[212Pb]VMT-Alpha-NET in Metastatic or Inoperable Somatostatin-Receptor Positive Gastrointestinal Neuroendocrine Tumors, Pheochromocytoma/Paragangliomas, Small Cell Lung, Renal Cell, and Head and Neck Cancers

NCT ID: NCT06479811

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-08-19

Study Completion Date

2032-01-01

Brief Summary

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Background:

Some cancers have high levels of proteins called somatostatin receptors (SSTRs) on the surface of the tumors. These tumors can be in the lung, head and neck, digestive tract, kidneys, and in or near the adrenal glands. Researchers want to know if drug treatments that target SSTRs can help shrink these types of tumors.

Objective:

To test a study drug (\[212Pb\]VMT-Alpha-NET) in people with tumors that have SSTRs.

Eligibility:

People aged 18 years and older with tumors of the lung, kidneys, head and neck, digestive tract, or adrenal glands that have SSTRs. Their tumors must have spread to other organs and cannot be removed with surgery.

Design:

Participants will be screened. They will have a physical exam with blood and urine tests. They will have imaging scans and a test of their heart function. A sample of tumor tissue may be collected if one is not already available.

\[212Pb\]VMT-Alpha-NET is given through a tube attached to a needle inserted into a vein. The drug will be given on the first day of four 8-week cycles. Participants will stay in the hospital for a few nights after each dose. They will have blood tests once a week during each cycle.

Some participants will also get a related study drug (\[203Pb\]VMT-Alpha-NET). They will receive this drug a few days before the first 2 cycles. At 4, 24, and 48 hours after each infusion, they will have whole body scans. These scans will show where the study drug went in their body.

Follow-up visits will continue up to 6 years after the last treatment.

Detailed Description

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Background:

* Somatostatin receptors (SSTR) have been shown to be over-expressed in a number of human tumors, including gastrointestinal (GI) neuroendocrine tumors (NET), pheochromocytoma/paragangliomas (PPGL), small cell lung cancers (SCLC), kidney cancers (KC), and some head and neck (H\&N) cancers.
* Targeted radioligand therapy (TRT) is a class of cancer therapeutic agents formed by attaching a radioactive isotope to a ligand that can target specific surface receptors such as SSTR on a tumor cell membrane. Efficacy is typically determined by the radiation dose deposited onto a tumor, which is determined by the radioactive isotope being used as well as the binding characteristics of the ligand-receptor/transporter pair.
* Alpha emitters such as 212Pb emit alpha particles that are more damaging to tumor cells than beta emitters such as 177Lu. Therefore, TRT agents using alpha emitters are considered to be more potent than beta-emitting TRTs.
* VMT-Alpha-NET is a peptide that binds to SSTR, which when attached to 212Pb becomes an alpha particle-emitting TRT that can be used to treat tumors that have SSTR surface expression.
* \[203Pb\]VMT-Alpha-NET is the chemically identical imaging surrogate for \[212Pb\]VMT-Alpha-NET and has the same mechanism of action via binding to SSTR2. The nuclide 203Pb contained in \[203Pb\]VMT-Alpha-NET emits gamma radiation suitable for single-photon emission computerized tomography (SPECT) imaging. These images can be used to assess drug product biodistribution throughout the body.

Objective:

-To determine the maximum tolerated dose (MTD) of \[212Pb\]VMT-Alpha-NET (dose escalation cohort) and assess the safety of \[212Pb\]VMT-Alpha-NET at the MTD (dose expansions cohorts).

Eligibility:

* Age \>= 18 years.
* Histopathologically confirmed GI NET, PPGL, SCLC, KC, or H\&N (nasopharyngeal carcinoma \[NPC\], olfactory neuroblastoma \[ONB\], sinonasal neuroendocrine carcinoma \[SNEC\]) cancers that are metastatic or inoperable.
* No prior systemic radioligand therapy.
* Eastern Cooperative Oncology Group (ECOG) Performance Status \<= 1.

