Phase 2 study to evaluate safety and dosimetry of Lutathera in adolescent patients with GEP-NETs, pheochromocytoma and paragangliomas

Phase 1

Conditions: somatostatin receptor positive gastroenteropancreatic neuroendocrine (GEP-NET) tumors, pheochromocytoma and paragangliomas
MedDRA version: 21.0Level: PTClassification code 10052399Term: Neuroendocrine tumourSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0Level: PTClassification code 10077559Term: Gastroenteropancreatic neuroendocrine tumour diseaseSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0Level: LLTClassification code 10077560Term: Gastroenteropancreatic neuroendocrine tumor diseaseSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.1Level: LLTClassification code 10034876Term: PheochromocytomaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0Level: LLTClassification code 10073860Term: ParagangliomaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Therapeutic area: Diseases [C] - Cancer [C04]

Registration Number: EUCTR2020-002951-39-PT

Lead Sponsor: Advanced Accelerator Applications SA

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

1. 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.
PPGL cohort: presence of metastasized or locally advanced, inoperable (curative intent), histologically proven PPGL.
2. Patients from 12 to < 18 years of age at the time of enrollment.
3. 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.
4. Performance status as determined by Karnofsky score = 50 or Lansky Play-Performance Scale score = 50.

Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: - To evaluate organ absorbed radiation doses from PRRT with Lutathera in adolescent patients with SSTR-positive GEP-NETs<br> - To evaluate safety and tolerability of Lutathera in adolescents with SSTR-positive GEP-NETs<br>;Secondary Objective: - To evaluate cumulative safety of Lutathera in adolescents with SSTR-positive GEP-NETs<br> - To evaluate long-term safety of Lutathera in adolescents with SSTR-positive GEP-NETs<br> - To perform comparative assessment of dosimetry and pharmacokinetics (PK) between adolescent patients with GEP-NET and adult patients using the extrapolation model developed for the clinical study;Primary end point(s): - Target organ (e.g. kidney and bone marrow) absorbed radiation doses in adolescents with SSTR-positive GEP-NETs<br> - The incidence of adverse events (AEs) and laboratory toxicities after the 1st Lutathera administration in adolescents with SSTR-positive GEP-NETs;Timepoint(s) of evaluation of this end point: After 1st administration

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - 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<br> - The incidence of adverse events (AEs) and laboratory abnormalities during the long term follow-up of 5 years after the last Lutathera dose in adolescents with SSTR-positive GEP-NETs<br> - Calculated organ absorbed doses and PK parameters based on imaging/blood radioactivity concentration data from adolescent patients with SSTR-positive GEP-NETs compared to the predicted distribution / organ absorbed doses;Timepoint(s) of evaluation of this end point: After 1st administration, until 6 months after the last Lutathera dose and until 5 years after the last Lutathera dose

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