Thymic Epithelial TumorPhase 2NCT05104736
PT-112 in Subjects With Thymoma and Thymic Carcinoma
A Phase 2 study testing PT-112 in people with Thymic Epithelial Tumor and Recurrent Thymoma, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: There are no approved drugs to treat recurrent thymoma and thymic carcinoma. New therapies are needed for people with these cancers. Researchers want to see if the drug PT-112 can help. PT-112 kills cancer cells. It also helps the body s immune system fight cancer. Read more about this study
- Who is it for?
- Ages 18 to 120 years
- Conditions the study is for
- Thymic Epithelial Tumor, Recurrent Thymoma, Thymic Cancer
- What is being tested?
- PT-112
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Apr 2022to 30 Jun 2028
- 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 to 120 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must have histologically confirmed thymoma or thymic carcinoma.
- Participants should have received at least one prior line of platinum-based chemotherapy. For participants who have refused cytotoxic chemotherapy, a rationale for refusal to receive standard first-line therapy will be captured in the case report form and the medical record. Progressive disease must be documented prior to study entry and participants must have advanced, unresectable disease that is not amenable to surgical resection.
- Disease must be measurable with at least 1 unidimensional measurable lesion by RECIST 1.1.
- Participants must be aged >=18 years.
- ECOG performance status <=1.
- Participants must have adequate organ and marrow function as defined below:
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- absolute neutrophil count >= 1,500/mm3 OR >= 1.5 x 10(9)/L
- platelets >=100,000/mm3 OR (Bullet) 100 x 10(9)/L
- hemoglobin >= 9g/dL (may have been transfused, at least 7 days prior)
- total bilirubin <= 1.5 x the upper limit of normal range (ULN)
- AST(SGOT)/ALT(SGPT) <= 2.5 x ULN OR <= 5 x ULN for participants with documented metastatic disease to the liver
- creatinine <= 1.5x ULN OR:
- creatinine clearance >= 60 mL/min/1.73 m2 calculated by calculated using eGRF in the clinical lab
- Negative serum or urine pregnancy test at screening for individuals of childbearing potential (IOCBP). NOTE: IOCBP is defined as any individual who has experienced menarche and who has not undergone successful surgical sterilization or who is not postmenopausal. Absence of pregnancy must be demonstrated unless there is proven menopause (age >= 50 years and last menarche >= 3 years, or documented menopausal sex hormone profile, or surgical castration) at screening.
- Participants must not become pregnant or start breast feeding during the study. Breastfeeding should be discontinued if the mother is treated with PT-112.
- Individuals of child-bearing potential and those that can father children with a sexual partner of childbearing potential must use medically effective contraception during the study and for 6 months after the last dose of study medication.
- Participants with previously treated brain or CNS metastases are eligible provided that the participant has recovered from any acute side effects of radiotherapy and does not require treatment with steroids, and any whole brain radiation therapy was completed at least 2 weeks prior to initiation of study therapy.
- Ability of participant to understand and the willingness to sign a written informed consent document.
Who cannot join
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to PT-112. Since there is no definitive list of compounds of similar chemical or biologic composition to PT-112, the principal investigator if in doubt, will report known allergies to the pharmacist to make a determination as to whether it is safe to enroll a participant.
- Concurrent treatment with a non-permitted drug.
- Concurrent anticancer treatment within 14 days before initiation of study therapy (includes radiotherapy; however, palliative bone-directed radiotherapy is permitted).
- Major surgery within 14 days before enrollment (excluding prior diagnostic biopsy).
- Concurrent systemic therapy with immunosuppressive agents within 14 days (or 5 half-lives of a drug, whichever is shorter) before initiation of study therapy.
- Use of hormonal agents for anti-cancer therapy within 14 days before initiation of study therapy; or use of any investigational drug within 14 days before initiation of study therapy.
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- History of previous malignant disease within the last 2 years with the following exceptions: basal or squamous cell carcinoma of the skin, cervical carcinoma in situ, ductal carcinoma in situ of the breast, papillary or follicular thyroid carcinoma, and non-muscle invasive bladder cancer.
- Active infection requiring systemic therapy or significant acute or chronic infections including, among others:
- Hepatitis B virus (HBV) or hepatitis C virus (HCV) infection at screening (positive HBV surface antigen or HCV RNA if anti-HCV antibody screening test positive).
- Known history of testing positive for HIV or known acquired immunodeficiency syndrome with a detectable viral load. However, participants with HIV who have an undetectable viral load and are on stable doses of Highly Active Antiretroviral Therapy (HAART) can be screened for the study.
- Persisting toxicity related to prior therapy (NCI CTCAE v. 5 Grade > 1) with the exception of, alopecia, sensory neuropathy Grade <= 2 and hearing loss Grade <=2.
- Known alcohol or drug abuse.