Design:

* This is an open-label, single-arm, single-center, phase I study evaluating the safety, preliminary efficacy, and pharmacokinetic properties of \[212Pb\]VMT-Alpha-NET in GI NET, PPGL, SCLC, KC, or H\&N cancers.
* First, participants will be accrued in Dose Escalation Part with 4 dose levels to estimate MTD of \[212Pb\]VMT-Alpha-NET. Once MTD is estimated, the following participants with GI NET, PPGL, SCLC, KC, or H\&N cancers will be accrued in separate cohorts and treated at MTD of \[212Pb\]VMT-Alpha-NET.
* \[212Pb\]VMT-Alpha-NET will be given IV every 8 weeks for a total of 4 administrations.
* A subset of participants (Dosimetry Arm 1) will have \[203Pb\]VMT-Alpha-NET administration followed by whole-body gamma scans combined with dosimetry SPECT/ Computed Tomography (CT) scans and collection of blood and urine samples prior to each cycle.
* Participants will have timed clinical laboratory evaluations, imaging studies, and research blood, and urine samples while on the study therapy for safety and efficacy evaluations.
* Following completion of treatment, participants will be seen at the NIH Clinical Center approximately 30 days later, every 12 weeks for 3 years after that for safety and efficacy assessments. Beyond 3 years, participants will be contacted annually through any NIH-approved platform to assess for overall survival and health status.

Conditions

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Head and Neck Tumors Kidney Cancers Small Cell Lung Cancers Pheochromocytoma/Paragangliomas Gastrointestinal Neuroendocrine Tumors Somatostatin Receptor Positive

Keywords

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212Pb Targeted alpha Therapy Image-Guided Dosimetry VMT-alpha-NET

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1/Dosimetry Arm 1

Escalating doses of \[212Pb\]VMT-alpha-NET, imaging with \[203Pb\]VMT-alpha-NET

Group Type EXPERIMENTAL

68Ga-DOTATATE

Intervention Type DRUG

68Ga-DOTATATE PET/CT whole-body scanning will be done at at different intervals to monitor disease.

[203Pb]VMT-alpha-NET

Intervention Type DRUG

\[203Pb\]VMT-alpha-NET will be given IV 7 days prior to \[212Pb\]VMT-alpha-NET.

[212Pb]VMT-alpha-NET

Intervention Type DRUG

\[212Pb\]VMT-alpha-NET will be given IV on Day 1 of every cycle for 4 cycles total at escalating doses in Phase I and at MTD during dose expansion. One cycle is 8 weeks.

2/Arm 2

Escalating doses of \[212Pb\]VMT-alpha-NET

Group Type EXPERIMENTAL

68Ga-DOTATATE

Intervention Type DRUG

68Ga-DOTATATE PET/CT whole-body scanning will be done at at different intervals to monitor disease.

[212Pb]VMT-alpha-NET

Intervention Type DRUG

\[212Pb\]VMT-alpha-NET will be given IV on Day 1 of every cycle for 4 cycles total at escalating doses in Phase I and at MTD during dose expansion. One cycle is 8 weeks.

3/Arm 3

\[212Pb\]VMT-alpha-NET at MTD

Group Type EXPERIMENTAL

68Ga-DOTATATE

Intervention Type DRUG

68Ga-DOTATATE PET/CT whole-body scanning will be done at at different intervals to monitor disease.

[212Pb]VMT-alpha-NET

Intervention Type DRUG

\[212Pb\]VMT-alpha-NET will be given IV on Day 1 of every cycle for 4 cycles total at escalating doses in Phase I and at MTD during dose expansion. One cycle is 8 weeks.

Interventions

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68Ga-DOTATATE

68Ga-DOTATATE PET/CT whole-body scanning will be done at at different intervals to monitor disease.

Intervention Type DRUG

[203Pb]VMT-alpha-NET

\[203Pb\]VMT-alpha-NET will be given IV 7 days prior to \[212Pb\]VMT-alpha-NET.

Intervention Type DRUG

[212Pb]VMT-alpha-NET

\[212Pb\]VMT-alpha-NET will be given IV on Day 1 of every cycle for 4 cycles total at escalating doses in Phase I and at MTD during dose expansion. One cycle is 8 weeks.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Participants must have histopathologically confirmed gastrointestinal neuroendocrine tumors (GI NET), pheochromocytoma/paraganglioma (PPGL), small cell lung cancers (SCLC), kidney cancers (KC), or Head \& Neck cancers (nasopharyngeal carcinoma \[NPC\], olfactory neuroblastoma \[ONB\], sinonasal neuroendocrine carcinoma \[SNEC\]) that are metastatic or inoperable per Standard of Care. Note: for KC, all histopathologies of kidney cancers are eligible as long as it is a primary renal neoplasm.
* Required prior therapies:
* GI NET, PPGL, H\&N: no specific prior therapy is needed.
* SCLC: At least one prior line of standard of care systemic treatment such as chemotherapy and/or immunotherapy.
* KC: Renal cell carcinoma (RCC) participants should have received at least one line of prior therapy in the metastatic setting and should have received at least one Programmed cell death protein 1 (PD1) / Programmed death-ligand 1 (PDL1)-targeted immune checkpoint inhibitor as well as one agent targeting the VEGF pathway. Participants with fumarate hydratase (FH) deficient RCC should have received at least one prior line of systemic therapy (such as bevacizumab plus erlotinib). No prior therapy is needed for participants with other histologic subtypes.
* Have NOT received prior systemic radioligand therapy for definitive therapeutic purposes. Prior external beam radiation therapy is allowed.
* History of disease progression by imaging (e.g., RECIST 1.1) or clinically (defined as increase in severity or frequency of symptoms related to disease) within the past 36 months prior to the first dose of \[203Pb\]VMT-Alpha-NET.
* Evidence of somatostatin receptors (SSTR) expression on at least 50% of the radiographically identifiable (i.e., visible on an anatomic scan such as CT or magnetic resonance imaging \[MRI\]) tumor, as indicated by a positive (uptake qualitatively identifiable as above the local background) on SSTR PET scan.
* Age \>= 18 years.
* ECOG performance status \<=1.
* Participants must have adequate organ and marrow function as defined below:

* Leukocytes: 3,000/microliter
* Absolute Neutrophil Count: 1,500/microliter
* Platelets 100,000/microliter
* Hemoglobin \>= 9.0 g/dL
* Total bilirubin: within normal institutional limits. Note: \<= 5 X institutional upper limit of normal (ULN) if bilirubin elevation is due to a benign process such as Gilbert syndrome
* AST: \<= 2.5 X institutional ULN
* ALT: \<= 2.5 X institutional ULN
* Creatinine: within normal institutional limits

OR

* Calculated creatinine clearance (glomerular filtration rate (eGFR): \>= 60 mL/min/1.73 m\^2 for participants with creatinine levels above institutional normal

* Participants with treated brain metastases are eligible if follow-up brain imaging after central nervous system (CNS)-directed therapy shows no evidence of progression at screening.
* Participants with new or progressive brain metastases or leptomeningeal disease are eligible as long as the participant is asymptomatic and not requiring medication for symptom control from the brain lesions at screening.
* Participants seropositive for human immunodeficiency virus (HIV) must:
* be on effective anti-retroviral therapy; and
* have an undetectable viral load at screening.

* Participants seropositive for hepatitis B virus (HBV), must have HBV viral load undetectable at screening.
* Participants seropositive for hepatitis C virus (HCV) must:
* received curative treatment; and
* have an undetectable HCV viral load at screening.

* Individuals of child-bearing potential (IOCBP) and individuals who can father children must agree to use an effective method of contraception (barrier, hormonal, intrauterine device \[IUD\], surgical sterilization, abstinence) at study entry and up to 6 months after the last dose of the study agent(s).
* Nursing participants must be willing to discontinue nursing from study treatment initiation through 6 months after the last dose of the study agents.
* The ability of the participant to understand and the willingness to sign a written informed consent document.

Exclusion Criteria

* Any investigational agents should be stopped at least 28 days prior to the first dose of \[203Pb\]VMT-Alpha-NET.
* Systemic therapy should be stopped at least 28 days prior to the first dose of \[203Pb\]VMT-Alpha-NET (participants with prior systemic therapies for their malignancy only, except participants with SCLC).
* Systemic therapy should be stopped at least 14 days prior to the first dose of \[203Pb\]VMT-Alpha-NET (participants with SCLC only).
* History of allergic reactions attributed to compounds of similar chemical or biologic composition to VMT-Alpha-NET.
* Positive Beta human chorionic gonadotropin (Beta-HCG) serum or urine pregnancy test performed in IOCBP at screening.
* QTc \> 450 ms on electrocardiogram (EKG) at screening. Note: Framingham correction for QTc will be used
* History of or detection at screening of active/untreated secondary malignancy except nonmelanoma skin cancer and carcinoma in situ of the uterine cervix.
* Uncontrolled intercurrent illness, factors, evaluated by medical history and physical exam which would potentially increase in the risk of the participant.
Minimum Eligible Age

18 Years

Maximum Eligible Age

120 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Frank I Lin, M.D.

Role: PRINCIPAL_INVESTIGATOR

National Cancer Institute (NCI)

Locations

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National Institutes of Health Clinical Center

Bethesda, Maryland, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Joy H Zou, R.N.

Role: CONTACT

Phone: (240) 760-6153

Email: [email protected]

Frank I Lin, M.D.

Role: CONTACT

Phone: (240) 760-6166

Email: [email protected]

Facility Contacts

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National Cancer Institute Referral Office

Role: primary

Related Links

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Other Identifiers

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001709-C

Identifier Type: -

Identifier Source: secondary_id

10001709

Identifier Type: -

Identifier Source: org_study_id