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NCT Number: NCT05789394

Allogenic Adipose-Derived Mesenchymal Stem Cells for the Treatment of Recurrent Glioblastoma or Recurrent Astrocytoma in Patients Undergoing Craniotomy

This phase I trial tests the safety, side effects, and best dose of allogenic adipose-derived mesenchymal stem cells (AMSCs) in treating patients with glioblastoma or astrocytoma that has come back (recurrent) who are undergoing brain surgery (craniotomy). Glioblastoma is the most common and most aggressive form of primary and malignant tumor of the brain. Currently, the standard of care for this disease includes surgical resection, followed by radiation with chemotherapy and tumor treating fields. Despite this aggressive therapy, the survival after finishing treatment remains low and the disease often reoccurs. Unfortunately, the available therapy options for recurrent glioblastoma are minimal and do not have a great effect on survival. AMSCs are found in body fat and when separated from the fat, are delivered into the surgical cavity at the time of surgery. When in direct contact with tumor cells, AMSCs affect tumor growth, residual tumor cell death, and chemotherapy resistance. The use of AMSCs delivered locally into the surgical cavity of recurrent glioblastoma during a craniotomy could improve the long-term outcomes of these patients by decreasing the progression rate and invasiveness of malignant cells.

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Key information

Conditions

Recurrent Glioblastoma, IDH-Wildtype Astrocytoma Astrocytoma, Grade IV Glioblastoma Glioma Neoplasms Neoplasms by Histologic Type Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroectodermal Tumors Recurrent Astrocytoma, IDH-Mutant, Grade 4

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Mayo Clinic in Florida

Jacksonville, Florida, 32224-9980, United States

Location status: Recruiting

Location contact

Alfredo Quinones-Hinojosa, MD

PRINCIPAL_INVESTIGATOR

Clinical Trials Referral Office

CONTACT

[email protected]

855-776-0015

About this study

PRIMARY OBJECTIVE:

I. To establish the maximum tolerated dose (MTD) of locally delivered adipose-derived mesenchymal stem cells (AMSCs) in patients with recurrent glioblastoma (GBM).

SECONDARY OBJECTIVES:

I. To assess the safety and toxicity profile of locally delivered AMSCs in patients with recurrent GBM.

II. To assess overall survival (OS) in recurrent GBM patients treated with locally delivered AMSCs.

III. To assess progression free survival (PFS) in recurrent GBM patients treated with locally delivered AMSCs.

CORRELATIVE OBJECTIVES:

I. To explore the systemic immune response after application of AMSCs through cytokine analysis on peripheral blood samples.

II. To explore the local changes on the brain parenchyma by analyzing tissue at recurrence.

III. To explore the presence of AMSCs on brain tissue at recurrence.

OUTLINE: This is a dose-escalation study.

Patients receive AMSCs intratumorally (IT) and undergo Ommaya reservoir placement during a craniotomy on study. Patients also undergo magnetic resonance imaging (MRI) on study and during follow-up, as well as blood sample, tissue sample and cerebrospinal fluid (CSF) sample collection on study.

After completion of study treatment patients are followed up every 2 months for 1 year.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participants >= 18 years
  • Karnofsky Performance Scale (KPS) >= 60
  • Negative pregnancy test done =< 7 days prior to registration, for persons of childbearing potential only
  • Patients with a previous histological diagnosis of glioblastoma multiforme, isocitrate dehydrogenase (IDH) wildtype (WT) or astrocytoma, IDH-mutant World Health Organization (WHO) grade IV according to the 2021 WHO classification of tumors of the central nervous system , who are candidates to- and will undergo a redo craniotomy for excision of recurrent tumor
  • There is measurable disease according to the immunotherapy response assessment in neuro-oncology (iRANO) criteria
  • Serum creatinine and urea <= 2 times the upper limit of normal (=< 3 weeks prior to registration)
  • Alanine transaminase (ALT), aspartate transferase (AST) and alkaline phosphatase =< 3 times the upper limit of normal, and bilirubin =< 2.5 mg/dL (=< 3 weeks prior to registration)
  • Prothrombin time =< 1.5 times upper limit of normal (=< 3 weeks prior to registration)
  • International normalized ratio (INR) and partial thromboplastin time (PTT) =< 1.5 times the upper limit of normal (=< 3 weeks prior to registration)
  • Hemoglobin >= 9 g/dL (=< 3 weeks prior to registration)
  • Platelets >= 100 x 10^9/L (=< 3 weeks prior to registration)
  • Absolute neutrophil count (ANC) >= 1.5 x 10^9/L (=< 3 weeks prior to registration)
  • Willing to return to enrolling institution for follow-up (during the active monitoring phase of the study)
  • Patient or legally authorized representative (LAR) is able to fully understand and provide written and verbal consent for the protocol
  • Willingness to provide mandatory blood specimens for correlative research
  • Willingness to provide mandatory tissue specimens for correlative research
  • Willingness to undergo Ommaya reservoir placement and provide cerebrospinal fluid (CSF) samples for correlative research

