Head and Neck Squamous Cell CarcinomaPhase 2MIT-001NCT04651634
MIT-001 for Prevention of CCRT-Induced OM in HNSCC Patients
A Phase 2 study testing MIT-001 plus CCRT in people with Head and Neck Squamous Cell Carcinoma and Oral Mucositis, run by MitoImmune Therapeutics at 16 sites in 2 countries.
Trial summary
The proposed study in patients with previously untreated locally advanced head and neck squamous cell carcinoma (HNSCC) is designed to evaluate the efficacy and safety of three different doses of MIT-001 compared to the placebo in prevention of oral mucositis (OM) in patients with HNSCC who are undergoing concurrent… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Head and Neck Squamous Cell Carcinoma, Oral Mucositis
- What is being tested?
- MIT-001 plus CCRT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 21 Jun 2021to 31 Dec 2025
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
- Histologically confirmed HNSCC (The American joint committee on cancer [AJCC] 8th edition, Stage II, III, IVA, or IVB), involving either the oral cavity or oropharynx, or HPV-positive Stage I oropharyngeal cancer.
- Treatment plan to receive a continuous course of intensity-modulated radiation therapy (IMRT) for definitive treatment of HNSCC delivered as single daily fractions of 1.8 to 2.5 Gy with a cumulative radiation dose between 60 and 72 Gy (EQD2 of 60 to 72 Gy, α/β ratio=10): Planned radiation treatment fields must include at least 30% of oral cavity that are planned to receive a total of 50 Gy or higher.
- CCRT plan to receive standard cisplatin monotherapy: Standard cisplatin monotherapy administered weekly (30 to 40 mg/m2), once per week for 5 to 7 continuous weeks.
- Eastern Cooperative Oncology Group Performance Status (ECOG-PS) 1 or less
- Serum pregnancy test negative for women of childbearing potential (woman of childbearing potential [WOCBP]
Who cannot join
- Patients who have active mucositis at screening.
- Planned to receive Erbitux™ (Cetuximab) or other targeted or immune therapy during the study.
- Tumor of the lips, sinuses, or salivary glands or unknown primary tumors.
- Metastatic disease (M1) Stage.
- Known history of severe vascular toxicity or allergies or intolerance to cisplatin and similar platinum-containing compounds.
- Any clinically significant and/or active infection, other systemic illness or condition (other than HNSCC) that would preclude them from participating in the study in the opinion of the Investigator.
- Prior resective surgery (4 weeks or less than 4 weeks from receiving surgery to randomization) for primary tumor under treatment for HNSCC.
Trial locations and contacts
16 sites in 2 countries.
16 sites in 2 countries
South Korea10 sites
Keimyung University Dongsan Hospital
Daegu
Chungnam National University Hospital
Daejeon
National Cancer Center
Goyang-si
Seoul National University Bundang Hospital
Gyeonggi-do, 13620
Inha University Hospital
Incheon
Jeonbuk National University Hospital
Jeonju, Jeollabuk-do, 54907
Seoul National University Hospital
Seoul, 03082
Korea University Guro Hospital
Seoul, 08308
Hanyang University Seoul Hospital
Seoul
The Catholic University of Korea Saint Vincent's Hospital
Suwon, Gyeonggi-do, 16247
United States6 sites
James Cancer Hospital Solove Research Institute
Columbus, Ohio, 43210
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234
Norris Comprehensive Cancer Center
Los Angeles, California, 90033
James P. Wilmot Cancer Center
Rochester, New York, 14642
Cancer Center of Kansas
Wichita, Kansas, 67208
Wake Forest Baptist Health - Comprehensive Cancer Center
Winston-Salem, North Carolina, 27103
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo Controlled Trial to Evaluate the Safety and Efficacy of MIT-001 in Prevention of Oral Mucositis in Patients Receiving CCRT for Locally Advanced HNSCC
The proposed study in patients with previously untreated locally advanced head and neck squamous cell carcinoma (HNSCC) is designed to evaluate the efficacy and safety of three different doses of MIT-001 compared to the placebo in prevention of oral mucositis (OM) in patients with HNSCC who are undergoing concurrent chemoradiotherapy (CCRT).
Read the detailed description
Oral mucositis associated with cancer therapy carries a significant morbidity. OM is a common complication in patients receiving CCRT used for treating HNSCC. Mucositis lesions can be painful, affect nutrition and quality of life (QoL), and have a significant economic impact. However, a definitive intervention regime has not been established. Therefore, it is essential to develop appropriate treatment.
