Clinical trials: Single Dose
Systemic Lupus Erythematosus (SLE), Lupus Nephritis (LN)
A Phase IIa Study of ICG318 CAR-T in Refractory Systemic Lupus Erythematosus With or Without Lupus Nephritis
This Phase IIa, open-label, single-arm study includes an initial Safety Run-In followed by dose expansion to evaluate the safety, tolerability, cellular kinetics, pharmacodynamics, and preliminary clinical activity of a single…
- Trial name
- NCT07328581
- Testing
- ICG318, BCMA-CD19-IL-15/IL-15 sushi Compound CAR T following lymphodepletion
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- iCell Gene Therapeutics
Hematologic Malignancies
Study of SLS009 (Formerly GFH009) a Potent Highly Selective CDK9 Inhibitor in Patients With Hematologic Malignancies and High-Risk Newly Diagnosed AML
SLS009 (formerly GFH009) is a potent and highly selective CDK9 inhibitor. In this study the safety, tolerability, and antitumor activity of single agent SLS009 are assessed in two dose escalation groups (Group 1 in patients with…
- Trial name
- NCT04588922
- Testing
- SLS009, venetoclax, azacitidine
- Where
- 32 sites in United States, China
- Volunteers
- 160 people
- Sponsor
- Sellas Life Sciences Group
Recessive Dystrophic Epidermolysis Bullosa
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Protocol PTR-01-001 is a Phase 1/2 study of PTR-01. The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an 8-week Follow-up Period.
- Trial name
- PTR-01-001 (NCT03752905)
- Testing
- PTR-01, Normal saline
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Phoenix Tissue Repair, Inc.
Migraine in Children, Migraine
A Study in Children and Young People With Migraine to Learn What the Body Does to Eptinezumab
The purpose of the study is to investigate how the body absorbs, distributes, and get rid of eptinezumab when given directly into a vein.
- Trial name
- NCT04537429
- Testing
- Eptinezumab
- Where
- 9 sites in United States
- Volunteers
- 28 people
- Sponsor
- H. Lundbeck A/S
Gram Positive Bacterial Infection
Pharmacokinetics (PK) and Safety Evaluation of Daptomycin in Children Ages 3-24 Months With Proven or Suspected Gram-positive Infections or Peri-Operative Subjects Receiving Prophylactic Antibiotics
This is a research study designed to look at the pharmacokinetics (distribution, breakdown, and removal) and tolerability of a single dose of daptomycin in patients aged 3 months to 24 months who have proven or suspected…
- Trial name
- NCT01019395
- Testing
- Daptomycin
- Where
- 8 sites in United States
- Volunteers
- 23 people
- Sponsor
- Cubist Pharmaceuticals LLC, a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Type 2 Diabetes
A Study to Assess the Pharmacokinetics, Safety and Tolerability of Sitagliptin in Adolescents (0431-081)
This study will assess the safety, tolerability and pharmacokinetics of sitagliptin in 10 to 17 year old diabetic patients.
- Trial name
- NCT00730275
- Testing
- Sitagliptin phosphate
- Volunteers
- 35 people
- Sponsor
- Merck Sharp & Dohme LLC
Cystic Fibrosis
Safety, Tolerability and Pharmacokinetics of Tiotropium in Cystic Fibrosis Patients
Study to obtain information about the safety and tolerability of tiotropium bromide administered via the Respimat® inhalation device in pediatric (≤11 y.o.) and adolescent/adult (≥12 y.o.) cystic fibrosis (CF) patients after…
- Trial name
- NCT02172534
- Testing
- Tiotropium bromide low, Tiotropium bromide medium, Tiotropium bromide high
- Volunteers
- 113 people
- Sponsor
- Boehringer Ingelheim
Asthma
Dose-Ranging Study of LQ036 Single-Domain Antibody Nebulization Solution in Poorly Controlled Asthma
What is the goal of this study? The goal of this clinical trial is to find out whether the study drug LQ036 (a nebulized solution taken by inhaling through a nebulizer) works to improve lung function and asthma control in people…
- Trial name
- NCT07589439
- Testing
- LQ036
- Where
- China
- Volunteers
- 216 people
- Sponsor
- Shanghai Novamab Biopharmaceuticals Co., Ltd.
