PAMSARC study
Overview
- The PAMSARC study is recruiting patients with desmoplastic small and round cell tumours or synovial sarcoma.
- The study is investigating the drug pasireotide: it is intended to support treatment success as a long-term (maintenance) therapy following chemotherapy.
- The aim of the PAMSARC study is to determine whether the drug can slow the progression of the disease or further shrink the tumour.
Study status: The study is currently recruiting patients (recruitment open).
Desmoplastic small and round cell tumours (DSRCT) and synovial sarcoma (SySa) are rare, aggressive tumours found in muscle, fat or connective tissue. They mostly affect children, adolescents or young adults. As part of standard treatment, patients usually receive chemotherapy. This is intended to slow the growth of the tumour or reduce its size. However, the disease usually progresses again after chemotherapy has finished.
Pasireotide (trade name Signifor®) is a medicine that acts in a similar way to one of the body’s own hormones, somatostatin. This hormone can slow down cell growth by binding to specific binding sites on the cell surface known as somatostatin receptors. The drug is provided by the pharmaceutical company RECORDATI, the manufacturer of the investigational drug pasireotide (Signifor®).
Previous studies have shown that cells of DSRCT and synovial sarcoma carry a particularly high number of these somatostatin receptors on their surface. Because there are so many binding sites, scientists believe that pasireotide can inhibit the growth of tumour cells. Researchers in the PAMSARC study are therefore investigating whether pasireotide can support the therapeutic success of chemotherapy.
Detailed information: How does pasireotide work?
Pasireotide is an active substance that has the same structure as the hormone somatostatin. In the body, somatostatin is responsible for maintaining the balance of various processes. Somatostatin generally has an inhibitory effect.
When somatostatin binds to binding sites on the cell surface, it can slow down cell growth. In previous studies, researchers have shown that tumour cells from DSRCT or synovial sarcoma often carry many of these binding sites (also known as somatostatin receptors) on their cell surface.
Based on this property, the scientists assume that tumour cells with many somatostatin receptors are more sensitive to the drug than healthy body cells. It is therefore believed that pasireotide may be able to slow down the growth of tumour cells.
Pasireotide is already approved for other conditions, but not yet for the treatment of DSRCT and synovial sarcoma. Experts refer to this as off-label use.
Which types of cancer are covered by PAMSARC?
The PAMSARC study treats patients with certain soft tissue tumours, namely desmoplastic small and round cell tumours (DSRCT) or synovial sarcoma.
Participants must have received and completed chemotherapy.
What is the aim of the PAMSARC study?
The full title of the PAMSARC study is: Pasireotide as Maintenance Treatment with monthly deep intramuscular injection in SSTR2/3/5-Expressing Synovial Sarcoma and Desmoplastic Small Round Cell Tumour.
In other words:
- The drug pasireotide is being tested as maintenance therapy.
- This study involves patients with desmoplastic small round cell tumours (DSRCT) or synovial sarcoma.
- Their tumour cells have many binding sites (somatostatin receptors) on their surface.
- Pasireotide is injected into the muscle every 4 weeks.
You can read more about this in the next section on the objectives of the PAMSARC study.
Objectives of the PAMSARC study
The PAMSARC study aims to investigate whether, and to what extent, the drug pasireotide can inhibit tumour growth following chemotherapy in patients with DSRCT or synovial sarcoma.
To this end, researchers aim to determine:
- Whether patients treated with pasireotide live longer overall.
- Whether the drug pasireotide can prevent the cancer from progressing further over a longer period of time.
- How well the drug is tolerated and whether any adverse reactions occur during pasireotide treatment.
- How many cancer cells are still detectable in the body – before, during and after pasireotide treatment.
- Whether the cancer cells develop resistance to the treatment.
- How participants’ quality of life develops before, during and after treatment. This includes, for example, sleep quality, psychological stress or anxiety.
To be eligible to participate in the PAMSARC study, there are criteria that all patients must meet equally. It does not matter whether they have a desmoplastic small and round cell tumour (DSRCT) or a synovial sarcoma. In addition, there are eligibility criteria that apply specifically to DSRCT or synovial sarcomas.
The criteria that ALL participants must meet include:
- The tumour cells have a high number of binding sites (somatostatin receptors) on the cell surface. This is verified by molecular genetic testing of the tissue within the master’s program.
- The disease has remained stable or improved following chemotherapy.
