Clinical trials involving children

FOP

• Description:  Phase 3 randomized, placebo-controlled study to assess safety, tolerability and efficacy of garetosmab in patients with fibrodysplasia ossificans progressiva
• Principal Investigator: Matti Hero, HUS
• Sponsor: Regeneron

MELD-ATG

• Description: Efficacy study of low dose anti-thymocyte globulin treatment in people recently diagnosed with type 1 diabetes 
• Principal Investigator: Mari Pulkkinen
• Sponsor: University hospital UZ Leuven (Universitaire Ziekenhuizen Leuven)
• EudraCT number: 2019-003265-17

Minimed 780g study for young children

The effect of Minimed 780G insulin pump to glycemic control and safety in children aged 2–6 years

• Description: The purpose of this study is to evaluate the effectiveness of advanced hybrid-closed loop system (so-called smart insulin pump) to glycemic control and avoidance of hypoglycemic values. Additionally, the study evaluates the perceived burden of the diabetes treatment to parents (diabetes distress) during a 3-month treatment.
• Official title: Glycemic outcomes and safety with Minimed 780G system in children with type 1 diabetes aged 2-6 years
• Linkki: https://clinicaltrials.gov/(opens in new window, links to another website)
• Principal Investigator: Anna-Kaisa Tuomaala, HUS
• EudraCT number: 2021-000403-21

Real 8

• Description: A study comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® as well as evaluating long-term safety of somapacitan in a basket study design in children with short stature either born small for gestational age or with Turner syndrome, Noonan syndrome, or idiopathic short stature. 
• Principal Investigator: Matti Hero, HUS
• EudraCT number: 2021-005607-13

Prevalence of hypoglycemia in long QT syndrome in pediatric patients on beta-blocker therapy

• Description: The study investigates whether asymptomatic hypoglycemia occurs in children with long QT syndrome while taking propranolol. In addition, risk factors predisposing to low blood sugar will be investigated and, if low blood sugars are detected, it will be investigated whether they can be prevented by nutritional means.
• Principal investigator: Elina Hakonen
   

A-MORE

• A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health (A-MORE)
• Sponsor: Swedish Orphan Biovitrum AB (Sobi)
• https://clinicaltrials.gov/ct2/show/NCT04293523(opens in new window, links to another website)
• National Principal Investigator: Pasi Huttunen, HUS
• Principal Investigator: Pasi Huttunen, HUS

MOTIVATE 

• MOdern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study
• https://clinicaltrials.gov/ct2/show/NCT04023019(opens in new window, links to another website)
• National Principal Investigator: Minna Koskenvuo, HUS
• Principal Investigator: Minna Koskenvuo, HUS

HemiNorth

• A multicenter, non-interventional study evaluating health-related quality of life, physical activity and bleeds in patients with severe hemophilia A without inhibitors on FVIII prophylaxis within the Nordic countries
• EudraCT Number 2020-003256-32
• National Principal Investigator: Riitta Lassila (adult patients), Satu Långström (pediatric patients)
• Principal Investigator: Satu Långström, HUS

RSV MAT-009

• A Phase III, randomized, double-blind, placebo-controlled multi-country study to demonstrate efficacy of a single dose of unadjuvanted RSV Maternal vaccine, administered IM to pregnant women 18 to 49 years of age, for prevention of RSV associated LRTIs in their infants up to 6 months of age
• https://clinicaltrials.gov/ct2/show/NCT04605159
• National Principal Investigator: Anu Kantele
• New Children’s Hospital’s Principal Investigator: Santtu Heinonen

FinNissle trial

Preventing children's UTIs with probiotic E. coli Nissle 

• Official title: Preventing urinary tract infections in infants and young children with probiotic E. coli Nissle: FinNissle trial
• Link: https://clinicaltrials.gov/
• Principal Investigator: Tea Nieminen, HUS
• EudraCT number: 2020-001769-35

MK-1654-007

A phase 3, randomized, partially blinded, palivizumab-controlled study to evaluate the safety, efficacy, and pharmacokinetics of MK-1654 in infants and children who are at increased risk for severe RSV disease 

• Official title: A Phase 3, Multicenter, Randomized, Partially Blinded, Palivizumab-Controlled Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of MK-1654 in Infants and Children at Increased Risk for Severe RSV Disease
• Link: https://clinicaltrials.gov/
• Principal Investigator: Santtu Heinonen, HUS
• EudraCT number: 2020-005996-11
• Description: This is a phase 3, randomized, partially blinded, palivizumab-controlled study to evaluate the safety, efficacy, and pharmacokinetics of MK-1654 in infants and children who are at increased risk for severe RSV disease.

