Study to treat adult patients with Very High-Risk T-cell Acute Lymphoblastic Leukemia (ALL) with Daratumumab in addition to the National ALL treatment program

Start 17 Oct 2024 · Status Ongoing, recruitment ended · 1 EU/EEA countries · 23 sites · Protocol ALL3024

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)

Status Ongoing, recruitment ended

Participants planned 31

Countries 1

Sites 23

Very High-Risk T-Lineage Acute Lymphoblastic Leukemia (ALL)

The primary objective of the trial is to evaluate the impact of the addition of daratumumab to the national standard of care, based on the pediatric-inspired treatment (i.e. LAL1913) in increasing the MRD-negativity rate (<10-4) at time point 1 (TP1), i.e. after the first induction cycle with chemotherapy plus daratumu…

Key facts

Sponsor: Fondazione Gimema Franco Mandelli Onlus
Participant type: Patients
Age range: 18-64 years, 65+ years
Gender: Male and Female
Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
Trial duration: 17 Oct 2024 → ongoing
Decision date (initial): 2024-07-26
Transition trial: No
Low-intervention: No
Rare-disease indication: Yes
Vulnerable population: No
Funding sources: Fondazione GIMEMA Franco Mandelli Onlus · JANSSEN-CILAG

External identifiers

EU CT number: 2024-511627-34-00
ClinicalTrials.gov: NCT06253637

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

Secondary objectives 8

To evaluate MRD at later timepoints
To evaluate the feasibility of transplant
To evaluate survival outcomes (DFS, EFS, OS) of patients treated with daratumumab and chemotherapy
To evaluate the feasibility of a combination approach with daratumumab and chemotherapy, in terms of side effects, adverse events and serious adverse events, both hematologic and non-hematologic and treatment-related mortality.
To investigate the impact of CD38 expression on outcome endpoints
To correlate the therapeutic effect of daratumumab with genomic profile
To explore the immunomodulatory activity of daratumumab in ALL
To perform a refined Ig/TR marker screening with NGS assays and NGS-based MRD monitoring

Conditions and MedDRA coding

Very High-Risk T-Lineage Acute Lymphoblastic Leukemia (ALL)

Version	Level	Code	Term	System organ class
21.1	LLT	10066105	T-cell lymphoblastic leukaemia acute	10029104

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

Age 18-65 years.
A diagnosis of T-ALL according to the 2022 International Consensus Classification (ICC) is required, either de novo or secondary to chemo-radiotherapy for another cancer. Pre-treatment with low-dose corticosteroids +/- cyclophosphamide in patients presenting with hyperleukocytosis is allowed.
Availability of fresh bone marrow (BM) (or peripheral blood (PB) in patients with hyperleukocytosis) samples to perform diagnostic procedures).
Bone marrow blast percentage at diagnosis ≥20%.
CD38 positivity on ALL blasts (any level of positivity).
ETP and near ETP at diagnosis according to internationally accepted criteria (appendix G) at diagnosis or other VHR T-ALL subtypes (WBC count >100 x109/L; complex karyotype with ≥5 unrelated anomalies; other CD1a-negative immunophenotypes). T-Myeloid MPAL according to the 2022 ICC of Acute Leukemias of Ambiguous Lineage (appendix H) can also be eligible and considered as VHR.
Availability of full cytological, cytochemical, immunophenotypic, cytogenetic and molecular disease characterization according to the EGIL and WHO classifications.
An ECOG performance status 0-2, unless a performance of 3 is unequivocally caused by the disease itself, (and not by pre-existing comorbidities,) and is considered and/or documented to be reversible following the application of anti-leukemic therapy and appropriate supportive measures.
For females of childbearing potential, a negative pregnancy test must be documented. Female and male patients who are fertile must agree to use an effective form of contraception with their sexual partners from enrollment through 12 months after the end of treatment.
Signed written informed consent according to ICH/E U/GCP and national local laws.

