Clinical trials: suspended
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.
Blood Pressure, Lipid Profile
A Study Of Starting Strength Barbell Training
The purpose of this study is to determine the effect of a 12-week coached Starting Strength barbell training program on systolic and diastolic blood pressure.
- Conditions
- Blood Pressure, Lipid Profile, Fasting Glucose
- Trial name
- NCT07578285
- Testing
- Progressive Barbell Strength Training
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Medium-chain Acyl-CoA Dehydrogenase Deficiency
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
This is a medical research study to test a medication in patients 10 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A>G (K304E) mutation.
- Trial name
- NCT06069375
- Testing
- Sodium phenylbutyrate
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Jerry Vockley, MD, PhD
Leukodystrophy, Globoid Cell
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease
PBKR03 is a gene therapy for Krabbe Disease (Globoid cell leukodystrophy) intended to deliver a functional copy of the GALC gene to the brain and peripheral tissues.
- Trial name
- GALax-C (NCT04771416)
- Testing
- PBKR03
- Where
- 9 sites in United States, Brazil and 4 more
- Volunteers
- 24 people
- Sponsor
- Gemma Biotherapeutics
First Episode Psychosis
OnTrackNY's Learning Healthcare System
This application proposes OnTrackNY as a regional scientific hub for the Early Psychosis Intervention Network (EPINET) program as part of the National Institute of Mental Health (NIMH)'s creation of a national learning health…
- Trial name
- NCT04021719
- Testing
- Stakeholder feedback, Data infrastructure
- Where
- United States
- Volunteers
- 2,100 people
- Sponsor
- New York State Psychiatric Institute
Hyperphosphatemia Related to Chronic Kidney Disease
Study to Evaluate the Safety and Tolerability of Ferric Citrate in Children With Hyperphosphatemia Related to Chronic Kidney Disease
This study will be conducted to assess the safety and tolerability of ferric citrate in pediatric participants with hyperphosphatemia related to chronic kidney disease (CKD).
- Trial name
- NCT04523727
- Testing
- ferric citrate
- Where
- 14 sites in United States
- Volunteers
- 45 people
- Sponsor
- Keryx Biopharmaceuticals
Cerebral Adrenoleukodystrophy
MT2022-01: MSCs for ALD
This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to…
- Trial name
- NCT06030648
- Testing
- Mesenchymal stem cells (IV-MSC)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Preterm Infant, Growth Delay
The Milk, Growth and Microbiota Study
Late preterm infants, who are born at 34, 35 or 36 weeks gestation, often have difficulty feeding, establishing growth, and fighting off infection.
- Conditions
- Preterm Infant, Growth Delay, Microbial Colonization, Breastfeeding
- Trial name
- MGM (NCT03220282)
- Testing
- Donor milk, Preterm infant formula
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- University of California, San Francisco
Advanced or Unresectable Locally Advanced Bone and Soft Tissue Sarcoma
A Single-arm, Open-label, Single-center Prospective Study of Fruquintinib Combined With Envafolimab in the Treatment of Advanced or Unresectable Locally Advanced Bone and Soft Tissue Sarcoma
Many osteosarcomas are cured with a variety of combined chemotherapy and surgery, but a significant number will still relapse.VEGF promotes an immunosuppressive microenvironment and contributes to immune checkpoint inhibitor…
- Trial name
- NCT05941325
- Testing
- fruquintinib, Envafolimab
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shanghai 6th People's Hospital
Acute Liver Failure, Mitochondrial Diseases
Longitudinal Study of Mitochondrial Hepatopathies
The specific aims of this study are (1) to determine the clinical phenotypes and natural history of hepatic RC and FAO disorders, (2) to determine the correlation between genotype and phenotype, (3) to determine if circulating…
- Conditions
- Acute Liver Failure, Mitochondrial Diseases, End Stage Liver Disease, Respiratory Chain Deficiencies, Mitochondrial
- Trial name
- MITOHEP (NCT01148550)
- Where
- 16 sites in United States, Canada
- Volunteers
- 90 people
- Sponsor
- Arbor Research Collaborative for Health
Liver Diseases, Alagille Syndrome
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Cholestasis is a condition in which bile is not properly transported from the liver to the small intestine. Cholestasis can be caused by an array of childhood diseases, including the genetic diseases Alagille syndrome (ALGS)…
- Conditions
- Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency
- Trial name
- LOGIC (NCT00571272)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,675 people
- Sponsor
- Arbor Research Collaborative for Health
Anticholinergic Toxicity
Rivastigmine to Prevent Recurrence of Antimuscarinic Delirium
Antimuscarinic delirium (AMD) is a common and dangerous toxicology condition caused by poisoning by medications and other chemicals that block muscarinic receptors.
