Clinical trials: open to volunteers, in United Kingdom
Autosomal Recessive Polycystic Kidney (ARPKD)
A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
To evaluate the pharmacodynamics and safety of tolvaptan in pediatric subjects with ARPKD
- Trial name
- NCT04782258
- Testing
- Tolvaptan Suspension, Tolvaptan Tablets
- Where
- 23 sites in United States, Belgium and 4 more
- Volunteers
- 20 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Dravet Syndrome
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to <36 months (Part 1A), aged ≥48 months to <18 years (Part 1B), and…
- Trial name
- ENDEAVOR (NCT05419492)
- Testing
- ETX101
- Where
- 14 sites in United States, Australia and 1 more
- Volunteers
- 113 people
- Sponsor
- Encoded Therapeutics
Epilepsy
An Observational Study of Epilepsy: Biology and Outcomes Using Real-world Data
The goal of this observational study is to learn more about phenotypic, genetic, biochemical, neurophysiological and radiological patterns in epilepsy.
- Trial name
- NCT05632978
- Where
- United Kingdom
- Volunteers
- 2,000 people
- Sponsor
- Cardiff and Vale University Health Board
Phenylketonuria
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
- Trial name
- EPIPHENY (NCT06302348)
- Testing
- Sepiapterin
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 56 people
- Sponsor
- PTC Therapeutics
Crohn's Disease
A Study to Evaluate the Efficacy and Safety of Tulisokibart (MK-7240) in Participants With Moderate to Severe Crohn's Disease (MK-7240-008)
The purpose of this protocol is to evaluate the efficacy and safety of tulisokibart in participants with moderately to severely active Crohn's disease.
- Trial name
- NCT06430801
- Testing
- IV Tulisokibart, SC Tulisokibart
- Where
- 499 sites in United States, Australia and 45 more
- Volunteers
- 1,200 people
- Sponsor
- Merck Sharp & Dohme LLC
Pruritus, Chronic Kidney Diseases
Safety and Tolerability of Difelikefalin in Adolescents on Haemodialysis With Moderate-to-Severe Pruritus
Rationale: People with long term kidney disease who are on haemodialysis (a procedure for removing waste products from the blood) commonly develop a condition that makes their skin very itchy.
- Trial name
- NCT06593392
- Testing
- Difelikefalin
- Where
- 18 sites in China, Greece and 5 more
- Volunteers
- 18 people
- Sponsor
- Vifor Fresenius Medical Care Renal Pharma
Pmm2-CDG, Phosphomannomutase 2 Deficiency
Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
The goal of this clinical trial is to provide continued access to GLM101 to treat PMM2-CDG in people who have previously received GLM101 in other trials and learn about the long term effect of GLM101.
- Trial name
- NCT06657859
- Testing
- GLM101
- Where
- 15 sites in United States, Czechia and 6 more
- Volunteers
- 90 people
- Sponsor
- Glycomine, Inc.
Enthesitis-related Arthritis, Juvenile Psoriatic Arthritis
An Open-label Study to Evaluate the Pharmacokinetics and Safety of Bimekizumab in Pediatric Study Participants With Active Juvenile Idiopathic Arthritis Subtypes Enthesitis-related Arthritis (Including Juvenile-onset Ankylosing Spondylitis) and Juvenile Psoriatic Arthritis
The purpose of this study is to assess plasma bimekizumab concentrations following subcutaneous (sc) bimekizumab administration.
- Trial name
- NCT06668181
- Testing
- Bimekizumab
- Where
- 23 sites in Canada, France and 4 more
- Volunteers
- 40 people
- Sponsor
- UCB Biopharma SRL
Generalized Myasthenia Gravis
A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)
The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of…
- Trial name
- THYME (NCT06987539)
- Testing
- Inebilizumab
- Where
- 14 sites in United States, Argentina and 6 more
- Volunteers
- 15 people
- Sponsor
- Amgen
Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia
Study of AZD3632 Monotherapy or in Combination With Anticancer Agents in Participants With Advanced Haematologic Malignancies With KMT2Ar, NPM1m, or Other Genotypes Associated With HOX Overexpression
The purpose of this study is to understand the safety, tolerability, efficacy, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy of orally administered AZD3632 in participants with advanced haematologic…
- Conditions
- Acute Lymphoblastic Leukaemia, Acute Myeloid Leukaemia, Higher-risk Myelodysplastic Syndromes
- Trial name
- MOMENTUM (NCT07155226)
- Testing
- AZD3632, Posaconazole
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 85 people
- Sponsor
- AstraZeneca
Fibrodysplasia Ossificans Progressiva (FOP)
To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia…
- Trial name
- Progress (NCT05090891)
- Testing
- INCB000928
- Where
- 24 sites in United States, Argentina and 15 more
- Volunteers
- 98 people
- Sponsor
- Incyte Corporation
Fabry Disease
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to < 12 years of age with Fabry disease and with amenable GLA variants.
