Safety and Tolerability of Fb-PMT in Recurrent Glioblastoma

NCT05226494 | Recruiting Phase 1 FDA Drug

• 1 other identifier: NP-100-101
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

Brief Summary

Glioblastoma is a highly aggressive and fatal form of primary malignant brain tumor with limited treatment options. fb-PMT affects a large group of cancer cell signaling pathways and thus may be effective in heterogeneous, treatment-resistant tumors such as Glioblastoma. fb-PMT also is actively transported across the blood-brain barrier into the brain. This study is being conducted to determine the dose level for further clinical development of fb-PMT to treat recurrent Glioblastoma.

Conditions

Glioma, Malignant

Keywords

Glioblastoma; Glioblastoma Multiforme

Outcome Measures

Primary Outcomes (2)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

  Determined by the number of Treatment-Emergent Adverse Events, including Dose-Limiting Toxicities per patient.

  15 months

• Incidence of Dose Limiting Toxicities [Safety and Tolerability]

  Number of participants with a dose-limiting toxicity during the first cycle (28 days) of treatment at their highest dose level administered.

  28 Days

Secondary Outcomes (1)

• Establishment of Recommended Phase 2 Dose

  28 Days

Study Arms (1)

Treatment (fb-PMT)

EXPERIMENTAL

Daily subcutaneous injection of fb-PMT in four escalating cohorts to determine maximum tolerated dose, followed by treatment of up to 10 additional patients at maximum tolerated dose.

Drug: fb-PMT

Interventions

fb-PMT DRUG

Daily dosing based on patient weight

Also known as: NP-100, NP751

Treatment (fb-PMT)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically proven intracranial glioblastoma, with first or second recurrence
• On stable or decreasing dose of steroids, if taken prior to screening
• Baseline MRI (with and without contrast) completed with 5 days of starting fb-PMT
• Prior completion of and recovery from the effects of standard of care for glioblastoma management with surgery/biopsy and radiotherapy
• Confirmation of true progressive disease for patients previously treated with interstitial brachytherapy or stereotactic radio surgery
• Life expectancy of more than three months
• Karnofsky Performance Status of ≥ 70
• Hypertension must be well controlled (≤ 95th percentile) on stable doses of medication
• Adequate bone marrow and organ function, confirmed by laboratory testing at screening
• Patient or caregiver must be able to store drug under refrigerated conditions, prepare and administer daily subcutaneous injections on a set schedule, and record information in a daily treatment diary
• Women of childbearing potential must agree to ongoing pregnancy testing and to use medically acceptable contraception for the duration of the study and for 2 months after their last dose of study drug
• Males must agree to use medically acceptable contraception and refrain from donating sperm for the duration of the study and for 2 months after their last dose of study drug

You may not qualify if:

• Significant medical illness that is uncontrolled, may obscure toxicity, may dangerously alter drug metabolism, or may compromise ability for study participation
• History of any other cancer (except non-melanoma skin cancer or carcinoma in-situ of the cervix), unless in complete remission and off all therapy for that disease for at least 3 months prior to first dose of study drug
• Use of bevacizumab or any other experimental drug or therapy within 28 days of study treatment
• Prior therapy with fb-PMT or related drugs
• Currently pregnant or breastfeeding
• Active infection or serious intercurrent medical illness
• Surgery of any type within the preceding 28 days that has not fully healed
• A serious or non-healing wound, ulcer, or bone fracture
• A known bleeding diathesis or coagulopathy, or a history of bleeding diathesis within 28 days of study treatment
• A known thrombophilic condition (i.e., protein S, protein C, or antithrombin III deficiency, Factor V Leiden, Factor II G20210A mutation, homocysteinemia or antiphospholipid antibody syndrome). Testing is not required in patients without thrombophilic history.
• Evidence of new central nervous system hemorrhage on baseline MRI obtained within 14 days prior to study enrollment
• Clinically significant cardiovascular event such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of screening.
• New York Heart Association classification of heart disease greater than Class 2
• QTc interval \> 450 msec in males or \> 470 msec in females at screening
• Use of concomitant medications that prolong the QT/QTc interval or risk inducing Torsades de Pointes

Sponsors & Collaborators

• NanoPharmaceuticals LLC: lead

Study Sites (1)

Smilow Cancer Hospital

New Haven, Connecticut, 06511, United States

RECRUITING

MeSH Terms

Conditions

Glioma; Glioblastoma

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Astrocytoma

Study Officials

• Nicholas Blondin, MD

  Yale University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Amy L Rodrigues, CCRC

CONTACT

(203) 260-9632; amy.rodrigues@yale.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 3+3 Dose Escalation with option for cohort expansion

Study Record Dates

• First Submitted: December 3, 2021
• First Posted: February 7, 2022
• Study Start: June 23, 2022
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): October 1, 2027
• Last Updated: June 4, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)