Tumor, Desmoplastic Small Round Cell, AdultPhase 2ABRADESNCT03275818
Trial of Nab-paclitaxel in Patients With Desmoid Tumors and Multiply Relapsed/Refractory Desmoplastic Small Round Cell Tumors and Ewing Sarcoma
A Phase 2 study testing nab paclitaxel in people with Tumor, Desmoplastic Small Round Cell, Adult and Tumor, Desmoplastic Small Round Cell, Childhood, run by Grupo Espanol de Investigacion en Sarcomas at 7 sites in Spain.
Trial summary
A two-cohort, fase II, open-label, non-randomized, multicenter clinical trial. 14 sites in Spain. Cohort 1: Subjects with desmoid tumor (DT) Cohort 2: Subjects with desmoplastic small round cell tumor or Ewing sarcoma (DSRCT and ES) Nab-paclitaxel (ABRAXANE) will be administered as follows: Age ≥ 21 and ≤ 80 years… Read more about this study
- Who is it for?
- Ages 6 months to 80 years
- Conditions the study is for
- Tumor, Desmoplastic Small Round Cell, Adult, Tumor, Desmoplastic Small Round Cell, Childhood, Sarcoma, Ewing
- What is being tested?
- nab paclitaxel
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 69 participants
- Where
- 7 sitesSpain
- Study dates
- 9 May 2017to 30 Sep 2021
- 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 6 months to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects (parent or legal guardian if subject under 18 years) must voluntarily sign the informed consent form before any study test is conducted that is not part of routine subject care.
- Subjects with pathologic diagnosis of deep desmoid tumor of extremities, trunk wall or head and neck region.Intra-abdominal desmoid tumor cases could be enrolled if harboring betacatenin mutation.
- Subjects must be symptomatic (pain) due to tumor desmoid mass or lack physical function due to desmoid tumor mass, or in RECIST progression in the last 6 months.
- Age: ≥ 6 months.
- Subjects could have received one previous chemotherapy line if the scheme was methotrexate plus vinca alkaloids. Patients who received prostaglandin inhibitors or hormone therapy are also eligible.
- Availability of archive tumor block.
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- Measurable disease, according to RECIST 1.1 criteria.
- Performance status ≤1 (ECOG).
- Normal ECG values.
- Adequate bone marrow function (hemoglobin ≥ 9 g/dL, leukocytes ≥ 3.000/mm3, neutrophils ≥ 1.500/mm3, platelets ≥ 100.000/mm3). Subjects with plasma creatinine ≤ 1.6 mg/dl, transaminases ≤ 2.5 times the ULN, total bilirubin ≤ 1.25 times the ULN are acceptable.
- Men or women of childbearing potential must use an effective method of contraception before entry into the study and throughout the same and for 6 months after ending the study treatment. Women of childbearing potential must have a negative urine or serum pregnancy test before study entry.
- HBV and HCV serologies must be performed prior to inclusion. If HbsAg is positive it is recommended to reject the existence of replicative phase (HbaAg+, DNA VHB+) remaining at investigators' discretion the preventive treatment with lamivudine. If a potential subject is positive for anti-HCV antibodies, presence of the virus should be ruled out with a qualitative PCR, or the subject should NOT be included in the study (if a qualitative PCR cannot be performed then subject will not be able to enter the study).
- Subjects or legal guardian must voluntarily sign the informed consent form before any study test is conducted that is not part of routine subject care.
- Subject diagnosed of relapsed/refractory desmoplastic small round cell tumor (DSRCT) or Ewing´s sarcoma.
- DSRCT subjects must have received at least one previous poli-chemotherapy line.
- Ewing´s sarcoma subjects must have received at least two standard chemotherapy lines.
- Age ≥ 6 months
- Availability of archive tumor blocks or slides (new biopsy recommended).
- Measurable disease, according to RECIST 1.1 criteria.
- Performance status ≤1 (ECOG).
- Adequate respiratory functions: FEV1 > 1L.
- Normal ECG values.
