Clinical trials: Pharmacokinetics and Pharmacodynamics
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD)/preliminary efficacy of…
- Trial name
- Unite-CNM (NCT04033159)
- Testing
- DYN101
- Where
- 8 sites in Belgium, Denmark and 4 more
- Volunteers
- 14 people
- Sponsor
- Dynacure
Obese Adolescents, Obesity
A Study of XW003 Injection in Chinese Adolescents With Obesity
The aim of the study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of XW003 injection in Chinese adolescents with obesity
- Trial name
- NCT07143227
- Testing
- XW003 injection
- Where
- China
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Hangzhou Sciwind Biosciences Co., Ltd.
Sepsis, Antibiotic Reaction
PK/PD of Ceftazidime Avitbatan Sodium in Children With Severe Infection
Ceftazidime avitbatan sodium is a broad-spectrum antibiotic with an antimicrobial spectrum covering resistant gram-negative bacteria. Its use in pediatric intensive care for severe infections is not unusual.
- Trial name
- NCT04961580
- Testing
- Ceftazidime Avitbatan Sodium
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Children's Hospital of Fudan University
Myelodysplastic Syndrome, Leukemia, Myelomonocytic, Juvenile
Study With Azacitidine in Pediatric Subjects With Newly Diagnosed Advanced Myelodysplastic Syndrome (MDS) and Juvenile Myelomonocytic Leukemia (JMML)
Indication Treatment of pediatric subjects with newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT).
- Trial name
- NCT02447666
- Testing
- Azacitidine
- Where
- 39 sites in Austria, Belgium and 11 more
- Volunteers
- 28 people
- Sponsor
- Celgene
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
Leukemia, Acute Lymphoblastic, Leukemia
Vincristine PK and PD in the AYA Population Compared to Younger Children
The trial is to determine if a difference exists in the way that adolescents and young adults metabolize the chemotherapy agent vincristine compared to younger children.
- Trial name
- NCT02360930
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital Los Angeles
Healthy
Study of ALXN1820 in Healthy Adult Participants
This is a Phase 1, randomized, double-blind, placebo-controlled single and multiple ascending dose study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of ALXN1820…
- Trial name
- NCT04631562
- Testing
- ALXN1820 SC, ALXN1820 IV
- Where
- 3 sites in Australia, United Kingdom
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Healthy Volunteers, Propionic Acidemia
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.
- Conditions
- Healthy Volunteers, Propionic Acidemia, Methylmalonic Acidemia, Organic Acidemia
- Trial name
- NCT04836494
- Testing
- BBP-671
- Where
- 3 sites in United States
- Volunteers
- 79 people, healthy volunteers welcome
- Sponsor
- CoA Therapeutics, Inc., a BridgeBio company
Respiratory Syncytial Virus Infections
A Study of ALS-008176 in Infants Hospitalized With RSV
This study will assess the safety, tolerability, pharmacokinetics (PK) and anti-viral effect of single and multiple doses of ALS-008176 in infants hospitalized with respiratory syncytial virus (RSV)
- Trial name
- NCT02202356
- Testing
- ALS-008176
- Where
- 77 sites in United States, Australia and 12 more
- Volunteers
- 183 people
- Sponsor
- Alios Biopharma Inc.
Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B
A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously
This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who…
- Trial name
- CL01-T (NCT02618512)
- Testing
- SBC-103
- Where
- United Kingdom
- Volunteers
- 3 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Sepsis
Prolonged Intravenous Infusion of β-lactam Antibiotics in Early Septic Patients
The prolonged β-lactam Antibiotics intravenous infusion strategy has emerged as the standard treatment for sepsis despite its unknown efficacy.
- Trial name
- PROBES (NCT05024565)
- Testing
- prolonged intravenous infusion of β-lactams Antibiotics
- Volunteers
- 2,600 people
- Sponsor
- Qilu Hospital of Shandong University
Intubated and Mechanically Ventilated Pediatric Subjects
Pharmacokinetics and Pharmacodynamics of Dexmedetomidine in Pediatrics Subjects
The objective of this study is to characterize the pharmacokinetic and pharmacodynamic profile of dexmedetomidine administered as an intravenous loading dose followed by a continuous intravenous infusion in pediatric subjects…
- Trial name
- NCT00652028
- Testing
- Dexmedetomidine, midazolam; fentanyl
- Where
- 9 sites in United States, Guatemala
- Volunteers
- 69 people
- Sponsor
- Hospira, now a wholly owned subsidiary of Pfizer
Venous Thromboembolism (VTE), Pulmonary Embolism
Hokusai Study in Pediatric Patients With Confirmed Venous Thromboembolism (VTE)
This is an event driven Phase 3, prospective, randomized, open-label, blinded endpoint evaluation (PROBE) parallel group study in subjects with confirmed VTE.
