DesmoidPhase 2NCT03966742
Doxorubicin Eluting Intra-arterial Embolization for Aggressive Desmoid Fibromatosis
A Phase 2 study testing Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis in people with Desmoid and Desmoid Fibromatosis of Skin, run by Rabin Medical Center at 1 site in Israel.
Trial summary
In this study Drug-eluting microbeads (DEB) loaded with Doxorubicin will be delivered into the target Desmoid Fibromatoses (DF) tissue via selective arterial embolization by angiographic technique. The objective of the study is to demonstrate the safety and efficacy of this treatment. Read more about this study
- Who is it for?
- Ages 3 to 80 years
- Conditions the study is for
- Desmoid, Desmoid Fibromatosis of Skin, Desmoid Neoplasm of Chest Wall
- What is being tested?
- Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteIsrael
- Study dates
- 11 Oct 2018to 18 Dec 2020
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age 3-80 years.
- Histologically confirmed diagnosis of Desmoids Fibromatosis.
- After at least one systemic treatment line. Standard first line systemic treatment may include: Methotrexate, Vinblastine, Doxorubicin, Liposomal Doxorubicin (Doxil), NSAIDS or hormonal treatment. If first line treatment is renounced, this treatment decision must be documented. Considering the trend of avoiding surgical treatment, the documentation must include that the treatment decision is not associated to the resectability of the tumor.
- Karnofsky performance status (PS)>50% for patients older than 16 years or Lansky PS >50% for patients under 16 years.
- At least one measurable lesion, with a long diameter of at least 30mm, with an anatomical location accessible for endovascular treatment.
- T2 signal increase on MRI.
Show all 12Show fewer
- No evidence of prior treatment toxicity, adequate washout period after prior treatment:
- 14 days after myelosuppressive chemotherapy treatment.
- 7 days after GCSF (Granulocyte colony-stimulating factor), 14 days after Neulastim.
- 7 days after targeted/biologic treatment.
- Female patients of childbearing potential must be willing to use an adequate method of contraception (hormonal, barrier or abstinence) for the treatment period and up to 90 days after the treatment completion.
- Willing and able to provide written informed consent for the trial.
Who cannot join
- Participation in another interventional study.
- Congestive heart failure, characterised by LVEF (Left Ventricular Ejection Fraction) < 50% or Shortening fracture < 27%.
- Previous treatment with anthracycline of a accumulative dose of more than 360 mg/m2.
- History of allergic reaction attributed to Doxil or doxorubicin treatment.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure (New York Heart Association Class III or IV), cardiac arrhythmia, or psychiatric illness, social situations that would limit compliance with study requirements.
Trial locations and contacts
1 site in 1 country.
About this study
In this study Drug-eluting microbeads (DEB) loaded with Doxorubicin will be delivered into the target Desmoid Fibromatoses (DF) tissue via selective arterial embolization by angiographic technique. The objective of the study is to demonstrate the safety and efficacy of this treatment.
Read the detailed description
Desmoid Fibromatoses (DF) are locally aggressive lesions associated with substantial morbidity and potentially mortality, due to invasion of adjacent neurovascular structures and vital organs. They have no potential for metastasis. Histologically, they are characterised by mature fibroblasts within a matrix of abundant fibrous stroma. While 5-15% of cases are seen in patients with Familial Adenomatous Polyposis (FAP) syndrome, the vast majority arise sporadically.
The etiology of Desmoids remains poorly understood and the therapeutic approaches in their management remain very diverse. For resectable lesions, surgery is recommended but reported cure rates range broadly from 12-80%. Systemic treatments range from non-steroidal anti-inflammatories and anti-estrogenic therapy to targeted tyrosine kinase inhibitors and cytotoxic chemotherapy, most commonly methotrexate, vinblastine and doxorubicin.
Doxorubicin is an anthracycline with demonstrated efficacy in treating desmoids at systemic IV doses of 50- 75mg/m2 over 3-4 week cycles. Extended use is limited by dose - dependent cardiotoxicity which can be seen in up to 36% of patients receiving doses in excess of 550mg/m2. Delayed cardiotoxicity is particularly common and less predictable among pediatric cancer survivors.
Selective trans-arterial chemo-embolization (TACE) is a method to achieve high tissue drug concentration with minimal systemic toxicity. Historically, this has been achieved by mixing doxorubicin with embolic agents such as lipiodol or gelatin sponge in the treatment of hepatocellular carcinoma (HCC).
Drug-eluting microbeads (DEB) ionically loaded with doxorubicin have shown sustained release in TACE target tissues with substantially lower serum drug concentrations when compared to lipiodol TACE.