- Adequate bone marrow function (hemoglobin ≥ 9 g/dL, leukocytes ≥ 3,000/mm3, neutrophils ≥ 1,500/mm3, platelets ≥ 100,000/mm3). Subjects with plasma creatinine ≤ 1.6 mg/dL, transaminases ≤ 2.5 times the ULN, total bilirubin ≥ 1.25 times ULN, CPK ≤ 2.5 times ULN, alkaline phosphatase ≤ 2.5 times the ULN are acceptable. If alkaline phosphatase is > 2.5 times the ULN, then the alkaline phosphatase liver fraction and/or 5' nucleotidase and/or GGT must be ≤ ULN.
- Men or women of child bearing potential should be using an effective method of contraception before entry into the study and throughout the same and for 6 months after ending the study. Women of childbearing potential must have a negative urine pregnancy test before study entry.
- HBV and HCV serologies must be performed prior to inclusion. If HbsAg is positive it is recommended to reject the existence of replicative phase (HbaAg+, DNA VHB+) remaining at investigators' discretion the preventive treatment with lamivudine. If a potential subject is positive for anti-HCV antibodies, presence of the virus should be ruled out with a qualitative PCR, or the subject should NOT be included in the study (if a qualitative PCR cannot be performed then subject will not be able to enter the study).
- Prior taxane therapy for any indication is accepted.
- > Grade 3 (intense and diffuse) expression of SPARC by immunohistochemistry.
Who cannot join
- Prior taxane therapy for any indication.
- Less than 4 weeks elapsed since prior exposure to chemotherapy.
- More than one previous chemotherapy line.
- Subjects with desmoid tumor of abdominal cavity (abdominal wall is not an exclusion criterion)
- Desmoid tumor with ill-defined margins.
- Unavailability to undergo MRI.
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- Previously irradiated target lesion (if radiation dose exceeded 50 Gy).
- Pre-existing neuropathy greater than grade 1.
- Other active invasive malignancy requiring ongoing therapy or expected to require systemic therapy within two years. However, localized squamous cell carcinoma of the skin, basal cell carcinoma of the skin, carcinoma in situ of the cervix or other malignancies requiring only locally ablative therapy, will not result in exclusion.
- Concomitant anticancer therapy, immunotherapy or radiation therapy within prior 4 weeks.
- Uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring IV antibiotic, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, psychiatric illness or social situations that would limit compliance with study requirements
- Hb < 9 g/dL.
- Women who are pregnant or breast-feeding.
- Known hypersensitivity to protein bound paclitaxel
- Any other concurrent condition that in the investigators opinion would jeopardize compliance with the protocol
- Known positive test for infection by human immunodeficiency virus (HIV).
- Subjects participating in another clinical trial or receiving any other investigational product.
- DSRCT and ES Cohort
- Less than 4 weeks elapsed since prior exposure to chemotherapy.
- Pre-existing neuropathy greater than Grade 1.
- Other active invasive malignancy requiring ongoing therapy or expected to require systemic therapy within two years. However, localized squamous cell carcinoma of the skin, basal cell carcinoma of the skin, carcinoma in situ of the cervix or other malignancies requiring only locally ablative therapy, will not result in exclusion.
- Concomitant anticancer therapy, immunotherapy or radiation therapy within prior 4 weeks.
- Uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring IV antibiotic, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, psychiatric illness or social situations that would limit compliance with study requirements
- Hb < 9 g/dL.
- Women who are pregnant or breast-feeding.
- Known hypersensitivity to protein bound paclitaxel.
- Any other concurrent condition that in the investigators opinion would jeopardize compliance with the protocol.
- Known positive test for infection by human immunodeficiency virus (HIV).
- Subjects participating in another clinical trial or receiving any other investigational product.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Spain7 sites
H. de la Santa Creu i Sant Pau
Barcelona
H. Sant Joan de Déu
Barcelona
H.U. 12 de Octubre
Madrid
H.U.Niño Jesús
Madrid
H.U. Virgen del Rocío
Seville, 41013
H.U i Politècnic La Fe
Valencia
H.U. Miguel Servet
Zaragoza
About this study
Official title: Phase II Trial of Nab-paclitaxel for the Treatment of Desmoid Tumors and Multiply Relapsed/Refractory Desmoplastic Small Round Cell Tumors and Ewing Sarcoma
A two-cohort, fase II, open-label, non-randomized, multicenter clinical trial. 14 sites in Spain.