STEPS-EA-trial

• Intralesional steroid injections to prevent refractory strictures in patients with esophageal atresia - a randomized controlled trial
• National Principal Investigator: Janne Suominen
• New Children’s Hospital’s Principal Investigator: Janne Suominen

FIONA OLE

• This study gathers information on the safety and effects of finerenone in long-term use.
• EudraCT 2021-002905-89
• Sponsor: Bayer
• Principal Investigator: Tuula Hölttä, HUS

FIONA

• A 6-month multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and pharmacokinetics/ pharmacodynamics of an age- and body weight-adjusted oral finerenone regimen, adjusted for age and weight, in addition to an ACE inhibitor or ATR blocker, for the treatment of children between the ages of 6 months and 18 years with chronic kidney disease and proteinuria
• Link: https://clinicaltrials.gov/
• Principal Investigator: Tuula Hölttä, HUS
• EudraCT 2021-002071-19

DINKY

• A single-arm, multi-centre trial to evaluate efficacy and safety of imlifidase in highly sensitised children (1–17 years) receiving a kidney transplant with positive cross-match against a living or deceased donor converted to negative after imlifidase treatment
• The study recruits children and adolescents (1–17 years) with end-stage renal disease who are waiting for a renal transplant and have a large amount of antibodies. The primary aim of the study is to assess how efficiently imlifidase cleaves IgG antibodies and converts a positive cross-match result to a negative, which will enable renal transplantation.
• Principal Investigator: Timo Jahnukainen, HUS
• Sponsor: Hansa

Roxadustat study

• The objective of the study is to determine whether roxadustat is an effective and safe treatment for anemia associated with chronic kidney disease. Roxadustat increases erythropoietin production in the body and improves blood iron availability. These mechanisms help to increase hemoglobin and the number of red blood cells. Roxadustat is used in adults to treat anemia associated with chronic kidney disease. 
• Participation in the study lasts up to 14 months. During the study, the subjects receive the investigational drug Roxadustat, and blood and urine samples are taken, medical examinations are performed, and vital signs are measured. The study also includes an electrocardiogram. The subjects are asked to answer questions related to health, previous medication, and possible symptoms and side effects and fill in questionnaires that are used to evaluate different aspects of the study.
• Principal Investigator: Deputy Chief Physician Elisa Ylinen, HUS
• Sponsor: Astellas Pharma

PREV-IS

• PREDICTION AND PREVENTION OF INFANTILE SPASMS IN HIGH RISK CHILDREN
• National Principal Investigator: Kirsi Mikkonen
• New Children’s Hospital’s Principal Investigator: Kirsi Mikkonen

AGU trial

Cystadane for the treatment of aspartylglucosaminuria: trial evaluating efficacy and safety (cystadane for the treatment of AGU).

• Principal Investigator: Minna Laine
• EudraCT number: 2017-000645-48

SKYPP

• An Open-label, Sequential, Ascending, Repeated Dose-finding Study of Sarilumab, Administered with Subcutaneous (SC) Injection, in Children and Adolescents, Aged 2 to 17 Years, with Polyarticular-course Juvenile Idiopathic Arthritis (pcJIA) Followed by an Extension Phase
• https://clinicaltrials.gov/ct2/show/NCT02776735
• National Principal Investigator: Kristiina Aalto
• New Children’s Hospital’s Principal Investigator: Kristiina Aalto

ALBINO

• Effect of ALlopurinol in addition to hypothermia for hypoxic-ischemic Brain Injury on Neurocognitive Outcome – a blinded randomized placebo-controlled parallel group multicenter trial for superiority (Phase III)
• https://clinicaltrials.gov/ct2/show/NCT03162653
• National Principal Investigator: Marjo Metsäranta
• New Children’s Hospital’s Principal Investigator: Marjo Metsäranta

Carescape patient monitoring system R&D study

• The aim of the study is to evaluate the operation and usability of the Carescape patient monitoring system in a hospital environment. In addition, data are collected on variables describing the functioning of the body in graph and numerical form for the development of research equipment.
• Principal investigator: Tero Varpula
• Sponsor: GE Healthcare Finland Oy

Product development study for wireless patient monitoring system

• The purpose of the study is to evaluate the usability, functionality, and ease of use of the wireless patient monitoring system in a hospital environment.
• Principal investigator: Markku Pentikäinen
• Sponsor: GE Healthcare Finland Oy

Study of the accuracy of blood pressure measurement in children (NIBP Accuracy Neonate and Pediatrics)

• The objective is to collect cuff-measured blood pressure values and arterial blood pressure values from subjects in clinical settings to demonstrate accuracy according to the standard for cuff blood pressure.
• Principal investigator: Otto Rahkonen
• Sponsor: GE Healthcare Finland Oy

