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Completed

NCT Number: NCT06254781

Luspatercept in Metastatic AGCT of the Ovary

This is a single participant study of luspatercept for the treatment of a patient with dult granulosa cell tumor (AGCT) of the ovary.

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Key information

Conditions

Adult Granulosa Cell Tumor of the Ovary Granulosa cell tumor of the ovary

Age range

18 year and older

Sex eligibility

Female

Study type

Interventional

Phase

Not applicable

Primary location

Princess Margaret Cancer Centre

Toronto, Ontario, M5G 2M9, Canada

About this study

This patient has AGCT driven by a somatic oncogenic FOXL2 mutation identified through a molecular profiling study. One of the ways in which this mutation drives tumorigenesis is via increased activity of SMAD3, which results in decreased expression of follistatin and allows increased signalling of activin, a TGFB ligand that activates the TGFB pathway. Anti-TGFB approaches have shown promise in preclinical studies of AGCTs and several early phase trials are investigating different levels of TGFB pathway inhibition1.

As an activin receptor ligand trap, luspatercept has been shown to reduce SMAD2/3 signaling and improve anemia in disorders characterized by ineffective erythropoiesis, such as B-thalassemia and myelodysplastic syndromes (MDSs). This study aims to determine if we can apply this rationale to a patient with recurrent AGCT with oncogenic FOXL2 mutation known to drive TGFB/SMAD pathway overactivity.

Luspatercept has not been investigated in AGCT and therefore the efficacy of this specific agent as a cancer therapeutic is not yet known. Other TFGB ligand-trapping agents are in development and early phase clinical trials. One such example is AVID200, a TGFB ligand trap and selective inhibitor of TGFB1 and advanced solid tumors. There were 19 patients included in a phase I study of AVID200 monotherapy in patients with advanced solid tumors. A best response of stable disease for over 12 weeks seen in two patients (adenoid cystic carcinoma and breast carcinoma). The maximum tolerated dose was not reached; no Grade 3 adverse events were seen and only three adverse events were reported overall including diarrhea and lipase elevation.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • N/A

Exclusion criteria

  • N/A

This patient's case was recently discussed in the hospital expert molecular tumor board rounds consisting of representatives from specialist genomic profiling and gynecologic medical oncology departments. The discussion surrounded the best next therapeutic option in this rare cancer subtype without clear standard of care guidelines. The recommendation from this discussion was to investigate targets of the TGFβ pathway, such as luspatercept. Therefore, specific eligibility criteria aside from individual patient's medical history is not applicable.

Treatment and study plan

Luspatercept

Drug

Erythroid Maturation Agent

Other names: Reblozyl

Primary outcomes

  1. Objective response

    Time frame: From baseline CT until disease progression or until the investigator deems that it is in the patient's best interest to stop study treatment, whichever came first, assessed up to 3 months.

    By computed tomography (CT) scans

  2. Eastern Cooperative Oncology Group (ECOG) score

    Time frame: From baseline ECOG until disease progression or until the investigator deems that it is in the patient's best interest to stop study treatment, whichever came first, assessed up to 3 months.

    • Fully active, able to carry on all pre-disease performance without restriction
    • Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work
    • Ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
    • Capable of only limited selfcare; confined to bed or chair more than 50% of waking hours
    • Completely disabled; cannot carry on any selfcare; totally confined to bed or chair
    • Death

  3. Circulating tumor DNA (ctDNA) levels

    Time frame: From baseline ctDNA until disease progression or until the investigator deems that it is in the patient's best interest to stop study treatment whichever came first, assessed up to 3 months..

    Observe change between baseline ctDNA until disease progression

Sponsors and collaborators

Lead sponsor

University Health Network, Toronto

Other

Registry information

Official study title

Luspatercept in Metastatic Adult Granulosa Cell Tumor (AGCT) of the Ovary

Important dates

Study start
2022
Primary completion
2022
Study completion
2022
First posted
Feb 12, 2024
Registry last updated
Feb 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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