Cohort 1: Subjects with desmoid tumor (DT) Cohort 2: Subjects with desmoplastic small round cell tumor or Ewing sarcoma (DSRCT and ES)
Read more
Nab-paclitaxel (ABRAXANE) will be administered as follows:
Age ≥ 21 and ≤ 80 years: 125 mg/m2 days 1, 8 and 15 in cycles of 28 days Age ≥ 6 months and ≤ 20 years: 240 mg/m2 days 1, 8 and 15 in cycles of 28 days
Subjects in the DT cohort will receive a maximum of three cycles. Subjects in the DSRCT and ES cohort will receive unlimited cycles until disease progression, the subject begins a new anticancer treatment, withdrawal of parent/guardian/subject consent/assent, parent/guardian/subject refusal, physician decision, toxicity that cannot be managed by dose delay or dose reduction alone or the study ends for any reason.
The main goal is to determine the objective response rate (ORR), using RECIST 1.1 criteria and to determine the clinical benefit rate (CBR), defined as CR+PR+SD for 3 months with improvement of pain with at least minimally important difference (MID) of 2 in subjects with desmoid tumors (DT cohort) and to determine the objective response rate (ORR) in subjects with desmoplastic small round cell tumor and Ewing sarcoma, using RECIST 1.1 criteria (DSRCT and ES cohort)
- Conditions
- Tumor, Desmoplastic Small Round Cell, Adult, Tumor, Desmoplastic Small Round Cell, Childhood, Sarcoma, Ewing, Sarcoma, Desmoid
- Being tested
- nab paclitaxel
- Keywords
- sarcoma, Desmoid tumor, Desmoplastic small round cell tumor, Ewing sarcoma, relapsed, refractory
- Medical subject headings
- Neoplasms, Sarcoma, Ewing, Sarcoma, Desmoid Tumors, Desmoplastic Small Round Cell Tumor, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nab paclitaxelDrug
Also called Abraxane
Subjects in the DT cohort will receive a maximum of three cycles. Subjects in the DSRCT and ES cohort will receive unlimited cycles until disease progression, the subject begins a new anticancer treatment, withdrawal of parent/guardian/subject consent/assent, parent/guardian/subject refusal, physician decision, toxicity that cannot be managed by dose delay or dose reduction alone or the study ends for any reason
| Study group | Role | What they receive |
|---|---|---|
| nab-paclitaxel | Experimental | nab-paclitaxel (ABRAXANE) will be administered as follows:
* Age ≥ 21: 125 mg/m2 days 1, 8 and 15 in cycles of 28 days
* Age ≥ 6 months and ≤ 20 years: 240 mg/m2 (for patients weighing > 10 kg) and 11.5 mg/kg (for patients weighing ≤ 10 kg) on days 1, 8 and 15 in cycles of 28 days Receives: Drug: nab paclitaxel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR) - cohort 1
TD cohort: Overall response rate (ORR) according to RECIST 1.1 and/or clinical benefit rate (CBR) at 3 months with pain improvement of at least 2 points in the Brief Pain Inventory - Short Form (BPI-SF)
Measured: 3 months
Objective response rate (ORR) - cohort 2
DSRCT and SE cohort: To determine the objective response rate (ORR) in subjects with desmoplastic small round cell tumor and Ewing's sarcoma, using RECIST 1.1 criteria
Measured: 2 months
Secondary outcomes (6)
Pattern of radiological response - cohort 1
TD cohort: To define the pattern of radiological response according to MRI parameters and to correlate it with CBR and Brief Pain Inventory (BPI) parameters.
Measured: 3 months
Progression-free survival - cohort 1
TD cohort: To estimate the efficacy of nab-paclitaxel as measured by the progression-free survival (PFS) assessed by median time.