ALLTogether1

• A Treatment study protocol of the ALLTogether Consortium for children and young adults (1-45 years of age) with newly diagnosed acute lymphoblastic leukaemia (ALL)
• Sponsor: Karolinska University Hospital, Sweden
• EUCT number: 2022-501050-11-01
• National Principal Investigator: Riitta Niinimäki (children, OYS) ja Ulla Wartiovaara-Kautto (adults, HUS)
• New Children’s Hospital, Principal Investigator: Antti Kyrönlahti

EsPhALL2017 / COGAALL1631 

• International phase III trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones
• Sponsor: University of Milano-Bicocca, Italy 
• EUCT number: 2024-515499-12-00
• National Principal Investigator: Olli Lohi (TAYS)
• New Children’s Hospital, Principal Investigator: Jukka Kanerva

Interfant-21 

• International collaborative treatment protocol for infants under one year with KMT2A-rearranged acute lymphoblastic leukemia or mixed phenotype acute leukemia
• Sponsor: Princess Máxima Center, Netherlands
• EUCT number: 2022‐502503‐30‐00  
• National Principal Investigator: Olli Lohi (TAYS)
• New Children’s Hospital, Principal Investigator: Antti Kyrönlahti

AMGEN 20180130 

• Evaluation of Long-term Safety in Paediatric Patients With B-precursor Acute Lymphoblastic Leukemia (ALL) who Have Been Treated With Either Blinatumomab or Chemotherapy, Followed by Transplantation
• Sponsor: Amgen Inc. / Amgen AB
• EUCT number: not exist
• National Principal Investigator: Samppa Ryhänen (HUS)
• New Children’s Hospital, Principal Investigator: Samppa Ryhänen 

PAVO 

• Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy
• Sponsor: Novartis Pharma Services AG
• EUCT number: 2023-508128-37-00
• National Principal Investigator: Samppa Ryhänen (HUS)
• New Children’s Hospital, Principal Investigator: Samppa Ryhänen 

IntReALL HR 2010 

• International Study for Treatment of High Risk Childhood Relapsed ALL 2010
• Sponsor: Charité - Universitätsmedizin Berlin, Germany 
• EUCT number: 2024-513070-21-00
• National Principal Investigator: Päivi Lähteenmäki (TYKS) 
• New Children’s Hospital, Principal Investigator: Mervi Taskinen

B1931036 

• A prospective, randomized, open-label phase 2 study to evaluate the superiority of Inotuzumab Ozogamicin monotherapy versus ALLR3 for induction treatment of childhood high risk first relapse B-cell precursor acute lymphoblastic leukaemia
• Sponsor: Pfizer Inc.
• EUCT number: 2023-509810-13-00
• National Principal Investigator: Samppa Ryhänen (HUS)
• New Children’s Hospital, Principal Investigator: Samppa Ryhänen

ITCC-101 APAL2020D 

• A randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML
• Sponsor: Princess Máxima Center, Netherlands 
• EUCT number: 2023-510160-12-00
• National Principal Investigator: Pasi Huttunen (HUS)
• New Children’s Hospital, Principal Investigator: Pasi Huttunen

NOPHO-DBH-AML 2012 

• Research Study for Treatment of Children and Adolescents With Acute Myeloid Leukemia 0-18 Years (AML2012)
• Sponsor: Västra Götaland Regionen, Sweden
• EUCT number: 2024-518254-16-00
• National Principal Investigator: Kirsi Jahnukainen (HUS)
• New Children’s Hospital, Principal Investigator: Kirsi Jahnukainen

SCRIPT-AML

• A Randomized, Multi-Center Phase III Trial comparing two conditioning regimens (CloFluBu and BuCyMel) in children with Acute Myeloid Leukemia undergoing allogeneic stem cell transplantation.
• Sponsor: Västra Götaland Regionen, Sweden
• EUCT number: 2023-505512-37-00
• National Principal Investigator: Samppa Ryhänen (HUS)
• New Children’s Hospital, Principal Investigator: Samppa Ryhänen

ALCL-VBL

• International cooperative prospective study for children and adolescents with standard risk ALK-positive anaplastic large cell lymphoma (ALCL) estimating the efficacy of Vinblastine
• Sponsor: German Paediatric Oncology Group GPOG gGmbH, Germany
• EUCT number: 2022-501454-11-00
• National Principal Investigator: Kaisa Vepsäläinen (KYS)
• New Children’s Hospital, Principal Investigator: Pasi Huttunen

