Ignite Creation Date:
2024-05-06 @ 8:06 PM
Last Modification Date:
2024-10-26 @ 3:20 PM
Study NCT ID:
NCT06254781
Status:
COMPLETED
Last Update Posted:
2024-02-12
First Post:
2022-05-26
Brief Title:
Luspatercept in Metastatic AGCT of the Ovary
Sponsor:
University Health Network Toronto
Organization:
University Health Network Toronto
Study Overview
Official Title:
Luspatercept in Metastatic Adult Granulosa Cell Tumor AGCT of the Ovary
Status:
COMPLETED
Status Verified Date:
2024-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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 SMAD23 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 TGFBSMAD 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
As an activin receptor ligand trap luspatercept has been shown to reduce SMAD23 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 TGFBSMAD 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 22-5326 | OTHER | University Health Network | None |