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NeuroblastomaPhase 1NCT01169584

Safety Study of Recombinant Vaccinia Virus to Treat Refractory Solid Tumors in Pediatric Patients

A Phase 1 study testing Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594) in people with Neuroblastoma and Rhabdomyosarcoma, run by Jennerex Biotherapeutics at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a Phase I, open-label, dose-escalation trial of JX-594 (Pexa-Vec) in pediatric patients with advanced/metastatic, unresectable solid tumors refractory to standard therapy and/or the patient does not tolerate standard therapies. Read more about this study

Who is it for?
Ages 2 to 21 years
Conditions the study is for
Neuroblastoma, Rhabdomyosarcoma, Lymphoma
What is being tested?
Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
2 sitesUnited States
Study dates
Aug 2010to Nov 2014
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age between 2 and 21 years
  • Histologically-confirmed, advanced/metastatic non-CNS solid tumor that is relapsed and/or refractory to standard therapy (progressive disease despite therapy) and/or the patient does not tolerate standard therapy. Non-CNS solid tumors are eligible and are likely to include such histologies as neuroblastoma, Wilms' tumor, rhabdomyosarcoma, Ewing's sarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue sarcomas, and malignant peripheral nerve sheath tumors.
  • Cancer is not surgically resectable for cure
  • At least one measurable tumor mass by CT/MRI (i.e. lesion that can accurately be measured in at least one dimension with longest diameter ≥ 1 cm) and that can be injected by direct visualization/palpitation or by imaging-guidance (CT or ultrasound)
  • Expected survival for approximately 8 weeks or longer
  • Lansky Score ≥ 50
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  • Total bilirubin ≤ 2.5 × ULN
  • AST, ALT ≤ 2.5 × ULN (if liver tumor(s) present: AST/ALT ≤ 5 x ULN)
  • Serum creatinine ≤ 1.8 x ULN
  • INR ≤ 1.5 x ULN
  • Hematologic parameters: Patients can be transfused to meet these entry criteria.
  • Hemoglobin ≥ 9 g/dL
  • For bone marrow negative patients: ANC ≥ 750 cells/ mm3 and platelet count ≥ 75,000 plts/mm3
  • For bone marrow positive patients: ANC ≥ 750 cells/ mm3. Platelet count recovery is not a requirement, but platelets should be transfused to ≥ 75,000 plts/ mm3 prior to treatment.
  • CD4 count ≥ 200/mm3. Patients who demonstrate intact delayed-type hypersensitivity (DTH) via skin immune response to common antigens (e.g. candida, mumps) are also eligible.
  • For patients who are sexually active, able and willing to abstain from sexual activity for 3 weeks following treatment with JX-594. Thereafter, able and willing to use accepted birth control methods through 3 months after last treatment with JX-594. [Acceptable birth control methods include contraceptive pills, condom, IUD, diaphragm or sponge + spermicide, or other methods with >97% effectiveness]
  • Able and willing to sign an Institutional Review Board (IRB)/Research Ethics Board (REB)-approved written consent form (patient and/ or parents/guardians).
  • Able and willing to comply with study procedures and follow-up examinations, including compliance with the "Infection Control Guidelines for Patients" contained within the written consent form (patient and/ or parents/guardians).

Who cannot join

  • Pregnant or nursing infant
  • Injected tumor(s) in location that would potentially result in significant clinical adverse effects if post-treatment tumor swelling were to occur or if deemed unsafe by investigator (e.g. tumors impinging on the upper airway or affecting biliary tract drainage, adherent to and/or invading a major vascular structure, CNS, etc.)
  • Brain metastases, unless surgically resected and/or irradiated. (Brain metastases cannot be considered as a site for injection).
  • Patients with lymphomas
  • Use of high dose systemic corticosteroids or other immune suppressive medication within 3 weeks of first treatment (e.g. cortisone, dexamethasone, hydrocortisone, prednisone, prednisolone, interferon, cisplatin, doxorubicin, fluorouracil, etc.). * Note: patients taking low-dose corticosteroids for the treatment of nausea and/or taking maintenance corticosteroids for adrenal insufficiency are permitted to enroll.
  • Known infection with HIV or known underlying genetic immunodeficiency disease
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  • Treatment of the injected tumor(s) with radiotherapy, chemotherapy, surgery, or an investigational drug within 3 weeks prior to first treatment
  • Clinically significant active infection or uncontrolled medical condition considered high risk for investigational new drug treatment (e.g. pulmonary, neurological, cardiovascular, gastrointestinal, genitourinary)
  • History of exfoliative skin condition (e.g. severe eczema, ectopic dermatitis, or similar skin disorder) requiring systemic therapy
  • Clinically significant and/or rapidly accumulating ascites, peri-cardial and/or pleural effusions (e.g. requiring drainage for symptom control)
  • Severe or unstable cardiac disease which may include, but is not limited to, any of the following within 6 months prior to screening: myocardial infarct, unstable angina, congestive heart failure, myocarditis, arrhythmias diagnosed and requiring medication, or any clinically-significant change in cardiac status
  • Current, active, progressing CNS malignancy, including carcinomatosis meningitis (definitively surgically resected or irradiated metastases allowed)
  • Pulse oximetry O2 saturation <90% at rest
  • Use of anti-viral, anti-platelet or anti-coagulation medication (for example, heparin, warfarin, aspirin, ticlopidine, clopidogrel, dipyridamole) [Patients who discontinue such medications within 7 days prior to first treatment may be eligible for this study. Any required, chronic medications indicated for other medical issues should not be discontinued in order to meet eligibility criteria for this trial without consultation with both the patient and the treating physician.] Note: Low Dose Heparin to maintain patency of venous catheters is permitted.
  • Patients with benign tumors
  • Inability or unwillingness to give informed consent (patient or parent/guardian) or comply with the procedures required in this protocol
  • Vaccination with a live virus (i.e. measles, mumps, rubella, etc) < 30 days prior to first treatment
  • Patients with household contacts who meet any of these criteria will be excluded unless alternate living arrangements can be made during the patient's active dosing period and for three weeks following the last dose of study medication:
  • Women who are pregnant or nursing an infant
  • Children < 1 years old
  • People with skin disease (eczema, atopic dermatitis and related diseases)
  • Immunocompromised hosts (severe deficiencies in cell-mediated immunity, including AIDS, organ transplant recipients, hematologic malignancies)

