Clinical trials: Phase 1
Sex Chromosome Disorders
A Multiple Baseline Feasibility Trial of Social Management Training for People With Sex Chromosome Aneuploidies
We will examine is Social Management Training, a 10-session group-based psychosocial intervention will improve the primary outcome mental health, and secondary outcomes executive functions and autism symptoms, in a multiple…
- Trial name
- TRICXY-SMT (NCT07718997)
- Testing
- Social Management Training
- Where
- Norway
- Volunteers
- 30 people
- Sponsor
- University of Oslo
In-stent Restenosis
Pilot Study of the Coronary Spur Stent for In-stent Restenosis (DEEPER CORONARY)
To demonstrate acceptable short term safety rates of the Drug-eluting Coronary Spur Stent System as a primary treatment for coronary in-stent restenosis.
- Trial name
- NCT06117150
- Testing
- Drug-eluting Coronary Spur Stent System
- Where
- 2 sites in New Zealand
- Volunteers
- 9 people
- Sponsor
- ReFlow Medical, Inc.
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Relapsed Neuroblastoma, Refractory Neuroblastoma
NKG2D.Zeta-NK Cell Conditioning With C7R.GD2.CAR-T Cells for Patients With Relapsed or Refractory Osteosarcoma or Neuroblastoma
The purpose of this study is to find the largest safe dose of i15.NKG2D.zeta-NK cells in combination with C7R.GD2.CAR-T cells, and additionally to evaluate how long they can be detected in patients' blood and what affect they…
- Conditions
- Relapsed Neuroblastoma, Refractory Neuroblastoma, Relapsed Osteosarcoma, Refractory Osteosarcoma
- Trial name
- NCT07211737
- Testing
- i15.NKG2D.zeta NK cells and C7R.GD2.CARTs cells
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Baylor College of Medicine
Beta-Thalassemia
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT).
- Trial name
- NCT05577312
- Testing
- BRL-101
- Where
- 7 sites in China
- Volunteers
- 39 people
- Sponsor
- Bioray Laboratories
High Grade Glioma, Meningioma
Lutathera for Treatment of Recurrent or Progressive High-Grade CNS Tumors
This study will evaluate the safety and efficacy of Lutathera (177Lu-DOTATATE) in patients with progressive or recurrent High-Grade Central Nervous System (CNS) tumors and meningiomas that demonstrate uptake on DOTATATE PET.
- Conditions
- High Grade Glioma, Meningioma, Embryonal Tumor, Medulloblastoma
- Trial name
- NCT05278208
- Testing
- LUTATHERA® (Lutetium Lu 177 dotatate)
- Where
- 4 sites in United States
- Volunteers
- 65 people
- Sponsor
- Nationwide Children's Hospital
Gastro-Intestinal Disorder
Feasibility, Safety, and Potential Efficacy of Fecal Microbiota Transplantation (FMT) for Gastrointestinal Dysfunction in Children Following Hematopoietic Cell Transplant (HCT).
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant has Gastrointestinal (GI) symptoms following a Hematopoietic Cell Transplant (HCT).
- Trial name
- NCT05664113
- Testing
- Fecal microbiota transplant (FMT)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- St. Jude Children's Research Hospital
RSV Infection
Evaluating the Infectivity, Safety and Immunogenicity of a Recombinant Live-Attenuated Respiratory Syncytial Virus Vaccine, LID/ΔM2-2/1030s, in RSV-Seronegative Infants and Children 6 to 24 Months of Age
The purpose of this study is to evaluate the infectivity, safety, and immunogenicity of a single dose of a recombinant, live-attenuated respiratory syncytial virus (RSV) vaccine, LID/ΔM2-2/1030s, in RSV-seronegative infants and…
- Trial name
- NCT04520659
- Testing
- RSV LID/ΔM2-2/1030s
- Where
- 3 sites in United States
- Volunteers
- 81 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Constitutional Mismatch Repair Deficiency Syndrome, Lynch Syndrome
Pembrolizumab in Treating Younger Patients With Recurrent, Progressive, or Refractory High-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma or Medulloblastoma
This phase I trial studies the side effects and best dose of pembrolizumab and to see how well it works in treating younger patients with high-grade gliomas (brain tumors that are generally expected to be fast growing and…
- Conditions
- Constitutional Mismatch Repair Deficiency Syndrome, Lynch Syndrome, Malignant Glioma, Recurrent Brain Neoplasm
- Trial name
- NCT02359565
- Testing
- Biospecimen Collection, Conventional Magnetic Resonance Imaging, Diffusion Tensor Imaging
- Where
- 12 sites in United States, Canada
- Volunteers
- 71 people
- Sponsor
- National Cancer Institute (NCI)
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
- Trial name
- NCT05682144
- Testing
- Autologous Plasmablasts (B cells)
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Immusoft of CA, Inc.
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
Rhabdomyosarcoma
Mocetinostat With Vinorelbine in Children, Adolescents & Young Adults With Refractory and/or Recurrent Rhabdomyosarcoma
This phase I trial studies the side effects and best dose of mocetinostat when given together with vinorelbine to see how well it works in treating children, adolescents, and young adults with rhabdomyosarcoma that has spread to…
- Trial name
- NCT04299113
- Testing
- Vinorelbine, Mocetinostat
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Jonsson Comprehensive 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
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
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
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
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
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.
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
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