The present study utilizes DEB's loaded with Doxorubicin delivered into the target DF tissue via selective arterial embolization by angiographic technique. This study follows a successful feasibility study.
- Conditions
- Desmoid, Desmoid Fibromatosis of Skin, Desmoid Neoplasm of Chest Wall, Desmoid Tumor Caused by Somatic Mutation, Aggressive Fibromatoses, Fibromatosis Desmoid
- Being tested
- Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis
- Medical subject headings
- Desmoid Tumors, Desmoid disease, hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Drug Eluting Bead Embolization (DEB) for Desmoid FibromatosisProcedure or surgery
Delivery of Doxorubicin selectively into Desmoid Fibromatosis utilizing its vascular supply as a conduit, and ionic loading the doxorubicin onto embolized particles as the drug delivery vehicle.
Maximal dose is 75 mg/m2 and at least 50 mg. The bead size is 75-300 Mµ in a 2 CC syringe.
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Doxorubin-eluting particle embolization for treatment of Desmoid Fibromatosis. Receives: Procedure: Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate of tumor size.
Response to treatment. Measured by change in tumor size according to RECIST (Response Evaluation Criteria in Solid Tumors) version 1.1 criteria.
Measured: At baseline ; 6-10 weeks after each treatment
Objective response rate of tumor biological activity.
Response to treatment. Tumor biological activity measured by change in MRI T2 signal intensity.
Measured: At baseline ; 6-10 weeks after each treatment
Patient reported outcomes.
Change in clinical symptoms measured by standard clinical patient questionnaires - EORTC QLQ-C30. (Quality of Life Questionnaire).
All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus a high score for a functional scale represents a high level of functioning, a high score for the global health status / QoL represents a high QoL. Similarly, a high score for a symptom scale represents a high level of symptomatology.
Measured: At baseline ; 6-10 weeks after each treatment
Secondary outcomes (1)
Adverse event profile (safety)
Treatment safety measured by standard patient questionnaires and clinical evaluation using CTCAE (Common Terminology Criteria for Adverse Events) version 5.0.
Measured: At baseline ; 6-10 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 May 2019
- Study start11 Oct 2018
- Primary completion18 Dec 2020
- Study completion18 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for DesmoidStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- All patients
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rabin Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Eldad Elnekave, MD, Rabin Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03966742
- Sponsor's protocol ID
- RMC-0485-17
- Status last verified
- Mar 2022
- First submitted
- 8 Apr 2019
- Last updated
- 25 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Elnekave E, Atar E, Amar S, Bruckheimer E, Knizhnik M, Yaniv I, Dujovny T, Feinmesser M, Ash S. Doxorubicin-Eluting Intra-Arterial Therapy for Pediatric Extra-Abdominal Desmoid Fibromatoses: A Promising Approach for a Perplexing Disease. J Vasc Interv Radiol. 2018 Oct;29(10):1376-1382. doi: 10.1016/j.jvir.2018.04.009. Epub 2018 Jul 31.
PubMed 30075974
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Doxorubicin Eluting Intra-arterial Embolization for Aggressive Desmoid Fibromatosis
- ClinicalTrials.gov ID
- NCT03966742
- Acronym / sponsor ID
- RMC-0485-17
- Conditions
- Desmoid, Desmoid Fibromatosis of Skin, Desmoid Neoplasm of Chest Wall, Desmoid Tumor Caused by Somatic Mutation, Aggressive Fibromatoses, Fibromatosis Desmoid
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Rabin Medical CenterUniversity, hospital or other
- Study start
- 11 Oct 2018 (actual)
- Primary completion
- 18 Dec 2020 (actual)
- Study completion
- 18 Dec 2020 (actual)
- First posted
- 29 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this study Drug-eluting microbeads (DEB) loaded with Doxorubicin will be delivered into the target Desmoid Fibromatoses (DF) tissue via selective arterial embolization by angiographic technique. The objective of the study is to demonstrate the safety and efficacy of this treatment.
Detailed description
Desmoid Fibromatoses (DF) are locally aggressive lesions associated with substantial morbidity and potentially mortality, due to invasion of adjacent neurovascular structures and vital organs. They have no potential for metastasis. Histologically, they are characterised by mature fibroblasts within a matrix of abundant fibrous stroma. While 5-15% of cases are seen in patients with Familial Adenomatous Polyposis (FAP) syndrome, the vast majority arise sporadically.
The etiology of Desmoids remains poorly understood and the therapeutic approaches in their management remain very diverse. For resectable lesions, surgery is recommended but reported cure rates range broadly from 12-80%. Systemic treatments range from non-steroidal anti-inflammatories and anti-estrogenic therapy to targeted tyrosine kinase inhibitors and cytotoxic chemotherapy, most commonly methotrexate, vinblastine and doxorubicin.
