Glioblastom WHO Grade 4Phase 1CV01-104NCT07862647
TMZ + RT + 5-ALA + LIDU SDT System for Newly Diagnosed Butterfly Glioma
A Phase 1 study testing 5-ALA and Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound in people with Glioblastom WHO Grade 4 and GBM, run by Alpheus Medical, Inc. at 1 site in United States.
Trial summary
This study is assessing the safety and tolerability of 5-ALA- mediated Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound, in patients who have just been diagnosed with bihemispheric (in both sides of the brain) glioblastoma, sometimes called butterfly glioblastoma, before they complete their standard of care… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Glioblastom WHO Grade 4, GBM, bGBM
- What is being tested?
- 5-ALA and Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound
- Type of treatment
- Pill or oral medicine, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Oct 2026to 30 Mar 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
- Patient, or the patient's legally authorized representative, must provide informed consent, stating understanding of the procedures and investigational nature of the study treatment, and willingness to comply with study requirements
- ≥ 18 and ≤ 80 years of age
- WHO performance status of ≤ 2 at screening
- Newly diagnosed histologically proven glioblastoma (WHO criteria 2021), with absence of IDH mutation demonstrated by negative IDH1 R132H staining on immunohistochemistry or equivalent molecular testing.
- Patients must have a radiographic butterfly glioma phenotype on MRI, defined as a contiguous enhancing glioma involving both cerebral hemispheres with midline extension through the corpus callosum (genu, body, and/or splenium) or other commissural pathway. Patients must not have started TMZ/RT. Bilateral involvement must reflect continuous tumor spread across the midline and not merely bilateral edema, mass effect, multifocal non-contiguous disease, or separate bilateral lesions. Note:
- patients must have undergone prior biopsy or decompressive surgery with histopathologic confirmation of butterfly glioma.
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- Adequate bone marrow and organ function, defined by the following laboratory values:
- Absolute neutrophil count (ANC) ≥ 1000 cells/mm3
- Platelet count ≥ 50,000 cells/mm3
- Hemoglobin (Hgb) ≥ 8 g/dL
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x upper limit of normal (ULN)
- Total bilirubin ≤ 1.5 x ULN (unless gilbert's syndrome, then patients may be eligible if total serum bilirubin is ≤ 3.0 x ULN or direct bilirubin is ≤ 1.5 x ULN)
- Creatinine clearance (CrCl) as estimated by Cockcroft-Gault equation of ≥ 50 mL/min
- Adequate coagulation function defined as PT (prothrombin time)/PTT (partial thromboplastin time) defined as either results within normal institutional values or not considered clinically significant <1.5 x ULN.
- Non-pregnant, non-lactating females who are postmenopausal, surgically sterile (bilateral tubal ligation with surgery at least 6 weeks prior to study initiation or hysterectomy), or who agree to use effective contraceptive methods as defined by the protocol during the study and for 30 days after the last investigational treatment, see Appendix 1. Postmenopausal is defined as at least 12 months natural spontaneous amenorrhea and a serum follicle stimulating hormone (FSH) concentration ≥ 40 IU/L, or at least 6 weeks following surgical menopause (bilateral oophorectomy). See Appendix 1, females on HRT and whose menopausal status is in doubt will be required to use one of the nonestrogen hormonal highly effective contraception methods if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of postmenopausal status before study enrollment.
- Women of childbearing potential must have a negative serum human chorionic gonadotropin (hCG) pregnancy test within 7 days prior to first 5-ALA administration
- Male patients must be surgically sterile (i.e., > 3 months post-vasectomy) or willing to use a highly effective double-barrier contraception method (e.g., male condom with diaphragm or male condom with cervical cap) for the duration of the study and for at least 30 days following SDT treatment. Male patients must not donate sperm from the time of study drug dosing until 30 days following SDT treatment.
Who cannot join
- Any component of the tumor in the infratentorial location (cerebellar or brainstem tumors are excluded)
- Commencing TMZ + RT per SOC before the first SDT treatment
- Leptomeningeal disease
- Corticosteroid use > 4 mg/day of dexamethasone (or equivalent), steroid dose should be stable or decreasing for ≥1 week prior to randomization
- Inability to undergo MRI or receive gadolinium (Gd)-based contrast agents
- Hypersensitivity to 5-ALA or porphyrins
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- Patient head size > 16.6 cm in breadth (max width of head, above the ears) and > 21.3 cm in length (front to back) to ensure fit within the helmet transducer array.