Asthma
Safety, Tolerability, and Pharcodynamics of AMG 853 in Adolescents With Asthma
The purpose of this study is to study the safety and tolerability in adolescent and adult subjects with intermittent or mild to moderate persistent asthma.
- Trial name
- NCT01137565
- Testing
- AMG 853
- Volunteers
- 23 people
- Sponsor
- Amgen
Muscular Dystrophies
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
- Trial name
- DEMAND I (NCT01128855)
- Testing
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968
- Where
- 2 sites in United States, France
- Volunteers
- 20 people
- Sponsor
- GlaxoSmithKline
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pain
To Evaluate the Safety and Efficacy of Acetaminophen 650 mg and 1000mg in Dental Pain After Surgery
The objective of this study is to evaluate the safety and efficacy of acetaminophen 650 mg and acetaminophen 1000 mg in dental surgery.
- Trial name
- NCT01115673
- Testing
- Acetaminophen
- Where
- United States
- Volunteers
- 540 people
- Sponsor
- McNeil Consumer Healthcare Division of McNEIL-PPC, Inc.
Meningococcal Disease
Safety of One Dose of Meningococcal ACWY Conjugate Vaccine in Subjects 2 Years of Age and Older
A multicenter, single arm, postmarketing surveillance study. This study is a postlicensure requirement of the Philippine Food and Drug Administration (FDA) to provide continued safety evaluation of MenACWY-CRM in Philippine…
- Trial name
- NCT01705379
- Testing
- Novartis Meningococcal ACWY Conjugate Vaccine
- Volunteers
- healthy volunteers welcome
- Sponsor
- Novartis Vaccines
Constipation
Single Dose Pharmacokinetics of Prucalopride in Paediatric Subjects, With Functional Faecal Retention
The purpose of this study is characterize the pharmacokinetics of a single oral dose of 0.03 mg/kg prucalopride in paediatric subjects aged >= 4 to <= 12 years with functional faecal retention.
- Trial name
- NCT01674166
- Testing
- prucalopride
- Volunteers
- 38 people
- Sponsor
- Movetis
Advanced Solid Tumor
Study of FCN-011 in Patients With Advanced Solid Tumor（Phase I）and NTRK Fusion Positive Advanced Solid Tumor （Phase II）
A multicenter, open, single-arm phase I dose exploration and phase II extended study was conducted to evaluate the safety, tolerability, pharmacokinetic characteristics, and primary antitumor activity of FCN-011 in patients with…
- Trial name
- NCT04687423
- Testing
- FCN-011
- Where
- China
- Volunteers
- 82 people
- Sponsor
- Fochon Pharmaceuticals, Ltd.
Beta-Thalassemia, Thalassemia
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT05356195
- Testing
- CTX001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
MPS IIIA, Sanfilippo Syndrome
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
Open-label, clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
- Conditions
- MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
- Trial name
- NCT04088734
- Testing
- ABO-102
- Where
- 3 sites in United States, Australia and 1 more
- Volunteers
- 5 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Dental Pain
Study of Long-Acting Acetaminophen in Postoperative Dental Pain
The purpose of the study is to evaluate the efficacy and safety of acetaminophen ER 1500 mg (two 750 mg tablets) over 10 to 12 hours in the dental pain model following third molar extraction(s) and to evaluate the…
- Trial name
- NCT01960114
- Testing
- Acetaminophen ER
- Where
- United States
- Volunteers
- 403 people
- Sponsor
- McNeil Consumer Healthcare Division of McNEIL-PPC, Inc.
GM1 Gangliosidosis, GM1 Gangliosidosis, Type I
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues.
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Trial name
- Imagine-1 (NCT04713475)
- Testing
- PBGM01
- Where
- 7 sites in United States, Brazil and 2 more
- Volunteers
- 26 people
- Sponsor
- Gemma Biotherapeutics
Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory and non-ambulatory subjects with…
- Trial name
- NCT03362502
- Testing
- PF-06939926
- Where
- 22 sites in United States
- Volunteers
- 23 people
- Sponsor
- Pfizer