Clinical trials involving children

Lenny device study

• Description: The purpose of this study is to demonstrate the safety and performance of the MiniMed™ 780G system in pediatric subjects (2–6 years old) with type 1 diabetes in a home setting.
• Principal Investigator: Anna-Kaisa Tuomaala, HUS
• Sponsor: Medtronic

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

A-SURE

• Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor VIII Products
• Sponsor: Swedish Orphan Biovitrum AB (Sobi)
• https://clinicaltrials.gov/ct2/show/NCT02976753(opens in new window, links to another website)
• National Principal Investigator: Minna Koskenvuo, HUS
• Principal Investigator: Minna Koskenvuo, HUS

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.

KD-Caap trial

Preventing coronary artery aneurysms with corticosteroids in Kawasaki disease

• Principal Investigator: Tea Nieminen

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

PANORAMA

• En öppen multicenterstudie för att bedöma säkerhet och tolerabilitet på lång sikt av sakubitril/valsartan hos barnpatienter som har hjärtsvikt orsakad av systemisk systolisk vänsterkammardysfunktion och som har fullföljt studien CLCZ696B2319 (PANORAMA-HF-E1) 
• Huvudforskare: Tiina Ojala
• Uppdragsgivare: Novartis Pharma

PAI

• Paracetamol and ibuprofen/indomethacin in closing persistent ductus arteriosus
• https://clinicaltrials.gov/ct2/show/NCT03648437
• National Principal Investigator: Outi Aikio
• New Children’s Hospital’s Principal Investigator: Mirka Lumia

TREOCAPA

• Prophylactic treatment of the ductus arteriosus in preterm infants by acetaminophen
• https://clinicaltrials.gov/ct2/show/NCT04459117
• Sponsor           INSERM
• National Principal Investigator: Outi Aikio
• New Children’s Hospital’s Principal Investigator: Mirka Lumia

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

ALLTogether

• 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)
• EudraCT number: 2018-001795-38
• https://clinicaltrials.gov/ct2/show/NCT03911128
• National Principal Investigator: Mervi Taskinen and Ulla Wartiovaara-Kautto
• New Children’s Hospital, Principal Investigator: Mervi Taskinen

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
• EudraCT number: 2017-000705-20
• https://clinicaltrials.gov/ct2/show/NCT03007147
• National Principal Investigator: Olli Lohi
• New Children’s Hospital, Principal Investigator: Jukka Kanerva

CASSIOPEIA

• A Phase II Trial of Tisagenlecleucel in First-line High-risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy
• Sponsor Novartis
• EudraCT number: 2017-002116-14
• https://www.clinicaltrials.gov/ct2/show/NCT03876769
• National Principal Investigator: Kim Vettenranta
• New Children’s Hospital, Principal Investigator: Kim Vettenranta

ITCC-059

• A phase I/II study of Inotuzumab Ozogamicin as a single agent and in combination with chemotherapy for pediatric Cd22 positive relapsed ALL
• Sponsor: Erasmus Medical Center Rotterdam
• EudraCt number:  2016-000227-71 
• https://clinicaltrials.gov/ct2/show/NCT02981628
• National Principal Investigator: Mervi Taskinen
• New Children’s Hospital, Principal Investigator: Mervi Taskinen

BRIGATINIB (STARTING IN AUTUMN 2021)

• A Phase I/II study of Brigatinib as monotherapy in pediatric and young adult patients with ALK-positive Anaplastic Large Cell Lymphoma (ALCL), Inflammatory Myofibroblastic Tumors (IMT) or other solid tumors
• Sponsor Princess Maxima Centrum
• Phase of the study: feasibility questionnaire answered+, Helsinki National Coordination Center

LELU study

• Skeletal complications in childhood leukemia - tissue-level mechanisms
• (Lapsuusiän leukemian luustovaikutukset - kudostason mekanismit) 
• New Children’s Hospital, Principal Investigator: Pauliina Utriainen

NOPHO-DBH AML 2012

• Research Study for Treatment of Children and Adolescents With Acute Myeloid Leukaemia 0-18 Years (AML2012)
• Sponsor: Västra Götaland Regionen, Queen Silvias Childrens and Adolescents Hospital
• EudraCT number: 2012-002934-35
• https://www.clinicaltrials.gov/ct2/show/NCT01828489
• National Principal Investigator: Kirsi Jahnukainen
• New Children’s Hospital, Principal Investigator: Kirsi Jahnukainen

IntreALL HR 2010

• International Study for Treatment of High Risk Childhood Relapsed ALL 2010
• EudraCT number: 2012-000810-12
• https://clinicaltrials.gov/ct2/show/NCT03590171
• National Principal Investigator: Päivi Lähteenmäki (TYKS)
• New Children’s Hospital, Principal Investigator: Mervi Taskinen

