ChordomaPhase 2NCT02383498
QUILT-3.011 Phase 2 Yeast-Brachyury Vaccine Chordoma
A Phase 2 study testing GI-6301 Vaccine (Yeast- Brachyury), Radiotherapy and wGT3X-BT Actigraph in people with Chordoma, run by NantCell, Inc. at 1 site in United States.
Trial summary
This is a phase 2 clinical trial to determine whether the yeast-brachyury vaccine GI-6301 improves the effectiveness of radiation for patients with localized chordoma. Chordoma patients with inoperable or residual tumor who do not have metastases and are planning to be treated with definitive (>70Gy) radiation were… Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Chordoma
- What is being tested?
- GI-6301 Vaccine (Yeast- Brachyury), Radiotherapy and wGT3X-BT Actigraph
- Type of treatment
- Vaccines, Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 1 May 2015to 1 Sep 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must meet the following criteria for participation:
- Diagnosis: Patients must have histologically confirmed chordoma by the Laboratory of Pathology, NCI, which is localized (no evidence of metastatic disease), unresectable and they must have planned radiation therapy, to at least one targeted lesion with evidence of growth prior to enrollment. The tentative radiation plan at enrollment must be in compliance with the required radiation doses. This can be given in standard or hypofractionated dosing with any technique deemed most appropriate by the treating radiation oncologist if other requirements are not met.
- Patients must have disease that is measurable by RECIST version 1.1.
- Fresh or archived tumor specimen must be available for correlative studies.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 at study entry (Karnofsky greater than or equal to 70)
- Age greater than or equal to 18 years. Because no dosing or adverse event data are currently available on the use of GI-6301 (Yeast Brachyury vaccine) in patients <18 years of age, children are excluded from this study, but will be eligible for future pediatric trials.
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- Prior Therapy: Patients must have fully recovered from prior surgery before enrollment. Prior radiation therapy is allowed provided the radiation field can safely irridated in the opinion of the treating radiation oncologist.
- Patients must have normal organ and marrow function as defined below:
- Serum creatinine less than or equal to 1.5 X upper limit of normal OR creatinine clearance on a 24-h urine collection of greater than or equal to 60 mL/min.
- ALT and AST less than or equal to 3 X the upper limits of normal.
- Total bilirubin less than or equal to 1.5 X upper limit of normal OR in patients with Gilbert s syndrome, a total bilirubin less than or equal to 3.0.
- Hematological eligibility parameters (within16 days of starting therapy):
- Granulocyte count greater than or equal to 1,500/mm3
- Platelet count greater than or equal to 100,000/mm3
- Men and women of child-bearing potential must agree to use effective birth control or abstinence during and for a period of 4 months after the last vaccination therapy.
- Patients must not have a history of yeast allergy. If patient has a questionable history of allergy to yeast, a yeast skin test can be performed. Patients would be eligible if skin test is negative.
- Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- Patients with any of the following will not be eligible for participation in this study:
- 1. Patients should have no evidence of immune dysfunction as listed below.
- Human immunodeficiency virus (HIV) positivity due to the potential for decreased immune response to the vaccine.
- Active autoimmune diseases requiring treatment or a history of autoimmune disease that might be stimulated by vaccine treatment. This requirement is due to the potential risks of exacerbating autoimmunity. However, patients with vitiligo, diabetes mellitus, and Hashimoto thyroiditis on appropriate replacement therapy may be enrolled.
- History of allergy or untoward reaction to yeast-based products (any hypersensitivity to yeast-based products will be excluded).
- Pregnant or breast-feeding women, due to the unknown effects of the Yeast-brachyury vaccine on the fetus or infant.
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- Serious intercurrent medical illness which would interfere with the ability of the patient to carry out the treatment program, including, but not limited to, inflammatory bowel disease, Crohn's disease, ulcerative colitis, or active diverticulitis.
- Chronic hepatitis infection, including B and C, because potential immune impairment caused by these disorders may diminish the effectiveness of this immunologic therapy.
- Any significant disease that, in the opinion of the investigator, may impair the patient s tolerance of study treatment.
- Significant dementia, altered mental status, or any psychiatric condition that would prohibit the understanding or rendering of informed consent.
- Patients may not be on systemic steroids within 4 weeks of enrolling on study with the exception of physiologic replacement doses (for instance in the case of adrenal insufficiency) or steroid premedication for baseline MRI and/or CT in the case of subjects with known contrast dye allergies.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Double-Blind, Phase 2 Trial of GI-6301 (Yeast-Brachyury Vaccine) Versus Placebo in Combination With Standard of Care Definitive Radiotherapy in Locally Advanced, Unresectable, Chordoma
This is a phase 2 clinical trial to determine whether the yeast-brachyury vaccine GI-6301 improves the effectiveness of radiation for patients with localized chordoma. Chordoma patients with inoperable or residual tumor who do not have metastases and are planning to be treated with definitive (>70Gy) radiation were enrolled. Patients were randomized to receive radiation plus the vaccine or radiation plus a blinded placebo. The study compared the outcomes of patients treated with radiation with and without the vaccine to determine whether the vaccine can increase the chances of shrinking the tumor and/or preventing further tumor growth.