- Skull voids/defects > 10 mm diameter if defect is open, or > 14 mm if defect is covered with titanium burr hole cover, titanium mesh, or similar repair hardware. Note, Longeviti Neuro Solutions or other sonolucent implants should be treated as open defects and therefore may not exceed 10 mm in diameter.
- Hemorrhagic or ischemic stroke (including transient ischemic attacks) and central nervous system bleeding in the preceding 6 months that are not related to glioma surgery. History of prior intratumoral bleeding prior to screening is not an exclusion criterion; however, patients with a history of prior intratumoral or intracranial bleeding will undergo a non contrast head CT to exclude acute bleeding.
- Has any history of glioma, a concurrent malignancy, or malignancy within 3 years of randomization, unless definitive therapy is completed, with the exception of basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the cervix or breast that has completed curative therapy
- Patients who have clinically significant cerebral edema requiring urgent intervention (e.g., surgery, unstable or escalating high doses of steroids).
- Patients with rapid clinical deterioration that, in the opinion of the investigator, is likely to worsen during the first cycle of treatment.
- Acute or chronic types of porphyria.
- Gastrointestinal disorder that negatively affects absorption.
- Known active hepatitis B or C (Note: testing is not required).
- Known human immunodeficiency virus (HIV) infection (Note: testing is not required).
- Participation in another interventional clinical trial for butterfly glioma/GBM. (Observational studies are allowable)
- Unable to avoid phototoxic drugs (e.g., St. John's wort, griseofulvin, thiazide diuretics, sulfonylureas, phenothiazines, sulfonamides, quinolones, and tetracyclines) for 24 hours prior to and following 5-ALA administration.
- Any other concurrent severe or uncontrolled concomitant medical condition that could compromise participation in the study (e.g., clinically significant pulmonary disease, cardiac disease, clinically significant psychiatric or neurological disorder, active or uncontrolled infection).
- Women who are pregnant or breastfeeding
- Patients with a condition the Investigator believes would interfere with the ability to comply with study instructions, or that might confound the interpretation of the study results or put the patient at undue risk.
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
Dent Neurologic Institute
Buffalo, New York, 14226
Andrea Bryan, 716-250-2000, ABryan@dentinstitute.com
About this study
Official title: A Phase 1 Clinical Trial Evaluating the Safety and Tolerability of Bilateral Hemispheric 5-ALA-Mediated Sonodynamic Therapy Using the LIDU System Before Standard Radiochemotherapy and During Adjuvant Temozolomide in Patients With Histologically Proven Newly Diagnosed Butterfly Glioma
This study is assessing the safety and tolerability of 5-ALA- mediated Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound, in patients who have just been diagnosed with bihemispheric (in both sides of the brain) glioblastoma, sometimes called butterfly glioblastoma, before they complete their standard of care chemotherapy and radiation and after their standard of care chemotherapy and radiation while they are receviing their standard of care adjuvant chemotherapy.
- Conditions
- Glioblastom WHO Grade 4, GBM, bGBM, Butterfly GBM, Bihemispheric GBM, Glioblastoma
- Being tested
- 5-ALA, Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound
- Medical subject headings
- Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5-ALADrug
5-ALA is an oral drug that is taken prior to PoMA
Porphyrin Metabolite Activation (PoMA) with LIDU ultrasoundDevice
Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound
| Study group | Role | What they receive |
|---|---|---|
| 5-ALA + LIDU SDT System for newly diagnosed butterfly glioma | Experimental | Patients with newly diagnosed bihemispheric GBM will receive up to 2 treatments of 5-ALA + PoMA with the LIDU ultrasound device prior to receiving standard chemotherapy radiation treatment. Patients will then complete 6 weeks of SOC chemoradition (CRT). After completion of CRT, patients will receive 4 more treatments, 1 treatment every 28 days, of 5-ALA + PoMA. Receives: Drug: 5-ALA; Device: Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
To assess the number of participants with treatment-related adverse events as assessed by CTCAE v4.0 in patients receiving bi-hemispheric 5-ALA- mediated Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound.
Measured: up to 30 weeks
Secondary outcomes (4)
To assess overall response rate
Overall Response Rate (ORR), defined as the percentage of participants achieving a complete response or partial response according to RANO 2.0 criteria.
