Royal Manchester Childrens Hospital

Manchester M27 4HA

15 studies enrolling now · 72 studies all time

What they study most

ALK Fusion-positive Solid or CNS Tumors, ALL, Adult and Pediatric, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Acid Maltase Deficiency Disease, Adenovirus, Adenovirus Infection, Adenovirus Infections, Adolescent, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Anemia, Arthritis, Juvenile

Studies at this site

• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis — Recruiting now
• A Phase 3 Trial to Compare IV BCV Versus IV CDV for Treatment of Adenovirus Infection After Allo-HCT — Recruiting now
• A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young Adults With Cancer — Recruiting now
• A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors — Recruiting now
• A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC) — Recruiting now
• EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety — Recruiting now
• A Study of Guselkumab in Pediatric Participants With Moderately to Severely Active Crohn's Disease — Recruiting now
• Phase 1 Study of INBRX-109 in Subjects With Locally Advanced or Metastatic Solid Tumors Including Sarcomas — Recruiting now
• Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1) — Recruiting now
• A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C — Recruiting now
• The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency — Recruiting now
• A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL) — Recruiting now
• Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD). — Recruiting now
• A Study of the Efficacy and Safety of DMX-200 in Patients With FSGS Who Are Receiving an ARB — Recruiting now
• An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 — Recruiting now
• Simple Bone Cysts in Kids — Recruiting now
• CAMPFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Desmoplastic Small Round Cell Tumor — Completed
• A Study of Ustekinumab or Guselkumab in Pediatric Participants With Active Juvenile Psoriatic Arthritis — Completed
• A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy — Running, not enrolling
• A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow — Running, not enrolling
• An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007 — Enrolling by invitation
• A Research Study in Children Born Small and Who Stayed Small. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day — Running, not enrolling
• Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta — Running, not enrolling
• A Research Study in Children With a Low Level of Hormone to Grow. Treatment is Somapacitan Once a Week Compared to Norditropin® Once a Day (REAL4) — Completed
• A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia — Stopped early
• ADH1 and ADH2 Disease Monitoring Study (DMS) — Running, not enrolling
• Setrusumab vs Placebo for Osteogenesis Imperfecta — Running, not enrolling
• A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP) — Completed
• Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis — Completed
• Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF) — Completed
• Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B — Completed
• A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) — Running, not enrolling
• Evaluate the Safety and Efficacy of Ferric Maltol Oral Suspension vs. Ferrous Sulfate Oral Liquid in Children and Adolescents Aged 2 to 17 Years With Iron-deficiency Anaemia, With a Single Arm Study in Infants Aged 1 Month to Less Than 2 Years — Completed
• A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy — Completed
• Long-term Follow-up of Patients Treated With Genetically Modified Autologous T Cells — Enrolling by invitation
• Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 — Completed
• Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy — Stopped early
• A Study to Evaluate the Safety, Tolerability and Immunogenicity of an Investigational RSV Vaccine Candidate (Ad26.RSV.preF) in Adults 18 to 50 Years of Age, and RSV-seropositive Toddlers 12 to 24 Months of Age — Completed
• A Study to Evaluate the Efficacy and Safety of Daratumumab in Pediatric and Young Adult Participants Greater Than or Equal to (>=)1 and Less Than or Equal to (<=) 30 Years of Age With Relapsed/Refractory Precursor B-cell or T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma — Completed
• The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency — Running, not enrolling
• A Study of ALS-008176 in Infants Hospitalized With RSV — Completed
• Safety and Efficacy Study of Viaskin Peanut in Peanut-allergic Young Children 1-3 Years of Age — Completed
• Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) — Completed
• A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation — Completed
• A Study to Evaluate the Efficacy and Safety of NexoBrid in Children With Thermal Burns Compared the Standard of Care — Completed
• A Study to Compare the Efficacy and Safety of Ifosfamide and Etoposide With or Without Lenvatinib in Children, Adolescents and Young Adults With Relapsed and Refractory Osteosarcoma — Completed
• A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation — Completed
• Posoleucel (ALVR105) for the Treatment of Adenovirus Infection in Pediatric and Adult Participants Receiving Standard of Care Following Allogeneic Hematopoietic Cell Transplantation — Stopped early
• N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) — Completed
• Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis — Completed