MitoImmune Therapeutics Inc. (hereafter referred to as Sponsor) has developed MIT-001 which can scavenge abnormal levels of reactive oxygen species (ROS), enabling the cells to retain mitochondrial membrane permeability and mitochondrial function. This eventually inhibits additional ROS production, indicating that MIT-001 can prevent excessive inflammation caused by ROS. In addition, MIT-001 may possibly 1) block inflammatory cytokine production via inhibiting nuclear factor kappa B (NF kB) or inflammasome dependent pathways, 2) inhibit necrosis/necroptosis via blocking high mobility group box 1 (HMGB1) mediated cytokine production, and 3) balance regulation between T helper type 1/17 (Th1/17) and regulatory T cells.
Based on the pathophysiological progression of CCRT-associated OM, initiated by direct injury to basal epithelial cells which experience deoxyribonucleic acid (DNA) damage and increased ROS levels, Sponsor expects the prevention of OM in patients receiving CCRT of locally advanced HNSCC with MIT 001 by effectively scavenging increased ROS induced by CCRT.
- Being tested
- MIT-001 plus CCRT
- Keywords
- MIT-001, Oral Mucositis, OM, NecroX-7, CCRT-associated OM, CCRT-induced OM, Mitochondria-targeted, ROS scavenger
- Medical subject headings
- Squamous Cell Carcinoma of Head and Neck, Stomatitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MIT-001 plus CCRTDrug
MIT-001 IV-infusion plus CCRT
| Study group | Role | What they receive |
|---|---|---|
| 20 mg | Experimental | MIT-001 20 mg Receives: Drug: MIT-001 plus CCRT |
| 40 mg | Experimental | MIT-001 40 mg Receives: Drug: MIT-001 plus CCRT |
| 60 mg | Experimental | MIT-001 60 mg Receives: Drug: MIT-001 plus CCRT |
| Placebo | Placebo comparator | Matching placebo Receives: Drug: MIT-001 plus CCRT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of severe OM
severe OM (WHO criteria Grade 3 or higher) at a cumulative radiation dose of 60 Gy
Measured: From first treatment to 2 months of safety follow-up period after CCRT completion
Secondary outcomes (4)
Incidence of OM
Incidence of OM of each Grade (WHO criteria)
Measured: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Time to onset of severe OM
Time to onset of severe OM, defined as Grade 3 or higher (WHO criteria)
Measured: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Mouth pain and discomfort
Patient-reported mucositis-related mouth pain and discomfort
Measured: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Analgesic use for OM
Frequency and Cumulative dose (in morphine mg equivalent)
Measured: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2020
- Study start21 Jun 2021
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Head and Neck Squamous Cell CarcinomaStudies 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
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The three active treatment groups will receive either 20, 40, and 60 mg/day of MIT-001 IV infusion for 30 minutes, two times per week for 5 to 7 continuous weeks until a cumulative radiation dose of between 60 and 72 Gy, with CCRT. Placebo group will receive placebo, manufactured with the same properties and appearance as MIT 001, at same treatment frequency as MIT-001 with CCRT.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- MitoImmune Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jinsang Jung, M.Pharm, Study director, MitoImmune Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04651634
- Sponsor's protocol ID
- MIT001-OM-01
- Acronym
- MIT-001
- Status last verified
- Apr 2025
- First submitted
- 26 Nov 2020
- Last updated
- 6 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 6 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo Controlled Trial to Evaluate the Safety and Efficacy of MIT-001 in Prevention of Oral Mucositis in Patients Receiving CCRT for Locally Advanced HNSCC
- ClinicalTrials.gov ID
- NCT04651634
- Acronym / sponsor ID
- MIT-001 / MIT001-OM-01
- Keywords
- MIT-001, Oral Mucositis, OM, NecroX-7, CCRT-associated OM, CCRT-induced OM, Mitochondria-targeted, ROS scavenger
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- MitoImmune TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 21 Jun 2021 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 3 Dec 2020 (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.
The proposed study in patients with previously untreated locally advanced head and neck squamous cell carcinoma (HNSCC) is designed to evaluate the efficacy and safety of three different doses of MIT-001 compared to the placebo in prevention of oral mucositis (OM) in patients with HNSCC who are undergoing concurrent chemoradiotherapy (CCRT).
Detailed description
Oral mucositis associated with cancer therapy carries a significant morbidity. OM is a common complication in patients receiving CCRT used for treating HNSCC. Mucositis lesions can be painful, affect nutrition and quality of life (QoL), and have a significant economic impact. However, a definitive intervention regime has not been established. Therefore, it is essential to develop appropriate treatment.