Trial locations and contacts

2 sites in 1 country.

United States2 sites

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Texas Children's Hospital

    Houston, Texas, 77030-2399

Other trials in United States

About this study

Official title: A Phase I, Open-Label, Dose Escalation Study of JX-594 (Vaccinia GM-CSF/Thymidine Kinase-Deactivated Virus) Administered by Intratumoral Injection in Pediatric Patients With Unresectable Refractory Solid Tumors.

This is a Phase I, open-label, dose-escalation trial of JX-594 (Pexa-Vec) in pediatric patients with advanced/metastatic, unresectable solid tumors refractory to standard therapy and/or the patient does not tolerate standard therapies. Tumors are likely to include neuroblastoma, lymphoma, Wilms' tumor, rhabdomyosarcoma, Ewing's sarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue sarcomas, and malignant peripheral nerve sheath tumors. Benign tumors are excluded. These tumor types were selected because evidence of biological activity was observed in cancer cells lines and ex vivo infected primary human tissue samples, specifically pediatric cancer types such as sarcomas and neuroblastomas.

Being tested
Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594)
Keywords
Phase I, advanced metastatic pediatric solid tumors, pediatric, vaccinia virus, oncolytic virus, neuroblastoma, rhabdomyosarcoma, lymphoma, Wilm's tumor, Ewing's sarcoma, osteosarcoma, non-rhabdomyosarcoma soft tissue sarcomas, malignant peripheral nerve sheath tumors, Pexa-Vec
Medical subject headings
Neuroblastoma, Rhabdomyosarcoma, Lymphoma, Wilms Tumor, Sarcoma, Ewing, Vaccinia, Osteosarcoma, Neurofibrosarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594)Drug

    Also called Pexa-Vec

    Intratumoral Injection Dosage from 1 x 10^6 pfu/kg to 3 x 10^7 pfu/kg is administered once to 1-3 injectable tumors in pediatric patients.

Study groups in NCT01169584
Study groupRoleWhat they receive
Single Arm - JX-594ExperimentalIntratumoral injection of JX-594
Receives: Drug: Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Determine the maximally-tolerated dose (MTD) and/or maximum-feasible dose (MFD) of JX-594

    Determine the maximally-tolerated dose (MTD) and/or maximum-feasible dose (MFD) of JX-594 administered by intratumoral (IT) injection in pediatric patients with advanced/metastatic, unresectable refractory solid tumors

    Measured: 3 weeks

  2. Determine the safety/toxicity of JX-594 administered by IT injection in this patient population

    Measured: 3 weeks

Secondary outcomes (2)

  1. Determine the JX-594 pharmacokinetics and pharmacodynamics over time following IT injection in this patient population

    Measured: 3 weeks

  2. Determine the immune response to JX-594 following IT injection in this patient population

    Measured: 3 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Jul 2010 (estimated)
  2. Study startAug 2010
  3. Primary completionMar 2012
  4. Study completionNov 2014

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01169584
Sponsor's protocol ID
JX594-IT-P009
Status last verified
Jan 2016
First submitted
22 Jul 2010
Last updated
21 Jan 2016
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594) in people with Neuroblastoma and Rhabdomyosarcoma, run by Jennerex Biotherapeutics at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 21 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Determine the maximally-tolerated dose (MTD) and/or maximum-feasible dose (MFD) of JX-594 (time frame: 3 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01169584.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jan 2016.

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