Clinical trials: Early Phase 1, open to volunteers
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay
Aprepitant for the Treatment of CLIFAHDD
There are no drug treatments for individuals with Congenital Contractures of the Limb and Face, Hypotonia, and Developmental Delay (CLIFAHDD), an ultra-rare and severe neurodevelopmental disease.
- Conditions
- CLIFAHDD, Congenital Contractures of the Limbs and Face, Hypotonia, and Developmental Delay, NALCN Channelopathy, Ataxia - Other
- Trial name
- NCT07845825
- Testing
- Aprepitant Powder for Oral Suspension
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- The University of Texas Health Science Center at San Antonio
Chronic Disease, Type 2 Diabetes Mellitus (T2DM), Obesity, Overweight
Truway Diagnostic Tools in Primary Care
This study evaluates the effectiveness of Truway Health-sourced portable diagnostic devices, including ultrasound and blood glucose monitors, in early detection of chronic conditions in outpatient primary care settings.
- Conditions
- Chronic Disease, Type 2 Diabetes Mellitus (T2DM), Obesity, Overweight, Hypertension, Cardiovascular Disease Acute
- Trial name
- TruDxPC (NCT07209527)
- Testing
- Truway Portable Ultrasound Device, Truway Blood Glucose Monitor, Standard Oral Hypoglycemic Agent
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Truway Health, Inc.
Malaria Prevention
Focal Mass Drug Administration for the Prevention of Malaria in Pregnancy
The purpose of this study is to demonstrate the feasibility, acceptability, and preliminary effectiveness of a focal mass drug administration program for household members of pregnant women to protect against malaria in pregnancy.
- Trial name
- NCT07021430
- Testing
- Dihydroartemisinin Piperaquine
- Where
- Uganda
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
CNS Embryonal Tumor, CNS, Medulloblastoma
Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation
Pilot study to determine feasibility of adding intrathecal chemotherapy and maintenance therapy after high dose chemotherapy for treatment of newly diagnosed HR-EBTs in patients less than 6 years of age.
- Conditions
- CNS Embryonal Tumor, CNS, Medulloblastoma, Atypical Teratoid Rhabdoid Tumor, Medulloblastoma, Childhood
- Trial name
- NCT06942039
- Testing
- Cytarabine IT, hydrocortisone, Cisplatin
- Where
- 13 sites in Canada
- Volunteers
- 15 people
- Sponsor
- C17 Council
Sarcoma Metastatic
Pilot Study of TumorGlow for Intraoperative Molecular Imaging of Sarcoma Metastases to the Lungs in Pediatric Patients
Subjects will undergo infusion of 5 mg/kg indocyanine green, ICG ("TumorGlow") intravenously up to 5-days prior to surgery. Then, during the surgical procedure the next day, the subjects will undergo standard-of-care surgery.
- Trial name
- TumorGlow (NCT07658768)
- Testing
- ICG INFUSION 5MG/KG
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Abramson Cancer Center at Penn Medicine
Solid Tumor
Indocyanine Green (ICG) Guided Sentinel Lymph Node Mapping for Pediatric Visceral Solid Tumors
The purpose of the study is to find out the usefulness and safety of a dye called Indocyanine Green (ICG for short). This dye will be used to help the surgeon find lymph nodes draining solid tumors inside the abdomen that need to…
- Trial name
- NCT05645523
- Testing
- Indocyanine Green
- Where
- 3 sites in United States
- Volunteers
- 35 people
- Sponsor
- St. Jude Children's Research Hospital
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Intra-Bony Defects in Periodontitis Patients
The Efficacy of Exosomes Combined With Synthetic Bone Graft for the Treatment of Periodontal Intra-bony Defects
The aim of this study will be to evaluate clinically, radiographically and biochemically the efficacy of exosomes combined with synthetic bone graft for the treatment of periodontal intra-bony defects.
- Trial name
- NCT07897292
- Testing
- single flap approach, single flap approach combined with nanocrystalline hydroxyapatite bone substitute, Exosomes
- Where
- 2 sites in Egypt
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Khoshoe Al-mokhtar Mohammed
Radiation Dermatitis
Adapalene Gel for Acute Radiation-Induced Skin Injury
his study aims to evaluate whether adapalene gel is effective and safe for treating acute radiation-induced skin injury in adults receiving radiotherapy.