MitoImmune Therapeutics Inc. (hereafter referred to as Sponsor) has developed MIT-001 which can scavenge abnormal levels of reactive oxygen species (ROS), enabling the cells to retain mitochondrial membrane permeability and mitochondrial function. This eventually inhibits additional ROS production, indicating that MIT-001 can prevent excessive inflammation caused by ROS. In addition, MIT-001 may possibly 1) block inflammatory cytokine production via inhibiting nuclear factor kappa B (NF kB) or inflammasome dependent pathways, 2) inhibit necrosis/necroptosis via blocking high mobility group box 1 (HMGB1) mediated cytokine production, and 3) balance regulation between T helper type 1/17 (Th1/17) and regulatory T cells.
Based on the pathophysiological progression of CCRT-associated OM, initiated by direct injury to basal epithelial cells which experience deoxyribonucleic acid (DNA) damage and increased ROS levels, Sponsor expects the prevention of OM in patients receiving CCRT of locally advanced HNSCC with MIT 001 by effectively scavenging increased ROS induced by CCRT.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- The three active treatment groups will receive either 20, 40, and 60 mg/day of MIT-001 IV infusion for 30 minutes, two times per week for 5 to 7 continuous weeks until a cumulative radiation dose of between 60 and 72 Gy, with CCRT. Placebo group will receive placebo, manufactured with the same properties and appearance as MIT 001, at same treatment frequency as MIT-001 with CCRT.
| Arm | Type | Receives |
|---|---|---|
| 20 mg | Experimental | Drug: MIT-001 plus CCRTMIT-001 20 mg |
| 40 mg | Experimental | Drug: MIT-001 plus CCRTMIT-001 40 mg |
| 60 mg | Experimental | Drug: MIT-001 plus CCRTMIT-001 60 mg |
| Placebo | Placebo comparator | Drug: MIT-001 plus CCRTMatching placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Histologically confirmed HNSCC (The American joint committee on cancer [AJCC] 8th edition, Stage II, III, IVA, or IVB), involving either the oral cavity or oropharynx, or HPV-positive Stage I oropharyngeal cancer.
- Treatment plan to receive a continuous course of intensity-modulated radiation therapy (IMRT) for definitive treatment of HNSCC delivered as single daily fractions of 1.8 to 2.5 Gy with a cumulative radiation dose between 60 and 72 Gy (EQD2 of 60 to 72 Gy, α/β ratio=10): Planned radiation treatment fields must include at least 30% of oral cavity that are planned to receive a total of 50 Gy or higher.
- CCRT plan to receive standard cisplatin monotherapy: Standard cisplatin monotherapy administered weekly (30 to 40 mg/m2), once per week for 5 to 7 continuous weeks.
- Eastern Cooperative Oncology Group Performance Status (ECOG-PS) 1 or less
- Serum pregnancy test negative for women of childbearing potential (woman of childbearing potential [WOCBP]
Key exclusion criteria (7 listed)
- Patients who have active mucositis at screening.
- Planned to receive Erbitux™ (Cetuximab) or other targeted or immune therapy during the study.
- Tumor of the lips, sinuses, or salivary glands or unknown primary tumors.
- Metastatic disease (M1) Stage.
- Known history of severe vascular toxicity or allergies or intolerance to cisplatin and similar platinum-containing compounds.
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 |
|---|---|---|
| MIT-001 plus CCRTMIT-001 IV-infusion plus CCRT | Drug | 20 mg, 40 mg, 60 mg, Placebo |
Primary outcome
Incidence of severe OM
Time frame: From first treatment to 2 months of safety follow-up period after CCRT completion
severe OM (WHO criteria Grade 3 or higher) at a cumulative radiation dose of 60 Gy
4 secondary outcomes
Incidence of OM
Time frame: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Time to onset of severe OM
Time frame: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Mouth pain and discomfort
Time frame: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Analgesic use for OM
Time frame: From first treatment to 2 months of short-term safety follow-up period after CCRT completion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Apr 2025.
- 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.
Jinsang Jung, M.Pharm study director
MitoImmune Therapeutics
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MIT-001 plus CCRT in people with Head and Neck Squamous Cell Carcinoma and Oral Mucositis, run by MitoImmune Therapeutics at 16 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 5 inclusion and 7 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: under way, not looking for participants, as of the sponsor's last update on 6 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in South Korea, 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?
This study is still under way. Results are not available in the source record yet.
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?
Incidence of severe OM (time frame: From first treatment to 2 months of safety follow-up period after CCRT completion). 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 6 Apr 2025.