Doxorubicin is an anthracycline with demonstrated efficacy in treating desmoids at systemic IV doses of 50- 75mg/m2 over 3-4 week cycles. Extended use is limited by dose - dependent cardiotoxicity which can be seen in up to 36% of patients receiving doses in excess of 550mg/m2. Delayed cardiotoxicity is particularly common and less predictable among pediatric cancer survivors.
Selective trans-arterial chemo-embolization (TACE) is a method to achieve high tissue drug concentration with minimal systemic toxicity. Historically, this has been achieved by mixing doxorubicin with embolic agents such as lipiodol or gelatin sponge in the treatment of hepatocellular carcinoma (HCC).
Drug-eluting microbeads (DEB) ionically loaded with doxorubicin have shown sustained release in TACE target tissues with substantially lower serum drug concentrations when compared to lipiodol TACE.
The present study utilizes DEB's loaded with Doxorubicin delivered into the target DF tissue via selective arterial embolization by angiographic technique. This study follows a successful feasibility study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All patients
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Procedure: Drug Eluting Bead Embolization (DEB) for Desmoid FibromatosisDoxorubin-eluting particle embolization for treatment of Desmoid Fibromatosis. |
Who may qualify
- Ages
- 3 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Age 3-80 years.
- Histologically confirmed diagnosis of Desmoids Fibromatosis.
- After at least one systemic treatment line. Standard first line systemic treatment may include: Methotrexate, Vinblastine, Doxorubicin, Liposomal Doxorubicin (Doxil), NSAIDS or hormonal treatment. If first line treatment is renounced, this treatment decision must be documented. Considering the trend of avoiding surgical treatment, the documentation must include that the treatment decision is not associated to the resectability of the tumor.
- Karnofsky performance status (PS)>50% for patients older than 16 years or Lansky PS >50% for patients under 16 years.
- At least one measurable lesion, with a long diameter of at least 30mm, with an anatomical location accessible for endovascular treatment.
Key exclusion criteria (5 listed)
- Participation in another interventional study.
- Congestive heart failure, characterised by LVEF (Left Ventricular Ejection Fraction) < 50% or Shortening fracture < 27%.
- Previous treatment with anthracycline of a accumulative dose of more than 360 mg/m2.
- History of allergic reaction attributed to Doxil or doxorubicin treatment.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure (New York Heart Association Class III or IV), cardiac arrhythmia, or psychiatric illness, social situations that would limit compliance with study requirements.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Drug Eluting Bead Embolization (DEB) for Desmoid FibromatosisDelivery of Doxorubicin selectively into Desmoid Fibromatosis utilizing its vascular supply as a conduit, and ionic loading the doxorubicin onto embolized particles as the drug delivery vehicle. Maximal dose is 75 mg/m2 and at least 50 mg. The bead size is 75-300 Mµ in a 2 CC syringe. | Procedure or surgery | Treatment |
Primary outcome
Objective response rate of tumor size.
Time frame: At baseline ; 6-10 weeks after each treatment
Response to treatment. Measured by change in tumor size according to RECIST (Response Evaluation Criteria in Solid Tumors) version 1.1 criteria.
Objective response rate of tumor biological activity.
Time frame: At baseline ; 6-10 weeks after each treatment
Response to treatment. Tumor biological activity measured by change in MRI T2 signal intensity.
Patient reported outcomes.
Time frame: At baseline ; 6-10 weeks after each treatment
Change in clinical symptoms measured by standard clinical patient questionnaires - EORTC QLQ-C30. (Quality of Life Questionnaire). All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus a high score for a functional scale represents a high level of functioning, a high score for the global health status / QoL represents a high QoL. Similarly, a high score for a symptom scale represents a high level of symptomatology.
1 secondary outcome
Adverse event profile (safety)
Time frame: At baseline ; 6-10 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2022.
- Background: PubMed 30075974Elnekave E, Atar E, Amar S, Bruckheimer E, Knizhnik M, Yaniv I, Dujovny T, Feinmesser M, Ash S. Doxorubicin-Eluting Intra-Arterial Therapy for Pediatric Extra-Abdominal Desmoid Fibromatoses: A Promising Approach for a Perplexing Disease. J Vasc Interv Radiol. 2018 Oct;29(10):1376-1382. doi: 10.1016/j.jvir.2018.04.009. Epub 2018 Jul 31.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis in people with Desmoid and Desmoid Fibromatosis of Skin, run by Rabin Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective response rate of tumor size. (time frame: At baseline ; 6-10 weeks after each treatment). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03966742, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2022; AzTrials last refreshed its copy on 8 Nov 2026.