro in pediatric mode for Continuous Venovenous Hemodiafiltration (CVVHD) in children.
- Trial name
- ECHOped (NCT06603324)
- Testing
- CVVHD treatment in paediatric mode with multiFiltratePRO
- Where
- 2 sites in Czechia, Germany
- Volunteers
- 43 people
- Sponsor
- Fresenius Medical Care Deutschland GmbH
Hemophilia B
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events.
- Trial name
- Honey-B (NCT05568459)
- Testing
- Non-Interventional
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Regeneron Pharmaceuticals
Acute Kidney Injury
Nafamostat Efficacy in Phase 3 Registrational CRRT Study
A prospective, randomized, placebo-controlled clinical study to investigate the safety and efficacy of Niyad (nafamostat mesylate) for anticoagulation of extracorporeal blood circulating through a dialysis filter in patients…
- Trial name
- NEPHRO (NCT06150742)
- Testing
- Niyad (nafamostat mesylate)
- Where
- 10 sites in United States
- Volunteers
- 70 people
- Sponsor
- Talphera, Inc
Hunter Syndrome
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do…
- Trial name
- NCT00882921
- Testing
- Idursulfase
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 26 people
- Sponsor
- Shire
Muscular Atrophy, Spinal
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)
This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in…
- Trial name
- OFELIA (NCT05073133)
- Testing
- OAV101
- Where
- 5 sites in Argentina, Brazil
- Volunteers
- 16 people
- Sponsor
- Novartis Pharmaceuticals
Alpha 1-Antitrypsin Deficiency
Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)
The goal of this trial was to explore the utility of evaluating emphysema progression through CT scans measuring lung density during a 2 year period of weekly infusions of either placebo or human alpha-1-antitrypsin (AAT…
- Trial name
- NCT00263887
- Testing
- Alpha1-Proteinase Inhibitor (Human), Albumin (Human) 20%, United States Pharmacopeia (USP)
- Where
- 3 sites in Denmark, Sweden and 1 more
- Volunteers
- 77 people
- Sponsor
- Grifols Therapeutics LLC
Hunter Syndrome, Mucopolysaccharidosis II
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II, MPS II
- Trial name
- NCT00607386
- Testing
- Idursulfase
- Where
- 3 sites in Brazil, Poland and 1 more
- Volunteers
- 28 people
- Sponsor
- Shire
Smoking Cessation
Safety and Efficacy Study of the NRT CQSS2 System for Smoking Cessation in Treatment-Seeking Subjects
Transdermal nicotine delivery systems are a cigarette smoking cessation aid designed to deliver nicotine into the systemic circulation via the skin.
- Trial name
- NCT03178422
- Testing
- CQSS2, NicoDerm CQ Patch
- Where
- 5 sites in United States
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- Chrono Therapeutics, Inc.
Continuous Renal Replacement Therapy, Pulmonary Edema
C2Rx Hemofiltration Treatment in Severe or Critically Ill Adults With COVID-19
The clinical trial will evaluate the short term and extended impact on on respiration, pulmonary function and cardiovascular function in C2Rx treatment verse Standard of Care (SOC) in critically ill adults with COVID-19…
- Conditions
- Continuous Renal Replacement Therapy, Pulmonary Edema, COVID-19
- Trial name
- NCT04537975
- Testing
- C2Rx, Standard of Care (SOC)
- Sponsor
- SeaStar Medical
Mucopolysaccharidosis IVA, Morquio A Syndrome
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01697319
- Testing
- BMN 110
- Where
- 7 sites in United States, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- BioMarin Pharmaceutical
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Hypogonadism
Study of Serum Testosterone Levels in Non-dosed Females After Secondary Exposure
To further characterize the transfer potential of marketed testosterone gel and new 1.62% formulation.
- Trial name
- NCT00998933
- Testing
- Testosterone gel 1.62%
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Solvay Pharmaceuticals
Renal Insufficiency
Registry of Pre-mixed Solutions in Critically Ill Patients on the Continuous Renal Replacement Therapy
This is a multicenter, prospective, registry study. By collecting relevant demographic information and clinical data of patients receiving continuous renal replacement therapy (CRRT) with pre-mixed solutions, the clinical…
- Trial name
- PrioR (NCT06983509)
- Testing
- Regiocit, Biphozyl, Prismasol 2
- Where
- 10 sites in China
- Volunteers
- 228 people
- Sponsor
- Vantive Health LLC
NGLY1 Deficiency
Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency
A non-randomized, open-label, Phase 1/2/3 study of a single intracerebroventricular (ICV) administration of a gene replacement therapy (GS-100) in participants who are 2 to 18 years old with NGLY1 Deficiency.
- Trial name
- NCT06199531
- Testing
- GS-100
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Grace Science, LLC
MPS IV A, Mucopolysaccharidosis IVA
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS…
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Trial name
- NCT00787995
- Where
- 15 sites in United States, Argentina and 8 more
- Volunteers
- 353 people
- Sponsor
- BioMarin Pharmaceutical
Iron Deficiency Anaemia, Iron Deficiency Anemia
Iron Isomaltoside/Ferric Derisomaltose vs Iron Sucrose for the Treatment of Iron Deficiency Anemia (IDA)
Evaluate safety and efficacy of iron isomaltoside/ferric derisomaltose (Monofer®/Monoferric®) compared with iron sucrose (Venofer®), in subjects diagnosed with IDA.
- Trial name
- NCT02940886
- Testing
- Iron isomaltoside/ferric derisomaltose, Iron sucrose
- Where
- 114 sites in United States
- Volunteers
- 1,512 people
- Sponsor
- Pharmacosmos A/S