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Childhood CNS TumorEarly Phase 1NCT03554707

SGT-53 in Children With Recurrent or Progressive CNS Malignancies

A Early Phase 1 study testing SGT-53, Radiation and Irinotecan in people with Childhood CNS Tumor, run by SynerGene Therapeutics, Inc. at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

An early phase 1 for pediatric patients with recurrent or progressive CNS malignancies Read more about this study

Who is it for?
Ages 1 year to 21 years
Conditions the study is for
Childhood CNS Tumor
What is being tested?
SGT-53, Radiation and Irinotecan
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
1 Jun 2022to 1 Jun 2022
Phase
Early 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 1 year to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Patients must have a recurrent, progressive, or refractory CNS malignancy for which there are not known curative options. Low-grade glioma, craniopharyngioma, and other non-malignant CNS tumors are excluded.
  • Tumor must be measureable, defined as a tumor that can be accurately measured in two perpendicular dimensions on MRI.
  • Patients with metastatic disease are eligible but must have at least one target lesions which is measurable.
  • Patients must have available archival (formalin-fixed paraffin embedded) or fresh tumor tissue for correlative studies.
  • Patients must be >1yrs and <21 years of age.
  • Must have recovered from all surgical interventions prior to the start of the Radiation and Chemotherapy Phases.
Show all 19Show fewer
  • Patients must have recovered from the acute effects of prior therapy.
  • There is a maximum of 3 previous myelosuppressive therapy regimens. However, there is no maximum number of therapeutic courses.
  • Patients must have received their last dose of known myelosuppressive therapy at least three (3) weeks prior to receipt of SGT-53.
  • Patients must have received their last dose of biological agent >7 days prior to receipt of SGT-53.
  • Patients must be far enough from previous irradiation that in the opinion of a radiation oncologist using standard fractionation is deemed to be reasonable from a clinical standard of care perspective.
  • Patients who are receiving dexamethasone or other corticosteroids must be on a stable or decreasing dose for at least one (1) week prior to enrollment.
  • Patients must have received their last dose of any short acting growth factor at least one week prior to treatment, for long acting or pegylated growth factors, the last dose must be at least two (2) weeks prior to start of treatment.
  • Patients with neurologic deficits must have deficits that have been stable in grade for a minimum of one week prior to enrollment.
  • Performance status (Karnofsky PS for >16yrs, or Lansky PS for <16yrs) assessed within two weeks must be >50.
  • Patients must have normal organ and marrow function.
  • All patients of childbearing or child fathering potential must be willing to use an acceptable form of birth control while being treated on this study.
  • Female patients must not be pregnant or nursing. Female patients must also have a negative serum pregnancy test at the time of enrollment.
  • Patient and/or guardian have the ability to understand and the willingness to sign a written informed consent document according to institutional guidelines.

Who cannot join

  • Patients with any clinically significant unrelated systemic illness (serious infection or significant cardiac, pulmonary, hepatic, or other organ dysfunction) that is likely to interfere with ability to tolerate study therapy or study procedure results.
  • Patients with low-grade gliomas, craniopharyngioma, or extracranial tumors with CNS metastases.
  • Patients who are receiving any other investigational drug therapy.
  • Patients who require therapeutic anti-coagulation.
  • Patients who in the opinion of the investigator cannot adhere to protocol requirements.
  • Patients with history of clinically significant clot or hemorrhage are eligible but will not receive bevacizumab during chemotherapy regimen.
  • Unavailability of the chemotherapy due to insurance coverage or other logistical issues is an ineligibility criterion.
  • Patients may not be on immunosuppressive therapy, including corticosteroids (with the exception of physiologic replacement, defined as 0.75mg/m2/day) at time of enrollment. However, patients who require intermittent use of bronchodilators or local steroid injections will not be excluded from the study.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: A Pilot Study of SGT-53 in Conjunction With Irradiation and Chemotherapy in Children With Recurrent or Progressive CNS Malignancies

An early phase 1 for pediatric patients with recurrent or progressive CNS malignancies

Read the detailed description

This clinical trial is a early phase 1, open label, single center, single arm study of the combination of intravenously administered SGT-53 and irradiation and/or chemotherapy in pediatric patients with recurrent or progressive CNS malignancies. The objective of the study is to establish the safety and feasibility of administration of SGT-53 in conjunction with conventional radiotherapy and/or chemotherapy in children with recurrent or refractory CNS malignancies.