B-NHL 2013 

• Treatment Protocol of the NHL-BFM and the NOPHO Study Groups for Mature Aggressive B-cell Lymphoma and Leukemia in Children and Adolescents (B-NHL 2013)
• Sponsor: Universitätsklinikum Münster, Germany
• EUCT number: 2023-505509-18-00
• National Principal Investigator: Pasi Huttunen (HUS)
• New Children’s Hospital, Principal Investigator: Pasi Huttunen

LBL 2018 

• International co-operative treatment protocol for children and adolescents with lymphoblastic lymphoma
• Sponsor: Universitätsklinikum Münster, Germany
• EUCT number: 2023-508101-24-00
• National Principal Investigator: Kaisa Vepsäläinen (KYS)
• New Children’s Hospital, Principal Investigator: Pasi Huttunen

PNET5MB 

• An international prospective trial on medulloblastoma (MB) in children older than 3 to 5 years with WNT biological profile (PNET 5 MB - LR and PNET 5 MB - WNT-HR), average-risk biological profile (PNET 5 MB -SR), or TP53 mutation, and registry for MB occurring in the context of genetic predisposition
• Sponsor: Medical Center Hamburg-Eppendorf, Germany
• EUCT number: 2024-513724-42-00
• National Principal Investigator: Virve Pentikäinen (HUS)
• New Children’s Hospital, Principal Investigator: Virve Pentikäinen

SIOP-HRMB 

• International prospective trial on high-risk medulloblastoma in patients older than 3 years
• Sponsor: The University of Birmingham, Great Britain 
• EUCT number: 2024-510578-25-00
• National Principal Investigator: Virve Pentikäinen (HUS)
• New Children’s Hospital, Principal Investigator: Virve Pentikäinen

SIOP Ependymoma II 

• An international clinical program for the diagnosis and treatment of children, adolescents and young adults with ependymoma
• Sponsor: Centre Léon Bérard, France
• EUCT number: 2024-512222-28-00
• National Principal Investigator: Satu Långström (HUS)
• New Children’s Hospital, Principal Investigator: Satu Långström

SIOPE ATRT01 

• An international prospective umbrella trial for children with atypical teratoid/rhabdoid tumors (ATRT), including A randomized phase III study evaluating the non-inferiority of three courses of high-dose chemotherapy (HDCT) compared to focal radiotherapy as consolidation therapy
• Sponsor: German Paediatric Oncology Group, GPOH gGmbH, Germany 
• EUCT number: 2022-501456-28-00
• National Principal Investigator: Satu Långström (HUS)
• New Children’s Hospital, Principal Investigator: Satu Långström

LOGGIC Core 

• BioClinical Data Bank, LOGGIC: Low Grade Glioma In Children
• Sponsor: Hopp Children’s Cancer Center Heidelberg (KiTZ), German Cancer Research Center (DKFZ), Germany
• EUCT number: not exist
• National Principal Investigator: Päivi Lähteenmäki (TYKS)
• New Children’s Hospital, Principal Investigator: Satu Långström 

Day One DAY101-002 

• LOGGIC/FIREFLY-2: A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients with Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy
• Sponsor: Day One Biopharmaceuticals, Inc., USA
• EUCT number: 2024-510742-13-00
• National Principal Investigator: Kristiina Nordfors (TAYS)
• New Children’s Hospital, Principal Investigator: Satu Långström

iEuroEwing

• International Euro Ewing trial for treatment optimisation in patients with Ewing sarcoma
• Sponsor: German Paediatric Oncology Group, GPOH gGmbH, Germany
• EUCT number: 2022-501180-40-00 
• National Principal Investigator: Jukka Kanerva (HUS)
• New Children’s Hospital, Principal Investigator: Jukka Kanerva

rEECur 

• International Randomised Controlled Trial of Chemotherapy for the Treatment of Recurrent and Primary Refractory Ewing Sarcoma
• Sponsor: University of Birmingham, Great Britain
• EUCT number: 2024-516078-31-00
• National Principal Investigator: Jukka Kanerva (HUS)
• New Children’s Hospital, Principal Investigator: Jukka Kanerva

ITCC-098 BrigaPED  

• A Phase I/II study of Brigatinib in pediatric and young adult patients with ALK+ Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumors or other solid tumors
• Sponsor: Princess Máxima Center, Netherlands
• EUCT number: 2024-513412-10-00
• National Principal Investigator: Antti Kyrönlahti
• New Children’s Hospital, Principal Investigator: Antti Kyrönlahti

ON-TRK  

• ON-TRK: PrOspective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with Larotrectinib
• Sponsor: Bayer AG
• EUCT number: not exist
• National Principal Investigator: Virve Pentikäinen (HUS)
• New Children’s Hospital, Principal Investigator: Virve Pentikäinen