Adult Granulosa Cell Tumor of the OvaryNCT06254781
Luspatercept in Metastatic AGCT of the Ovary
A study testing Luspatercept in people with Adult Granulosa Cell Tumor of the Ovary, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
This is a single participant study of luspatercept for the treatment of a patient with dult granulosa cell tumor (AGCT) of the ovary. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Adult Granulosa Cell Tumor of the Ovary
- What is being tested?
- Luspatercept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteCanada
- Study dates
- 30 May 2022to 29 Aug 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- N/A
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Luspatercept in Metastatic Adult Granulosa Cell Tumor (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.
Read the detailed description
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.
- Conditions
- Adult Granulosa Cell Tumor of the Ovary
- Being tested
- Luspatercept
- Medical subject headings
- Granulosa cell tumor of the ovary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LuspaterceptDrug
Also called Reblozyl
Erythroid Maturation Agent
| Study group | Role | What they receive |
|---|---|---|
| Luspatercept in metastatic AGCT of the ovary | Experimental | Luspatercept, 1.0 mg/kg, subcutaneously, every three weeks Receives: Drug: Luspatercept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response
By computed tomography (CT) scans
Measured: 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.
Eastern Cooperative Oncology Group (ECOG) score
- 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
Measured: 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.
Circulating tumor DNA (ctDNA) levels
Observe change between baseline ctDNA until disease progression
Measured: 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..
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2024
- Study start30 May 2022
- Primary completion22 Aug 2022
- Study completion29 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adult Granulosa Cell Tumor of the OvaryStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Amit Oza, M.D., Principal investigator, Princess Margaret Cancer Centre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06254781
- Sponsor's protocol ID
- OZUHN-015
- Other IDs
- 22-5326
- Status last verified
- Feb 2024
- First submitted
- 26 May 2022
- Last updated
- 12 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Luspatercept in Metastatic Adult Granulosa Cell Tumor (AGCT) of the Ovary
- ClinicalTrials.gov ID
- NCT06254781
- Acronym / sponsor ID
- OZUHN-015
- Conditions
- Adult Granulosa Cell Tumor of the Ovary
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 1 participants (actual)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Study start
- 30 May 2022 (actual)
- Primary completion
- 22 Aug 2022 (actual)
- Study completion
- 29 Aug 2022 (actual)
- First posted
- 12 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single participant study of luspatercept for the treatment of a patient with dult granulosa cell tumor (AGCT) of the ovary.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Luspatercept in metastatic AGCT of the ovary | Experimental | Drug: LuspaterceptLuspatercept, 1.0 mg/kg, subcutaneously, every three weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- N/A
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LuspaterceptAlso: ReblozylErythroid Maturation Agent | Drug | Luspatercept in metastatic AGCT of the ovary |
Primary outcome
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
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.
0. Fully active, able to carry on all pre-disease performance without restriction 1. 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 2. Ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours 3. Capable of only limited selfcare; confined to bed or chair more than 50% of waking hours 4. Completely disabled; cannot carry on any selfcare; totally confined to bed or chair 5. Death
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2024.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Amit Oza, M.D. principal investigator
Princess Margaret Cancer Centre
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Luspatercept in people with Adult Granulosa Cell Tumor of the Ovary, run by University Health Network, Toronto at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Feb 2024; AzTrials last refreshed its copy on 8 Nov 2026.