Exclusion criteria

  • Patients who are undergoing needle biopsy only or non-eligible for a surgical intervention
  • Tumors located solely in the brain stem, midbrain, or thalamus without inclusion/involvement of surrounding brain matter
  • Previous treatment with bevacizumab
  • Radiographic evidence of leptomeningeal disease

Treatment and study plan

Allogeneic Adipose-derived Mesenchymal Stem Cells

Biological

Receive IT

Other names: Allogeneic Adipose-derived MSCs, Allogeneic Adipose-derived Stem/Stromal Cells, Allogeneic Mesenchymal Stem/Stromal Cells

Biospecimen Collection

Procedure

Undergo blood and tissue sample collection

Other names: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Craniotomy

Procedure

Undergo craniotomy

Other names: incision of the skull, Open Craniotomy

Magnetic Resonance Imaging

Procedure

Undergo MRI

Other names: Magnetic Resonance, Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), MRIs, sMRI, Structural MRI

Ommaya Reservoir Tap

Procedure

Undergo Ommaya reservoir placement for collection of CSF

Other names: Ommaya Reservoir Access

Primary outcomes

  1. Maximum tolerated dose (MTD)

    Time frame: Up to 4 weeks

    Defined as the dose level below the lowest dose that induces dose-limiting toxicity in at least one-third of patients (at least 2 of a maximum of 6 new patients). Will use the standard cohort 3+3 design.

Secondary outcomes

  1. Incidence of adverse events (AEs)

    Time frame: Up to 1 year

    All patients who have received any treatment will be considered evaluable for assessing adverse event rates. The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration. Will follow the Common Terminology Criteria for Adverse Events version 5.0 to record and grade (1-5) any potential AEs.

  2. Best response

    Time frame: Up to 5 years

    Best response per patient is defined to be the best objective status recorded from treatment application until disease progression or death (taking as reference for progressive disease the smallest measurements recorded since the treatment started). Best response assignment will depend on the achievement of both measurement and confirmation criteria.

  3. Response rate (RR)

    Time frame: Up to 5 years

    RR is defined as the number of patients who have achieved complete response or partial response per immunotherapy response assessment for neuro-oncology for recurrent glioblastoma after application of allogeneic adipose-derived mesenchymal stem cells (AMSCs) divided by total number of evaluable patients. Responses will be summarized by simple descriptive summary statistics delineating complete and partial responses as well as stable and progressive disease in this patient population.

  4. Progression free survival

    Time frame: From time of AMSCs application until the first occurrence of progression or death, assessed up to 5 years

    The quality of survival will be measured by performance status. The data on time-related variables will be summarized descriptively.

  5. Overall survival

    Time frame: From beginning the time of AMSCs application to the date of death, assessed up to 5 years

    The quality of survival will be measured by performance status. The data on time-related variables will be summarized descriptively.

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Trials Referral Office

CONTACT

[email protected]

855-776-0015

Sponsors and collaborators

Lead sponsor

Mayo Clinic

Other

Registry information

Official study title

Phase 1, Dose Escalation, Non-Randomized, Open Label, Clinical Trial Evaluating the Safety and Preliminary Efficacy of Allogenic Adipose-Derived Mesenchymal Stem Cells (AMSCs) for Recurrent Glioblastoma

Important dates

Study start
2023
Primary completion
2027
Study completion
2027
First posted
Mar 29, 2023
Registry last updated
Apr 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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