Clinical trials: Phase 1
Amaurosis of Leber, Retinal Diseases
Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations
A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber…
- Trial name
- LCA (NCT00481546)
- Testing
- rAAV2-CBSB-hRPE65
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- University of Pennsylvania
Pneumonia, Bacterial
A Trial to Evaluate the Safety and Reactogenicity of an Investigational Pneumococcal Vaccine in Infants Receiving 3-dose Primary Dosing Series Followed by a Booster Dose at 12 to 15 Months of Age
The main purpose of this study is to evaluate safety and reactogenicity of the investigational pneumococcal vaccine (called Pn-MAPS30plus). PCV20 will be used as a comparator for this study
- Trial name
- NCT07406347
- Testing
- Pn-MAPS30plus, PCV20
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Advanced Cancer
Sirolimus or Everolimus or Temsirolimus and Vorinostat in Advanced Cancer
The goal of this clinical research study is to find the highest tolerable dose of the combination vorinostat given in combination with either sirolimus, everolimus or temsirolimus that can be given to patients with advanced…
- Trial name
- NCT01087554
- Testing
- Sirolimus, Vorinostat, Everolimus
- Where
- United States
- Volunteers
- 249 people
- Sponsor
- M.D. Anderson Cancer Center
Mucopolysaccharidosis IH
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of…
- Trial name
- TigetT10_MPSIH (NCT03488394)
- Testing
- Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Orchard Therapeutics
FSHD, FSHD1
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with…
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE (NCT05747924)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 90 people
- Sponsor
- Avidity Biosciences, Inc.
Sickle Cell Disease, Sickle Cell Anemia in Children
Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study
NOHARM MTD is an extension of a previous study for children with Sickle Cell Anemia (SCA) who were enrolled in the NOHARM study.
- Trial name
- NOHARM MTD (NCT07708714)
- Testing
- hydroxycarbamide
- Where
- Uganda
- Volunteers
- 250 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Teeth Staining
Clinical Efficacy of Antivet Versus Phosphoric Acid and Hydrochloric Acid in Microabrasion
This clinical trial will be conducted to assess the clinical efficacy of using Antivet cleaning enamel solution or hydrochloric acid and phosphoric acid in microabrasion technique for mild to moderate intrinsic stained teeth.
- Trial name
- NCT07709286
- Testing
- (Antivet, MDC DENTAL, Zapopan Jalisco México), Opalustre, phosphoric acid 37% and aluminium oxid 50 microns
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Misr University for Science and Technology
Severe Combined Immunodeficiency Due to RAG1 Deficiency
Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
This trial is a prospective, non-randomized, open-label, multicentre single-arm phase I/II intervention trial in children up to 24 months of age with RAG1-deficient SCID and an indication for allogeneic hematopoietic stem cell…
- Trial name
- RAG1-SCID (NCT04797260)
- Testing
- Gene therapy
- Where
- 4 sites in Netherlands, Poland and 2 more
- Volunteers
- 10 people
- Sponsor
- Videja B.V.
Remimazolam, Tonsillectomy
Remimazolam for Emergence Delirium Prevention in Children Undergoing Tonsillectomy
The aim of this study is to confirm the effectiveness of remimazolam in preventing delirium during emergence from anesthesia in children who will undergo elective procedures of tonsillectomy, adenotonsillectomy or adenoidectomy…
- Conditions
- Remimazolam, Tonsillectomy, Emergence Delirium
- Trial name
- NCT07704437
- Testing
- Remimazolam 20 MG Injection [Byfavo], NaCl (0,9%)
- Where
- Croatia
- Volunteers
- 110 people
- Sponsor
- University Hospital of Split
Asthma
A Study to Investigate How Budesonide and Formoterol Move Through the Body (Pharmacokinetics) When Delivered With Different Devices in Participants Aged 4 to Less Than 12 Years Old With Asthma
The purpose of this study is to assess the pharmacokinetics (PK) and safety of symbicort aerosphere and symbicort pressurized metered dose inhaler (pMDI) in participants with asthma aged 4 to less than 12 years.
- Trial name
- COMPAIR (NCT07433569)
- Testing
- Budesonide/formoterol fumarate Aerosphere, Budesonide/formoterol fumarate pMDI
- Where
- 6 sites in United States
- Volunteers
- 16 people
- Sponsor
- AstraZeneca
Hematologic Diseases
Bispecific CD19/CD20-Targeted Chimeric Antigen Receptor (CAR) Modified T Cells With 4-1BB and Mutated CD28 Costimulatory Domains in Patients With Relapsed or Refractory CD19+ Hematologic Malignancies
To determine the safety, toxicity, and maximum tolerated dose (MTD) of CD19/CD20 bispecific CAR-T cells with 4-1BB and mutated CD28 costimulatory domains (20-19 Tan BB06z) in patients with relapsed or refractory CD19+ aggressive…
- Trial name
- NCT07701889
- Testing
- 20-19 Tan BB06oz CAR T
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Roswell Park Cancer Institute
Malignant Neoplasm of Bone and Articular Cartilage, Malignant Neoplasms of Female Genital Organs
Study of Aerosol Gemcitabine in Patients With Solid Tumors and Pulmonary Metastases
Any time the words "you," "your," "I," or "me" appear, it is meant to apply to the potential participant. The goal of this clinical research study is to find the highest tolerable dose of gemcitabine that can be given by…
- Conditions
- Malignant Neoplasm of Bone and Articular Cartilage, Malignant Neoplasms of Female Genital Organs, Malignant Neoplasms of Independent (Primary) Multiple Sites, Malignant Neoplasms of Lip Oral Cavity and Pharynx
- Trial name
- NCT03093909
- Testing
- Gemcitabine
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- M.D. Anderson Cancer Center
Respiratory Syncytial Virus Infections
Safety and Immunogenicity of CodaVax-RSV in Seropositive and Seronegative Children
This study is a Phase 1, randomized, double-blind, placebo-controlled, dose-escalation clinical trial to evaluate the safety of and immune response to CodaVax-RSV in healthy children.
