Desmoid TumorPhase 1NCT03459469
Phase I, Open-label, Non-randomized Study to Evaluate Safety of BC2059
A Phase 1 study testing Tegavivint in people with Desmoid Tumor, run by Iterion Therapeutics at 7 sites in 2 countries.
Trial summary
Phase I, open-label, non-randomized study to evaluate safety of BC2059 administered intravenously to subjects with proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Desmoid Tumor
- What is being tested?
- Tegavivint
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 15 Jul 2018to 1 May 2022
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with histologically proven primary or recurrent desmoid tumor with currently bi-dimensionally measurable tumor by WHO criteria.
- Patients with disease that is either unresectable or for which the patient refuses surgery but is currently progressing, as defined by:
- 20% increase in tumor volume within 6 months OR
- Recurrent disease within 1 year of surgery OR
- Desmoid related symptoms as documented by a PRO questionnaire and documentation that symptoms are related to desmoid and not prior therapies.
- Willingness to provide tumor biopsies prior to treatment and while on treatment
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- Patients may have been previously treated with local therapies such as surgery, radiation, radiofrequency ablation, or cryosurgery provided this has been completed at least 4 weeks prior to registration and recovered from therapy related toxicity to less than CTCAE grade 2 and show no improvement in tumor size or symptom score.
- Patients may have been treated with systemic therapies such as tyrosine kinase inhibitors, hormone inhibitors or nonsteroidal anti-inflammatory drugs (NSAIDs) provided this has been completed at least 4 weeks prior to registration and recovered from any therapy related toxicity to less than CTCAE grade 2 and show no improvement in tumor size or symptom score.
- Patients may have been treated with systemic therapies such as cytotoxics, biologics or other unclassified experimental therapies provided this has been completed at least 8 weeks prior to registration and recovered from any therapy related toxicity to less than CTCAE grade 2 and show no improvement in tumor size or symptom score.
- Patients who have been treated with immune therapies such as vaccines, dendritic or other whole cell therapies, oncolytic or other viral approaches within the preceding 12 months should be discussed with the Medical Monitor prior to screening and enrollment into the study to determine eligibility.
- Age: 18 and over (no pre-pubertal patients)
- ECOG Performance status: 0-1
- Women of child-bearing potential (WOCBP) and men who are sexually active with WOCBP must agree to use one highly effective method of contraception, including hormonal contraceptives (e.g. combined oral contraceptives, patch, vaginal ring, injectables, and implants); intrauterine device (IUD) or intrauterine system (IUS); vasectomy or tubal ligation; and one effective method of contraception, including male condom, female condom, cervical cap, diaphragm or contraceptive sponge or abstain from sex for the duration of study participation and for 4 months following completion of BC2059 administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. See section 8.7.3 for more information.
- Contraception includes:
- Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
- Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy or tubal ligation at least 6 weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
- Male sterilization (at least 6 months prior to screening). For female patients on the study the vasectomized male partner should be the sole partner for that patient.
- Use of oral (estrogen and progesterone), injected or implanted combined hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS) or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.
- Sexually active males must use a condom during intercourse while taking drug and for 4 months after stopping study treatment and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.
- In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.
- Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least 6 weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential.
- Hematopoietic:
- Absolute granulocyte count ≥ 1,500/mm3
- Platelet count ≥ 100,000/mm3
- Hemoglobin at least 10.0 g/dL (transfusion allowed, subjects that require transfusion or growth factor need to demonstrate stable hemoglobin for at least 7 consecutive days of hemoglobin ≥ 10 g/dL)
- Hepatic:
- Bilirubin no greater than 1.5 times institutional upper limit of normal, in the absence of documented Gilbert's syndrome
- Transaminases no greater than 3 times upper limit of normal (ULN)
- Alkaline phosphatase no greater than 3 times ULN
- Renal: Creatinine clearance ≥75 mL/min by Cockcroft-Gault
- Pulmonary:
- Diffusing capacity of the lung for carbon monoxide (DLCO) greater than 75% predicted by single breath test
- Capillary oxygen saturation (O2 sat) > 95% by pulse oximetry
Who cannot join
- Patients who have not recovered to grade 1 from adverse events related to prior therapy excluding those considered not clinically significant (ex. Lymphopenia).
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to BC2059 or other agents used in study
- Patients with metabolic bone disease (ex. Hyperparathyroidism, Paget's disease, or osteomalacia)
- Clinically significant, uncontrolled heart disease and/or cardiac repolarization abnormality or QTc > 480 msec
- Uncontrolled concurrent illness including, but not limited to: ongoing or active infection (Viral, bacterial, fungal or other)
- Psychiatric illness/social situations that would limit compliance with study requirements
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- Pregnant and breastfeeding women are excluded from this study. The effects of BC2059 on the developing human fetus have the potential for teratogenic or abortifacient effects. There is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with BC2059.
- HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with BC2059
- Patients with abnormal serum chemistry values other than the specific limits detailed above, that in the opinion of the Investigator is considered to be clinically significant, should be discussed with the Medical Monitor before being enrolled in the study.
- Lack of peripheral venous or central venous access or any condition that would interfere with drug administration or collection of study samples
- Personal history of malignancy except:
- Cervical intraepithelial neoplasia;
- Skin basal cell carcinoma;
- Treated localized prostate carcinoma with PSA <1 ng/mL;
- Neoplasia treated with curative intent, in remission for at least five years and considered at low risk of relapse.
- Patients with familial adenomatous polyposis.
Trial locations and contacts
7 sites in 2 countries.