Keywords
Childhood CNS tumor, Recurrent CNS malignancies, Progressive CNS malignancies, Refractory CNS malignancies
Medical subject headings
Central Nervous System Neoplasms

What participants receive

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

  • SGT-53Genetic

    2.1 mg DNA/m2 or 2.8 mg DNA/m2 twice weekly

  • RadiationRadiation

    Standard radiation plan

  • IrinotecanDrug

    Also called Camptosar, Onivyde

    50mg/m2/dose IV daily for five days in a 4-week cycle

  • TemozolomideDrug

    Also called Temodar

    100mg/m2 PO daily for five days in a 4-week cycle

  • BevacizumabDrug

    Also called Avastin

    10mg/kg IV every two weeks in a 4-week cycle

Study groups in NCT03554707
Study groupRoleWhat they receive
SGT-53 with radiation or drugsExperimentalRadiation phase: SGT-53 will be given at 2.1 mg DNA/m2 twice weekly for the first week of radiation therapy, and then increase to 2.8 mg DNA/m2 twice weekly. Radiation therapy will be administered as per clinical care, with a target of fifteen (15) fractions, but patients with other clinically-determined radiation plans will be allowed. Chemotherapy phase: SGT-53 will be administered at the highest tolerated dose given during radiation phase. Irinotecan will be given at a dose of 50mg/m2/dose IV daily for five days in a 4-week cycle. Temozolomide will be given at a dose of 100mg/m2 PO daily for five days in a 4-week cycle and bevacizumab will be given at a dose of 10mg/kg IV every two weeks in a 4-week cycle.
Receives: Genetic: SGT-53; Radiation: Radiation; Drug: Irinotecan; Drug: Temozolomide; Drug: Bevacizumab

What is being measured

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

  1. Incidence of Adverse Events

    An adverse event (AE) was any untoward medical occurrence that began or worsened in grade after the start of study drug through 30 days after the last dose. The safety will be assessed by the number and severity of any AE or serious adverse events (SAE) experienced by the patients, and by their relationship to the study drug SGT-53 (e.g. definitely, probably, possibly, unlikely or unrelated). Severity will be graded according to NCI CTCAE version 4.0.

    Measured: up to 13 months

Secondary outcomes (6)

  1. Response Rate

    The response rate will be calculated from the percentage of patients whose cancer shrinks or disappears after treatment.

    Measured: 36 months

  2. Duration of Response

    The duration of response will be the time calculated from documentation of tumor response as indicated by response criteria, to disease progression.

    Measured: 36 months

  3. Overall Survival

    Overall survival will be calculated from date of original diagnoses to death and also from the date of study registration to death.

    Measured: 36 months

  4. Progressive-Free Survival (PFS)

    PFS will be calculated at all times during follow-up, with particular interest in the 6-month time point. Progression free survival will be calculated from date of first treatment to the date of first observation of progressive disease, non-reversible neurological progression or increasing steroid requirements (applies to stable disease only), death due to any cause, or early discontinuation of treatment.

    Measured: 36 months

  5. Characterization of Phenotype of Patients

    Tissue will be obtained from each enrolling patient for subsequent testing for TP53 pathway functionality, including assessment of mdm2, p21, and other mutation or expression alterations. This analysis will also include measures of commonly assessed polymorphisms, including MGMT methylation assessment, ATRX mutation among others.

    Measured: 4-5 days

  6. Feasibility of Droplet PCR Assays to Monitor for Tumor Burden

    Droplet PCR will be used to assess for the presence of circulating tumor DNA

    Measured: 12 months

Timeline

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

  1. First posted13 Jun 2018
  2. Study start1 Jun 2022
  3. Primary completion1 Jun 2022
  4. Study completion1 Jun 2022

Why it stopped: The grant intended to support this trial as ultimately not funded. This trial was not initiated,. No patients were enrolled.

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
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03554707
Sponsor's protocol ID
SGT53-00-1
Status last verified
May 2026
First submitted
17 May 2018
Last updated
8 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing SGT-53, Radiation and Irinotecan in people with Childhood CNS Tumor, run by SynerGene Therapeutics, Inc. at 1 site 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 19 inclusion and 8 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: withdrawn, as of the sponsor's last update on 8 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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 was withdrawn before enrolling participants, so there are no results.

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?

Incidence of Adverse Events (time frame: up to 13 months). 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 NCT03554707.

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 8 May 2026.

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