Qarziba for Patients in Relapsed/Refractory High-grade Osteosarcoma

NCT05558280 | Suspended Phase 2

• 1 other identifier: MaQ Study
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 1

Brief Summary

Limited progress has been made in identifying novel targets that may be therapeutic for Osteosarcoma(OS) and there remains an urgent need for the development of new agents that are effective in improving survival. From this perspective, repurposing already proven targets in other tumors may offer new opportunities for OS in children and young adults. Anecdotal evidence of anti-GD2 therapy exists in OS from prior Phase 1 trials that included patients with OS.

Conditions

Osteosarcoma in Children

Outcome Measures

Primary Outcomes (1)

• Efficacy of Dinutuximab Beta as maintenance therapy in patients with OS relapsed/refractory that have achieved Complete Response (CR) or Partial Response (PR)

  • Disease Control Rate (DCR) at 12 months from the last episode of relapse/refractory patients with CR or PR OS R/R treated with a maintenance therapy with Dinutuximab Beta.

  12months

Study Arms (1)

Dinutuximab beta

EXPERIMENTAL

The treatment phase foresees 5 cycles of Dinutuximab Beta. Dinutuximab Beta administration is restricted to hospital-use only and must be administered under the supervision of a physician experienced in the use of oncological therapies. It must be administered by a healthcare professional prepared to manage severe allergic reactions including anaphylaxis in an environment where full resuscitation services are immediately available. The study treatment will be administered with a continuous intravenous infusion at a dose of 14 mg/mq/day, days 1-5, a total of 60 hours (cumulative dose/cycle: 70 mg/mq). Each cycle lasts 28 days.

Drug: Dinutuximab beta

Interventions

Dinutuximab beta DRUG

The study treatment will be administered with a continuous intravenous infusion at a dose of 14 mg/mq/day, days 1-5, a total of 60 hours (cumulative dose/cycle: 70 mg/mq). Each cycle lasts 28 days.The treatment phase foresees 5 cycles of Dinutuximab Beta.

Also known as: QARZIBA

Dinutuximab beta

Eligibility Criteria

• Age: 1 Year - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Provision of signed and dated, written informed consent from each patient or patient's legally acceptable representative, parent (s) or legal guardian in accordance with regional laws or regulations and the patient's assent, when applicable, before any study-specific activity, including screening evaluation, is performed. For patients who reach the age of legal consent during the clinical study, notification may be required and a new consent form may need to be signed by the patient.
• Histologically confirmed high-grade osteosarcoma which is relapsed or refractory (ONLY patients in first or second relapse will be eligible). Histological confirmation from initial diagnosis or relapse is acceptable.
• Disease status: subjects must have achieved a complete or partial response after a second or further line of systemic therapy (with or without surgery) as defined by the following criteria:
• Complete Response = disappearance of all target and non-target lesions
• Partial Response = at least 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD of the last episode of relapse or recurrence. If a partial response is achieved, only patients with a focal localized disease at any site must be enrolled (for patients with lung disease a maximum of 2 lung unilateral nodules will be accepted).
• Age: ≥ 1 to \< 25 years old at the time of signing the informed consent form.
• Performance Level: Karnofsky Performance Status ≥ 60% for participants \> 16 years old or Lansky Play Score ≥ 60% for pediatric participants ≤ 16 years old. Subjects who are unable to walk because of paralysis and/or previous surgeries will be considered ambulatory for the purpose of assessing their performance score.
• Subjects must have recovered to \< Grade 2 per the NCI CTCAE v 4.03 or to baseline from any non-hematologic toxicities (except alopecia) due to previous therapy.
• Life expectancy ≥ 3 months.
• Completed previous line of treatment (systemic therapy with or without surgery) within 28 days prior the first dose of Dinutuximab Beta.
• Adequate bone marrow function independent of transfusion for at least 7 days prior to screening and independent of growth factor support for at least 14 days prior to screening
• Adequate organ function
• Normal ventricular ejection fraction (LV ejection fraction \> 50%).
• Availability of paraffin tumor material and/or fresh frozen tumor sample of the most recent biopsy.

You may not qualify if:

• Any other malignancy that required treatment within 2 years prior to study drug administration;
• Concomitant Medications:
• Anticancer agents: subjects who are currently receiving other anticancer agents
• Corticosteroid: Due to their immunosuppressive activity, concomitant treatment with corticosteroids is not recommend within 2 weeks prior to the first treatment course until 1 week after the last treatment course with Dinutuximab Beta, except for life-threatening conditions
• Prior Therapies and/or Procedures:
• Major surgery within 28 days prior to the first dose of Dinutuximab Beta.
• Minor surgery within 14 days prior to the first dose of Dinutuximab Beta.
• Received prior to treatment with Dinutuximab Beta or any other monoclonal antibody GD2.
• Received prior to treatment with chimeric antigen receptor anti GD2 therapy (CAR-T anti GD2) within 28 days prior to the first dose of Dinutuximab Beta.
• Received any anti-cancer drug within 28 days prior to the first dose of Dinutuximab Beta.
• Received any investigational drug within 28 days prior to the first dose of Dinutuximab Beta.
• Received radiotherapy or proton-therapy within 28 days prior to the first dose of Dinutuximab Beta.
• Received any immunotherapy within 28 days prior to the first dose of Dinutuximab Beta.
• Received any live (including attenuated) vaccines within 28 days prior the first dose of Dinutuximab Beta.
• Disease progression or presence of a multifocal disease after the induction therapy (except for the presence of only 2 unilateral lung diseases).

Sponsors & Collaborators

• Prof. Franca Fagioli: lead

Study Sites (1)

AOU Città della Salute e della Scienza di Torino - Presidio Infantile Regina Margherita

Turin, 10126, Italy

Location

MeSH Terms

Interventions

dinutuximab

Study Officials

• Franca Fagioli, MD

  A.O.U. Città della Salute e della Scienza

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: September 23, 2022
• First Posted: September 28, 2022
• Study Start: November 30, 2023
• Primary Completion: December 15, 2025
• Study Completion (Estimated): December 15, 2026
• Last Updated: November 18, 2023

Record last verified: 2023-11

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)