7 sites in 2 countries
United States6 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
The Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43202
MD Anderson Cancer Center
Houston, Texas, 770030
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Seattle Cancer Care Alliance
Seattle, Washington, 98109
About this study
Official title: Phase 1 Trial of BC2059 (Tegavivint) in Patients With Unresectable Desmoid Tumor
Phase I, open-label, non-randomized study to evaluate safety of BC2059 administered intravenously to subjects with proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive.
Read the detailed description
This study is a phase I, open-label, non-randomized study to evaluate safety of BC2059 administered intravenously to subjects with proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive. This study will utilize single patient cohorts for the first two dose levels in order to minimize sub-optimal drug exposures, followed by a conventional 3+3 dose escalation phase to achieve MTD or RP2D determined by pharmacokinetics or biologically relevant activity. Once MTD or RP2D is determined, that dose level cohort will expand to 14 patients enrolled to collect additional safety PK and PD data. If at least 1 patient has clinical benefit, the dose expansion phase will be expanded by a further 11 patients (25 total in at RP2D). The total duration of study for each subject will be dependent upon the safety, tolerability and efficacy of BC2059
- Conditions
- Desmoid Tumor
- Being tested
- Tegavivint
- Medical subject headings
- Desmoid Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TegavivintDrug
Also called BC2059
This is an Investigational drug
| Study group | Role | What they receive |
|---|---|---|
| Investigational drug | Experimental | An open-label, non-randomized study to evaluate safety of Tegavivint administered intravenously to subjects with proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive. Receives: Drug: Tegavivint |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability
Adverse events, Serious adverse events and Dose limiting toxicities
Measured: 12 Months
Secondary outcomes (1)
1. To determine the durability of response (DOR) to BC2059 after the achievement of best response
Assessing CR and PR
Measured: 12 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Mar 2018
- Study start15 Jul 2018
- Primary completion1 May 2022
- Study completion1 May 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Desmoid TumorStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Iterion Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03459469
- Sponsor's protocol ID
- BCI-001-DT17
- Status last verified
- Feb 2023
- First submitted
- 1 Mar 2018
- Last updated
- 1 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Trial of BC2059 (Tegavivint) in Patients With Unresectable Desmoid Tumor
- ClinicalTrials.gov ID
- NCT03459469
- Acronym / sponsor ID
- BCI-001-DT17
- Conditions
- Desmoid Tumor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Iterion TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 15 Jul 2018 (actual)
- Primary completion
- 1 May 2022 (actual)
- Study completion
- 1 May 2022 (actual)
- First posted
- 9 Mar 2018 (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.
Phase I, open-label, non-randomized study to evaluate safety of BC2059 administered intravenously to subjects with proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive.
Detailed description
This study is a phase I, open-label, non-randomized study to evaluate safety of BC2059 administered intravenously to subjects with proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive. This study will utilize single patient cohorts for the first two dose levels in order to minimize sub-optimal drug exposures, followed by a conventional 3+3 dose escalation phase to achieve MTD or RP2D determined by pharmacokinetics or biologically relevant activity. Once MTD or RP2D is determined, that dose level cohort will expand to 14 patients enrolled to collect additional safety PK and PD data. If at least 1 patient has clinical benefit, the dose expansion phase will be expanded by a further 11 patients (25 total in at RP2D). The total duration of study for each subject will be dependent upon the safety, tolerability and efficacy of BC2059
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Investigational drug | Experimental | Drug: TegavivintAn open-label, non-randomized study to evaluate safety of Tegavivint administered intravenously to subjects with proven primary or recurrent desmoid tumor that is unresectable and symptomatic or progressive. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (33 listed)
- Patients with histologically proven primary or recurrent desmoid tumor with currently bi-dimensionally measurable tumor by WHO criteria.
- Patients with disease that is either unresectable or for which the patient refuses surgery but is currently progressing, as defined by:
- Willingness to provide tumor biopsies prior to treatment and while on treatment
- Patients may have been previously treated with local therapies such as surgery, radiation, radiofrequency ablation, or cryosurgery provided this has been completed at least 4 weeks prior to registration and recovered from therapy related toxicity to less than CTCAE grade 2 and show no improvement in tumor size or symptom score.
- Patients may have been treated with systemic therapies such as tyrosine kinase inhibitors, hormone inhibitors or nonsteroidal anti-inflammatory drugs (NSAIDs) provided this has been completed at least 4 weeks prior to registration and recovered from any therapy related toxicity to less than CTCAE grade 2 and show no improvement in tumor size or symptom score.
Key exclusion criteria (16 listed)
- Patients who have not recovered to grade 1 from adverse events related to prior therapy excluding those considered not clinically significant (ex. Lymphopenia).
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to BC2059 or other agents used in study
- Patients with metabolic bone disease (ex. Hyperparathyroidism, Paget's disease, or osteomalacia)
- Clinically significant, uncontrolled heart disease and/or cardiac repolarization abnormality or QTc > 480 msec
- Uncontrolled concurrent illness including, but not limited to: ongoing or active infection (Viral, bacterial, fungal or other)
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 |
|---|---|---|
| TegavivintAlso: BC2059This is an Investigational drug | Drug | Investigational drug |
Primary outcome
To evaluate the safety and tolerability
Time frame: 12 Months
Adverse events, Serious adverse events and Dose limiting toxicities
1 secondary outcome
1. To determine the durability of response (DOR) to BC2059 after the achievement of best response
Time frame: 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 1 Mar 2023.
- 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.
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Tegavivint in people with Desmoid Tumor, run by Iterion Therapeutics at 7 sites in 2 countries.
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 33 inclusion and 16 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 1 Mar 2023. 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 United States, Canada. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
To evaluate the safety and tolerability (time frame: 12 Months). 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 1 Mar 2023.