Clinical trials: Hormone Replacement
Hypoplasminogenemia
A Treatment Protocol for Expanded Access Administration of Prometic Plasminogen Due to Closure of Clinical Trial
The clinical trial is no longer enrolling and is currently closing and Prometic will continue to provide Plasminogen (Human) under a treatment protocol to subjects in the United States (US) with hypoplasminogenemia requiring…
- Trial name
- NCT03642691
- Testing
- Ryplazim
- Where
- 3 sites in United States
- Sponsor
- Prometic Biotherapeutics, Inc.
Spinal Cord Compression, Mucopolysaccharidosis I
Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
This is a one-year extension study of the use of laronidase into the spinal fluid to treat spinal cord compression in mucopolysaccharidosis I.
- Conditions
- Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00786968
- Testing
- laronidase
- Where
- 2 sites in United States, Finland
- Volunteers
- 3 people
- Sponsor
- Patricia I. Dickson, M.D.
Surfactant Deficiency Syndrome Neonatal, Respiratory Distress Syndrome in Premature Infant
Fast Assessment of Surfactant Deficiency in Preterm Infants to Speed up Treatment - Validation Study
The aim is to re-validate a FTIR spectroscopy test for measuring lung maturity/Respiratory Distress Syndrome (RDS) before conducting a RCT using this test to guide surfactant treatment of preterm infants.
- Trial name
- FAST2 (NCT05615428)
- Testing
- LS-ratio
- Where
- 3 sites in Denmark
- Volunteers
- 68 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Primary Immunodeficiency Diseases (PID)
A Study About How TAK-881 is Processed by the Body and Side Effects in People With Primary Immunodeficiency Diseases
The main aim of this study is to evaluate the PK, safety, tolerability and immunogenicity of subcutaneous (SC) administration of TAK-881 in adult and pediatric participants with PIDD and compare them to HYQVIA in participants 16…
- Trial name
- NCT05755035
- Testing
- TAK-881, HYQVIA
- Where
- 32 sites in United States, Czechia and 6 more
- Volunteers
- 65 people
- Sponsor
- Takeda
Prostate Cancer
Testosterone in Castration-Resistant Prostate Cancer
Primary Objective: 1. To assess the prostate-specific antigen (PSA)-response (50% decline) to Testosterone Replacement Therapy (TRT) in men with "intermediate and good-risk" Castration-Resistant Prostate Cancer (CRPC).
- Trial name
- NCT00577980
- Testing
- Testosterone
- Sponsor
- M.D. Anderson Cancer Center
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
Shiga Toxin-Producing Escherichia Coli (E. Coli) Infection, Hemolytic-Uremic Syndrome
Hyperhydration in Children With Shiga Toxin-Producing E. Coli Infection
The objective of this study is to determine if early high volume intravenous fluid administration (hyperhydration) may be effective in mitigating or preventing complications of shiga toxin-producing E.
- Trial name
- HIKO-STEC (NCT05219110)
- Testing
- Infusion of 200% maintenance fluids as balanced crystalloid IV solution, Oral fluids; infusion of up to 110% maintenance fluids as balanced crystalloid IV solution
- Where
- 26 sites in United States, Canada
- Volunteers
- 1,040 people
- Sponsor
- University of Calgary
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
Cerebral Vein Thrombosis
Rivaroxaban vs. Warfarin in CVT Treatment
Cerebral venous thrombosis (CVT) is an uncommon venous-type of stroke tends to affect younger patients with somewhat different risk factors and much better outcome compared to arterial strokes.
- Trial name
- RWCVT (NCT04569279)
- Testing
- Rivaroxaban, Warfarin
- Where
- Syria
- Volunteers
- 71 people
- Sponsor
- Damascus University
Postmenopause
Use of the Synthetic Hormone CDB-2914 in Treating Symptoms of Menopause
This study will examine the safety and effectiveness of a synthetic hormone, CDB-2914, for treating symptoms of menopause. It will compare the effects of estrogen and CDB-2914 with those of estrogen and progesterone in…
- Trial name
- NCT00009659
- Testing
- CDB 2914
- Where
- United States
- Volunteers
- 58 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Gaucher Disease, Type III
Eliglustat on Gaucher Disease Type IIIB
Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.
- Trial name
- NCT03519646
- Testing
- Eliglustat
- Where
- Taiwan
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Mucopolysaccharidosis IV A, Morquio A Syndrome
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This Phase 3 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
- Conditions
- Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01415427
- Testing
- BMN 110 - Weekly, BMN 110 - Every Other Week
- Where
- 43 sites in United States, Argentina and 18 more
- Volunteers
- 173 people
- Sponsor
- BioMarin Pharmaceutical
Fabry Disease
China Post-marketing Surveillance (PMS) Study of Fabrazyme®
This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.
- Trial name
- NCT05054387
- Testing
- Agalsidase beta
- Where
- 6 sites in China
- Volunteers
- 22 people
- Sponsor
- Genzyme, a Sanofi Company
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Renal Replacement Therapies
Evaluating the Usability of New Dialysis Bags
Objective: To compare the usability, efficiency, and ergonomics of new dialysis bags with current bags from the nursing perspective in ICU settings. Study Type: Prospective, single-center observational study.
- Trial name
- NCT07426575
- Where
- Netherlands
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Erasmus Medical Center
Replacement Therapy in Preterm Infants
Efficacy and Safety of Bile Salt Stimulated Lipase (BSSL) as Replacement Therapy in Infant Formula for Preterm Infants
The purpose of this study is to evaluate the efficacy in preterm infants following treatment with BSSL when administered in infant formula.
- Trial name
- NCT00658905
- Testing
- rhBSSL
- Where
- 6 sites in Italy
- Volunteers
- 33 people
- Sponsor
- Swedish Orphan Biovitrum
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
Lysosomal Acid Lipase Deficiency
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid…
- Trial name
- ARISE (NCT01757184)
- Testing
- Sebelipase Alfa
- Where
- 40 sites in United States, Argentina and 13 more
- Volunteers
- 66 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Menopause, Postmenopausal Bleeding
Observational Study to Investigate the Occurrence of Bleeding in Postmenopausal Women Treated With Estradiol/NETA for 12 Months
This study is conducted in Europe. The primary aim of this observational study is to investigate the occurrence of bleeding in women taking 0.5 mg estradiol and 0.1 mg norethisterone acetate (NETA) for 12 months.
- Trial name
- NCT01076621
- Testing
- 0.5 mg estradiol / 0.1 mg norethisterone acetate (NETA)
- Where
- 2 sites in Norway, Sweden
- Volunteers
- 176 people
- Sponsor
- Novo Nordisk A/S
Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II)
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Hypothesis: Children diagnosed with a lysosomal disease will exhibit developmental, adaptive, and behavioral strengths and difficulties depending upon 1) biomedical risk factors (i.e.
- Conditions
- Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI)
- Trial name
- NCT01938014
- Where
- 3 sites in United States
- Volunteers
- 19 people
- Sponsor
- University of Chicago