RecruitingPhase 3ACTRN12608000214336

A Pivotal Trial to Determine the Efficacy and Safety of AP23573 when Administered as Maintenance Therapy to Patients with Metastatic Soft Tissue or Bone Sarcomas.

A Phase 3, Randomised, Multi-Centre, Double Blinded, Placebo Controlled Trial to Determine the Efficacy and Safety of Oral AP23573 Versus Oral Placebo in Patients with Metastatic Sarcoma when Administered as Maintenance Therapy.


Sponsor

ARIAD Pharmaceuticals, Inc.

Enrollment

650 participants

Start Date

Sep 1, 2007

Study Type

Interventional

Conditions

Summary

Phase 3 This is a pivotal study to determine the effectiveness and safety of AP23573 (deforolimus) when administered as maintenance therapy to people with metastatic cancers of either soft tissue or bone. Who is it for? This trial is for you is you have metastatic cancers of either soft tissue or bone connective tissue, and have already had your cancer controlled by chemotherapy. Trial Details Participants will be randomly divided into two groups. One group will receive maintenance treatment with oral AP23573 (deforolimus) and the other will receive standard treatment. The trial aims to see whether those people receiving oral AP23573 (deforolimus) have better disease control compared to those receiving standard treatment. Deforolimus has shown promising activity in early phase trials and its side effects are generally mild and reversible and include mouth sores, fatigue and nausea.’ Deforolimus inhibits the protein mTOR, which is a ‘master switch’ in cancer cells. Blocking mTOR effectively ‘starves’ cancer cells by interfering with cell growth, division, metabolism, and blood vessel growth. If you have metastatic cancers of either soft tissue or bone connective tissue, you usually receive chemotherapy until the disease is controlled or until chemotherapy causes significant side effects. Chemotherapy is then stopped and your cancer specialist observes you regularly. This trial is testing whether, once the disease has been controlled with chemotherapy, treatment with oral AP23573 (deforolimus) can control the cancer for longer compared to standard treatment.


Eligibility

Sex: Both males and femalesMin Age: 13 Yearss

Plain Language Summary

Simplified for easier understanding

This study is testing an oral medication called deforolimus (AP23573) as a maintenance therapy for people with soft tissue or bone sarcoma (a type of cancer affecting connective tissue) whose disease has been controlled after chemotherapy. The drug works by blocking a key protein in cancer cells called mTOR, which helps 'starve' cancer cells and slow their growth. Participants will be randomly assigned to receive either deforolimus or standard care. You may be eligible if: - You have been diagnosed with metastatic (spread) soft tissue or bone sarcoma - Your cancer was controlled after at least 4 cycles of chemotherapy - You are 13 years of age or older (if aged 13-17, you must weigh at least 45 kg) - You have good general health (ECOG performance status of 0 or 1) - Your organ and bone marrow function are adequate - Your last chemotherapy was 3 to 8 weeks before joining the study You may NOT be eligible if: - You are pregnant or breastfeeding - You have active brain or central nervous system metastases - You have previously been treated with rapamycin or similar drugs - You have ongoing side effects from previous cancer treatment (grade 2 or higher) - You have had another cancer diagnosis in the past 3 years - You have a severe allergy to macrolide antibiotics - You have significant uncontrolled heart disease or an active infection - You are HIV positive Talk to your doctor about whether this trial might be right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

This is a multi-centre, randomised Phase 3, double-blinded, placebo-controlled trial to determine the efficacy and safety of oral AP23573 (MK-8669) versus oral placebo in patients with metastatic sarc

This is a multi-centre, randomised Phase 3, double-blinded, placebo-controlled trial to determine the efficacy and safety of oral AP23573 (MK-8669) versus oral placebo in patients with metastatic sarcoma. Eligible patients include those with metastatic soft-tissue or bone sarcoma who have achieved a complete response, partial response or stable disease following 1st, 2nd or 3rd line chemotherapy for metastatic sarcoma. The trial is designed to test the hypothesis that a clinically significant improvement in progression-free survival will be induced in patients treated with AP23573 compared to placebo. It is anticipated that approximately 650 patients, age from 13 years will participate in this trial. AP23573 will be administered once daily as oral tablets for 5 consecutive days followed by a 2-day dosing holiday each week at a dose of 40 mg per day. Patients randomised to the placebo arm will receive matched tablets consisting of excipients without active drug Patients must have achieved an ongoing complete response, partial response or stable disease following prior therapy as defined by Response Evaluation Criteria in Solid Tumours (RECIST) guidelines. The disease status at study entry will be confirmed through an independent radiographic review during the screening period (prior to randomisation). Only eligible patients will be randomized. Patients will be randomly assigned (1:1) to receive either AP23573 or placebo by oral administration. Treatment will continue until progressive disease by RECIST guidelines is documented or other discontinuation criteria are met. Patients will be stratified by geographical region, histological category (soft-tissue or bone sarcoma), and prior treatment (1st line or 2nd/3rd line). Disease assessments will be performed every 8 weeks and assessed in accordance with the RECIST guidelines. Patients will be followed for overall survival for at least 24 months and up to 60 months following randomisation.


Locations(26)

United States of America

Canada

Belgium

Czech Republic

France

Germany

Greece

Israel

Netherlands

Poland

Romania

Slovakia

Spain

Sweden

United Kingdom

India

Korea, Democratic People's Republic Of

New Zealand

Argentina

Colombia

Mexico

Peru

Chile

South Africa

Brazil

Italy

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ACTRN12608000214336