IntreALL SR 2010

• International Study for Treatment of Standard Risk Childhood Relapsed ALL 2010
• EudraCT number: 2012-000793-30
• https://clinicaltrials.gov/ct2/show/NCT01802814
• National Principal Investigator: Päivi Lähteenmäki (TYKS)
• New Children’s Hospital, Principal Investigator: Mervi Taskinen

SCRIPT-AML (upcoming)

• Randomized trial of AML preliminary conditioning comparing BuCyMel and BuFluClo pre-treatments. Donors allowed in the randomized main protocol include sib, MUD and poles; haplos are recruited in the observational branch (upcoming)

SCD-Haplo (upcoming)

• A Regensburg-sponsored researcher-driven trial comparing transplant results in sickle patients conforming to transplantation indications with design sib vs. a/b TCR dep haplo.

ALL SCTped 2012 FORUM (ENDED)

• Open, randomised, multicentre, controlled, prospective phase III study for therapy and therapy optimisation in patients with acute lymphoblastic leukaemia (ALL) and an indication for allogeneic haematopoietic stem cell
• EudraCT number: 2012-003032-22
• https://clinicaltrials.gov/ct2/show/NCT01949129
• National Principal Investigator: Kim Vettenranta
• New Children’s Hospital, Principal Investigator: Kim Vettenranta

LCOMPLEMENT

• Complement in pediatric hematopoietic stem cell transplantation
• Reactions of the complement system in children’s stem cell transplants
• Doctoral researcher: Lilli Leimi
• New Children’s Hospital, Principal Investigator: Kim Vettenranta

LALLOIMMU

• Immune reconstitution in children after hematopoietic stem cell transplantation and their susceptibility to viral infections with special reference to monitor hemostasis and the risk of thrombosis
• Doctoral researcher: Adam Alexandersson
• Principal Investigator: Minna Koskenvuo

Lung complications after allogeneic stem cell transplant

• Doctoral researcher: Elli-Maija Ukonmaanaho
• Principal Investigator: Mervi Taskinen

B-NHL 2013 2.2

• 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)
• EudraCT number: 2013-003253-21
• https://clinicaltrials.gov/ct2/show/NCT03206671
• National Principal Investigator Pasi Huttunen
• New Children’s Hospital, Principal Investigator Pasi Huttunen

LBL 2018

• International Cooperative Treatment Protocol for Children and Adolescents With Lymphoblastic Lymphoma (LBL 2018)
• EudraCT number: 2017-001691-39
• https://www.clinicaltrials.gov/ct2/show/NCT04043494
• National Principal Investigator Kaisa Vepsäläinen (KYS)
• New Children’s Hospital, Principal Investigator Pasi Huttunen

EuroNet-PHL C2 (to be opened)

• Second International Inter-Group Study for Classical Hodgkin Lymphoma in Children and Adolescents
• EudraCT number: 2012-004053-88
• https://clinicaltrials.gov/ct2/show/NCT02684708
• National Principal Investigator
• New Children’s Hospital, Principal Investigator Pasi Huttunen

Medulloblastoma

PNET5

• International Society of Paediatric Oncology (SIOP) PNET 5 Medulloblastoma
• EudraCT number: 2011-004868-30
• https://clinicaltrials.gov/ct2/show/NCT02066220
• National Principal Investigator: Virve Pentikäinen
• New Children’s Hospital, Principal Investigator: Virve Pentikäinen

Ependymoma

SIOP EPENDYMOMA

• An International Clinical Program for the Diagnosis and Treatment of Children With Ependymoma (SIOP-EP-II)
• EudraCT number: 2013-002766-39
• https://clinicaltrials.gov/ct2/show/NCT02265770
• National Principal Investigator: Satu Långström
• New Children’s Hospital, Principal Investigator: Satu Långström

Central nervous system germ cell tumors

(SIOP CNS GCT II)

• Prospective Trial for the Diagnosis and Treatment of Intracranial Germ Cell Tumors (SIOPCNSGCTII)
• EudraCT number: 2009-018072-33
• https://clinicaltrials.gov/ct2/show/NCT01424839
• National Principal Investigator: Virve Pentikäinen
• New Children’s Hospital, Principal Investigator: Virve Pentikäinen

Ewing’s sarcoma

rEECur

• An international randomised controlled trial of chemotherapy for the treatment of recurrent and primary refractory Ewing sarcoma
• EudraCT number: 2014-000259-99
• https://www.birmingham.ac.uk/research/crctu/trials/reecur/index.aspx
• National Principal Investigator: Jukka Kanerva
• New Children’s Hospital, Principal Investigator: Jukka Kanerva

Neuroblastoma

HR-NBL-1.8 / SIOPEN (closed), HR-NBL 2.0/ SIOPEN (to be opened)

