Pasireotide as Maintenance Treatment in Synovial Sarcoma and Desmoplastic Small Round Cell Tumor

NCT06456359 | Recruiting Phase 2 DMC

• 1 other identifier: PAMSARC
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 4

Brief Summary

PAMSARC is a non-commercial interventional Phase 2 clinical trial of academic research institutions, with its primary goal being to improve medical treatment of fusion driven Desmoplastic small round cell tumor (DSRCT) and Synovial sarcoma (SySa) in young adults and adolsecents with male predominance. Current management of DSRCT and SySa includes chemotherapy, radiation and aggressive cytoreductive surgery. Despite advances in multimodal therapy, outcomes remain poor with frequent disease recurrence and very limited options for patients with advanced disease. Selected somatostatin receptor (SSTR) family members, i.e., SSTR2, SSTR3 and SSTR5, are frequently overexpressed in DSRCT and SySa, providing the rationale for treatment with somatostatin analogues (SSA). Pasireotide is a SSA with high affinity for SSTR1, -2, -3, and -5 and is approved for the treatment of Cushing's disease and acromegaly and has also shown activity in other cancers. In patients with advanced stage DSRCT and SySa, conventional chemotherapeutic approaches frequently lead to disease response, however, the duration of progression-free time after chemotherapy is short. The targeted approach with pasireotide after initial intensive multimodal treatment may have the potential to significantly improve outcome.

Conditions

Desmoplastic Small Round Cell Tumor; Synovial Sarcoma

Keywords

SSTR; somatostatin receptor; SSTR2, SSTR3, SSTR5; somatostatin analog; pasireotide; fusion-gene driven; SS18-SSX1, SS18-SSX2, SS18-SSX4; EWSR1-WT1; extremely rare, aggressive sarcoma; maintaince therapy

Outcome Measures

Primary Outcomes (1)

• progression-free survival (PFS)

  Assessment of clinical efficacy of pasireotide maintenance therapy

  PFS, measured from study registration to radiologically confirmed disease progression according to Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1, or death from any cause, whichever occurs first up to 4 years

Secondary Outcomes (1)

• overall survival (OS)

  OS, measured from study registration to death from any cause (censoring of patients without an event at date of last follow-up) up to 4 years

Other Outcomes (1)

• measurable residual disease (MRD)

  MRD before, during, and after pasireotide maintenance therapy up to 4 years

Study Arms (1)

Pasireotid treatment single arm

EXPERIMENTAL

Adults receive 60 mg via intragluteal depot injection every 28±3 days, adolescents 60 mg (body surface area \[BSA\] \>1.6 m²) or 40 mg (BSA 1.1-1.6 m2) via intragluteal depot injection every 28±3 days.

Drug: Signifor

Interventions

Signifor DRUG

Signifor 40 mg: each vial contains 40 mg pasireotide (as pasireotide pamoate). Signifor 60 mg: each vial contains 60 mg pasireotide (as pasireotide pamoate).

Also known as: ATC-Code: H01CB05

Pasireotid treatment single arm

Eligibility Criteria

• Age: 13 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients meeting all of the following criteria are considered for enrolment into the trial:
• Reference pathological proven diagnosis of DSRCT in any stage; or Reference pathological proven diagnosis of SySa, IRS III, metastatic or relapsed disease
• High SSTR2/3/5 mRNA expression, as determined by RNA sequencing in the DKFZ/NCT/DKTK MASTER (ClinicalTrials.gov ID: NCT05852522) or INFORM programs.
• Stable disease, partial or complete response after completion of standard treatment
• Age from 13 to 50 years
• For patients (≥16 years): Karnofsky-Index ≥ 80% For patients (\<16 years): Lansky-Index ≥ 80%
• No curative treatment option
• Bodyweight ≥ 30kg and BSA ≥ 1.1m²
• Time from last chemotherapy (at least 2 chemotherapy cycles) to enrollment \<8 weeks
• Ability of patient to understand character and individual consequences of the clinical trial
• Written informed consent (for individuals \<18 years of age an ICF for adolescents and their parents is needed)
• For women of childbearing potential negative urine pregnancy test at screening as well as highly effective forms of contraception have to be in place thereafter
• Evidence of childbearing potential is defined as fertile, following menarche and until becoming post-menopausal unless permanently sterile
• Postmenopausal or evidence of non-childbearing status is defined as:
• Amenorrhea for 1 year or more without an alternative medical cause following cessation of exogenous hormonal treatments plus follicle stimulating hormone (FSH) levels in the postmenopausal range in women not using hormonal contraception or hormonal replacement therapy.