- Trial name
- RIVA-AP (NCT06399679)
- Testing
- Rivastigmine
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Washington University School of Medicine
Otolaryngologic Surgery
Superficial Cervical Plexus Block for Improved Outcomes in Pediatric Otolaryngologic Surgery
Literature on the use of superficial cervical plexus blocks for ear surgery is sparse in general, and almost non-existent in the pediatric population.
- Trial name
- NCT06023329
- Testing
- ropivacaine, Saline
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- University of Minnesota
Acute Lymphobkastic Leukemia
Immunotherapy Combined With Auto-HSCT and CD22/CD19 CAR-T Sandwich Strategy for B-ALL
Chimeric antigen receptor T-cell (CAR-T) therapy has achieved remarkable efficacy in B-cell acute lymphoblastic leukemia (B-ALL). However, relapse after CAR-T has been a major issue.
- Trial name
- NCT06985498
- Testing
- Sandwich stratergy
- Where
- China
- Volunteers
- 40 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Dyslexia
taVNS for Letter Learning in Dyslexia
This is a pilot study to establish feasibility of transcutaneous auricular vagus nerve stimulation (taVNS) for improving letter-learning in adolescents with dyslexia.
- Trial name
- NCT06127550
- Testing
- active taVNS
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Florida
Caesarean Delivery
Regenerative Effects of Birth Material Derived Extracellular Vesicles
Stem cells offer great hope for a wide range of disorders, including age-related brain disease such as dementia. Mesenchymal stem cells (MSCs) are special types of adult stem cells found in various tissues like the umbilical…
- Trial name
- ReGAIN (NCT06866184)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Aberdeen
Postoperative Chylothorax, Congenital Heart Disease
Propranolol Treatment for Postoperative Chylothorax
Postoperative chylothorax is a serious complication after open heart surgery for pediatric patients with congenital heart disease (CHD).
- Conditions
- Postoperative Chylothorax, Congenital Heart Disease, Open Heart Surgery, Chylothorax
- Trial name
- NCT06833320
- Testing
- Propranolol Hydrochloride
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- June Wu
Performance, Recovery
Effects of Root Extract Ashwagandha (KSM-66) on Physiological Stress, Perception of Recovery and Muscle Strength in Youth Footballers
This trial examines if six weeks of taking 450 mg Ashwagandha root extract affects exercise performance and recovery in youth male elite football players.
- Conditions
- Performance, Recovery, Strength, Aerobic Endurance
- Trial name
- NCT07343219
- Testing
- Root extract Ashwagandha, Corn Starch
- Where
- United Kingdom
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Sports Performance and Applied Research in Trials and Analysis
PTEN Gene Mutation
Cancer Risk Assessment in Patients With a Constitutional Alteration of the PTEN Gene
This is a multicentric, observational, retrospective and prospective study, aiming to estimate the risk of cancer occurrence in subjects carrying a PTEN mutation, based on the constitution of a national cohort.
- Trial name
- COCO (NCT05630105)
- Where
- 45 sites in France
- Volunteers
- 430 people
- Sponsor
- Institut Bergonié
Neoplasm
A Study of SGT-53 in Children With Refractory or Recurrent Solid Tumors
The purpose of this study is to determine the dose limiting toxicities and recommended phase 2 dose of SGT-53 alone and in combination with topotecan and cyclophosphamide in pediatric patients with recurrent or refractory solid…
- Trial name
- NCT02354547
- Testing
- SGT-53, Topotecan, Cyclophosphamide
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- SynerGene Therapeutics, Inc.