- Trial name
- NCT04919109
- Testing
- CodaVax-RSV, Normal Saline
- Where
- 5 sites in United States, South Africa
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Codagenix, Inc
Allergic Reaction
A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of DESF in Pediatric Subjects
An open-label study to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of epinephrine administered as a single dose of DESF in pediatric subjects weighing ≥30 kg with a personal history of…
- Trial name
- PARKS (NCT07703982)
- Testing
- DESF
- Where
- 8 sites in United States, Canada
- Volunteers
- 32 people
- Sponsor
- Aquestive Therapeutics
Acute Myeloid Leukemia, Myelodysplastic Syndromes
Vorinostat Dose-escalation After Allogeneic Hematopoietic Cell Transplantation
The objective of this study is to evaluate the maximum tolerated (MTD) of vorinostat used in combination with low-dose azacitidine after allogeneic hematopoietic cell transplantation (alloHCT) for prevention of relapse of…
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndromes, Mixed Phenotype Acute Leukemia, Juvenile Myelomonocytic Leukemia
- Trial name
- NCT03843528
- Testing
- Vorinostat, Azacitidine Injection
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Johns Hopkins All Children's Hospital
Juvenile Idiopathic Arthritis
Study to Measure Filgotinib in the Blood of Children and Teenagers With Arthritis Taking Filgotinib (SCALESIA)
A Study to evaluate the pharmacokinetics, safety, and tolerability in paediatric population for treating juvenile idiopathic arthritis (JIA).
- Trial name
- NCT06222034
- Testing
- Filgotinib
- Where
- 10 sites in France, Germany and 3 more
- Volunteers
- 9 people
- Sponsor
- Alfasigma S.p.A.
Obesity, Adolescent Obesity
Physiologic Response to Bariatric Surgery and the Impact of Adjunct Semaglutide in Adolescents
The study plans to learn more about what happens to the body after bariatric surgery in people 12 to 24 years old. The study aims to understand why people respond differently to bariatric surgery and how to define success beyond…
- Conditions
- Obesity, Adolescent Obesity, Body-Weight Trajectory, Weight Loss Trajectory
- Trial name
- PRESSURE (NCT06575738)
- Testing
- Injectable semaglutide or injectable tirzepatide, Standard postoperative care
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of Colorado, Denver
Bronchopulmonary Dysplasia (BPD), Premature Births
PK, Safety and Preliminary Efficacy Study of Montelukast in Critically Ill Infants With Developing Bronchopulmonary Dysplasia
The purpose of the study is to learn how safe montelukast may be in premature infants at significant risk for Bronchopulmonary Dysplasia (BPD) and to determine how much and how quickly montelukast moves from the stomach into the…
- Conditions
- Bronchopulmonary Dysplasia (BPD), Premature Births, Critical Illness
- Trial name
- PRISM (NCT07101640)
- Testing
- montelukast 4 mg granule
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Duke University
Atypical Teratoid/Rhabdoid Tumor, Epithelioid Sarcoma
Tiragolumab and Atezolizumab for the Treatment of Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
This phase I/II trial studies how well tiragolumab and atezolizumab works when given to children and adults with SMARCB1 or SMARCA4 deficient tumors that have either come back (relapsed) or do not respond to therapy (refractory).
- Conditions
- Atypical Teratoid/Rhabdoid Tumor, Epithelioid Sarcoma, Kidney Medullary Carcinoma, Malignant Solid Neoplasm
- Trial name
- NCT05286801
- Testing
- Atezolizumab, Biospecimen Collection, Computed Tomography
- Where
- 40 sites in United States, Australia and 1 more
- Volunteers
- 86 people
- Sponsor
- National Cancer Institute (NCI)
Advanced Solid Tumors
Study of mRNA-4359 Administered Alone and in Combination With Immune Checkpoint Blockade in Participants With Advanced Solid Tumors
The primary goal of this study is to assess the safety and tolerability of mRNA-4359 administered alone and in combination with pembrolizumab or ipilimumab and nivolumab.
- Trial name
- NCT05533697
- Testing
- mRNA-4359, Pembrolizumab, Ipilimumab
- Where
- 39 sites in United States, Australia and 5 more
- Volunteers
- 361 people
- Sponsor
- ModernaTX, Inc.
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