Exclusion criteria 8

Diagnosis of B-lineage ALL, and Ph+ ALL.
Down’s syndrome.
Prior systemic chemotherapy for ALL (excluding cyclophosphamide during pre-phase).
Pre-existing, uncontrolled pathology such as heart failure (congestive/ischemic, acute myocardial infarction within the past 3 months, untreatable arrhythmias, NYHA classes III and IV), FE<50% (unless attributable to ALL), severe liver disease with serum direct bilirubin >3 mg/dL (unless attributable to Gilbert’ syndrome or ALL) and/or ALT >5x upper normal limit (unless attributable to ALL), kidney function impairment with serum creatinine >2 mg/dL (unless attributable to ALL), severe lung disease with FEV1<50% (unless attributable to ALL) and severe neuropsychiatric disorder that impairs the patient’s ability to understand and sign the informed consent, or to cope with the intended treatment plan. N.B. For altered liver and kidney function tests, eligibility criteria can be reassessed at 24-96 hours, following the institution of adequate supportive measures.
Presence of serious, active, uncontrolled infections.
A history of cancer that is not in a remission phase following surgery and/or radiotherapy and/or chemotherapy, with a life expectancy <2 years.
Patients who have undergone major surgery ≤ 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.
Patients who are pregnant or breast feeding and adults of reproductive potential not employing an effective method of birth control (women of childbearing potential must have a negative serum pregnancy test within 48 hrs. prior treatment start). Postmenopausal women must be amenorrheic for at least 12 months to be considered of non-childbearing potential. Male and female patients must agree to employ two effective reliable methods of birth control throughout the study and for up to 12 months following discontinuation of study drugs.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

The primary endpoint of this study is to evaluate clinical response - in terms of MRD negativity (<10^-4) after induction (TP1) - in patients with very high-risk T-ALL treated with a daratumumab plus chemotherapy approach.

Secondary endpoints 8

The rate of MRD negativity at TP2, TP3, TP4 and before allo-SCT
The rate of allo-SCT allocation
DFS at 12 months
EFS at 18 months
CIR estimation from CR achievement at 18 months
OS at 18 months
Treatment-related mortality (TRM)
Rate of Adverse Events (AEs) and Serious AEs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

DARZALEX 1800 mg solution for injection

PRD8157846 · Product

Active substance: Daratumumab
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: SUBCUTANEOUS INJECTION
Max daily dose: 1800 mg milligram(s)
Max total dose: 23.4 g gram(s)
Max treatment duration: 13 Day(s)
Authorisation status: Authorised
ATC code: L01FC01 — -
Marketing authorisation: EU/1/16/1101/004
MA holder: JANSSEN-CILAG INTERNATIONAL NV
MA country: EU
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Auxiliary 38

Furosemide

SUB07849MIG · Substance

Active substance: Furosemide
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENIOUS INFUSION
Max daily dose: 40 mg milligram(s)
Max total dose: 40 mg milligram(s)
Max treatment duration: 1 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Granisetron

SUB07964MIG · Substance

Active substance: Granisetron
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENIOUS INFUSION
Max daily dose: 9 mg milligram(s)
Max total dose: 1.6 g gram(s)
Max treatment duration: 8 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Diphenhydramine

SUB07211MIG · Substance

Active substance: Diphenhydramine
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 50 mg milligram(s)
Max total dose: 650 mg milligram(s)
Max treatment duration: 13 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Allopurinol

SUB05338MIG · Substance

Active substance: Allopurinol
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 600 mg milligram(s)
Max total dose: 600 mg milligram(s)
Max treatment duration: 1 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Cytarabine

SUB06880MIG · Substance

Active substance: Cytarabine
Pharmaceutical form: POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS (IV) OR SUBCUTANEOUS (SC)
Max daily dose: 4000 mg/m2 milligram(s)/sq. meter
Max total dose: 16900 mg/m2 milligram(s)/sq. meter
Max treatment duration: 16 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Cytarabine

SUB06880MIG · Substance

Active substance: Cytarabine
Pharmaceutical form: POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS (IV) OR SUBCUTANEOUS (SC)
Max daily dose: 50 mg milligram(s)
Max total dose: 400 mg milligram(s)
Max treatment duration: 8 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Hydrocortisone

SUB08065MIG · Substance

Active substance: Hydrocortisone
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS
Max daily dose: 100 mg milligram(s)
Max total dose: 400 mg milligram(s)
Max treatment duration: 4 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Montelukast