- Conditions
- Venous Thromboembolism (VTE), Pulmonary Embolism, Deep Vein Thrombosis (DVT)
- Trial name
- NCT02798471
- Testing
- Edoxaban, Standard of Care
- Where
- 140 sites in United States, Argentina and 32 more
- Volunteers
- 290 people
- Sponsor
- Daiichi Sankyo
Thromboembolism
Study to Evaluate a Single Dose of Apixaban in Pediatric Participants at Risk for a Thrombotic Disorder
CV185118 is a single dose Apixaban PK/PD study in pediatric participants. The objective of this study is primarily to study the PK/PD of Apixaban in pediatric participants at risk for thrombosis
- Trial name
- NCT01707394
- Testing
- Apixaban
- Where
- 27 sites in United States, Australia and 3 more
- Volunteers
- 49 people
- Sponsor
- Bristol-Myers Squibb
Neonatal Herpes Simplex Infection
Valacyclovir in Neonatal Herpes Simplex Virus Disease
This is an open-label, single center, pharmacokinetic (PK) study to assess valacyclovir pharmacokinetics and pharmacodynamics in neonates and compare to the pharmacokinetics and pharmacodynamics of the standard of care treatment…
- Trial name
- NCT04448392
- Testing
- Valacyclovir
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University of Alabama at Birmingham
Osteochondrodysplasia
Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With Achondroplasia
This is a Phase 2, open-label, multicenter, study to evaluate safety, tolerability and efficacy of SAR442501 in children from birth up to 12 years of age with Achondroplasia.
- Trial name
- upreACH-2 (NCT06067425)
- Testing
- SAR442501
- Where
- 9 sites in Australia, China and 3 more
- Volunteers
- 16 people
- Sponsor
- Sanofi
Cushing Syndrome
Pharmacokinetic (PK), Pharmacodynamic (PD) and Tolerability of Osilodrostat in Pediatric Patients With Cushing's Syndrome
Multicenter, open-label, non-comparative study to evaluate the pharmacokinetics, pharmacodynamics, and tolerability of osilodrostat in children and adolescent patients with Cushing's syndrome.
- Trial name
- NCT03708900
- Testing
- LCI699
- Where
- 15 sites in United States, Belgium and 5 more
- Volunteers
- 12 people
- Sponsor
- RECORDATI GROUP
Juvenile Idiopathic Arthritis
A Study of Subcutaneously Administered Tocilizumab in Participants With Systemic Juvenile Idiopathic Arthritis
This open-label, multicenter study will evaluate the pharmacokinetics, pharmacodynamics, and safety of subcutaneously administered tocilizumab in participants with Systemic Juvenile Idiopathic Arthritis (sJIA).
- Trial name
- NCT01904292
- Testing
- Tocilizumab
- Where
- 42 sites in United States, Argentina and 10 more
- Volunteers
- 52 people
- Sponsor
- Hoffmann-La Roche
Congenital Adrenal Hyperplasia
Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia
This is a Phase 1, multicenter, open-label, single-dose study to evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-77860 in subjects with congenital adrenal hyperplasia (CAH).
- Trial name
- NCT02349503
- Testing
- NBI-77860
- Where
- 2 sites in United States
- Sponsor
- Neurocrine Biosciences
Depression - Major Depressive Disorder, Depressive Episodes
TDM-Guided Treatment With SSRIs in Hospitalized Adults and Children
Depression in childhood and adolescence is a serious and increasing public-health problem associated with profound short- and long-term consequences, including impaired social and educational functioning, increased risk of…
- Trial name
- NCT07478796
- Testing
- Fluoxetine, Escitalopram, Sertralin
- Where
- Poland
- Volunteers
- 180 people
- Sponsor
- Poznan University of Medical Sciences