AzTrials
AzTrials

Recurrent Glioblastoma, IDH-WildtypePhase 1NCT05789394

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

A Phase 1 study testing Allogeneic Adipose-derived Mesenchymal Stem Cells, Biospecimen Collection and Craniotomy in people with Recurrent Glioblastoma, IDH-Wildtype and Recurrent Astrocytoma, IDH-Mutant, Grade 4, run by Mayo Clinic at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record

Search all trials

Trial summary

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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Allogeneic Adipose-derived Mesenchymal Stem Cells, Biospecimen Collection and Craniotomy
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
20 participants
Where
1 siteUnited States
Study dates
16 Jun 2023to 24 Jul 2027
Phase
Phase 1

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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
Show all 17Show fewer
  • 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

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Clinical Trials Referral Office contact

855-776-0015

mayocliniccancerstudies@mayo.edu

United States1 site, 1 recruiting

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224-9980

    Clinical Trials Referral Office, 855-776-0015, mayocliniccancerstudies@mayo.edu

    Alfredo Quinones-Hinojosa, MD

    Recruiting

Other trials in United States

About this study

Official 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

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.

Read the detailed description

PRIMARY OBJECTIVE:

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

SECONDARY OBJECTIVES:

  1. 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:

  1. 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.

Being tested
Allogeneic Adipose-derived Mesenchymal Stem Cells, Biospecimen Collection, Craniotomy, Magnetic Resonance Imaging, Ommaya Reservoir Tap
Medical subject headings
Glioblastoma, Astrocytoma

What participants receive

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

  • Allogeneic Adipose-derived Mesenchymal Stem CellsBiological

    Also called Allogeneic Adipose-derived MSCs, Allogeneic Adipose-derived Stem/Stromal Cells, Allogeneic Mesenchymal Stem/Stromal Cells

    Receive IT

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo blood and tissue sample collection

  • CraniotomyProcedure or surgery

    Also called incision of the skull, Open Craniotomy

    Undergo craniotomy

  • Magnetic Resonance ImagingProcedure or surgery

    Also called Magnetic Resonance, Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR

    Undergo MRI

  • Ommaya Reservoir TapProcedure or surgery

    Also called Ommaya Reservoir Access

    Undergo Ommaya reservoir placement for collection of CSF

Study groups in NCT05789394
Study groupRoleWhat they receive
Treatment (AMSCs)ExperimentalPatients receive AMSCs IT and undergo Ommaya reservoir placement during a craniotomy on study. Patients also undergo MRI on study and during follow-up, as well as blood sample, tissue sample, and CSF sample collection on study.
Receives: Biological: Allogeneic Adipose-derived Mesenchymal Stem Cells; Procedure: Biospecimen Collection; Procedure: Craniotomy; Procedure: Magnetic Resonance Imaging; Procedure: Ommaya Reservoir Tap

What is being measured

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

  1. Maximum tolerated dose (MTD)

    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.

    Measured: Up to 4 weeks

Secondary outcomes (5)

  1. Incidence of adverse events (AEs)

    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.

    Measured: Up to 1 year

  2. Best response

    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.

    Measured: Up to 5 years

  3. Response rate (RR)

    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.

    Measured: Up to 5 years

  4. Progression free survival

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

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

  5. Overall survival

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

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

Timeline

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

  1. First posted29 Mar 2023
  2. Study start16 Jun 2023
  3. Primary completion24 Jul 2027 (estimated)
  4. Study completion24 Jul 2027 (estimated)

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
20 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05789394
Sponsor's protocol ID
MC220704
Other IDs
21-004561
Status last verified
Mar 2026
First submitted
16 Mar 2023
Last updated
2 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in