Recurrent GlioblastomaPhase 1GBM 001NCT06039709
Sonodynamic Therapy in Patients With Recurrent GBM
A Phase 1 study testing 5-ALA and Low-Intensity Focused Ultrasound (SDT) in people with Recurrent Glioblastoma and Glioblastoma Multiforme, run by Shayan Moosa, MD at 1 site in United States.
Trial summary
Patients diagnosed with glioblastoma (GBM) are faced with limited treatment options. This pilot study will evaluate the safety and feasibility of combining an investigational drug called 5-ALA with neuronavigation-guided low-intensity focused ultrasound (LIFU) for patients who have recurrent GBM. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Recurrent Glioblastoma, Glioblastoma Multiforme, GBM
- What is being tested?
- 5-ALA and Low-Intensity Focused Ultrasound (SDT)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Jan 2024to Jun 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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Disease status and Disease Parameters:
- Suspected recurrent glioblastoma that is clearly measurable based on the modified Response Assessment in Neuro-Oncology (RANO) criteria
- The tumor lesion needs to comprise at least 1 contrast-enhancing lesion with a volume of ≥ 2 cm3 and ≤ 20 cm3 of targeted treatment area
- Tumor tissue to be treated is in a surgically accessible brain region for resection
- The brain tumor to be treated must be in the treatment envelope of the NaviFUS system (30 mm to 90 mm from the inner skull table)
- Recurrence will be assessed by imaging and confirmed by consensus at tumor board
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- Men or women between the ages of 18-80 years of age at the time of consent
- No contraindication to repeat brain surgery
- Karnofsky Performance Score of 70-100
- Able to undergo an MRI with contrast
- Able to swallow oral medications
- Willingness and ability to comply with scheduled visits, treatment plans, lifestyle considerations, laboratory tests, and other procedures.
- Ability to understand and the willingness to sign a written informed consent document (personally or by the legally authorized representative, if applicable).
- Participants who received prior chemotherapy, radiation therapy, immunotherapy, and/or another investigational therapy must have recovered (Common Terminology Criteria for Adverse Events [CTCAE] Grade ≤1 or baseline) from the acute effects of the therapy or therapies) except for residual alopecia or Grade 2 peripheral neuropathy prior to registration.
- Has adequate bone marrow and organ function as defined by the following laboratory values (as assessed by the local laboratory for eligibility):
- Hematological
- Absolute neutrophil count (ANC) ≥1000/mm3
- Platelets ≥ 100,000/mm3
- Hemoglobin ≥ 11 g/dL for women and ≥ 12 g/dL for men Participants may receive erythrocyte transfusions to achieve this hemoglobin level at the discretion of the investigator.
- INR ≤ 1.4
- Renal \& Hepatic
- Creatinine clearance CrCl ≥ 60 mL/min/1.73 m2 as estimated by the Cockcroft-Gault (C-G) equation. If estimated CrCl is abnormal, accurate measurement should be obtained by 24- hour CrCl.
- Bilirubin ≤ 1.5 x ULN (except in patients with Gilbert's disease, where bilirubin to 2.0x ULN is allowed).
- AST and ALT ≤ 3 x ULN
- Alkaline phosphatase ≤ 3 x ULN
- Estimated glomerular filtration rate ≥30mL/min/1.73m2
Who cannot join
- Known sensitivity or allergy to 5-ALA
- Simultaneous use of other potentially phototoxic substances (e.g. tetracyclines, sulfonamides, fluoroquinolones, hypericin extracts)
- Diagnosis of porphyria
- Hypersensitivity against porphyrins
- Pregnancy
- Significant cardiac disease or coagulopathy
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- Herniation / intractable seizure / other clinical indications requiring urgent resection
- Known active systemic bacterial infection (requiring intravenous [IV] antibiotics at time of initiating study treatment), fungal infection, or detectable viral infection (such as known human immunodeficiency virus positivity or with known active hepatitis B or C (for example, hepatitis B surface antigen positive)
- Have had a recent (≤3 months prior to registration) transient ischemic attack or stroke
- Significant vascular disease (e.g. aortic aneurysm)
- Evidence of bleeding diathesis or coagulopathy
- Need for systemic anticoagulation which cannot be held for 7 days prior to SDT
- Unstable angina and/or congestive heart failure (NYH Class III or Class IV; see section 13.2) within 6 months prior to registration
- Severe hypertension (systolic ≥ 180 mm Hg; diastolic ≥ 120 mm Hg) despite anti-hypertensive medications
- Transmural myocardial infarction within 6 months prior to registration
- Serious and inadequately controlled cardiac arrhythmia
- Acute exacerbation of chronic obstructive pulmonary disease
- Has serious and/or uncontrolled preexisting medical condition(s) that, in the judgment of the investigator, would preclude participation in this study
- Treatment with another investigational drug or investigational procedure within 30 days prior to registration or within 5 half-lives of the investigational product, whichever is longer
- Brain edema and/or mass effect that causes midline shift of more than 15 mm
- Evidence of recent (within 30 days prior to registration) intracranial hemorrhage
- Calcifications or metallic implanted objects in the focused ultrasound sonication path
- Scalp atrophy or scars at the expected location of transducer
- Cerebral or systemic vasculopathy
- Need for or currently on dialysis
- Respiratory: chronic pulmonary disorders (e.g., severe emphysema, COPD, pulmonary vasculitis, or other causes of reduced pulmonary vascular cross-sectional area).
