Diffuse Midline Glioma (DMG)Phase 1/2TIGER-1NCT07767110
Targeted LSAM-Cisplatin Infusion in Gliomas, Evaluation of Response
A Phase 1/2 study testing LSAM-Cisplatin in people with Diffuse Midline Glioma (DMG) and Diffuse Intrinsic Pontine Glioma (DIPG), run by NanOlogy, LLC.
Trial summary
Open-label, dose-escalating, Phase 1/2a trial of Large Surface Area Microparticle (LSAM)-Cisplatin to treat participants with diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), via stereotactic infusion under intraoperative magnetic resonance imaging (MRI) guidance. Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Diffuse Midline Glioma (DMG), Diffuse Intrinsic Pontine Glioma (DIPG), Diffuse Midline Glioma or Diffuse Intrinsic Pontine Glioma
- What is being tested?
- LSAM-Cisplatin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Dec 2026to Dec 2029
- Phase
- Phase 1/2
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 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Aged 3 to ≤ 21 years.
- Diagnosis of diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), based on characteristic magnetic resonance imaging (MRI) findings and/or histopathologic confirmation.
- Prior radiation treatment must have included focal radiation therapy per institutional standard of care and must have been initiated within 6 weeks of diagnosis.
- At least 4 weeks, but no more than 12 weeks, post-completion of radiotherapy treatment.
- A standard of care post-radiation magnetic resonance imaging (MRI) performed 4 to 6 weeks after radiation therapy is required for confirmation of eligibility.
- Performance status [Karnofsky Performance Scale or Lansky Performance Score] within 14 days of Day 1 ≥ 60.
- Absence of other significant medical condition.
- A legal parent/guardian and/or participant must be able to understand and be willing to sign a written informed consent and/or assent document, as appropriate.
Who cannot join
- Untreated symptomatic hydrocephalus at the time of consent, has metastatic or disseminated disease, or leptomeningeal disease.
- Magnetic resonance imaging (MRI) findings that preclude stereotactic procedure.
- Intercurrent illnesses or conditions which preclude participation: active systemic infections, autoimmune disease requiring systemic immunomodulation, active or uncontrolled seizure disorder, central nervous system (CNS) vasculopathy or aneurysms, Grade ≥3 cardiac dysfunction, Fridericia-corrected QT interval (QTcF) ≥470 ms.
- Abnormal organ function:
- Renal insufficiency: glomerular filtration rate (GFR) ≤ 60 mL/min/1.73m² (with Schwartz equation)
- Hepatic dysfunction: aspartate aminotransferase (AST) / alanine aminotransferase (ALT) ≤ 1.5 x upper limit of normal (ULN) and 3 x upper limit of normal (ULN) in the presence of liver metastases; or bilirubin ≤ 1.5 x upper limit of normal (ULN) and 3 x upper limit of normal (ULN) in the presence of Gilbert disease
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- Bone marrow suppression:
- absolute neutrophil count (ANC) < 1,000/μL
- platelets < 100,000/μL
- Coagulopathy or international normalized ratio (INR) > 1.5
- Receiving any anticoagulants or antiplatelet drugs; any drugs known to cause ototoxicity and nephrotoxicity; receiving any other tumor-directed therapy.
- Known allergy or hypersensitivity to the study agent (including cisplatin and diluent components).
- Female participants of childbearing potential must not be pregnant or breast-feeding.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Phase 1/2a Study to Determine the Safety of Intratumoral Infusion of Large Surface Area Microparticle (LSAM)-Cisplatin in Participants With Diffuse Midline Glioma (DMG), Including Diffuse Intrinsic Pontine Glioma (DIPG)
Open-label, dose-escalating, Phase 1/2a trial of Large Surface Area Microparticle (LSAM)-Cisplatin to treat participants with diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), via stereotactic infusion under intraoperative magnetic resonance imaging (MRI) guidance.
