Clinical trials: 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
Lymphoma
Etoposide in Treating Patients With Relapsed Non-Hodgkin's Lymphoma
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of etoposide in treating patients with relapsed…
- Trial name
- NCT00002880
- Testing
- Etoposide phosphate
- Where
- 4 sites in United States
- Volunteers
- 53 people
- Sponsor
- Alliance for Clinical Trials in Oncology
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
Evidence of Liver Transplantation, Rejection
Safety and Efficacy of Alemtuzumab in Pediatric Intestinal Transplantation
This open-label clinical trial will evaluate the safety and efficacy of Alemtuzumab (Campath, Bayer Corp., Pittsburgh) in children (0-17) and adults (18-25) receiving intestinal transplant.
- Conditions
- Evidence of Liver Transplantation, Rejection, ALEMTUZUMAB
- Trial name
- NCT01208337
- Testing
- Alemtuzumab
- Volunteers
- 23 people
- Sponsor
- University of Pittsburgh
Leukaemia (Acute Lymphoblastic), Leukaemia (Acute Myeloid)
Phase I Clinical Trial of ThINKK Adoptive Immunotherapy After Allogeneic Hematopoietic Transplantation in Children With Leukemia or Neuroblastoma
A first-in-class adoptive immunotherapy we called ThINKK, for Therapeutic Inducers of Natural Killer (NK) cell Killing, have been designed for use after hematopoietic stem cell transplantation (HSCT), where the proper stimulation…
- Conditions
- Leukaemia (Acute Lymphoblastic), Leukaemia (Acute Myeloid), Neuroblastoma, Neuroblastoma, Metastatic
- Trial name
- ThINKK-01 (NCT07518654)
- Testing
- Therapeutic Inducers of Natural Killer Killing (ThINKK)
- Where
- 2 sites in Canada
- Volunteers
- 12 people
- Sponsor
- Michel Duval
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
Malignant Solid Tumour
Hydroxychloroquine + Vorinostat in Advanced Solid Tumors
This study is an open label non randomized study of hydroxychloroquine (HCQ) with histone deacetylase (HDAC) inhibitor Vorinostat in patients with advanced solid tumors to determine the maximum tolerated dose (MTD) and to…
- Trial name
- NCT01023737
- Testing
- Hydroxychloroquine, Vorinostat
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
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.
Solid Tumors
rhEndostatin Protein Involving Pediatric Patients With Cancer
The purpose of this study is to assess the safety of rhEndostatin, to determine how much of the drug stays in the patients blood (pharmacokinetics), to assess the development of proteins in the blood that are produced by the body…
- Trial name
- NCT00165373
- Testing
- rhEndostatin
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Dana-Farber Cancer Institute
Respiratory Syncytial Virus Infections
Study to Evaluate Safety and Antiviral Activity of Doses of JNJ-53718678 in Children (>=28 Days to <=3 Years) With Respiratory Syncytial Virus Infection
The purpose of this study is to evaluate the antiviral activity, clinical outcomes, safety, tolerability, and pharmacokinetic/pharmacodynamic relationships of different oral dose levels of JNJ-53718678 in children greater than or…
- Trial name
- NCT03656510
- Testing
- JNJ-53718678
- Where
- 169 sites in United States, Argentina and 20 more
- Volunteers
- 246 people
- Sponsor
- Janssen Research & Development, LLC
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
Acute Myeloid Leukemia
Crenolanib in Combination With Sorafenib in Patients With Refractory or Relapsed Hematologic Malignancies
PRIMARY OBJECTIVE: This is a pilot study to characterize the toxicity profile, to determine the maximum tolerated dose of the combination of crenolanib and sorafenib, and to determine the feasibility of administering these drugs…
- Trial name
- NCT02270788
- Testing
- Crenolanib, Sorafenib, methotrexate, hydrocortisone and cytarabine with leucovorin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- St. Jude Children's Research Hospital