- Receipt of radiotherapy ≤21 days prior to registration
- Receipt of chemotherapy ≤ 21 days prior to registration
- Prior treatment with sonodynamic therapy
- Concurrent use of Optune device
- Concurrent use of supplements or medications with substantial antioxidant effects (including sulfhydryl-containing medications such as captopril or supplements such as N-acetylcysteine, or high doses of vitamins with antioxidant activity such as C or E)
- Known sensitivity to gadolinium
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Virginia
Charlottesville, Virginia, 22903
Zachary Sturgill, 4342439986, FFM7RC@uvahealth.org
Judith Beenhakker, MS, 4349821856, jgb3p@uvahealth.org
About this study
Official title: Pilot Study of Sonodynamic Therapy With 5-ALA for the Treatment of Recurrent Glioblastoma Using Neuronavigation-Guided Low-Intensity Focused Ultrasound
Patients diagnosed with glioblastoma (GBM) are faced with limited treatment options. This pilot study will evaluate the safety and feasibility of combining an investigational drug called 5-ALA with neuronavigation-guided low-intensity focused ultrasound (LIFU) for patients who have recurrent GBM. Focused ultrasound (FUS) can be used to non-invasively destroy tumor tissue while preserving normal tissue. When FUS is combined with 5-ALA, this combinatorial approach is called sonodynamic therapy (SDT), and this investigational therapy is being tested for its ability to cause damage to GBM cells. SDT will take place prior to surgery for recurrent GBM.
Read the detailed description
The combination of 5-ALA (Gleolan) and LIFU is collectively known as sonodynamic therapy (SDT). SDT is an investigational therapy that will be administered 1-3 weeks before surgery for recurrent GBM. Researchers seek to determine the safety and feasibility of this therapy as well as measure its effectiveness to elicit tumor-cell death. All participants are expected to stay overnight in the hospital following administration of SDT to monitor for adverse events.
- Conditions
- Recurrent Glioblastoma, Glioblastoma Multiforme, GBM
- Being tested
- 5-ALA and Low-Intensity Focused Ultrasound (SDT)
- Keywords
- Sonodynamic Therapy, Low-Intensity Focused Ultrasound (LIFU), Focused Ultrasound, 5-ALA, Brain Cancer
- Medical subject headings
- Glioblastoma, Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5-ALA and Low-Intensity Focused Ultrasound (SDT)Combination product
5-ALA (20mg/kg orally) given ~6 hours prior to LIFU. Focused ultrasound will target a maximum of 50% of the tumor.
| Study group | Role | What they receive |
|---|---|---|
| Sonodynamic Therapy (5-ALA + LIFU) | Experimental | Administration of SDT occurs 1-3 weeks prior to GBM resection Receives: Combination Product: 5-ALA and Low-Intensity Focused Ultrasound (SDT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Per NCI Common Terminology Criteria for Adverse Events v5.0
Measured: From informed consent through 30 days after study intervention is complete
Severity of adverse events
Per NCI Common Terminology Criteria for Adverse Events v5.0
Measured: From informed consent through 30 days after study intervention is complete
Incidence of intracranial hemorrhage and/or worsening of edema
On post-SDT MRIs
Measured: From day after SDT (day 1) up to the time of surgery (day 7-day 21)
Extent of targeted tumor area receiving FUS
Use of NaviFUS system to target a maximum of 50% of the tumor volume of one contiguous lesion
Measured: Day 0
Ability to have participants undergo planned surgery without delay
A delay is defined as more than 3 weeks after SDT
Measured: within 3 weeks following SDT
Secondary outcomes (2)
Response of target tissue following SDT on imaging
Via MRI w/ use of modified Response Assessment in Neuro-Oncology (RANO) criteria
Measured: From day after SDT (day 1) up to 100 days after intervention is completed
Histologic tumor devitalization
Evaluating cell fate and cell death via histologic samples after GBM resection
Measured: Day 7-Day 21
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2023
- Study start31 Jan 2024
- Primary completionDec 2026 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent GlioblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 11 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shayan Moosa, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Shayan Moosa, MD, University of Virginia
- Investigators
- Shayan Moosa, MD, Principal investigator, UVA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06039709
- Sponsor's protocol ID
- HSR230064
- Acronym
- GBM 001
- Status last verified
- Apr 2026
- First submitted
- 10 Sep 2023
- Last updated
- 28 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No