Read the detailed description
Large Surface Area Microparticle (LSAM)-Cisplatin consists of large surface area microparticles of the chemotherapy drug cisplatin. These microparticles are administered as a single-magnetic resonance imaging (MRI) guided infusion directly into the tumor to target cancer at the site of the disease with less systemic exposure than intravenously administered chemotherapy. In this study, all participants with diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), will receive LSAM-Cisplatin and will be evaluated to determine whether it is safe and has an effect on the tumor.
- Conditions
- Diffuse Midline Glioma (DMG), Diffuse Intrinsic Pontine Glioma (DIPG), Diffuse Midline Glioma or Diffuse Intrinsic Pontine Glioma
- Being tested
- LSAM-Cisplatin
- Keywords
- Diffuse Midline Glioma, Diffuse Intrinsic Pontine Glioma, H3 K27 Mutation, Pediatric Brain Tumor, Convection-enhanced delivery
- Medical subject headings
- Diffuse Intrinsic Pontine Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LSAM-CisplatinDrug
Participants with diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG), will receive a single intratumoral infusion of LSAM-Cisplatin 6 mg/mL via MRI-guided delivery at an infusion rate of 5 µL/min.
Phase 1 (Dose Escalation): Participants will be enrolled sequentially into one of six dose levels. Volumes being delivered will range from 27 to 296 µL over a time period of 5 to 59 minutes.
Phase 2 (Dose Expansion): Participants will receive LSAM-Cisplatin 6 mg/mL at the recommended Phase 2 dose (RP2D) selected based on the safety and tolerability findings from the dose-escalation phase.
| Study group | Role | What they receive |
|---|---|---|
| LSAM-Cisplatin 6 mg/mL | Experimental | Phase 1 (Dose Escalation): Intratumoral infusion of LSAM-Cisplatin 6 mg/mL using sequential, dose escalating cohorts.
Phase 2 (Dose Expansion): Intratumoral infusion of LSAM-Cisplatin 6 mg/mL at the recommended Phase 2 dose (RP2D). Receives: Drug: LSAM-Cisplatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events (Safety and Tolerability)
Treatment Emergent Adverse Events will be assessed by changes in adverse events, changes in concomitant medications, changes in laboratory values, and physical exams.
Measured: Day 1 to Week 24
Secondary outcomes (5)
Objective Response Rate (ORR)
The proportion of participants with overall confirmed response of complete response (CR) or partial response (PR) as determined using Response Assessment in Pediatric Neuro-Oncology (RAPNO)/Response Assessment in Pediatric Neuro-Oncology for Diffuse Intrinsic Pontine Glioma (RAPNO-DIPG) criteria.
Measured: Weeks 4, 12, and 24
Progression-Free Survival (PFS)
Progression-Free survival (PFS) as assessed using Response Assessment in Pediatric Neuro-Oncology (RAPNO)/Response Assessment in Pediatric Neuro-Oncology for Diffuse Intrinsic Pontine Glioma (RAPNO-DIPG) criteria.
Measured: Day 1 to Week 24
Overall Survival (OS)
Overall survival (OS) as determined by survival time post-infusion of LSAM-Cisplatin.
Measured: Day 1 to Week 24
Concentration of Cisplatin in the Systemic Circulation Post-infusion
Cisplatin concentrations in plasma samples collected pre-infusion of LSAM-Cisplatin, and at 1, 2, 24, and 48 hours after completion of the LSAM-Cisplatin infusion, and at Weeks 1, 2, 3, 4, and 8.
Measured: Day 1 to Week 8
Concentration of Cisplatin in the Cerebrospinal Fluid (CSF) Post-infusion
Cisplatin concentrations in cerebrospinal fluid (CSF) samples collected prior to infusion of LSAM-Cisplatin, prior to discharge from the hospital post-infusion; and at Weeks 4, 12, and 24.
Measured: Prior to infusion to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2026
- Study startDec 2026 (estimated)
- Primary completionDec 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diffuse Midline Glioma (DMG)Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NanOlogy, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- WEP Clinical
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07767110
- Sponsor's protocol ID
- LSAMCIS-01
- Acronym
- TIGER-1
- Status last verified
- Aug 2026
- First submitted
- 11 Aug 2026
- Last updated
- 18 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No