Pasireotide as Maintenance Treatment with monthly deep intramuscular injection in SSTR2/3/5-Expressing Synovial Sarcoma and Desmoplastic Small Round Cell Tumor (PAMSARC)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Universitaetsklinikum Heidelberg AöR

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

18 Dec 2024 → ongoing

Decision date (initial)

2024-11-12

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2024-511935-86-00

ClinicalTrials.gov

NCT06456359

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy

To assess the clinical efficacy of pasireotide maintenance therapy for prolonging progression-free survival (PFS) in patients with SSTR2/3/5-expressing synovial sarcoma (SySa) and desmoplastic small round cell tumor (DSRCT)

Secondary objectives 1

1. To assess the clinical efficacy of pasireotide maintenance therapy for prolonging overall survival (OS) in patients with SSTR2/3/5-expressing SySa and DSRCT

Conditions and MedDRA coding

Locally advanced or metastatic synovial sarcoma ICD-O3, 9040/3, 9041/3, 9042/3, 9043/3 Locally advanced or metastatic desmoplastic small round cell tumor ICD-O3, 8806/3

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Reference pathological proven diagnosis of DSRCT in any stage; or Reference pathological proven diagnosis of SySa, IRS III, metastatic or relapsed disease
2. High SSTR2/3/5 mRNA expression, as determined by RNA sequencing within DKFZ/NCT/DKTK MASTER program
3. Stable disease, partial or complete response after completion of standard treatment
4. Age from 13 to 50 years
5. Bodyweight ≥ 30 kg
6. Lansky-Index ≥ 80% in patients < 16 years of age and Karnofsky-Index ≥ 80% in patients ≥ 16 years of age (including adults).
7. No curative treatment option
8. Time from last chemotherapy (at least 2 chemotherapy cycles) to enrollment <8 weeks
9. 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
10. Female patients of childbearing potential and male patients with partners of childbearing potential who are sexually active must agree to the use of two forms of contraception in combination (male condom and one highly effective method). These should be started immediately after signing the informed consent form and continued throughout the period of study treatment plus 3 months for female and male patients. Male patients should refrain from fathering a child or donating sperm during the trial and for at least 3 months following the last dose.
11. Adequate bone marrow, renal, and hepatic function defined by laboratory tests within 14 days prior to study treatment (for details refer to protocol)

Exclusion criteria 11

1. 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
2. Concurrent or previous treatment within 30 days in another interventional clinical trial / Participation in other ongoing clinical trials
3. Uncontrolled concurrent disease, in particular diabetes mellitus
4. Bleeding disorder
5. Therapeutic anticoagulation which cannot be paused temporarily in order to ensure safe intramuscular injection
6. Is taking or requiring any of the prohibited medication listed in 6.4.2
7. Heart rate at rest < 60/min
8. fasting glucose level > 110mg/dl
9. Severe neurologic or psychiatric disorder
10. Pregnancy/lactation
11. Prior treatment with somatostatin analog

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Progression free survival (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 (censoring of patients without an event at date of last follow-up)

Secondary endpoints 1

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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Universitaetsklinikum Heidelberg AöR

Sponsor organisation

Universitaetsklinikum Heidelberg AöR

Address

Im Neuenheimer Feld 672, Neuenheim Neuenheim

City

Heidelberg

Postcode

69120

Country

Germany

Scientific contact point

Organisation

Universitaetsklinikum Heidelberg AöR

Contact name

Coordinating Investigator

Public contact point

Organisation

Universitaetsklinikum Heidelberg AöR

Contact name

Projectmanagement

Locations

1 EU/EEA country · 4 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 19 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications