Oral MucositisPhase 1NCT00938080
Study to Assess Safety and Tolerability of AG013 in Oral Mucositis in Subjects Receiving Induction Chemotherapy for the Treatment of Cancers of the Head and Neck
A Phase 1 study testing AG013 in people with Oral Mucositis, run by ActoGeniX N.V. at 6 sites in United States.
Trial summary
The purpose of this study is to assess the safety and tolerability of AG013 (genetically modified L. lactis bacteria engineered to secrete human Trefoil Factor 1), and to explore the ability of AG013 to attenuate the course and severity of oral mucositis (OM) in subjects receiving induction chemotherapy for the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Oral Mucositis
- What is being tested?
- AG013
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 6 sitesUnited States
- Study dates
- Sep 2009to Sep 2012
- 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
- Willing and able to understand and sign the study specific Informed Consent Form (ICF) approved by the site's Institutional Review Board
- Males or females 18 years or older
- Recently diagnosed with pathologically-confirmed squamous cell carcinoma of the head and neck (e.g., oral cavity, oropharynx, hypopharynx, larynx, nasopharynx, lips, sinuses, salivary glands, unknown primary)
- Planned to receive at least two cycles of induction chemotherapy of the same regimen consisting of cisplatin/fluorouracil (PF) or cisplatin/fluorouracil/docetaxel (PFT). The planned CT cycles must be of the same length and must be a minimum of 14 days in length
- Karnofsky performance score ≥ 60%
- Screening laboratory assessments:
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- Hemoglobin ≥ 10g/dl
- White blood count ≥ 3500 cells/mm3
- Absolute neutrophil counts ≥ 1500 cells/ mm3
- Direct bilirubin ≤ 2x upper limit of normal (ULN)
- Serum AST and ALT ≤ 3 x ULN
- Serum creatinine ≤ 2 mg/dl
- Serum pregnancy test: negative for females of childbearing potential: A women is considered to be of child bearing potential unless she has had a tubal ligation or is postmenopausal (without a menstrual period for at least one year)
- Subjects of childbearing potential must agree to utilize effective contraceptive methods of birth control during study participation and for 30 days following the last treatment with IMP
- Have documented mouth pain during CT Cycle 1 (i.e., OMDQ question 2 score of ≥ 2 during CT Cycle 1)
Who cannot join
- Prior radiation to the head and neck
- Chemotherapy within 21 days prior to study start
- Presence of active infectious disease excluding oral candidiasis
- Current use of antibiotic rinses or troches
- Alcohol abuse syndrome; recovered alcoholics may be included
- Presence of OM (WHO Grade > 0)
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- Chronic immunosuppression
- Known seropositive for HIV or hepatitis B or C
- Use of investigational agent within 30 days of signing informed consent
- Teeth extractions within 7 days prior to the start of CT administration
- Female subjects who are pregnant or nursing
- Known sensitivity to any investigational agent
- Inability to give informed consent or comply with study requirements
- Unwilling or unable to complete subject diary
- Any other clinical condition, psychiatric condition or prior therapy that, in the opinion of the Investigator, would make the subject unsuitable for the study or unable or to comply with follow-up visits
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Dana Farber Cancer Institute
Boston, Massachusetts, 02115
Carolinas Medical Center
Charlotte, North Carolina, 28203
University of Illinois at Chicago
Chicago, Illinois, 60612
Vanderbilt Ingram Cancer Center
Nashville, Tennessee, 37232
Beth Israel Medical Center
New York, New York, 10003
Stanford University
Stanford, California, 94305
About this study
Official title: A Phase 1b, Multi-center, Single Blinded, Placebo-controlled, Sequential Dose Escalation Study to Assess the Safety and Tolerability of Topically Applied AG013 in Subjects Receiving Induction Chemotherapy for the Treatment of Cancers of the Head and Neck
The purpose of this study is to assess the safety and tolerability of AG013 (genetically modified L. lactis bacteria engineered to secrete human Trefoil Factor 1), and to explore the ability of AG013 to attenuate the course and severity of oral mucositis (OM) in subjects receiving induction chemotherapy for the treatment of head and neck cancer.
Read the detailed description
The purpose of this study is to investigate the safety and tolerability of AG013 in subjects who are at risk for oral mucositis (OM) and to assess clinical efficacy of AG013 in the reduction of signs and symptoms of OM in patients receiving induction chemotherapy for the treatment of their head and neck cancer.
AG013 is made up of genetically modified (GM) bacteria called Lactococcus lactis (L. lactis). Wild type L. lactis are commonly used to produce dairy products including cheeses and milk. To make AG013, the DNA of L. lactis has been changed in the laboratory to secrete a protein called human Trefoil Factor 1 (hTFF1). hTFF1 is normally secreted in saliva and intestines. Trefoil factors have been shown to be important in protecting and healing mucosal tissues, such as the tissue in the mouth, when these tissues are damaged by cancer therapies such as chemotherapy and radiation therapy.