SUB09054MIG · Substance

Active substance: Montelukast
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 10 mg milligram(s)
Max total dose: 10 mg milligram(s)
Max treatment duration: 1 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Paracetamol

SUB09611MIG · Substance

Active substance: Paracetamol
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 1000 mg milligram(s)
Max total dose: 13 g gram(s)
Max treatment duration: 13 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Co-Trimoxazole

SUB13477MIG · Substance

Active substance: Co-Trimoxazole
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 960 mg milligram(s)
Max total dose: 400.73 g gram(s)
Max treatment duration: 7 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Filgrastim

SUB07627MIG · Substance

Active substance: Filgrastim
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: SUBCUTANEOUS
Max daily dose: 5 µg/Kg microgram(s)/kilogram
Max total dose: 840 µg/Kg microgram(s)/kilogram
Max treatment duration: 24 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Foscarnet

SUB02258MIG · Substance

Active substance: Foscarnet
Pharmaceutical form: SOLUTION FOR INFUSION
Route of administration: INTRAVENOUS INFUSION
Max daily dose: 120 mg/kg milligram(s)/kilogram
Max total dose: 2520 mg/Kg milligram(s)/kilogram
Max treatment duration: 21 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Ciprofloxacin

SUB07470MIG · Substance

Active substance: Ciprofloxacin
Pharmaceutical form: FILM-COATED TABLET
Route of administration: ORAL
Max daily dose: 1000 mg milligram(s)
Max total dose: 28 g gram(s)
Max treatment duration: 4 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Vincristine Sulfate

SUB05101MIG · Substance

Active substance: Vincristine Sulfate
Pharmaceutical form: SOLUTION FOR INJECTION OR INFUSION
Route of administration: INTRAVENIOUS INFUSION
Max daily dose: 2 mg/m2 milligram(s)/sq. meter
Max total dose: 20 mg/m2 milligram(s)/sq. meter
Max treatment duration: 10 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Human Fibrinogen

SUB12502MIG · Substance

Active substance: Human Fibrinogen
Pharmaceutical form: POWDER FOR SOLUTION FOR INJECTION/INFUSION
Route of administration: INTRAVENIOUS INFUSION
Max daily dose: 2 g gram(s)
Max total dose: 20 g gram(s)
Max treatment duration: 10 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Amphotericin B

SUB05486MIG · Substance

Active substance: Amphotericin B
Pharmaceutical form: POWDER FOR CONCENTRATE FOR DISPERSION FOR INFUSION
Route of administration: IV INFUSION
Max daily dose: 2 mg/ml milligram(s)/millilitre
Max total dose: 28 mg/ml milligram(s)/millilitre
Max treatment duration: 14 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Folinic Acid

SUB13910MIG · Substance

Active substance: Folinic Acid
Pharmaceutical form: POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS INFUSION
Max daily dose: 37.5 mg/m2 milligram(s)/sq. meter
Max total dose: 787.5 mg/m2 milligram(s)/sq. meter
Max treatment duration: 6 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Levofloxacin

SUB08471MIG · Substance

Active substance: Levofloxacin
Pharmaceutical form: FILM COATED TABLETS
Route of administration: ORAL
Max daily dose: 500 mg milligram(s)
Max total dose: 14 g gram(s)
Max treatment duration: 4 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Methotrexate

SUB08856MIG · Substance

Active substance: Methotrexate
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENIOUS INFUSION
Max daily dose: 5000 mg/m2 milligram(s)/sq. meter
Max total dose: 15000 mg/m2 milligram(s)/sq. meter
Max treatment duration: 3 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Methotrexate

SUB08856MIG · Substance

Active substance: Methotrexate
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS INFUSION
Max daily dose: 15 mg milligram(s)
Max total dose: 120 mg milligram(s)
Max treatment duration: 8 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Prednisone

SUB10020MIG · Substance

Active substance: Prednisone
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 40 mg/m2 milligram(s)/sq. meter
Max total dose: 480 mg/m2 milligram(s)/sq. meter
Max treatment duration: 12 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Ondansetron