- Trial name
- YNSZLYY-RADGEL (NCT07886320)
- Testing
- Adapalene gel 0.1%, Biafine topical emulsion
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Yunnan Cancer Hospital
Rhabdomyosarcoma
Exploratory Study of Personalized mRNA Vaccine in Patients With Refractory Rhabdomyosarcoma
An Exploratory Study on the Use of mRNA Tumor Vaccines in the Treatment of Refractory Rhabdomyosarcoma
- Trial name
- NCT06932861
- Testing
- personalized neoantigen mRNA vaccine
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Xinxin Zhang
Arginine Vasopressin Deficiency, Oxytocin Deficiency
Identifying Oxytocin Deficiency in Pediatric Patients With Pituitary Disease
An open-labeled, interventional pilot trial, 10 youth with AVP-D and 10 PD matched for age, sex, and BMI will be recruited from Pediatric Endocrinology and Neuroendocrinology at Massachusetts General Hospital and in the…
- Conditions
- Arginine Vasopressin Deficiency, Oxytocin Deficiency, Pediatric Disease, Hypopituitarism
- Trial name
- Pedi-EVOLVE (NCT07568509)
- Testing
- Norethindrone Acetate-Ethinyl Estradiol
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Massachusetts General Hospital
Advanced Lung Cancer, Lung Metastasis
mRNA Vaccine for Advanced Lung Cancer and Pulmonary Metastases of Solid Tumors
BMD006 is an off-the-shelf antitumor mRNA vaccine targeting lung cancer and lung metastatic solid tumors. This is a single-center, open-label, dose-escalation clinical trial.
- Trial name
- NCT06928922
- Testing
- BMD006 monotherapy, BMD006 in combination with Immuno-Oncology (IO) therapy (Dose Escalation), BMD006 in combination with Immuno-Oncology (IO) therapy (Dose Expansion)
- Where
- China
- Volunteers
- 83 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Persistent Postural Perceptual Dizziness, Vestibular Migraine
Virtual Reality and Biofeedback for Adolescents With Chronic Dizziness
Both persistent postural-perceptual dizziness (PPPD) and vestibular migraine (VM) are common diagnoses in patients presenting to physicians for evaluation of vestibular symptoms such as dizziness and vertigo.
- Conditions
- Persistent Postural Perceptual Dizziness, Vestibular Migraine, Chronic Dizziness
- Trial name
- NCT07374640
- Testing
- Virtual Reality
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Boston Children's Hospital
Confluent and Reticulated Papillomatosis (CARP)
Topical Minocycline for CARP
This proof-of-concept trial evaluates the efficacy, safety, and patient satisfaction of topical minocycline foam (AMZEEQ®) in patients aged 9 and older with confluent and reticulated papillomatosis (CARP).
- Trial name
- NCT07462702
- Testing
- Topical Minocycline Foam FXFM244, Control
- Volunteers
- 35 people
- Sponsor
- Medical University of South Carolina
EBV Viremia, CMV Viremia
25BT056: Basket Study of Viral Cytotoxic T Lymphocytes
This study is evaluating the safety and effectiveness of virus-specific cytotoxic T lymphocytes (vCTLs) for treating difficult-to-control viral infections caused by cytomegalovirus (CMV), adenovirus (ADV), Epstein-Barr virus…
- Conditions
- EBV Viremia, CMV Viremia, BK Viremia, Adenovirus
- Trial name
- NCT07873970
- Testing
- Virus-Specific Cytotoxic T Lymphocytes (vCTLs)
- Volunteers
- 35 people
- Sponsor
- Children's Hospital of Philadelphia
Inherited Retinal Diseases
Extension Study of the PUMCH-E101 Clinical Trial：Safety and Efficacy Study of PUMCH-E101 Injection in the Contralateral Eye
The goal of this clinical trial is to evaluate the safety and efficacy of PUMCH-E101 injection in the contralateral eye of subjects with RDH12 retinopathy.
- Trial name
- NCT07878572
- Testing
- PUMCH-E101 Injection(High dose)
- Where
- China
- Volunteers
- 8 people
- Sponsor
- Peking Union Medical College Hospital
Graft-versus-host-disease (GVHD), Gastrointestinal
Vitamin C With Steroids for Gastrointestinal GVHD
After a transplant from another donor, one risk is graft versus host disease (GVHD) that happens because of differences between the donated cells (graft) and the patient's body cells (host).
- Trial name
- NCT07197749
- Testing
- Vitamin C (Ascorbic Acid)
- Volunteers
- 35 people
- Sponsor
- Baylor College of Medicine
Posttraumatic Stress Disorder (PTSD), Trauma Exposure
Predictors & Mechanisms of Adolescent PTSD
This study examines how adolescents with trauma-related symptoms respond to stress and strong emotions. The study assesses brain activity, physiological responses, and behavior during experimental tasks that involve responding to…
- Trial name
- NCT07537764
- Testing
- Repeated imaginal exposure: Script Driven Imagery (SDI) task
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- Medical University of South Carolina
Type 2 Diabetes
SMART 2D: Study of Montelukast's Effects on Renal and Cardiovascular Health in Adolescents and Young Adults With Type 2 Diabetes
Adolescents and young adults (AYA) with type 2 diabetes (T2D) have a more severe phenotype than what is seen in youth-onset type 1 diabetes or adult-onset T2D including earlier kidney and cardiovascular disease complications; and…
- Trial name
- NCT07409714
- Testing
- Montelukast
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Colorado, Denver