You may not qualify if:

• Patients presenting with any of the following criteria are not included in the trial:
• History of hypersensitivity to the investigational medicinal product or to any drug with similar chemical structure or to any excipient present in the pharmaceutical form of the investigational medicinal product
• Concurrent or previous treatment within 30 days in another interventional clinical trial / Participation in other ongoing clinical trials.
• Uncontrolled concurrent disease, in particular diabetes mellitus
• Bleeding disorder
• Therapeutic anticoagulation which cannot be paused temporarily in order to ensure safe intramuscular injection
• Is taking or requiring any of the prohibited medication listed in Table 5 (6.4.2)
• Heart rate at rest \< 60/min
• fasting glucose level \> 110mg/dl
• Severe neurologic or psychiatric disorder
• Pregnancy/lactation
• Prior treatment with somatostatin analog

Sponsors & Collaborators

• University Hospital Heidelberg: lead

Study Sites (4)

National Center for Tumour Diseases, University Hospital Heidelberg

Heidelberg, Baden-Wurttemberg, 69120, Germany

RECRUITING

Klinikum Stuttgart- Olga Hospital Zentrum für Kinder-, Jugend und Frauenmedizin

Stuttgart, Baden-Wurttemberg, 70174, Germany

NOT YET RECRUITING

Klinikum Stuttgart Studienzentrale Stuttgart Cancer Center, Tumorzentrum Eva-Mayr-Stihl

Stuttgart, Baden-Wurttemberg, 70175, Germany

NOT YET RECRUITING

Universitätsklinikum Essen Pädiatrische Hämatologie und Onkologie

Essen, North Rhine-Westphalia, 45147, Germany

NOT YET RECRUITING

Related Publications (1)

• Heilig CE, Heining C, Gnutzmann E, Roldan S, Heiligenthal L, Sparber-Sauer M, Hahn D, Dirksen U, Hamacher R, Florcken A, Thorwarth A, Deinzer CKW, Gaidzik VI, Pfaff E, Hubschmann D, Arndt K, Pfister SM, Glimm H, Frohling S, Schlenk RF. Rationale and design of the PAMSARC (pasireotide as maintenance treatment with monthly deep intramuscular injection in SSTR2/3/5-expressing synovial sarcoma and desmoplastic small round cell tumor) multicenter phase 2 trial. Cancer Treat Res Commun. 2025;45:100986. doi: 10.1016/j.ctarc.2025.100986. Epub 2025 Sep 3.

  PMID: 40934647 DERIVED

MeSH Terms

Conditions

Desmoplastic Small Round Cell Tumor; Sarcoma, Synovial

Interventions

pasireotide

Condition Hierarchy (Ancestors)

Sarcoma; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Connective Tissue

Study Officials

• Richard F. Schlenk, Professor

  National Center for Tumour Diseases, University Hospital Heidelberg

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Richard F. Schlenk, Professor

CONTACT

+49 (0)6221 56 6228; richard.schlenk@nct-heidelberg.de

Editha Gnutzmann, M.A.

CONTACT

+49 6221 36235; e.gnutzmann@nct-heidelberg.de

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. Dr. med. Richard F. Schlenk (Coordinating Investigator)

Model Details: This is a single arm prospective, open label, multi-centre phase II study of pasireoide maintenance therapy in patients with advanced/metastatic DSRCT and SySa. According to the staggered approach during a run-in phase feasibility is assessed in 3 (or, if necessary, more) adult patients before initiating recruitment in adolescents. Adults receive 60 mg via intragluteal depot injection every 28±3 days, adolescents 60 mg (body surface area \[BSA\] \>1.6 m²) or 40 mg (BSA 1.1-1.6 m2) via intragluteal depot injection every 28±3 days.

Study Record Dates

• First Submitted: May 22, 2024
• First Posted: June 13, 2024
• Study Start: December 19, 2024
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2028
• Last Updated: January 13, 2025

Record last verified: 2025-01

Locations

DE (4)