SUB09445MIG · Substance

Active substance: Ondansetron
Pharmaceutical form: SOLUTION FOR INJECTION/INFUSION IN PRE-FILLED SYRINGE
Route of administration: INTRAVENOUS
Max daily dose: 16 mg milligram(s)
Max total dose: 448 mg milligram(s)
Max treatment duration: 48 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Ganciclovir

SUB07881MIG · Substance

Active substance: Ganciclovir
Pharmaceutical form: POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSION
Route of administration: INTRAVENOUS INFUSION
Max daily dose: 10 mg/kg milligram(s)/kilogram
Max total dose: 245 mg/kg milligram(s)/kilogram
Max treatment duration: 28 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Mercaptopurine

SUB12149MIG · Substance

Active substance: Mercaptopurine
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 60 mg/m2 milligram(s)/sq. meter
Max total dose: 2050 mg/m2 milligram(s)/sq. meter
Max treatment duration: 40 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Cyclophosphamide

SUB06859MIG · Substance

Active substance: Cyclophosphamide
Pharmaceutical form: POWDER FOR SOLUTION FOR INFUSION
Route of administration: INTRAVENOUS
Max daily dose: 1000 mg/m2 milligram(s)/sq. meter
Max total dose: 4800 mg/m2 milligram(s)/sq. meter
Max treatment duration: 9 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Rasburicase

SUB04204MIG · Substance

Active substance: Rasburicase
Pharmaceutical form: POWDER AND SOLVENT FOR SOLUTION FOR INFUSION
Route of administration: INTRAVENOUS
Max daily dose: 0.2 mg/kg milligram(s)/kilogram
Max total dose: 1.4 mg/kg milligram(s)/kilogram
Max treatment duration: 7 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Chlorphenamine

SUB06201MIG · Substance

Active substance: Chlorphenamine
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS (IV) OR SUBCUTANEOUS (SC)
Max daily dose: 10 mg milligram(s)
Max total dose: 130 mg milligram(s)
Max treatment duration: 13 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Aprepitant

SUB20017 · Substance

Active substance: Aprepitant
Pharmaceutical form: CAPSULE, HARD
Route of administration: ORAL
Max daily dose: 125 mg milligram(s)
Max total dose: 1128 mg milligram(s)
Max treatment duration: 24 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Salbutamol

SUB10422MIG · Substance

Active substance: Salbutamol
Pharmaceutical form: PRESSURISED INHALATION, SUSPENSION
Route of administration: INHALATION
Max daily dose: 800 µg microgram(s)
Max total dose: 10.4 mg milligram(s)
Max treatment duration: 13 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Lenograstim

SUB02888MIG · Substance

Active substance: Lenograstim
Pharmaceutical form: SOLUTION FOR INJECTION/INFUSION
Route of administration: INTRAVENOUS (IV) OR SUBCUTANEOUS (SC)
Max daily dose: 5 µg/Kg microgram(s)/kilogram
Max total dose: 840 µg/Kg microgram(s)/kilogram
Max treatment duration: 24 Week(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Idarubicin Hydrochloride

SUB02635MIG · Substance

Active substance: Idarubicin Hydrochloride
Pharmaceutical form: POWDER FOR SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS
Max daily dose: 12 mg/m2 milligram(s)/sq. meter
Max total dose: 80 mg/m2 milligram(s)/sq. meter
Max treatment duration: 7 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Dexamethasone Sodium Phosphate

SUB01615MIG · Substance

Active substance: Dexamethasone Sodium Phosphate
Pharmaceutical form: SOLUTION FOR INJECTION/INFUSION
Route of administration: INTRAVENOUS
Max daily dose: 10 mg/m2 milligram(s)/sq. meter
Max total dose: 295 mg/m2 milligram(s)/sq. meter
Max treatment duration: 30 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Dexamethasone Sodium Phosphate

SUB01615MIG · Substance

Active substance: Dexamethasone Sodium Phosphate
Pharmaceutical form: SOLUTION FOR INJECTION
Route of administration: SOLUTION FOR INJECTION OR INFUSION
Max daily dose: 4 mg milligram(s)
Max total dose: 32 mg milligram(s)
Max treatment duration: 8 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Aciclovir