- Patients are aged between 13 and 50.
- Participants weigh more than 30 kilograms.
- The participants’ state of health is good enough for them to manage their daily lives independently and with only minor limitations.
- There are no remaining treatments that could potentially cure the cancer.
- Less than 8 weeks have passed since the last course of chemotherapy. The chemotherapy must have consisted of at least 2 cycles.
- The participant has understood what will happen during the study and the potential consequences of taking part.
- Patients have signed a written consent form. If the participant is under 18 years of age, a signed consent form from their legal guardian is available.
- Women of childbearing age must provide proof that they are not pregnant by means of a pregnancy test.
- People who are sexually active must undertake to use two highly effective methods of contraception simultaneously during the study. Doctors will provide information on suitable methods of contraception.
- Men taking part in the study must agree not to donate sperm during the study and for the following 3 months.
- Certain blood count and laboratory test results (such as kidney and liver function tests) must be sufficiently good.
In addition, the following applies to patients with DSCRT:
A tissue sample has confirmed the diagnosis of DSCRT.
For patients with synovial sarcoma: A tissue examination has confirmed the diagnosis of synovial sarcoma. In addition, one of the following situations must apply:
- The tumour has already been operated on and could not be completely removed.
- The synovial sarcoma has formed a metastasis.
- The disease has returned (relapse).
Patients cannot take part in the study if any of the following apply:
- The patient has had an allergic reaction to the drug or its components in the past.
- The participant has taken part in another clinical trial involving the testing of medicines within the last 30 days.
- The patient has a comorbidity that is untreated or poorly managed, such as diabetes.
- The participant has an increased tendency to bleed.
- The person is taking blood-thinning medication that cannot be paused. In this case, the injection of pasireotide into the muscle tissue would carry too high a risk of bleeding.
- Patients are taking medicines that have unacceptable interactions with pasireotide. For this reason, it is very important to tell the study doctor about all the medicines and preparations you are taking in addition to this. This includes over-the-counter medicines.
- Your resting heart rate is very low (below 60 beats per minute).
- Blood sugar is elevated (above 110 mg/dL) without having eaten or drunk anything beforehand.
- Participants have a severe neurological condition or a mental health condition.
- The person is pregnant or breastfeeding.
- Patients have already received a medication that works in a similar way to somatostatin.
The PAMSARC study is an interventional, single-arm phase II clinical trial:
- Single-arm study: All participants receive treatment with pasireotide (there is no control group).
- Phase II trial: Researchers are testing how pasireotide affects patients with DSRCT or synovial sarcoma.
- Intervention study: Participants are not merely observed but also receive treatment with pasireotide (“intervention”).
(1) Information session and informed consent
Once the patient has registered and is eligible to participate in the PAMSARC study, a detailed information session takes place with the study doctor at the relevant clinic. All patients must sign a consent form before any examinations for the study take place.
In the case of patients under the age of 18, their legal guardians must sign a consent form.
(2) Preliminary examinations to assess suitability
To determine whether patients are suitable for participation in the PAMSARC study, study doctors and healthcare professionals carry out several tests:
- They measure vital signs such as heart rate, blood pressure, body temperature, respiratory rate and oxygen saturation.
- They palpate and auscultate the body.
- Patients are weighed and their height is measured.
- They check whether the patient’s heart is functioning properly. To do this, they perform an electrocardiogram (recording of the heart rhythm) and an echocardiogram (ultrasound of the heart, short: ECG).
- They record information about any impairments (performance status according to Lansky or Karnofsky Performance Status).
- Blood is taken from participants, and they must provide a urine sample. The blood sample is tested for infectious diseases such as HIV or hepatitis, amongst other things.
- A pregnancy test is carried out for women of childbearing age.
- A tissue sample of the tumour is required for molecular genetic testing. Doctors can obtain this via a biopsy or during an operation. Using this tissue sample, genetic testing can determine whether the tumour cells carry an increased number of somatostatin receptors on their surface.
- Images of the tumour (and any possible metastases) are taken using computed tomography, CT or magnetic resonance imaging, MRI.
- Blood and tissue samples are stored for potential use in future studies.
In addition, the study team records further information. This includes:
- Age, gender and ethnicity.
- Details of current and past medical history.
- Information on medicines or treatments that patients are currently taking or have recently taken. Doctors use this to identify any potential interactions with previous medicines or treatments.
