Neoplasms

All Conditions

Skin and Connective Tissue Diseases

Rare Diseases

Sarcoma

Sarcoma, Ewing

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Osteosarcoma

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Neoplasms, Germ Cell and Embryonal

Neoplasms, Nerve Tissue

Neoplasms, Neuroepithelial

Neoplasms, Glandular and Epithelial

Soft Tissue Sarcoma

Ewing Sarcoma

Neuroepithelioma

Pharmaceutical Solutions

All Drugs and Chemicals

Zalypsis is provided as a lyophilized powder for concentrate for solution for infusion in a strength of 2.5 mg/vial.

Zalypsis

Fariba Navid, MD

Sant P Chawla, MD

Jean Yves Blay, MD

Stefano Ferrari, MD

Armando Santoro, Prof.

Paolo Casali, MD

Robin L. Jones, MD

This is a phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients with Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy to determine the antitumor activity of Zalypsis.

Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy

Phase II Multicenter, Open-label, Clinical and Pharmacokinetic Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy

Overall response rate (ORR), defined as the percentage of patients with confirmed objective response (OR), either CR or PR according to the RECIST v.1.1.

CR, complete response: disappearance of all lesions; PD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density; SD, stable disease: none of the CR, PR, or PD criteria met; RECIST, Response Evaluation Criteria in Solid Tumors

Best tumor response was defined as the best response achieved during the study according to RECIST v1.1 CR, complete response: disappearance of all lesions; PD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density; SD, stable disease: none of the CR, PR, or PD criteria met; RECIST, Response Evaluation Criteria in Solid Tumors

Progression-free survival (PFS), defined as the time from the first day of study treatment to the day of negative assessment (progression or death), start of subsequent antitumor therapy, or last tumor evaluation.

PD, disease progression: ≥10% increase in target lesion size and does not meet tumor density criteria of PR density; PR, partial response: ≥10% decrease in target lesion size or ≥15% decrease in tumor density

Overall survival (OS), defined as the time from the first day of treatment to the date of death (or the last day when the patient was known to be alive). Survival was to be followed for up to six months after the last treatment visit of the last patient, or 12 months after the last patient was included, whichever occurred first.

Cmax Maximum plasma concentration, directly determined from the experimental data

AUC Area under the plasma concentration-time curve from time zero to infinity

PharmaMar

Sixteen of 17 patients included in this study were treated with PM00104 and were therefore evaluable for safety

PM00104 was administered at a dose of 2 mg/m2 as a 1-hour i.v. infusion d1, d8 and d15 q4wk to patients with advanced and/or metastatic Ewing's sarcoma previously treated with at least one line of chemotherapy. A treatment cycle consisted of PM00104 administration on Days 1, 8 and 15, plus one week of follow-up. Study evaluations had to be completed during each cycle prior to subsequent PM00104 infusion. Treatment cycles were repeated every four weeks

PM00104

Age, Categorical

Age, Continuous

Sex: Female, Male

Race and Ethnicity were not collected from any participant.

Race and Ethnicity Not Collected

United States

Italy

France

Region of Enrollment

Eastern Cooperative Oncology Group Performance Status, ECOG PS 0 Fully active, able to carry on all pre-disease performance without restriction

1. Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature
2. Ambulatory and capable of all selfcare but unable to carry out any work activities. Up and about more than 50% of waking hours
3. Capable of only limited selfcare, confined to bed or chair more than 50% of waking hours
4. Completely disabled. Cannot carry on any selfcare. Totally confined to bed or chair
5. Dead

ECOG PS

Tumor diagnosis

Primary location at diagnosis

Metastatic disease

Time from diagnosis to first infusion

Time from last disease progression to first infusion

Sites of disease at diagnosis

Prior radiotherapy

Prior Antitumor surgery (palliative or curative)

Prior Systemic anticancer therapy

Clinical Developtment, Department of PharmaMar´s Oncology., Business Unit.

