Clinical trials: Open Label
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
The primary objective of the study was to evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD following 24 weeks of treatment.
- Conditions
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- NCT01886378
- Testing
- UX007
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 29 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Infectious Disease, Febrile Illness
A Rapid Triage Test to Improve Risk-stratification of Febrile Children (EChiLiBRiST, Clinical Trial 1, Outpatients)
The overall aim of the study is to provide evidence that introducing novel biomarkers evaluation at triaging (first clinical assessment), in combination with IMCI-based guidelines (SoC), is a viable strategy to enhance rapid and…
- Conditions
- Infectious Disease, Febrile Illness, Child, Only
- Trial name
- NCT06422338
- Testing
- IMCI-enhanced by suPAR levels (SoC + suPAR POC)
- Where
- 2 sites in Gabon, Mozambique
- Volunteers
- 5,212 people
- Sponsor
- Barcelona Institute for Global Health
Schizophrenia, Bipolar I Disorder
A Study to Evaluate the Pharmacokinetics, Safety and Tolerability of Sublingual Asenapine in a Pediatric Population With Schizophrenia or Bipolar I Disorder (P06522 AM1)
This study is an open label, sequential-group, two site, multiple dose escalating study of sublingual administered asenapine in a pediatric population with schizophrenia or bipolar I disorder; in one study cohort (3a)…
- Trial name
- NCT01206517
- Testing
- Asenapine 2.5 mg, Asenapine 5 mg, Asenapine 10 mg
- Volunteers
- 30 people
- Sponsor
- Organon and Co
Thrombosis Prophylaxis (Risk of Thrombosis Due to Central Venous Line (CVL)
Study of AVE5026 at Weight-adjusted Doses in Children With a Central Venous Line
Primary Objective: To assess the pharmacokinetic (PK) and pharmacodynamic (PD) parameters of Semuloparin [AVE5026] (assessed from the anti-Xa activity of Semuloparin) in children in order to determine the dose to be assessed in a…
- Trial name
- NCT01567904
- Testing
- Semuloparin sodium
- Where
- Hungary
- Volunteers
- 2 people
- Sponsor
- Sanofi
Chronic Graft Versus Host Disease
Safety and Efficacy of Oral Belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander Male and Female Participants Aged 12 Years and Above With Chronic Graft Versus Host Disease (cGVHD) After At Least 2 Prior Lines of Systemic Therapy
The purpose of this study is to measure safety and efficacy of oral belumosudil in Black or African American, American Indian or Alaska Native, and Native Hawaiian or Other Pacific Islander male and female participants with cGVHD…
- Trial name
- NCT05567406
- Testing
- Belumosudil
- Sponsor
- Kadmon, a Sanofi Company
NF2 Deficiency
Gene Therapy for Neurofibromatosis Type 2 (NF2) with ST002
This is an open-label, single-arm, dose-escalation pilot study to evaluate the safety, tolerability and preliminary efficacy of ST002 in the treatment of patients with NF2 mutation-related solid tumors.
- Trial name
- NCT06834438
- Testing
- ST002
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Gut Microbiota
Impact of Probiotics Supplement on the Gut Microbiota in Caesarean-born Infants
The purpose of this study is to evaluate the effect of probiotics on the gut microbiota of infants born by cesarean section.
- Trial name
- NCT05086458
- Testing
- probiotics
- Where
- China
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Shanghai 10th People's Hospital
Osteosarcoma, Survival
Comparison of MAPI+Camrelizumab Versus API+Apatinib Versus MAPI in Patients With a Poor Response to Preoperative Chemotherapy for Newly Diagnosed High-grade Osteosarcoma
Treatment strategies for high-grade osteosarcoma with multidrug chemotherapy and resection result in 3-year event-free survival of 60-70%.
- Conditions
- Osteosarcoma, Survival, Chemotherapy Effect, Toxicity, Drug
- Trial name
- MAPAC (NCT04351308)
- Testing
- MAPI chemotherapy, Apatinib Mesylate, Camrelizumab
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Peking University People's Hospital
Hemophilia B
A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B.