SUB05235MIG · Substance

Active substance: Aciclovir
Pharmaceutical form: TABLET
Route of administration: ORAL
Max daily dose: 400 mg milligram(s)
Max total dose: 70 g gram(s)
Max treatment duration: 175 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Methylprednisolone Sodium Succinate

SUB14562MIG · Substance

Active substance: Methylprednisolone Sodium Succinate
Pharmaceutical form: POWDER AND SOLVENT FOR SOLUTION FOR INJECTION
Route of administration: INTRAVENOUS
Max daily dose: 20 mg milligram(s)
Max total dose: 160 mg milligram(s)
Max treatment duration: 8 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Micafungin

SUB16444MIG · Substance

Active substance: Micafungin
Pharmaceutical form: POWDER FOR CONCENTRATE FOR SOLUTION FOR INFUSION
Route of administration: INTRAVENOUS INFUSION
Max daily dose: 50 mg/kg milligram(s)/kilogram
Max total dose: 1050 mg/kg milligram(s)/kilogram
Max treatment duration: 21 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Levocarnitine

SUB08466MIG · Substance

Active substance: Levocarnitine
Pharmaceutical form: ORAL SOLUTION
Route of administration: ORAL
Max daily dose: 100 mg/kg milligram(s)/kilogram
Max total dose: 16800 mg/kg milligram(s)/kilogram
Max treatment duration: 168 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Pegaspargase

SUB03666MIG · Substance

Active substance: Pegaspargase
Pharmaceutical form: SOLUTION FOR INJECTION/INFUSION
Route of administration: INTRAVENIOUS INFUSION
Max daily dose: 3750 IU international unit(s)
Max total dose: 14250 IU international unit(s)
Max treatment duration: 4 Day(s)
Authorisation status: Authorised
ATC code: - — -
Marketing authorisation: -
Paediatric formulation: No
Orphan designation: No
Modified vs. Marketing Authorisation: No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fondazione Gimema Franco Mandelli Onlus

Sponsor organisation: Fondazione Gimema Franco Mandelli Onlus
Address: Via Casilina 5
City: Rome
Postcode: 00182
Country: Italy

Scientific contact point

Organisation: Fondazione Gimema Franco Mandelli Onlus
Contact name: Data center

Public contact point

Organisation: Fondazione Gimema Franco Mandelli Onlus
Contact name: Data center

Third parties 5

Organisation	City, country	Duties
Laboratorio di Medicina Molecolare, Unità di Ematologia, Università di Perugia, CREO ORL-000006904	Perugia, Italy	Laboratory analysis
Laboratorio Ematologia, Azienda Policlinico "Umberto I", Diapartimento Medicina Traslaz. e di Precis ORL-000006908	Roma, Italy	Laboratory analysis
Laboratorio di Ematologia “Paolo Belli” ASST Papa Giovanni XXIII ORL-000006907	Bergamo, Italy	Laboratory analysis
Evidenze Health S.r.l. ORG-100042105	Milan, Italy	On site monitoring
U.O.S.D. Laboratorio di Oncoematologia e Manipolazione Cellulare,Ospedali Riuniti Villa S.-Cervello ORL-000006905	Palermo, Italy	Laboratory analysis

Locations

1 EU/EEA country · 23 investigational sites

By country

Country	MS status	Planned subjects	Sites
Italy	Ongoing, recruitment ended	31	23
Rest of world	—	0	—

Investigational sites

Italy

23 sites · Ongoing, recruitment ended

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

DIPARTIMENTO DI DIAGNOSTICA PER IMMAGINI, RADIOTERAPIA ONCOLOGICA ED EMATOLOGIA, Largo Agostino Gemelli 8, 00168, Rome

Azienda Ospedaliero-Universitaria Policlinico Umberto I

DIPARTIMENTO DI MEDICINA TRASLAZIONALE E DI PRECISIONE, Viale Del Policlinico 155, 00161, Rome

Azienda Ospedaliera Ospedali Riuniti Villa Sofia Cervello

UOC ONCOEMATOLOGIA, Via Trabucco 180, 90146, Palermo

Azienda Unita Sanitaria Locale Della Romagna

EMATOLOGIA, Viale Luigi Settembrini 2, 47923, Rimini

Azienda Sanitaria Locale Di Pescara

DIPARTIMENTO ONCOLOGICO-EMATOLOGICO, Via Renato Paolini 47, 65124, Pescara

Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII

DIPARTIMENTO DI ONCOLOGIA ED EMATOLOGIA, Piazza Oms 1, 24127, Bergamo

Azienda Ospedaliera Universitaria Federico II Di Napoli

DIPARTIMENTO DI MEDICINA CLINICA E CHIRURGIA, Via Sergio Pansini 5, 80131, Naples

ASST Grande Ospedale Metropolitano Niguarda

DIPARTIMENTO DI EMATOLOGIA,ONCOLOGIA E MEDICINA MOLECOLARE, Piazza Dell'ospedale Maggiore 3, 20162, Milan

Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli

EMATOLOGIA, Via Antonio Cardarelli 9, 80131, Naples

Azienda Sanitaria Universitaria Friuli Centrale

SOC CLINICA EMATOLOGICA, Piazzale Santa Maria Della Misericordia 15, 33100, Udine

Hospital Santa Maria Della Misericordia

Dipartimento di Medicina e Chirurgia- Unità di Ematologia, Piazzale Giorgio Menghini 1, 06129, Perugia

IRCCS Ospedale Policlinico San Martino

EMATOLOGIA E TRAPIANTI, Largo Rosanna Benzi 10, 16132, Genoa

Ospedale Vito Fazzi Lecce

ONCOLOGIA, Piazza Filippo Muratore 1, 73100, Lecce

ULSS3 SERENISSIMA - Ospedale dell'Angelo di Mestre

UO EMATOLOGIA, via Paccagnella 11, Italy

Azienda Ospedaliero-Universitaria Maggiore Della Carita

SCDU EMATOLOGIA, Corso Giuseppe Mazzini 18, 28100, Novara

Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia

EMATOLOGIA, Piazzale Spedali Civili 1, 25123, Brescia

Careggi University Hospital

DIPARTIMENTO DI MEDICINA SPERIMENTALE E CLINICA, Largo Giovanni Alessandro Brambilla 3, 50134, Florence

Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico

DIPARTIMENTO MALATTIE ONCOLOGICHE ED EMATOLOGICHE, Via Pietro Albertoni 15, 40138, Bologna

Azienda Ospedaliero Universitaria Policlinico G Rodolico San Marco Di Catania

UOC DI EMATOLOGIA, Via Santa Sofia 78, 95123, Catania

Azienda Ospedaliera Universitaria Integrata Verona

DIPARTIMENTO DI INGEGNERIA PER LA MEDICINA DI INNOVAZIONE- AREA DI EMATOLOGIA, Piazzale Ludovico Antonio Scuro 10, 37134, Verona

Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di Torino

DIPARTIMENTO DI ONCOLOGIA ED EMATOLOGIA- SC EMATOLOGIA 2, Corso Bramante 88, 10126, Turin

Azienda Ospedaliero Universitaria Parma

MEDICINA E CHIRURGIA- EMATOLOGIA, Viale Antonio Gramsci 14, 43126, Parma

Azienda Ospedaliera Universitaria Senese

Dipartimento di Scienze Mediche, Chirurgiche e Neuroscienze, Strada Delle Scotte 14, 53100, Siena

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country	Trial start	Trial end	Recruitment start	Recruitment end	Early termination
Italy	2024-10-17		2024-11-11	2026-02-04

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 4 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Type	Title	Version
Recruitment arrangements (for publication)	K1_Recruitment arrangements	1
Subject information and informed consent form (for publication)	L1_Dear doctor letter IT	1
Subject information and informed consent form (for publication)	L1_SIS and ICF study IT_redacted	1.1
Subject information and informed consent form (for publication)	L1_SIS and ICF translational study IT_redacted	1

Application history

3 submissions — initial application plus substantial / non-substantial modifications

#	Type	Code	Submitted	Reference MS	Conclusion	Decision date
1	INITIAL	IN	2024-05-03	Italy	Acceptable 2024-07-25	2024-07-26
2	SUBSTANTIAL MODIFICATION	SM-1	2024-09-20	Italy	Acceptable	2024-10-09
3	SUBSTANTIAL MODIFICATION	SM-2	2025-11-13	Italy	Acceptable	2025-12-23