Study on Atypical Teratoid/Rhabdoid Tumors in Children Using Dactinomycin, Carboplatin, and Cyclophosphamide

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What is this study about?

This clinical trial is focused on studying a rare type of brain tumor in children known as atypical teratoid/rhabdoid tumors (ATRT). The study aims to evaluate the effectiveness of different treatment approaches for children with ATRT. The treatments being tested include high-dose chemotherapy (HDCT) and focal radiotherapy, which are used as consolidation therapies. Consolidation therapy is a treatment given after the initial therapy to strengthen its effects and help prevent the cancer from returning.

The trial involves several medications, including dactinomycin, carboplatin, cyclophosphamide, ifosfamide, etoposide, vincristine sulfate, thiotepa, doxorubicin hydrochloride, and methotrexate. These medications are administered through intravenous use, meaning they are given directly into a vein. The study is designed to compare the outcomes of children receiving these treatments to those who receive standard care, as well as to historical data from previous patients.

The purpose of the study is to determine if the new treatment approaches are as effective as, or better than, the current standard treatments. The trial will follow participants over a period of time to assess their overall survival and other health outcomes. This research is important for improving treatment options and outcomes for children diagnosed with ATRT.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a series of tests such as MRI and CSF examination to evaluate the condition of the atypical teratoid/rhabdoid tumors (ATRT).

2 induction chemotherapy

The first phase involves receiving three courses of induction chemotherapy. This treatment uses a combination of medications administered through intravenous infusion or injection. The specific drugs include dactinomycin, carboplatin, cyclophosphamide, ifosfamide, etoposide, vincristine sulfate, thiotepa, doxorubicin hydrochloride, and methotrexate.

The purpose of this phase is to reduce the size of the tumors and assess the body’s response to the treatment.

3 evaluation and review

After completing the induction chemotherapy, another evaluation is conducted. This includes MRI and CSF examinations to determine the effectiveness of the treatment and to decide the next steps in the trial.

4 consolidation therapy

Depending on the age and response to the initial treatment, the next phase involves consolidation therapy. This may include high-dose chemotherapy (HDCT) or focal radiotherapy (RT).

For children aged 12-35 months, the trial evaluates the effectiveness of three courses of HDCT compared to RT. For children under 12 months, HDCT is used as a consolidation measure. For those aged 36 months or older, RT combined with conventional chemotherapy is assessed.

5 follow-up and monitoring

Throughout the trial, regular follow-up appointments are scheduled to monitor health and response to treatment. This includes assessments of overall survival, neurocognitive outcomes, quality of life, and any adverse effects.

These follow-ups occur at various intervals, including 2 and 5 years after the initial diagnosis, to ensure long-term health and well-being.

Who Can Join the Study?

Age at diagnosis must be less than 18 years.
For Part A: Must have an MRI and CSF (cerebrospinal fluid) examination after 3 courses of chemotherapy showing stable disease or better.
For Part A: Blood tests must show ALT or AST levels less than or equal to 3 times the upper limit of normal (ULN), and bilirubin levels less than or equal to 1.5 times the ULN.
For Part A: Creatinine levels must be less than or equal to 1.5 times the ULN, and kidney function (GFR) must be within normal age-related values.
For Part A: Heart function tests must show an ejection fraction (EF) of 50% or more, or a fractional shortening (FS) of 29% or more.
For Part B: Must be enrolled in the umbrella trial.
For Part B: Must have received 3 courses of induction chemotherapy according to the protocol.
For Part B: Radiotherapy is not allowed (for example, if under 12 months old or other reasons).
For Part B: Not eligible for the randomized trial in Part A (for example, if randomization is refused).
For Part B: Must have written informed consent or assent according to national laws.
For Part B: Central review must confirm the diagnosis of ATRT.
Pathology must be compatible with ATRT, and there must be a confirmed loss or deficiency of INI1, SMARCB1, or SMARCA4 by a local pathology lab.
For Part B: MRI and CSF examination after 3 courses of chemotherapy must show significant sensitivity to chemotherapy.
For Part B: Blood tests must show ALT or AST levels less than or equal to 3 times the ULN, and bilirubin levels less than or equal to 1.5 times the ULN.
For Part B: Creatinine levels must be less than or equal to 1.5 times the ULN, and kidney function (GFR) must be within normal age-related values.
For Part B: Heart function tests must show an ejection fraction (EF) of 50% or more, or a fractional shortening (FS) of 29% or more.
For Part C: Must be enrolled in the umbrella trial.
For Part C: Must have received 3 courses of induction chemotherapy according to the protocol.
For Part C: Must be aged 36 months or older, or high-dose chemotherapy (HDCT) is not possible, or not eligible for the randomized trial in Part A.
For Part C: Must have written informed consent or assent according to national laws.
For Part C: Central review must confirm the diagnosis of ATRT.
For Part C: MRI and CSF examination after 3 courses of chemotherapy must show stable disease or better.
Must have written informed consent or assent for study participation according to national laws.
For Part C: Blood tests must show ALT or AST levels less than or equal to 3 times the ULN, and bilirubin levels less than or equal to 1.5 times the ULN.
For Part C: Creatinine levels must be less than or equal to 1.5 times the ULN, and kidney function (GFR) must be within normal age-related values.
For Part C: Heart function tests must show an ejection fraction (EF) of 50% or more, or a fractional shortening (FS) of 29% or more.
Patient agrees to use effective contraception while on treatment.
For Part A: Must be enrolled in the umbrella trial.
For Part A: Must have received 3 courses of induction chemotherapy according to the protocol and show stable disease or better after induction.
For Part A: Expected age must be between 12-35 months at the time of consolidation therapy (radiotherapy or high-dose chemotherapy).
For Part A: Must have written informed consent or assent for randomization according to national laws.
For Part A: Central review must confirm the diagnosis of ATRT.