- Trial name
- BEYOND-9 (NCT06379789)
- Testing
- REGV131, LNP1265
- Where
- 52 sites in United States, Australia and 7 more
- Volunteers
- 130 people
- Sponsor
- Regeneron Pharmaceuticals
Acute Lymphoblastic Leukemia, Pediatric
A Prospective, Open-label, Randomized Controlled, Multicenter Clinical Study of MSD-HSCT Using a TBI or TMLI Conditioning Regimen for Pediatric ALL
This study aims to compare the effects of two different conditioning regimens on patients with acute lymphoblastic leukemia (ALL) undergoing matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT): Total Body…
- Trial name
- NCT06564493
- Testing
- TBI, TMLI, Cyclophosphamide
- Where
- China
- Volunteers
- 170 people
- Sponsor
- The First Affiliated Hospital of Zhengzhou University
Familial Hypercholesterolemia
Atorvastatin Three Year Pediatric Study
The purpose of this study is to characterize three year descriptive growth and development (ie, height, weight, body mass index, Tanner Stage) and efficacy of cholesterol reduction in pediatric subjects with Heterozygous Familial…
- Trial name
- NCT00827606
- Testing
- atorvastatin
- Where
- 40 sites in United States, Belgium and 13 more
- Volunteers
- 272 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Molluscum Contagiosum
Evaluation of Systemic Exposure to VP-102 in Subjects With Molluscum Contagiosum.
The primary objective of the study is to determine the presence or absence of systemic cantharidin exposure from a single 24-hour dermal application of VP-102 topical film-forming solution [0.7% (w/v) cantharidin] (VP-102) when…
- Trial name
- NCT03186378
- Testing
- VP-102 with applicator
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Verrica Pharmaceuticals Inc.
Hemophilia A
PF-05208756, Moroctocog Alfa (AF-CC), Xyntha For Hemophilia A
An open-label, single-arm, post- authorization pragmatic clinical trial on the safety and efficacy of Xyntha (Moroctocog-alfa (AF-CC), Recombinant FVIII) in subjects with hemophilia A in usual care settings in China for…
- Trial name
- NCT02492984
- Testing
- Intravenous infusions of Xyntha
- Where
- 15 sites in China
- Volunteers
- 85 people
- Sponsor
- Pfizer
Head Lice
A Pharmacokinetics (PK) Study in Lice Infested Children 6 Months to 3 Years of Age
The purpose of this study is to determine the bioavailability of 0.5% ivermectin cream in a pediatric population aged 6 months to 3 years.
- Trial name
- NCT00988533
- Testing
- 0.5% Ivermectin Cream
- Where
- 5 sites in United States
- Volunteers
- 30 people
- Sponsor
- Topaz Pharmaceuticals Inc
Duchenne Muscular Dystrophy
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data.
- Trial name
- NCT01239758
- Testing
- ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03), ACE-031 (Extension of cohort 3 from core study, A031-03)
- Where
- 4 sites in Canada
- Volunteers
- 11 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Graft Failure
Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT.
This study is designed as an open-label, single arm, proof of concept study in order to determine the appropriate emapalumab dosing regimen neutralizing IFNγ in patients at risk of GF.
- Trial name
- NCT04731298
- Testing
- Emapalumab
- Where
- 5 sites in Australia, Canada and 1 more
- Volunteers
- 2 people
- Sponsor
- Swedish Orphan Biovitrum
Molybdenum Cofactor Deficiency, Type A
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A
- Trial name
- NCT02629393
- Testing
- ORGN001 (formerly ALXN1101)
- Where
- 11 sites in United States, Israel and 4 more
- Volunteers
- 5 people
- Sponsor
- Origin Biosciences
Metastatic Uveal Melanoma
A Randomized, Phase 2/3 Study to Investigate the Efficacy and Safety of RP2 in Combination With Nivolumab in Immune Checkpoint Inhibitor-Naïve Adult Patients With Metastatic Uveal Melanoma
The purpose of this study is to measure the clinical benefits of the combination of RP2 and nivolumab as compared with the combination of nivolumab and ipilimumab in patients with metastatic uveal melanoma who have not been…
- Trial name
- NCT06581406
- Testing
- RP2, Ipilimumab, Nivolumab
- Where
- 33 sites in United States, Australia and 1 more
- Volunteers
- 280 people
- Sponsor
- Replimune, Inc.
Functional Abdominal Pain, Functional Dyspepsia
Efficacy of Open Label Placebo in Children With FGIDs
This study is aimed at investigating the efficacy of placebo for symptom relief in children with abdominal pain related functional gastrointestinal disorders.
- Conditions
- Functional Abdominal Pain, Functional Dyspepsia, Irritable Bowel Syndrome, Functional Gastrointestinal Disorders
- Trial name
- Placebo (NCT02389998)
- Testing
- Hyoscyamine
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Boston Children's Hospital
Epidermal Nevus Syndrome
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)
KRN23 is a fully human immunoglobulin monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum phosphorus levels.
- Trial name
- ENS (NCT04320316)
- Testing
- Crysvita (burosumab-twza) Treatment
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Alabama at Birmingham
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