Measured: 3 months
Variation of symptoms - cohort 1
TD cohort: To analyze the variation of symptoms during the first year from trial enrollment in accordance with BPI and Analgesic Quantification Algorithm (AQA).
Measured: during first year
Variation of physical function - cohort 1
TD cohort: To analyze the variation of physical function during the first year from trial enrollment in accordance with sponsor form.
Measured: during first year
Safety profile of nab-paclitaxel - cohort 1
TD cohort: To evaluate the safety profile of nab-paclitaxel according to CTCAE 4.0.
Measured: up to 12 months
Safety profile of nab-paclitaxel - cohort 2
DSRCT and SE cohort: To evaluate the safety profile of nab-paclitaxel according to CTCAE 4.0.
Measured: up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2017
- Study start9 May 2017
- Primary completion30 Sep 2021
- Study completion30 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tumor, Desmoplastic Small Round Cell, AdultStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 69 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grupo Espanol de Investigacion en Sarcomas
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Javier Martin Broto, MD, Study director, Hospitales Universitarios Virgen del RocíoJaume Mora, MD, Study director, Hospital Sant Joan de DeuJavier Martínez Trufero, MD, Principal investigator, Hospital Miguel ServetFrancisco Bautista, MD, Principal investigator, Hospital Universitario Niño JesúsAntonio López Pousa, MD, Principal investigator, Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant PauRoberto Díaz, MD, Principal investigator, Hospital Universitario La FeJosé Antonio López-Martín, MD, Principal investigator, Hospital Universitario 12 de Octubre
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03275818
- Sponsor's protocol ID
- GEIS-39
- Other IDs
- 2016-002464-14, 2016
- Acronym
- ABRADES
- Status last verified
- Apr 2022
- First submitted
- 3 Aug 2017
- Last updated
- 7 Jun 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 7 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Nab-paclitaxel for the Treatment of Desmoid Tumors and Multiply Relapsed/Refractory Desmoplastic Small Round Cell Tumors and Ewing Sarcoma
- ClinicalTrials.gov ID
- NCT03275818
- Acronym / sponsor ID
- ABRADES / GEIS-39
- Conditions
- Tumor, Desmoplastic Small Round Cell, Adult, Tumor, Desmoplastic Small Round Cell, Childhood, Sarcoma, Ewing, Sarcoma, Desmoid
- Keywords
- sarcoma, Desmoid tumor, Desmoplastic small round cell tumor, Ewing sarcoma, relapsed, refractory
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Grupo Espanol de Investigacion en SarcomasUniversity, hospital or other
- Study start
- 9 May 2017 (actual)
- Primary completion
- 30 Sep 2021 (actual)
- Study completion
- 30 Sep 2021 (actual)
- First posted
- 8 Sep 2017 (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.
A two-cohort, fase II, open-label, non-randomized, multicenter clinical trial. 14 sites in Spain.
Cohort 1: Subjects with desmoid tumor (DT) Cohort 2: Subjects with desmoplastic small round cell tumor or Ewing sarcoma (DSRCT and ES)
Nab-paclitaxel (ABRAXANE) will be administered as follows:
Age ≥ 21 and ≤ 80 years: 125 mg/m2 days 1, 8 and 15 in cycles of 28 days Age ≥ 6 months and ≤ 20 years: 240 mg/m2 days 1, 8 and 15 in cycles of 28 days
Subjects in the DT cohort will receive a maximum of three cycles. Subjects in the DSRCT and ES cohort will receive unlimited cycles until disease progression, the subject begins a new anticancer treatment, withdrawal of parent/guardian/subject consent/assent, parent/guardian/subject refusal, physician decision, toxicity that cannot be managed by dose delay or dose reduction alone or the study ends for any reason.
