Ignite Creation Date:
2024-06-16 @ 11:51 AM
Last Modification Date:
2025-12-17 @ 11:41 AM
Study NCT ID:
NCT06456359
Status:
None
Last Update Posted:
2025-01-13 00:00:00
First Post:
2024-05-22 00:00:00
Brief Title:
Pasireotide as Maintenance Treatment in Synovial Sarcoma and Desmoplastic Small Round Cell Tumor
Sponsor:
University Hospital Heidelberg
Organization:
University Hospital Heidelberg
Study Overview
Official Title:
Pasireotide as Maintenance Treatment With Monthly Deep Intramuscular Injection in SSTR2/3/5-Expressing Synovial Sarcoma and Desmoplastic Small Round Cell Tumor
Status:
None
Status Verified Date:
2025-01
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:
PAMSARC
Brief Summary:
Desmoplastic small round cell tumor (DSRCT) is an extremely rare, aggressive sarcoma. It originates from the serosal surface of the abdominal cavity and the hallmark characteristic of DSRCT is the EWSR1-WT1 gene fusion. Synovial sarcoma (SySa) is also a rare fusion-gene driven (SS18-SSX1, SS18-SSX2, or rarely, SS18-SSX4) soft-tissue sarcoma.
Selected somatostatin receptor (SSTR) family members, i.e., SSTR2, SSTR3 and SSTR5, were highly expressed in patients with available transcriptome data, providing the basis for treatment with a somatostatin analog such as pasireotide with high affinity for SSTR1, 2, 3, and 5.
The primary aim of the study is to assess the clinical efficacy of pasireotide maintenance therapy for prolonging progression-free (PFS) and overall survival (OS) in patients with SSTR2/3/5-expressing advanced SySa and DSRCT. Furthermore measurable residual disease (MRD) before, during, and after pasireotide maintenance therapy are assessed. Pasireotide is applied in adults with 60 mg and in adolescents 60 mg (body surface area \[BSA\] \>1.6 m²) or 40 mg (BSA 1.1-1.6 m²) via intragluteal via intragluteal depot injection every 28±3 days. The sample size is planned for the entire study population with subsequent sensitivity analysis in two subgroups, i.e., adolescents and adults. The primary efficacy analysis is be based on a two-sided, one-sample log-rank test using a significance level of 5%. The sample size was calculated assuming exponential data, planning for a power of 90% to detect a hazard ratio of 0.5. With a sample size of n=28, the expected number of events during the study is 22. Safety is assessed continuously according to CTCAE v5.0. The recruitment period is planned for 2 years starting in 2024 followed by a minimal follow-up of the last patient of 6 months leading to estimated trial completion in 2027.
Selected somatostatin receptor (SSTR) family members, i.e., SSTR2, SSTR3 and SSTR5, were highly expressed in patients with available transcriptome data, providing the basis for treatment with a somatostatin analog such as pasireotide with high affinity for SSTR1, 2, 3, and 5.
The primary aim of the study is to assess the clinical efficacy of pasireotide maintenance therapy for prolonging progression-free (PFS) and overall survival (OS) in patients with SSTR2/3/5-expressing advanced SySa and DSRCT. Furthermore measurable residual disease (MRD) before, during, and after pasireotide maintenance therapy are assessed. Pasireotide is applied in adults with 60 mg and in adolescents 60 mg (body surface area \[BSA\] \>1.6 m²) or 40 mg (BSA 1.1-1.6 m²) via intragluteal via intragluteal depot injection every 28±3 days. The sample size is planned for the entire study population with subsequent sensitivity analysis in two subgroups, i.e., adolescents and adults. The primary efficacy analysis is be based on a two-sided, one-sample log-rank test using a significance level of 5%. The sample size was calculated assuming exponential data, planning for a power of 90% to detect a hazard ratio of 0.5. With a sample size of n=28, the expected number of events during the study is 22. Safety is assessed continuously according to CTCAE v5.0. The recruitment period is planned for 2 years starting in 2024 followed by a minimal follow-up of the last patient of 6 months leading to estimated trial completion in 2027.
Detailed Description:
Desmoplastic small round cell tumor DSRCT is an extremely rare aggressive sarcoma It originates from the serosal surface of the abdominal cavity and the hallmark characteristic of DSRCT is the EWSR1-WT1 gene fusion Synovial sarcoma SySa is also a rare fusion-gene driven SS18-SSX1 SS18-SSX2 or rarely SS18-SSX4 soft-tissue sarcoma
Selected somatostatin receptor SSTR family members ie SSTR2 SSTR3 and SSTR5 were highly expressed in patients with available transcriptome data providing the basis for treatment with a somatostatin analog such as pasireotide with high affinity for SSTR1 2 3 and 5
The primary aim of the study is to assess the clinical efficacy of pasireotide maintenance therapy for prolonging progression-free PFS and overall survival OS in patients with SSTR235-expressing advanced SySa and DSRCT Furthermore measurable residual disease MRD before during and after pasireotide maintenance therapy are assessed Pasireotide is applied in adults with 60 mg and in adolescents 60 mg body surface area BSA 16 m² or 40 mg BSA 11-16 m² via intragluteal via intragluteal depot injection every 283 days The sample size is planned for the entire study population with subsequent sensitivity analysis in two subgroups ie adolescents and adults The primary efficacy analysis is be based on a two-sided one-sample log-rank test using a significance level of 5 The sample size was calculated assuming exponential data planning for a power of 90 to detect a hazard ratio of 05 With a sample size of n28 the expected number of events during the study is 22 Safety is assessed continuously according to CTCAE v50 The recruitment period is planned for 2 years starting in 2024 followed by a minimal follow-up of the last patient of 6 months leading to estimated trial completion in 2027
Selected somatostatin receptor SSTR family members ie SSTR2 SSTR3 and SSTR5 were highly expressed in patients with available transcriptome data providing the basis for treatment with a somatostatin analog such as pasireotide with high affinity for SSTR1 2 3 and 5
The primary aim of the study is to assess the clinical efficacy of pasireotide maintenance therapy for prolonging progression-free PFS and overall survival OS in patients with SSTR235-expressing advanced SySa and DSRCT Furthermore measurable residual disease MRD before during and after pasireotide maintenance therapy are assessed Pasireotide is applied in adults with 60 mg and in adolescents 60 mg body surface area BSA 16 m² or 40 mg BSA 11-16 m² via intragluteal via intragluteal depot injection every 283 days The sample size is planned for the entire study population with subsequent sensitivity analysis in two subgroups ie adolescents and adults The primary efficacy analysis is be based on a two-sided one-sample log-rank test using a significance level of 5 The sample size was calculated assuming exponential data planning for a power of 90 to detect a hazard ratio of 05 With a sample size of n28 the expected number of events during the study is 22 Safety is assessed continuously according to CTCAE v50 The recruitment period is planned for 2 years starting in 2024 followed by a minimal follow-up of the last patient of 6 months leading to estimated trial completion in 2027
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