Overall Response Rate (ORR)

One patient never treated Two patients were non-evaluable for efficacy because they were withdrawn due to toxicity without any tumor assessment after the start of study treatment and were considered as "treatment failures"

Best Tumor Response

Progression-free Survival

Progression-free Survival at 3 Months

Overall Survival

Overall Survival Rate at 6 Months

Overall Survival Rate at 12 Months

Fourteen of the 16 patients treated in this study had plasma profiles

PM00104 Plasma PK Parameters (Cmax) at First Infusion

PM00104 Plasma PK Parameters (AUC) at First Infusion

Eleven of the 16 patients treated in this study had plasma profiles at second infusion

PM00104 Plasma PK Parameters (Cmax) at Second Infusion

PM00104 Plasma PK Parameters (AUC) at Second Infusion

Overall Study

Digestive System Diseases

Blood and Lymph Conditions

Immune System Diseases

Esophagitis

Eosinophilic Esophagitis

Barrett Esophagus

Esophageal Diseases

Gastrointestinal Diseases

Gastroenteritis

Precancerous Conditions

Eosinophilia

Leukocyte Disorders

Hematologic Diseases

Hypersensitivity

Hypersensitivity, Immediate

Eosinophilic Gastroenteritis

Subject will swallow the SECM capsule and the imaging of the esophagus will be performed using a SECM optical system

Subjects will swallow a SECM capsule and the SECM imaging will be performed

SECM capsule

Anita Chung, RN

Nitasha Bhat, MD

Elizabeth Biddle, RN

Guillermo Tearney, MD, PhD

The goal of this validation study is to compare Spectrally Encoded Confocal Microscopy (SECM) Tethered Capsule Endoscopy (TCE) diagnosis of Eosinophilic Esophagitis to that of standard of care endoscopic biopsy.

Eosinophilic esophagitis is an inflammatory condition of the esophagus that occurs in response to certain foods or allergens. Approximately 300,000 people in the US have EoE, the incidence of newly diagnosed EoE is \~30,000/year, and EoE is found in 5-10% of all patients undergoing upper endoscopy. EoE is associated with debilitating symptoms, and is a common cause of dysphagia, strictures, and food impaction; it may even lead to esophageal perforation. Because of the impact of EoE on quality of life as well as the concern that this disease may progress to esophageal fibrosis with unknown long-term risks, experts recommend that EoE be treated until symptoms and eosinophilic infiltrate are resolved. Because the only objective method for diagnosing EoE is histopathologic assessment of multiple upper endoscopic biopsies, the processes of monitoring the esophagus during therapy and the food reintroduction involve a high number of repeat endoscopies. This process is both time consuming and frustrating for patients and their families. Because the endoscopic biopsy requires conscious sedation, it is also costly, exceeding $1000 per procedure. Given the rapid increase in the number of patients with this disease, there is a compelling need for a less invasive and more cost-effective means of identifying eosinophils in the esophagus.

Based on the extensive experience with Optical Coherence Tomography (OCT) and OFDI, the investigators at Tearney laboratory at MGH have developed a spectrally encoded confocal microscopy (SECM) technology that is able to acquire images at significantly higher frame rates than standard confocal microscopy. This SECM capability enables imaging of large portions of the esophagus at 2-µm resolution in realistic procedure times. Furthermore, the investigators at Tearney laboratory have created a tethered capsule SECM device that can be swallowed and used for EoE screening. The investigators at Tearney laboratory lab performed a pilot study using a SECM capsule on 58 subjects (2013-P-000863). The imaging was successful with no adverse or unanticipated events. SECM endoscopy capsule is similar to the approved endoscopy capsule.

A total of sixty (60) subjects will be recruited for this study. The imaging of the esophagus will be obtained. Subjects will not be sedated for the procedure.

The images obtained by the SECM capsule will be compared to the images obtained by the clinical endoscopy and biopsy. SECM imaging will be used for research purposes only and will not be used for diagnosis purposes.

Imaging of the Esophagus Using a SECM Capsule

comparing SECM imaging of the esophagus obtained with a SECM capsule to histopathology.

Massachusetts General Hospital

Principal Investigator

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy	NCT01222767	Ewing's Sarcoma Primitive Neuro... Askin's Tumor o... Extraosseous Ew...	Zalypsis	16 Years -	PharmaMar
Study of Zalypsis® (PM00104) in Patients With Unresectable Locally Advanced and/or Metastatic Ewing Family of Tumors (EFT) Progressing After at Least One Prior Line of Chemotherapy	NCT01222767	Ewing's Sarcoma Primitive Neuro... Askin's Tumor o... Extraosseous Ew...	Zalypsis	16 Years -	PharmaMar
Imaging of the Esophagus Using a SECM Capsule	NCT03546660	Eosinophilic Es... Barrett Esophag...	SECM capsule	12 Years -	Massachusetts General Hospital

Spots Global Cancer Trial Database for eoe

Every month we try and update this database with for eoe cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for eoe

Every month we try and update this database with for eoe cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for eoe cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.