The proposed Phase 1b clinical trial will enroll subjects with head and neck cancer who develop OM during their first cycle of treatment with induction chemotherapy. OM is a painful, common toxicity of many forms of drug and radiation therapy used for the treatment of cancer. Subjects with OM get soreness, irritation, and ulcers in the mouth and may have difficulties eating, drinking or swallowing as a result of their cancer treatment.
During the second cycle of induction chemotherapy, eligible subjects will receive AG013 or placebo for 14 consecutive days at a frequency of one rinse, three rinses or six rinses per day (sequential dose escalation design).
At least 21 subjects will be enrolled in the study. For each of the three daily dose levels, 5 subjects will be assigned to AG013 (n=5) and 2 subjects will be assigned to placebo (n=2).
Throughout the study, safety will be monitored by collecting and recording Adverse Events, laboratory assessments and the presence of sAGX0085 bacteria in blood.
An independent Data Safety Monitoring Board (DSMB) will be constituted before the enrollment of subjects in order to review safety data from each enrollment group.
- Conditions
- Oral Mucositis
- Being tested
- AG013, Placebo
- Medical subject headings
- Stomatitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AG013Biological
Mouth rinse once a day for 14 days
AG013Biological
Mouth rinse three times a day for 14 days
AG013Biological
Mouth rinse six times a day for 14 days
PlaceboOther
Mouth rinse once a day for 14 days
PlaceboOther
Mouth rinse three times a day for 14 days
PlaceboOther
Mouth rinse six times a day for 14 days
| Study group | Role | What they receive |
|---|---|---|
| AG013: one mouth rinse/day | Experimental | Not described Receives: Biological: AG013 |
| AG013: three mouth rinses/day | Experimental | Not described Receives: Biological: AG013 |
| AG013: six mouth rinses/day | Experimental | Not described Receives: Biological: AG013 |
| one mouth rinse/day | Placebo comparator | Not described Receives: Other: Placebo |
| three mouth rinses/day | Placebo comparator | Not described Receives: Other: Placebo |
| six mouth rinses/day | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety (Adverse Events, laboratory assessments and the presence of sAGX0085 bacteria in blood)
Up to 16 months
Measured: 16 months
Secondary outcomes (1)
Pharmacokinetics of serum samples, buccal smears and saliva samples
Day 1 CT cycle 2, Day 7 CT cycle 2, Day 14 CT cycle 2, End of Study
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2009 (estimated)
- Study startSep 2009
- Primary completionSep 2012
- Study completionSep 2012
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 Oral MucositisStudies 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
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- ActoGeniX N.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Barbara A Murphy, MD, Principal investigator, Vanderbilt-Ingram Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00938080
- Sponsor's protocol ID
- AG013-ODOM-101
- Other IDs
- RAC0810-942
- Status last verified
- Jan 2014
- First submitted
- 9 Jul 2009
- Last updated
- 7 Jan 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Robert S, Steidler L. Recombinant Lactococcus lactis can make the difference in antigen-specific immune tolerance induction, the Type 1 Diabetes case. Microb Cell Fact. 2014 Aug 29;13 Suppl 1(Suppl 1):S11. doi: 10.1186/1475-2859-13-S1-S11. Epub 2014 Aug 29.
PubMed 25185797
Study at a glance
From the registry record, last updated by the sponsor on 7 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Multi-center, Single Blinded, Placebo-controlled, Sequential Dose Escalation Study to Assess the Safety and Tolerability of Topically Applied AG013 in Subjects Receiving Induction Chemotherapy for the Treatment of Cancers of the Head and Neck
- ClinicalTrials.gov ID
- NCT00938080
- Acronym / sponsor ID
- AG013-ODOM-101
- Conditions
- Oral Mucositis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- ActoGeniX N.V.Industry (pharma, biotech, devices)
- Study start
- Sep 2009
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 13 Jul 2009 (estimated)
- 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.
The purpose of this study is to assess the safety and tolerability of AG013 (genetically modified L. lactis bacteria engineered to secrete human Trefoil Factor 1), and to explore the ability of AG013 to attenuate the course and severity of oral mucositis (OM) in subjects receiving induction chemotherapy for the treatment of head and neck cancer.
Detailed description
The purpose of this study is to investigate the safety and tolerability of AG013 in subjects who are at risk for oral mucositis (OM) and to assess clinical efficacy of AG013 in the reduction of signs and symptoms of OM in patients receiving induction chemotherapy for the treatment of their head and neck cancer.