The main goal is to determine the objective response rate (ORR), using RECIST 1.1 criteria and to determine the clinical benefit rate (CBR), defined as CR+PR+SD for 3 months with improvement of pain with at least minimally important difference (MID) of 2 in subjects with desmoid tumors (DT cohort) and to determine the objective response rate (ORR) in subjects with desmoplastic small round cell tumor and Ewing sarcoma, using RECIST 1.1 criteria (DSRCT and ES cohort)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| nab-paclitaxel | Experimental | Drug: nab paclitaxelnab-paclitaxel (ABRAXANE) will be administered as follows: * Age ≥ 21: 125 mg/m2 days 1, 8 and 15 in cycles of 28 days * Age ≥ 6 months and ≤ 20 years: 240 mg/m2 (for patients weighing > 10 kg) and 11.5 mg/kg (for patients weighing ≤ 10 kg) on days 1, 8 and 15 in cycles of 28 days |
Who may qualify
- Ages
- 6 months to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (27 listed)
- Subjects (parent or legal guardian if subject under 18 years) must voluntarily sign the informed consent form before any study test is conducted that is not part of routine subject care.
- Subjects with pathologic diagnosis of deep desmoid tumor of extremities, trunk wall or head and neck region.Intra-abdominal desmoid tumor cases could be enrolled if harboring betacatenin mutation.
- Subjects must be symptomatic (pain) due to tumor desmoid mass or lack physical function due to desmoid tumor mass, or in RECIST progression in the last 6 months.
- Age: ≥ 6 months.
- Subjects could have received one previous chemotherapy line if the scheme was methotrexate plus vinca alkaloids. Patients who received prostaglandin inhibitors or hormone therapy are also eligible.
Key exclusion criteria (29 listed)
- Prior taxane therapy for any indication.
- Less than 4 weeks elapsed since prior exposure to chemotherapy.
- More than one previous chemotherapy line.
- Subjects with desmoid tumor of abdominal cavity (abdominal wall is not an exclusion criterion)
- Desmoid tumor with ill-defined margins.
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 |
|---|---|---|
| nab paclitaxelAlso: AbraxaneSubjects in the DT cohort will receive a maximum of three cycles. Subjects in the DSRCT and ES cohort will receive unlimited cycles until disease progression, the subject begins a new anticancer treatment, withdrawal of parent/guardian/subject consent/assent, parent/guardian/subject refusal, physician decision, toxicity that cannot be managed by dose delay or dose reduction alone or the study ends for any reason | Drug | nab-paclitaxel |
Primary outcome
Overall response rate (ORR) - cohort 1
Time frame: 3 months
TD cohort: Overall response rate (ORR) according to RECIST 1.1 and/or clinical benefit rate (CBR) at 3 months with pain improvement of at least 2 points in the Brief Pain Inventory - Short Form (BPI-SF)
Objective response rate (ORR) - cohort 2
Time frame: 2 months
DSRCT and SE cohort: To determine the objective response rate (ORR) in subjects with desmoplastic small round cell tumor and Ewing's sarcoma, using RECIST 1.1 criteria
6 secondary outcomes
Pattern of radiological response - cohort 1
Time frame: 3 months
Progression-free survival - cohort 1
Time frame: 3 months
Variation of symptoms - cohort 1
Time frame: during first year
Variation of physical function - cohort 1
Time frame: during first year
Safety profile of nab-paclitaxel - cohort 1
Time frame: up to 12 months
Safety profile of nab-paclitaxel - cohort 2
Time frame: up to 12 months
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 7 Jun 2022.
- Individual participant dataSharing planned: Undecided
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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.
Javier Martin Broto, MD study director
Hospitales Universitarios Virgen del Rocío
Jaume Mora, MD study director
Hospital Sant Joan de Deu
Javier Martínez Trufero, MD principal investigator
Hospital Miguel Servet
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing nab paclitaxel in people with Tumor, Desmoplastic Small Round Cell, Adult and Tumor, Desmoplastic Small Round Cell, Childhood, run by Grupo Espanol de Investigacion en Sarcomas at 7 sites in Spain.
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 27 inclusion and 29 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 7 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Spain. 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?
Overall response rate (ORR) - cohort 1 (time frame: 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Jun 2022.