• High Risk Neuroblastoma Study 1.8 and 2.0 of SIOP-Europe (SIOPEN)
• EudraCT numbers: 2006-001489-17 and 2019-001068-31
• https://clinicaltrials.gov/ct2/show/NCT01704716
• https://clinicaltrials.gov/ct2/show/NCT04221035
• National Principal Investigator: Kim Vettenranta, Minna Koskenvuo
• New Children’s Hospital, Principal Investigator: Kim Vettenranta, Minna Koskenvuo

Liver tumors

PHITT

• Paediatric Hepatic International Tumour Trial (PHITT)
• EudraCT number: 2016-002828-85
• https://clinicaltrials.gov/ct2/show/NCT03017326
• National Principal Investigator: Jouni Pesola
• New Children’s Hospital, Principal Investigator: Jukka Kanerva

Renal tumors

SIOP Umbrella 2016 (to be opened)

• Integrated research and guidelines for standardized diagnostics and therapy of renal tumors
• EudraCT number: 2016-004180-39
• https://siop-rtsg.org/
• National Principal Investigator Marika Grönroos
• New Children’s Hospital, Principal Investigator Minna Koskenvuo

Tumor genetics and drug sensitivity

iCAN-PEDI

• iCAN Digital Precision Cancer Medicine for Discoveries and Improved Treatments
• Functional Precision Medicine For Pediatric Solid Tumors
• Sponsor Academy of Finland, University of Helsinki
• https://www.digitalprecisioncancermedicine.fi/
• National Principal Investigator: Minna Koskenvuo
• New Children’s Hospital, Principal Investigator: Minna Koskenvuo

INFORM

• Individualized Therapy for relapsed malignancies in childhood (INFORM)
• https://www.kitz-heidelberg.de/en/for-physicians/clinical-studies/molecular-diagnostics-studies/inform/
• National Principal Investigator: Olli Lohi
• New Children’s Hospital, Principal Investigator: Minna Koskenvuo

BAY27257556 (STARTING IN FALL OF 2021)

• ON-TRK: Prospective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with larotrectinib
• Sponsor: Bayer
• https://clinicaltrials.gov/ct2/show/NCT04142437
• National Principal Investigator: Virve Pentikäinen
• New Children’s Hospital, Principal Investigator: Virve Pentikäinen

Hemophilia

A-SURE

• Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
• Sponsor Swedish Orphan Biovitrum AB (Sobi)
• https://clinicaltrials.gov/ct2/show/NCT02976753
• National Principal Investigator: Minna Koskenvuo
• New Children’s Hospital, Principal Investigator: Minna Koskenvuo

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
• National Principal Investigator: Pasi Huttunen
• New Children’s Hospital, Principal Investigator: Pasi Huttunen

MOTIVATE (STARTING IN SUMMER 2021)  Modern Treatment of Inhibitor-PositiVe PATiEnts With Haemophilia A - An International Observational Study

• Sponsor Emory University
• https://clinicaltrials.gov/ct2/show/NCT04023019
• National Principal Investigator: Minna Koskenvuo
• New Children’s Hospital, Principal Investigator: Minna Koskenvuo

BAX855

• Phase 3, Prospective, Multi-center, Open Label Study to Investigate Safety, Immunogenicity and Hemostatic Efficacy of PEGylated Factor VIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years With Severe Hemophilia A (FVIII < 1%)
• Sponsor Baxalta now part of Shire
• https://clinicaltrials.gov/ct2/show/record/NCT02615691
• National Principal Investigator: Satu Långström
• New Children’s Hospital, Principal Investigator: Satu Långström

Hemoglobinopathies

STAND

• A Phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab Versus Placebo, With or Without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Vaso-Occlusive Crises (STAND)
• Sponsor Novartis Pharmaceuticals
• EudraCT number: 2017-001746-10
• https://clinicaltrials.gov/ct2/show/NCT03814746
• National Principal Investigator: Ulla Wartiovaara-Kautto, Kirsi Jahnukainen
• New Children’s Hospital, Principal Investigator: Kirsi Jahnukainen

Freezing of testicular tissue: with the aim of maintaining the fertility of a pre-puberty boy

• Researcher-driven study
• National Principal Investigator: Kirsi Jahnukainen
• New Children’s Hospital, Principal Investigator: Kirsi Jahnukainen

Preservation of fertility in cancer and stem cell transplant patients through freezing and in vitro maturation of ovarian tissue

• Researcher-driven study
• National Principal Investigator: Kirsi Jahnukainen
• New Children’s Hospital, Principal Investigator: Kirsi Jahnukainen

Socioeconomic long-term effects after childhood cancer treatment

• Researcher-driven study
• Doctoral researcher: Anniina Kyrönlahti
• New Children’s Hospital, Principal Investigator: Mervi Taskinen

Risk behavior after treatment of childhood cancer

• Researcher-driven study
• Doctoral researcher: Liisa Korhonen
• New Children’s Hospital, Principal Investigator: Mervi Taskinen