The aim of this study is to assess the efficacy of MR-HIFU as a treatment modality for desmoid-type fibromatosis (DTF).
ID
Source
Brief title
Condition
- Soft tissue neoplasms malignant and unspecified
- Soft tissue therapeutic procedures
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Primary outcome of the trial will be the patient satisfaction rate 12 months
after the completion of the MR-HIFU procedure(s). Patient satisfaction is being
defined as the number of patients achieving their own personal satisfaction
score.
Secondary outcome
Secondary outcomes include the presence of non-perfused volume on MRI after the
MR-HIFU procedure, change in tumour volume, the response rate according to the
Response Evaluation Criteria in Solid Tumors version 1.1. (RECIST v.1.1) and
mRECIST criteria, the number of patients who need a re-intervention, time to
(re)growth, duration of tumour response and patient satisfaction, adverse
events, change in symptoms, pain scores, and health-related quality of life
(HRQoL) in the first 12 months after treatment.
Background summary
Desmoid-type fibromatosis (DTF) is a rare, histologically benign soft tissue
tumour. Although incapable of metastasizing, the clinical course is
unpredictable and can be aggressive because of local invasive growth. Many
intensive treatments (i.e. surgery, systemic treatments, radiotherapy) may be
considered in patients with symptomatic disease but unfortunately, these
*traditional* treatment options do not guarantee success. Local recurrence
after surgery remains high and the response rates after systemic treatment and
radiotherapy are disappointing. Active surveillance is now recommended as a
first line management for most patients with DTF. However, symptoms can be
severe, and patients are often limited in their daily life because of pain,
functional deficits, and/or psychological problems. Therefore, development of
new treatment modalities which improve quality of life and/or achieve tumour
control, while minimising any possible harm, are warranted. MR-HIFU is a
promising non-invasive technique that uses focused ultrasound waves to
thermally ablate tumours, while minimizing side effects to surrounding healthy
tissues. Given the promising outcomes of previous studies of MR-HIFU as
treatment modality for DTF patients, we hypothesize that DTF patients could
benefit from treatment with MR-HIFU.
Study objective
The aim of this study is to assess the efficacy of MR-HIFU as a treatment
modality for desmoid-type fibromatosis (DTF).
Study design
This is a two-stage, open-label, single-arm, phase 2 prospective study.
Intervention
All patients will undergo an adequate MR-HIFU treatment procedure with the aim
of total tumour ablation (consisting of a single or multiple treatments).
Study burden and risks
Participation in the study will bring limited to moderate burden and risks to
the patient. In terms of benefits, patients participating in this study may
improve their personal satisfaction score and/or may experience durable control
or reduction of tumour volume as a result of the MR-HIFU intervention. Patients
treated with MR-HIFU are at (small) risk for skin burn, thermal damage to
adjacent structures, and pain caused by the MR-HIFU procedure. The eligibility
criteria will minimize the risk of damage of critical structures. Serious
adverse events due to the intervention are not to be expected. In terms of
burden, patients will need to pay a visit or visits to the University Medical
Center Utrecht (UMCU) for one pre-treatment out-patient clinic visit and the
MR-HIFU treatment procedure itself and they will be required to undergo an
intervention under sedation (maximum duration of 4 hours). The follow-up
schedule of this study states the same number of visits as the current
consensus-based guideline for desmoid tumours. Patients will be asked to fill
in the HRQoL questionnaires before and 6 and 12 months after completion of
treatment. Altogether, we consider the burden and risks to be limited to
moderate
Heidelberglaan 100 HP E01.335
Utrecht 3584CX
NL
Heidelberglaan 100 HP E01.335
Utrecht 3584CX
NL
Listed location countries
Age
Inclusion criteria
- Patients > 16 years
- Histological evidence of DTF
- Patients with failure of active surveillance* for their present manifestation
of DTF
- Desmoid tumour must be targetable with MR-HIFU device
- Desmoid tumour must be visible on pre-treatment MR-imaging
- Patient is able to fit in the MRI gantry
- Capable to understand and sign informed consent
*failure of active surveillance due to tumor growth and/or new or worsening
symptoms
Exclusion criteria
- Personal or family history of familial adenomatous polyposis (FAP)
- Intra-abdominal tumour localization
- Patients with a tumour greater than 12 centimetres (as measured on intake MRI
by a radiologist).If the tumor is larger than 10 cm in one dimension, the other
two dimensions of the tumor must not be greater than half of the largest
dimension (i.e. if the largest dimension is 12 cm, the other dimensions mustn*t
be larger than 6 cm).
- Patients who haveundergone prior systemic therapy or radiotherapy for the
present manifestation of DTF
- Patients weighing more than 140 kilograms
- Pregnancy
- Contra indications to MRI, MRI contrast agents or sedation
- Unavoidable critical structures or dense tissues in target area*
- Inability to tolerate stationary position for the duration of the
procedure
- Any other condition, which in the opinion of the investigators, would put the
patient at increased risk or otherwise make the patients unsuitable for this
study
*as judged by the operator. e.g.: nerve bundles, skin, extensive scarring,
non-targeted bones, air (e.g. hollow viscera), (external) fixation device,
surgical clips, implants, or prothesis
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
In other registers
| Register | ID |
|---|---|
| CCMO | NL76201.041.21 |
| Other | NTR NL9679 |