Using the data collected, the treating doctors can decide whether patients are suitable for the PAMSARC study.
(3) Treatment with pasireotide
Treatment with pasireotide takes place once a month at the study centre.
A doctor examines participants thoroughly before each pasireotide injection. This includes a consultation (symptoms, side effects, use of other medicines), a physical examination, blood tests, an ECG and for female patients a pregnancy test. Trained medical staff then inject the medicine into the gluteal muscle. If desired, the injection site can be locally anaesthetised beforehand to make the injection less uncomfortable.
The condition is monitored during treatment using imaging techniques such as MRI and CT scans. These scans are carried out after 8 and 16 weeks, and then every three months, or if there is any suspicion that the condition is progressing.
Study participants should allow between 3 and 6 hours for their monthly visit to the study centre.
At the time of the initial examination, as well as in weeks 8 and 16, and every 3 months thereafter, patients are asked to complete a digital questionnaire to assess their quality of life. This includes questions on, for example, pain, fatigue and psychological distress. Completing the questionnaires is voluntary.
(4) End of treatment
Treatment with pasireotide will be administered for a maximum of 36 months. The duration of a patient’s treatment within the PAMSARC study depends on their state of health and the progression of their condition.
Upon completion of the study treatment with pasireotide, the same examinations will be carried out as during the treatment appointments. The final appointment will take place within 7 days of the end of treatment.
(5) Examination after the end of treatment
After the end of treatment and the final examination, participants in the PAMSARC study will be examined again after 3 months and asked about their condition. If necessary, the examinations may be carried out by a GP. The medical history interview may take place over the telephone.
The doctor records any side effects and long-term effects that may have occurred.
(6) Follow-up examinations
Follow-up examinations will then take place at three-monthly intervals. Data on the current situation regarding the disease and any further cancer treatments will usually be collected by telephone.
These are the potential benefits of participating in the PAMSARC study:
- Researchers suspect that treatment with pasireotide may delay the progression of the disease and prolong patients’ lives.
- DSRCT and synovial sarcoma are very rare types of cancer – data from the PAMSARC study may be helpful to other patients in the future.
However, taking part in the PAMSARC study may also have disadvantages:
- The efficacy of pasireotide in the treatment of DSRCT or synovial sarcoma has not yet been proven. It is therefore possible that participants may not experience the hoped-for benefits.
- Treatment with pasireotide may cause side effects. These can vary in severity but are often treatable.
- The necessary examinations may also involve certain burdens or potential risks – for example, through blood tests or imaging diagnostics.
- Repeatedly completing the questionnaires may cause you to dwell on things and lead to psychological stress. However, no harm is expected to result from this.
Where is the study being conducted?
The PAMSARC trial is to be conducted at 6 different trial centres. Patients can currently be enrolled at NCT Heidelberg.
As soon as further study centres are added, these can be found on the NCT Heidelberg study website under Soft Tissue Sarcomas under the PAMSARC study.
What costs will participants incur?
Participation in the study is free of charge for patients. However, you will not receive any compensation for your time or expenses. Travel costs are not reimbursed as part of the study.
If you have any questions about the funding of the study, you can ask the study team, for example during the information session.
How many participants will the study accept? Where are there still places available?
In total, the PAMSARC study will enrol 28 patients. Those interested can find out whether and where there are still places available from the relevant study centres.
Referring doctors or patients wishing to take part in the PAMSARC study can contact the NCT Heidelberg by email (contact person: Barbara Stöhr, barbara.stoehr-at-nct-heidelberg.de).
The PAMSARC study is supported by patient representatives from the German Sarcoma Foundation.
Scientific publications (Selection)
Heilig CE et al. 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 tumour) multicentre phase 2 trial. Cancer Treatment and Research Communications (2025) 45: 100986, https://doi.org/10.1016/j.ctarc.2025.100986.
Schlenk RF, Heining C, Gnutzmann E et al. Pasireotide as maintenance treatment in SSTR2/3/5-expressing synovial sarcoma and desmoplastic small round cell tumor: The PAMSARC study (1784TiP). Oncol Res Treat. 2024;47(Suppl 2):Abstract.
Heilig CE, Heining C, Gnutzmann E et al. 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;100986. doi: 10.1016/j.ctarc.2025.100986.
This study description was prepared by authors from the Cancer Information Service, under the direction of Dr. S. Weg-Remers, based on the study documentation.