Lutetium (177Lu) oxodotreotide for Gastroenteropancreatic neuroendocrine tumor | Pheochromocytoma | Paraganglioma

Inclusion criteria

• {"criterion_text":"- GEP-NET cohort: presence of metastasized or locally advanced, inoperable (curative intent), histologically proven, G1 or G2 (Ki-67 index ≤20%), well differentiated GEP-NET.\n- PPGL cohort: presence of metastasized or locally advanced, inoperable (curative intent), histologically proven PPGL.\n- Patients from 12 to < 18 years of age at the time of enrollment.\n- Expression of somatostatin receptors confirmed by a somatostatin receptor imaging (SRI) modality within 3 months prior to enrollment, with tumor uptake observed in the target lesions more or equal to the normal liver uptake.\n- Performance status as determined by Karnofsky score ≥ 50 or Lansky Play-Performance Scale score ≥ 50.\n- Parent’s ability to understand and the willingness to sign a written informed consent document for adolescents as determined by local regulations. Adolescents will sign assent along with parental/legal guardian consent or will co-sign consent with parent/legal guardian in accordance with local regulation, prior to participation in the study."}

Exclusion criteria

• {"criterion_text":"- Laboratory parameters: • Estimated creatinine clearance calculated by the Cockroft-Gault method < 70 mL/min • Hb concentration <5.0 mmol/L (<8.0 g/dL); WBC <2x109/L; platelets <75x109/L. • Total bilirubin >3 x ULN for age. • Serum albumin <3.0 g/dL unless prothrombin time is within the normal range.\n- Patients with any other significant medical, psychiatric, or surgical condition, currently uncontrolled by treatment, which may interfere with the completion of the study.\n- Patient with known incompatibility to CT scans with I.V. contrast due to allergic reaction or renal insufficiency. If such a patient can be imaged with MRI, then the patient would not be excluded.\n- Patients who received any investigational agent within the last 30 days.\n- Prior therapies and procedures as detailed in Section 5.2.\n- Established or suspected pregnancy\n- Breastfeeding female patients unless they accept to discontinue breastfeeding from the 1st dose until 3 months after the last administration of study drug.\n- Female patients of child-bearing potential (female pediatric patients who are menarchal or who become menarchal during the study), unless they are using highly effective methods of contraception during treatment and for 7 months after the last dose of Lutathera (see details in the Appendix 1). If local regulations deviate from the listed contraception methods to prevent pregnancy, local regulations apply and will be described in the ICF.\n- Sexually active male patients, unless they agree to remain abstinent (refrain from heterosexual intercourse) or be willing to use condoms and highly effective methods of contraception with female partners of childbearing potential, and to use condoms with pregnant female partners during the treatment period and for at least 4 months after the last dose of Lutathera (see details in Appendix 1). In addition, male patients must refrain from donating sperm during this same period.\n- Patients for whom in the opinion of the investigator other therapeutic options are considered more appropriate than the therapy offered in the study, based on patient and disease characteristics.\n- Current spontaneous urinary incontinence.\n- Other known co-existing malignancies except non-melanoma skin cancer and carcinoma in situ of the uterine cervix, unless definitively treated and proven no evidence of recurrence for 5 years.\n- Hypersensitivity to the study drug active substance or to any of the excipients."}

Primary endpoints

• {"endpoint_text":"- Target organ (e.g. kidney and bone marrow) absorbed radiation doses in adolescents with SSTR-positive GEP-NETs and PPGLs as a pooled cohort.","definition_or_measurement_approach":"Calculated organ absorbed radiation doses from PRRT with Lutathera; measurement based on dosimetry (imaging and radioactivity measurements)."}
• {"endpoint_text":"- The incidence of adverse events (AEs) and laboratory toxicities after the 1st Lutathera administration in adolescents with SSTR-positive GEPNETs and PPGLs as a pooled cohort","definition_or_measurement_approach":"Incidence (count and rate) of AEs and laboratory toxicities occurring after the first administration of Lutathera; assessed by recorded adverse events and laboratory results following initial dosing."}

Secondary endpoints

• {"endpoint_text":"- The incidence of adverse events (AEs) and laboratory toxicities until 6 months after the last Lutathera dose (short-term follow-up) in adolescents with SSTR-positive GEP-NETs and PPGLs as a pooled cohort.","definition_or_measurement_approach":"Incidence of AEs and laboratory toxicities up to 6 months post last Lutathera dose, assessed by AE reporting and laboratory monitoring."}
• {"endpoint_text":"- The incidence of adverse events (AEs) and laboratory abnormalities during the long term follow-up of 5 years and 10 years after the last Lutathera dose in adolescents with SSTR-positive GEP-NETs and PPGLs as a pooled cohort.","definition_or_measurement_approach":"Incidence of AEs and laboratory abnormalities assessed during long-term follow-up visits at 5 and 10 years after last Lutathera dose."}
• {"endpoint_text":"- Calculated organ absorbed doses and PK parameters based on imaging/blood radioactivity concentration data from adolescent patients with SSTR-positive GEP-NETs and PPGLs as a pooled cohort compared to the predicted distribution /organ absorbed doses","definition_or_measurement_approach":"Organ absorbed dose calculations and PK parameter estimation based on imaging and blood radioactivity concentration data; comparison versus predicted distributions/organ absorbed doses using the extrapolation model."}

France

Earliest CTIS Part Ii Submission Date

28-03-2024

Latest Decision Or Authorization Date

14-04-2026

Processing Time Days

747

Number Of Sites

1

Number Of Participants

2

Spain

Earliest CTIS Part Ii Submission Date

28-03-2024

Latest Decision Or Authorization Date

14-04-2026

Processing Time Days

747

Number Of Sites

1

Number Of Participants

1

Poland

Earliest CTIS Part Ii Submission Date

28-03-2024

Latest Decision Or Authorization Date

13-04-2026

Processing Time Days

746

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

Advanced Accelerator Applications

Organisation Type

Pharmaceutical company

Country Of Registered Address

France

Contract research organisations

Name

IQVIA Limited

Responsibilities

Site Payment, Coordination of the logistics related to Drug shipment on site; additional operational roles (sponsorDuties codes: 1,12,15,2,5,6,8,9)

Name

Creative Development Enterprises Inc.

Responsibilities

Medical image analysis/review, primary/surrogate endpoint test, bioanalysis central laboratory testing (sponsorDuties code: 15)

Third parties

• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Creative Development Enterprises Inc.","duties_or_roles":"Medical Image analysis/review, primary/surrogate endpoint test, bioanalysis central laboratory testing (sponsorDuties code: 15)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"sponsorDuties codes: 1,12,15 (includes: Site Payment, Coordination of the logistics related to Drug shipment on site), 2,5,6,8,9","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}