Measured: 1 year
Duration of Response
The length of time a patient experiences stability, or shrinkage of tumor
Measured: 1 year
median PFS
The time from first treatment until disease progression or death from any cause. The median is the time by which 50% of patients have experienced progression
Measured: 1 year
Median Overall Survival
The time from the first treatment until death from any cause. The median is the time by which 50% of patients have died and 50% remain alive.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2026
- Study start29 Oct 2026 (estimated)
- Primary completion30 Oct 2027 (estimated)
- Study completion30 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glioblastom WHO Grade 4Studies 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
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alpheus Medical, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07862647
- Sponsor's protocol ID
- CV01-104
- Acronym
- CV01-104
- Status last verified
- Oct 2026
- First submitted
- 28 Sep 2026
- Last updated
- 9 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Clinical Trial Evaluating the Safety and Tolerability of Bilateral Hemispheric 5-ALA-Mediated Sonodynamic Therapy Using the LIDU System Before Standard Radiochemotherapy and During Adjuvant Temozolomide in Patients With Histologically Proven Newly Diagnosed Butterfly Glioma
- ClinicalTrials.gov ID
- NCT07862647
- Acronym / sponsor ID
- CV01-104 / CV01-104
- Conditions
- Glioblastom WHO Grade 4, GBM, bGBM, Butterfly GBM, Bihemispheric GBM, Glioblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Alpheus Medical, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Oct 2026 (estimated)
- Primary completion
- 30 Oct 2027 (estimated)
- Study completion
- 30 Mar 2028 (estimated)
- First posted
- 7 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is assessing the safety and tolerability of 5-ALA- mediated Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound, in patients who have just been diagnosed with bihemispheric (in both sides of the brain) glioblastoma, sometimes called butterfly glioblastoma, before they complete their standard of care chemotherapy and radiation and after their standard of care chemotherapy and radiation while they are receviing their standard of care adjuvant chemotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 5-ALA + LIDU SDT System for newly diagnosed butterfly glioma | Experimental | Drug: 5-ALA, Device: Porphyrin Metabolite Activation (PoMA) with LIDU ultrasoundPatients with newly diagnosed bihemispheric GBM will receive up to 2 treatments of 5-ALA + PoMA with the LIDU ultrasound device prior to receiving standard chemotherapy radiation treatment. Patients will then complete 6 weeks of SOC chemoradition (CRT). After completion of CRT, patients will receive 4 more treatments, 1 treatment every 28 days, of 5-ALA + PoMA. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Patient, or the patient's legally authorized representative, must provide informed consent, stating understanding of the procedures and investigational nature of the study treatment, and willingness to comply with study requirements
- ≥ 18 and ≤ 80 years of age
- WHO performance status of ≤ 2 at screening
- Newly diagnosed histologically proven glioblastoma (WHO criteria 2021), with absence of IDH mutation demonstrated by negative IDH1 R132H staining on immunohistochemistry or equivalent molecular testing.
- Patients must have a radiographic butterfly glioma phenotype on MRI, defined as a contiguous enhancing glioma involving both cerebral hemispheres with midline extension through the corpus callosum (genu, body, and/or splenium) or other commissural pathway. Patients must not have started TMZ/RT. Bilateral involvement must reflect continuous tumor spread across the midline and not merely bilateral edema, mass effect, multifocal non-contiguous disease, or separate bilateral lesions. Note:
Key exclusion criteria (21 listed)
- Any component of the tumor in the infratentorial location (cerebellar or brainstem tumors are excluded)
- Commencing TMZ + RT per SOC before the first SDT treatment
- Leptomeningeal disease
- Corticosteroid use > 4 mg/day of dexamethasone (or equivalent), steroid dose should be stable or decreasing for ≥1 week prior to randomization
- Inability to undergo MRI or receive gadolinium (Gd)-based contrast agents
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 5-ALA5-ALA is an oral drug that is taken prior to PoMA | Drug | 5-ALA + LIDU SDT System for newly diagnosed butterfly glioma |
| Porphyrin Metabolite Activation (PoMA) with LIDU ultrasoundPorphyrin Metabolite Activation (PoMA) with LIDU ultrasound | Device | 5-ALA + LIDU SDT System for newly diagnosed butterfly glioma |
Primary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: up to 30 weeks
To assess the number of participants with treatment-related adverse events as assessed by CTCAE v4.0 in patients receiving bi-hemispheric 5-ALA- mediated Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound.
4 secondary outcomes
To assess overall response rate
Time frame: 1 year
Duration of Response
Time frame: 1 year
median PFS
Time frame: 1 year
Median Overall Survival
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2026.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 5-ALA and Porphyrin Metabolite Activation (PoMA) with LIDU ultrasound in people with Glioblastom WHO Grade 4 and GBM, run by Alpheus Medical, 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 17 inclusion and 21 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: looking for participants now, as of the sponsor's last update on 9 Oct 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?
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ann-Marie Hulstine (484-252-1960, AMHulstine@alpheusmedical.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: up to 30 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Oct 2026.