Who Cannot Join the Study?

Patients who are not diagnosed with atypical teratoid/rhabdoid tumors (ATRT). ATRT is a rare and fast-growing type of brain tumor.
Children who are not within the specified age range for the study. The study is for children aged 12 to 35 months for Part A, and children under 12 months for Part B.
Children who are 36 months or older for Part C.
Patients who are not eligible for randomization within Part A of the study protocol.
Patients who are not suitable for the specific treatment plans outlined in the study, such as high-dose chemotherapy (HDCT) or radiation therapy (RT).

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country
University Hospital Jena KöR	Jena	Germany
Universitaetsmedizin Goettingen	Goettingen	Germany
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany
Universitaet Leipzig	Leipzig	Germany
Institut Gustave Roussy	Villejuif	France
Medizinische Hochschule Hannover	Hanover	Germany
Kuopio University Hospital	Kuopio	Finland
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz	Mainz	Germany
Technische Universitaet Dresden	Dresden	Germany
Katholieke Universiteit te Leuven	Leuven	Belgium
Institut Curie – Site Paris	Paris	France

Other Sites

Site Name	City	Country
Azienda Ospedaliera di Padova	Padua	Italy
Universitaetsklinikum Erlangen AöR	Erlangen	Germany
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Region Skane Skanes Universitetssjukhus	Lund	Sweden
Universitaetsklinikum Magdeburg AöR	Magdeburg	Germany
Hospital Universitario De Cruces	Barakaldo	Spain
Oslo University Hospital HF	Oslo	Norway
Turku University Hospital	Turku	Finland
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Universitaetsklinikum Ulm AöR	Ulm	Germany
Del-Pesti Centrumkorhaz Orszagos Hematologiai Es Infektologiai Intezet	Budapest	Hungary
Klinikum Kassel GmbH	Kassel	Germany
St. Olavs Hospital HF	Trondheim	Norway
Justus-Liebig-Universitaet Giessen	Giessen	Germany
CHC MontLegia	Liege	Belgium
Centre Hospitalier Universitaire De Rennes	Rennes	France
Rigshospitalet	Copenhagen	Denmark
Region Oestergoetland	Linkoping	Sweden
Universitair Ziekenhuis Gent	Gent	Belgium
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Universitaetsklinikum Schleswig-Holstein AöR	Kiel	Germany
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Oulu University Hospital	Oulu	Finland
Azienda Ospedaliera Santobono Pausilipon	Naples	Italy
Virgen del Rocío University Hospital	Sevilla	Spain
Universitaetsklinikum Regensburg AöR	Regensburg	Germany
Karolinska University Hospital	Solna	Sweden
Hospital Munich Schwabing	Munich	Germany
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
University Childrens Hospital Queen Fabiola	Brussels	Belgium
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Semmelweis University	Budapest	Hungary
Region Midtjylland	Aarhus	Denmark
University Of Pecs	Pecs	Hungary
Pirkanmaan hyvinvointialue	Tampere	Finland
Region Vaesterbotten	Umea	Sweden
Universitaetsklinikum Aachen AöR	Aachen	Germany
Deutsches Herzzentrum Berlin	Berlin	Germany
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Odense University Hospital	Odense	Denmark
Cyhwwtjwy Ucvjveqyrphtiv Sxjoaakoc	Woluwe-Saint-Lambert	Belgium
Agrhllcamv Pxlsbmhb Horqtjxl Dn Mmamrlfuz	Marseille	France
Hbtpe Btgedr Hk	Bergen	Norway
Hcxwudcq Uuxxjzvfrw Cotgmwi Hjneksij	Helsinki	Finland
Azfuava Oamueuhoucs Upeopfcpcnrpo Cklaptbvscgd Ddqxs Sioomt E Dfovm Sdpfygr Dh Tbuvze	Turin	Italy
Uhmmwzddhtrkbdjjcbwla Mierpfes Aex	Munster	Germany
Crtjzx Lcdn Bzwhnp	Lyon	France
Feuvvczc nbwsnwxff Mrbzq a Hykxchm	Prague	Czechia
Azselix Upvxw Shsoikwcb Lgxpqp Dh Bnqeepz	Bologna	Italy
Mzbtfafjxmuhjntzekokloxalf Hchxbydfbkfjzvqe	Halle (Saale)	Germany
Ucyigcspbsopzhtzrpvrk Aavemeqj	Augsburg	Germany
Ccgkjc Hsvjkxnweiw Rprbtbzo Donxufvdevrmtc	Angers	France
Fkwjbntoz Ppsr Lq Iaimxeaboyxwn Bhdyickeb Dii Hlltunvh Ufbdsulqzqbfa Lt Piz	Madrid	Spain
Usrgzyipnj Hlnqujjo Cgzgitz	Cologne	Germany
Uetswwj Uaanmuzrnt Hpjqvqud	Uppsala	Sweden
Lsvuww Movkiivbqy Uwqohuztpc Oz Mtisjx	Munich	Germany
Gdxlkj Usutqeyjhy Fhdfxwjeb	Frankfurt	Germany
Unqpqlbhqhdneupbiigiw Wncjiaypn Arq	Wuerzburg	Germany
Uahlqmgnxk Mzrhlyy Cpvyfw Hhdxjlqinxxtvoymt	Hamburg	Germany
Uegfwgetgxcouyilzsboa Ebiyt Ahs	Essen	Germany
Umvwhopismkj Modiwir Ckghqws Gstjjphxn	Groningen	The Netherlands
Hayvxgei Vnni dulszden	Barcelona	Spain
Hnrzqepm Ulqnwhhnghatxj Suytiaomon &aqtlnc Hzeqcgi dg Hlogwyjhkkq	STRASBOURG, Alsace	France