Read the detailed description
Chordoma is a rare disease, affecting about 3,000 people in the United States. There have been indications of clinical benefit in patients with chordoma enrolled on the phase I trial of GI-6301.
PRIMARY OBJECTIVE: To determine if there is a difference in overall response rate (ORR) defined as complete response (CR) or partial response (PR) by RECIST 1.1 after up to 24 months among patients with Chordoma who are treated with radiation plus placebo vs. radiation plus vaccine.
- Conditions
- Chordoma
- Being tested
- Placebo, GI-6301 Vaccine (Yeast- Brachyury), Radiotherapy, wGT3X-BT Actigraph
- Keywords
- Overall Response Rate, Immune Activation, Tumor Infiltrating Lymphocytes, T Cell Response, Cross-Over
- Medical subject headings
- Chordoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboOther
Placebo will consist of USP-grade or equivalent 0.9% Sodium Chloride for Injection. Doses of placebo will be drawn into labeled syringes by an independent, unblinded pharmacist or designee.
GI-6301 Vaccine (Yeast- Brachyury)Biological
GI-6301 Vaccine is a heat-killed, recombinant yeast-based vaccine engineered to express the transcription factor, Brachyury. The Brachyury gene is used to transfect the parental yeast strain (S. cerevisiae W303 - a haploid strain with known mutations from wildtype yeast) to produce the final recombinant vaccine product.
RadiotherapyRadiation
Standard of care
wGT3X-BT ActigraphDevice
wGT3X-BT is small, non-invasive, portable watch accelerometer worn on the subject's wrist for Cycle 1 through Cycle 9
| Study group | Role | What they receive |
|---|---|---|
| GI-6301 Vaccine | Experimental | Radiation + GI-6301 Vaccine + Actigraph Receives: Biological: GI-6301 Vaccine (Yeast- Brachyury); Radiation: Radiotherapy; Device: wGT3X-BT Actigraph |
| Placebo | Placebo comparator | Radiation + Placebo + Actigraph Receives: Other: Placebo; Radiation: Radiotherapy; Device: wGT3X-BT Actigraph |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate
Difference in overall response rate among patients with Chordoma who are treated with radiation plus placebo vs. radiation plus vaccine.
Overall response rate (ORR) defined as complete response (CR) or partial response (PR) by RECIST 1.1 in the irradiated tumor site after up to 24 months among patients with Chordoma who are treated with radiation plus vaccine vs. radiation plus placebo
Complete response (CR; disappearance of all target lesions and no new lesions) or partial response (PR; >=30% decrease in the sum of the greatest diameter).
Measured: Up to 24 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 2015 (estimated)
- Study start1 May 2015
- Primary completion1 Sep 2019
- Study completion1 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ChordomaStudies 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
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- NantCell, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02383498
- Sponsor's protocol ID
- QUILT-3.011
- Status last verified
- Oct 2019
- First submitted
- 4 Mar 2015
- Last updated
- 20 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Wedekind MF, Widemann BC, Cote G. Chordoma: Current status, problems, and future directions. Curr Probl Cancer. 2021 Aug;45(4):100771. doi: 10.1016/j.currproblcancer.2021.100771. Epub 2021 Jul 1.
PubMed 34266694
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Phase 2 Trial of GI-6301 (Yeast-Brachyury Vaccine) Versus Placebo in Combination With Standard of Care Definitive Radiotherapy in Locally Advanced, Unresectable, Chordoma
- ClinicalTrials.gov ID
- NCT02383498
- Acronym / sponsor ID
- QUILT-3.011
- Conditions
- Chordoma
- Keywords
- Overall Response Rate, Immune Activation, Tumor Infiltrating Lymphocytes, T Cell Response, Cross-Over
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- NantCell, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 1 May 2015 (actual)
- Primary completion
- 1 Sep 2019 (actual)
- Study completion
- 1 Sep 2019 (actual)
- First posted
- 9 Mar 2015 (estimated)
- Results
- Posted 20 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 2 clinical trial to determine whether the yeast-brachyury vaccine GI-6301 improves the effectiveness of radiation for patients with localized chordoma. Chordoma patients with inoperable or residual tumor who do not have metastases and are planning to be treated with definitive (>70Gy) radiation were enrolled. Patients were randomized to receive radiation plus the vaccine or radiation plus a blinded placebo. The study compared the outcomes of patients treated with radiation with and without the vaccine to determine whether the vaccine can increase the chances of shrinking the tumor and/or preventing further tumor growth.
