Safety and toxicity of Iopofosine I 131 (CLR 131) with external beam radiation therapy in recurrent or metastatic head and neck cancer: results of a phase 1 single-centre, open-label, single-arm, dose escalation and dose expansion study

EBioMedicine. 2025 Jan:111:105496. doi: 10.1016/j.ebiom.2024.105496. Epub 2024 Dec 12.

Abstract

Background: Re-irradiation of recurrent head and neck cancer (HNC) is often limited by tumour adherence to critical structures and/or radiation tolerance of critical normal tissues. Iopofosine I 131 (CLR 131) is a targeted small molecular phospholipid ether (PLE) drug conjugate that delivers iodine-131 selectively to tumour cells. We conducted a phase 1, single-centre, open-label study to determine whether CLR 131 given with reduced dose of external beam radiation therapy (EBRT) would be tolerable and feasible.

Methods: All participants received previous curative intent treatment with radiotherapy as primary or adjuvant treatment. Eligible participants demonstrated uptake of CLR 131 as indicated via single photon emission CT/CT (SPECT/CT) imaging following CLR 131 test dose. Participants received two therapeutic doses of CLR 131 (days 1 and 8) with SPECT/CT imaging performed to quantitate the biodistribution of CLR 131. Participants subsequently received EBRT to achieve the designated radiation dose (60-70 Gy). The primary endpoint was safety. This trial was registered with ClinicalTrials.gov, NCT04105543, and enrolment and follow-up are complete.

Findings: Twelve participants completed treatment with CLR 131 and EBRT. Eight participants experienced grade 4 non-DLT haematologic toxicities (2 anaemia, 8 leukopenia, 5 thrombocytopenia) at least probably attributed to CLR 131, consistent with the expected toxicity profile. Haematologic toxicities occurred during weeks 6-8 from the first dose of CLR 131 and resolved within three weeks without sequelae. There were no treatment-related grade 3-4 non-haematologic toxicities.

Interpretation: CLR 131 in combination with EBRT did not confer any safety concerns, and was tolerable in participants with recurrent/metastatic HNC. Myelosuppression was consistent with the known toxicity profile of CLR 131.

Funding: National Institutes of HealthP50 DE026787, National Cancer InstituteP30 CA014520, National Institutes of Health1UL1TR002373, Cellectar, NCT04105543.

Keywords: Head and neck cancer; Monte Carlo method; Radiopharmaceutical; Re-irradiation; Targeted radionuclide therapy.

Publication types

  • Clinical Trial, Phase I

MeSH terms

  • Adult
  • Aged
  • Female
  • Head and Neck Neoplasms* / pathology
  • Head and Neck Neoplasms* / radiotherapy
  • Humans
  • Iodine Radioisotopes* / administration & dosage
  • Iodine Radioisotopes* / adverse effects
  • Iodine Radioisotopes* / therapeutic use
  • Male
  • Middle Aged
  • Neoplasm Metastasis
  • Neoplasm Recurrence, Local* / radiotherapy
  • Radiotherapy Dosage
  • Tissue Distribution
  • Treatment Outcome

Substances

  • Iodine Radioisotopes
  • Iodine-131

Associated data

  • ClinicalTrials.gov/NCT04105543