- Trial name
- NCT06904261
- Testing
- Migalastat HCl 20 mg
- Where
- 12 sites in United States, Belgium and 4 more
- Volunteers
- 8 people
- Sponsor
- Amicus Therapeutics
Ulcerative Colitis
A Study to Learn More About How Risankizumab Works in Young Participants With Ulcerative Colitis
Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine).
- Trial name
- MIGHTY (NCT07071519)
- Testing
- Risankizumab
- Where
- 72 sites in United States, Belgium and 14 more
- Volunteers
- 120 people
- Sponsor
- AbbVie
Pre-eclampsia
Oxford Luteal Dysfunction and Placental Insufficiency Study
High blood pressure (BP) affects approximately 1 in 10 pregnancies. About half of women with high blood pressure in pregnancy develop a serious complication called preeclampsia, which kills over 70,000 women and 500,000 babies…
- Trial name
- OxLuPIn (NCT07072052)
- Testing
- No intervention planned
- Where
- United Kingdom
- Volunteers
- 360 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Achondroplasia
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH).
- Trial name
- ASPEN (NCT07441876)
- Testing
- BMN 333, Vosoritide Injection [Voxzogo]
- Where
- 28 sites in United States, Australia and 7 more
- Volunteers
- 160 people
- Sponsor
- BioMarin Pharmaceutical
Autoimmune Encephalitis (AE), Acute Disseminated Encephalomyelitis
OPM- MEG in Non-immune And iMmune-medIated Neurological Disease
Over the past 20 years, doctors have gotten much better at spotting and treating brain conditions caused by the immune system attacking the body. These include illnesses like autoimmune encephalitis, ADEM, and multiple sclerosis.
- Conditions
- Autoimmune Encephalitis (AE), Acute Disseminated Encephalomyelitis, Multiple Sclerosis
- Trial name
- O-MEG-A-MIND (NCT07689682)
- Testing
- OPM- Magnetoencephalography
- Where
- 2 sites in United Kingdom
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Aston University
Spinal Muscular Atrophy (SMA)
Service Evaluation for Newborn Screening for Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is a disease that causes progressive muscle weakness. There are many types- the most severe and most common, type 1, affects very young babies, and is life-threatening if untreated.
- Trial name
- SENS (NCT07888868)
- Where
- 3 sites in United Kingdom
- Volunteers
- 750,000 people
- Sponsor
- University of Oxford
Cardiac Arrest, Cardiopulmonary Arrest
Quality of Pediatric Resuscitation in a Multicenter Collaborative
This is a prospective, observational, multi-center cohort study of pediatric cardiac arrests. The purpose of the study is to determine the association between chest compression mechanics (rate, depth, flow fraction, compression…
- Trial name
- pediRES-Q (NCT02708134)
- Where
- 63 sites in United States, Australia and 13 more
- Volunteers
- 7,000 people
- Sponsor
- Children's Hospital of Philadelphia
Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumor
Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
This is a study to evaluate the efficacy and safety of belzutifan monotherapy in participants with advanced pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumor (pNET), von Hippel-Lindau (VHL) disease-associated…
- Conditions
- Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumor, Von Hippel-Lindau Disease, Advanced Gastrointestinal Stromal Tumor
- Trial name
- NCT04924075
- Testing
- Belzutifan
- Where
- 84 sites in United States, Australia and 20 more
- Volunteers
- 355 people
- Sponsor
- Merck Sharp & Dohme LLC
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