AG013 is made up of genetically modified (GM) bacteria called Lactococcus lactis (L. lactis). Wild type L. lactis are commonly used to produce dairy products including cheeses and milk. To make AG013, the DNA of L. lactis has been changed in the laboratory to secrete a protein called human Trefoil Factor 1 (hTFF1). hTFF1 is normally secreted in saliva and intestines. Trefoil factors have been shown to be important in protecting and healing mucosal tissues, such as the tissue in the mouth, when these tissues are damaged by cancer therapies such as chemotherapy and radiation therapy.
The proposed Phase 1b clinical trial will enroll subjects with head and neck cancer who develop OM during their first cycle of treatment with induction chemotherapy. OM is a painful, common toxicity of many forms of drug and radiation therapy used for the treatment of cancer. Subjects with OM get soreness, irritation, and ulcers in the mouth and may have difficulties eating, drinking or swallowing as a result of their cancer treatment.
During the second cycle of induction chemotherapy, eligible subjects will receive AG013 or placebo for 14 consecutive days at a frequency of one rinse, three rinses or six rinses per day (sequential dose escalation design).
At least 21 subjects will be enrolled in the study. For each of the three daily dose levels, 5 subjects will be assigned to AG013 (n=5) and 2 subjects will be assigned to placebo (n=2).
Throughout the study, safety will be monitored by collecting and recording Adverse Events, laboratory assessments and the presence of sAGX0085 bacteria in blood.
An independent Data Safety Monitoring Board (DSMB) will be constituted before the enrollment of subjects in order to review safety data from each enrollment group.
Study design
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| AG013: one mouth rinse/day | Experimental | Biological: AG013 |
| AG013: three mouth rinses/day | Experimental | Biological: AG013 |
| AG013: six mouth rinses/day | Experimental | Biological: AG013 |
| one mouth rinse/day | Placebo comparator | Other: Placebo |
| three mouth rinses/day | Placebo comparator | Other: Placebo |
| six mouth rinses/day | Placebo comparator | Other: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Willing and able to understand and sign the study specific Informed Consent Form (ICF) approved by the site's Institutional Review Board
- Males or females 18 years or older
- Recently diagnosed with pathologically-confirmed squamous cell carcinoma of the head and neck (e.g., oral cavity, oropharynx, hypopharynx, larynx, nasopharynx, lips, sinuses, salivary glands, unknown primary)
- Planned to receive at least two cycles of induction chemotherapy of the same regimen consisting of cisplatin/fluorouracil (PF) or cisplatin/fluorouracil/docetaxel (PFT). The planned CT cycles must be of the same length and must be a minimum of 14 days in length
- Karnofsky performance score ≥ 60%
Key exclusion criteria (15 listed)
- Prior radiation to the head and neck
- Chemotherapy within 21 days prior to study start
- Presence of active infectious disease excluding oral candidiasis
- Current use of antibiotic rinses or troches
- Alcohol abuse syndrome; recovered alcoholics may be included
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 |
|---|---|---|
| AG013Mouth rinse once a day for 14 days | Biological | AG013: one mouth rinse/day |
| AG013Mouth rinse three times a day for 14 days | Biological | AG013: three mouth rinses/day |
| AG013Mouth rinse six times a day for 14 days | Biological | AG013: six mouth rinses/day |
| PlaceboMouth rinse once a day for 14 days | Other | one mouth rinse/day |
| PlaceboMouth rinse three times a day for 14 days | Other | three mouth rinses/day |
| PlaceboMouth rinse six times a day for 14 days | Other | six mouth rinses/day |
Primary outcome
Safety (Adverse Events, laboratory assessments and the presence of sAGX0085 bacteria in blood)
Time frame: 16 months
Up to 16 months
1 secondary outcome
Pharmacokinetics of serum samples, buccal smears and saliva samples
Time frame: 28 days
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 Jan 2014.
- Derived from this study: PubMed 25185797Robert S, Steidler L. Recombinant Lactococcus lactis can make the difference in antigen-specific immune tolerance induction, the Type 1 Diabetes case. Microb Cell Fact. 2014 Aug 29;13 Suppl 1(Suppl 1):S11. doi: 10.1186/1475-2859-13-S1-S11. Epub 2014 Aug 29.
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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.
Barbara A Murphy, MD principal investigator
Vanderbilt-Ingram Cancer Center
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AG013 in people with Oral Mucositis, run by ActoGeniX N.V. at 6 sites 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 15 inclusion and 15 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 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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 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?
Safety (Adverse Events, laboratory assessments and the presence of sAGX0085 bacteria in blood) (time frame: 16 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 NCT00938080, 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 7 Jan 2014.