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	01.06.2021
Czechia	Recruiting	01.06.2021
Denmark	Recruiting	01.06.2021
Finland	Recruiting	01.06.2021
France	Recruiting	01.06.2021
Germany	Recruiting	01.06.2021
Hungary	Recruiting	01.06.2021
Italy	Recruiting	01.06.2021
Norway	Recruiting	01.06.2021
Spain	Recruiting	01.06.2021
Sweden	Recruiting	01.06.2021
The Netherlands	Recruiting	01.06.2021

Trial locations

Investigated drugs:

High-Dose Chemotherapy (HDCT) is a treatment that uses very high doses of powerful drugs to kill cancer cells. In this trial, it is used as a consolidation therapy, which means it is given after the initial treatment to help eliminate any remaining cancer cells and reduce the risk of the cancer coming back. The goal is to improve the chances of survival for children with atypical teratoid/rhabdoid tumors (ATRT).

Focal Radiotherapy (RT) is a treatment that uses high-energy rays to target and destroy cancer cells in a specific area of the body. In this trial, it is used as a consolidation therapy, similar to high-dose chemotherapy, to help ensure that any remaining cancer cells are destroyed after the initial treatment. This therapy is focused on a particular part of the body where the tumor was located.

Conventional Chemotherapy involves the use of standard cancer-fighting drugs that are given to patients to kill cancer cells or stop them from growing. In this trial, conventional chemotherapy is used alongside other treatments to help improve the overall effectiveness of the therapy and increase the chances of survival for children with ATRT.

Investigated diseases:

Atypical teratoid/rhabdoid tumour of CNS

Atypical Teratoid/Rhabdoid Tumors (ATRT) – Atypical Teratoid/Rhabdoid Tumors are rare, fast-growing tumors that typically occur in the brain and spinal cord. They are most commonly diagnosed in young children. These tumors are characterized by their aggressive nature and can spread quickly within the central nervous system. ATRT is composed of a mix of different cell types, which can make it challenging to study. The progression of ATRT involves rapid growth and potential spread to other areas of the brain or spine. Symptoms often depend on the tumor’s location and may include headaches, nausea, and changes in behavior or motor skills.

Trial ID:

2022-501456-28-00

Protocol code:

SIOPEATRT01

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Atypical Teratoid/Rhabdoid Tumors in Children Using Dactinomycin, Carboplatin, and Cyclophosphamide

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?