Detailed description
Chordoma is a rare disease, affecting about 3,000 people in the United States. There have been indications of clinical benefit in patients with chordoma enrolled on the phase I trial of GI-6301.
PRIMARY OBJECTIVE: To determine if there is a difference in overall response rate (ORR) defined as complete response (CR) or partial response (PR) by RECIST 1.1 after up to 24 months among patients with Chordoma who are treated with radiation plus placebo vs. radiation plus vaccine.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| GI-6301 Vaccine | Experimental | Biological: GI-6301 Vaccine (Yeast- Brachyury), Radiation: Radiotherapy, Device: wGT3X-BT ActigraphRadiation + GI-6301 Vaccine + Actigraph |
| Placebo | Placebo comparator | Other: Placebo, Radiation: Radiotherapy, Device: wGT3X-BT ActigraphRadiation + Placebo + Actigraph |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Diagnosis: Patients must have histologically confirmed chordoma by the Laboratory of Pathology, NCI, which is localized (no evidence of metastatic disease), unresectable and they must have planned radiation therapy, to at least one targeted lesion with evidence of growth prior to enrollment. The tentative radiation plan at enrollment must be in compliance with the required radiation doses. This can be given in standard or hypofractionated dosing with any technique deemed most appropriate by the treating radiation oncologist if other requirements are not met.
- Patients must have disease that is measurable by RECIST version 1.1.
- Fresh or archived tumor specimen must be available for correlative studies.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 at study entry (Karnofsky greater than or equal to 70)
- Age greater than or equal to 18 years. Because no dosing or adverse event data are currently available on the use of GI-6301 (Yeast Brachyury vaccine) in patients <18 years of age, children are excluded from this study, but will be eligible for future pediatric trials.
Key exclusion criteria (11 listed)
- 1. Patients should have no evidence of immune dysfunction as listed below.
- Human immunodeficiency virus (HIV) positivity due to the potential for decreased immune response to the vaccine.
- Active autoimmune diseases requiring treatment or a history of autoimmune disease that might be stimulated by vaccine treatment. This requirement is due to the potential risks of exacerbating autoimmunity. However, patients with vitiligo, diabetes mellitus, and Hashimoto thyroiditis on appropriate replacement therapy may be enrolled.
- History of allergy or untoward reaction to yeast-based products (any hypersensitivity to yeast-based products will be excluded).
- Pregnant or breast-feeding women, due to the unknown effects of the Yeast-brachyury vaccine on the fetus or infant.
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 |
|---|---|---|
| PlaceboPlacebo will consist of USP-grade or equivalent 0.9% Sodium Chloride for Injection. Doses of placebo will be drawn into labeled syringes by an independent, unblinded pharmacist or designee. | Other | Placebo |
| GI-6301 Vaccine (Yeast- Brachyury)GI-6301 Vaccine is a heat-killed, recombinant yeast-based vaccine engineered to express the transcription factor, Brachyury. The Brachyury gene is used to transfect the parental yeast strain (S. cerevisiae W303 - a haploid strain with known mutations from wildtype yeast) to produce the final recombinant vaccine product. | Biological | GI-6301 Vaccine |
| RadiotherapyStandard of care | Radiation | GI-6301 Vaccine, Placebo |
| wGT3X-BT ActigraphwGT3X-BT is small, non-invasive, portable watch accelerometer worn on the subject's wrist for Cycle 1 through Cycle 9 | Device | GI-6301 Vaccine, Placebo |
Primary outcome
Overall Response Rate
Time frame: Up to 24 months
Difference in overall response rate among patients with Chordoma who are treated with radiation plus placebo vs. radiation plus vaccine. Overall response rate (ORR) defined as complete response (CR) or partial response (PR) by RECIST 1.1 in the irradiated tumor site after up to 24 months among patients with Chordoma who are treated with radiation plus vaccine vs. radiation plus placebo Complete response (CR; disappearance of all target lesions and no new lesions) or partial response (PR; >=30% decrease in the sum of the greatest diameter).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34266694Wedekind MF, Widemann BC, Cote G. Chordoma: Current status, problems, and future directions. Curr Probl Cancer. 2021 Aug;45(4):100771. doi: 10.1016/j.currproblcancer.2021.100771. Epub 2021 Jul 1.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
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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 GI-6301 Vaccine (Yeast- Brachyury), Radiotherapy and wGT3X-BT Actigraph in people with Chordoma, run by NantCell, Inc. at 1 site in United States.
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 17 inclusion and 11 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 20 May 2024. 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 United States. 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 20 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 (time frame: Up to 24 months). 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 NCT02383498, 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 20 May 2024.