- Uncontrolled intercurrent illness including, but not limited to the following:
- Cardiovascular: SYMPTOMATIC congestive heart failure, unstable angina pectoris or cardiac arrhythmia, either active or within the past 6 months
- Respiratory: Pneumonitis or Idiopathic pulmonary fibrosis (not radiation-associated fibrosis), either active or within the past 6 months
- Gastrointestinal: Immune colitis or inflammatory bowel disease, either active or within the past 6 months
- Hematological: Bleeding diathesis or major bleeding events, either active or within the past 6 months
- Other: psychiatric illness/social situations that would limit compliance with study requirements, including active suicidal ideation or behavior, either active or within the past 12 months
- Administration of live vaccines within 4 weeks prior to treatment. COVID-19 vaccines are permitted at screening.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact National Cancer Institute Referral Office, 888-624-1937
About this study
Official title: A Phase II, Open-Label Trial of PT-112 in Subjects With Thymoma and Thymic Carcinoma
Background:
There are no approved drugs to treat recurrent thymoma and thymic carcinoma. New therapies are needed for people with these cancers. Researchers want to see if the drug PT-112 can help. PT-112 kills cancer cells. It also helps the body s immune system fight cancer.
Read more
Objective:
To see if the study drug PT-112 can cause tumors to shrink.
Eligibility:
People ages 18 and older who have thymoma or thymic cancer and whose disease returned or progressed after treatment with at least one platinum-containing chemotherapy, or who have refused standard treatment.
Design:
Participants will be screened with:
Review of medical history and medications
Physical exam
Blood and urine tests
CT or MRI scans of parts of the body, including the brain
Participants will get PT-112 in 28-day cycles, on days 1 and 15 of of the first cycle and on day 1 of each cycle after that. They will get the drug by infusion through a catheter. The catheter is a small plastic tube put into a vein. On days they receive the drug, participants will have physical exams and blood and urine tests. They will have an ECG to test heart function on day 1 of each cycle.
Participants will have scans every 8 weeks.
Participants may choose to have tumor biopsies on day 1 of cycles 1 and 3. Biopsies may be guided by an ultrasound or CT scan.
Participants will continue treatment as long as they can handle the side effects and their disease does not get worse, for up to 8 years.
Participants will have follow-up visits 2 weeks and 4 weeks after they stop therapy. Then the study team will check on participants every 3 months until 8 years after the participant joined the study.
Read the detailed description
Background
Platinum-based chemotherapy is the standard of care for advanced unresectable thymic epithelial tumors (TETs). However, more than half of these participants experience disease recurrence and require second-line therapy.
There are no approved drugs for treatment of recurrent thymoma and thymic carcinoma and new therapeutic options are needed for participants who have disease progression on or after platinum-containing therapy.
PT-112, a first-in-class metallo-pyrophosphate conjugate, offers a unique set of properties of both cellular interaction and molecular antitumor mechanisms, including resistance to DNA repair pathways and induction of immunogenic cell death.
PT-112 has been clinically proven to be safe and tolerable and has demonstrated efficacy.
Primary Objectives
To determine the objective response rate (ORR) based on RECIST criteria v1.1 to PT-112 in participants with relapsed or refractory TETs
Eligibility
Participants >= age 18 years with histologically confirmed, unresectable thymoma or thymic carcinoma who have previously been treated with at least one platinum-containing chemotherapy regimen or must have refused cytotoxic chemotherapy.
Participants must have progressive and measurable disease
Adequate renal, hepatic and hematopoietic function
Design
This will be a single-arm, open-label study with two cohorts (cohort 1: thymoma; cohort 2: thymic carcinoma) to determine the clinical activity and safety of PT-112 in participants with relapsed or refractory TETs.
PT-112 will be administered intravenously at a dose of 360 mg/m^2 on days 1 and 15 of cycle 1, and at 250 mg/m^2 on day 1 of each subsequent cycle, until disease progression or development of intolerable adverse events. Each cycle is 28 days.
For participants who develop intolerable toxicity at a dose of 360 mg/m^2, dose reduction will be permitted to 300 mg/m^2 after resolution of adverse events to <= grade 1 or baseline. For participants who develop intolerable toxicity at a dose of 250 mg/m^2, up to two dose reductions will be permitted to 200 mg/m^2 or 150 mg/m^2 after resolution of adverse events to <= grade 1 or baseline.
Toxicity will be assessed every cycle by CTCAE, version 5.0.
Tumor response will be assessed after completion of every other cycle (8 weeks) using RECIST criteria, version 1.1. Additionally, for participants with pleural dissemination tumor response will be assessed using International Thymic Malignancy Interest Group (ITMIG) modified RECIST criteria.
- Conditions
- Thymic Epithelial Tumor, Recurrent Thymoma, Thymic Cancer
- Being tested
- PT-112
- Keywords
- metallo-pyrophosphate, immunogenic cell death, osteotropism, damage-associated molecular patterns, Peripheral Neuropathy
- Medical subject headings
- Thymic epithelial tumor, Thymoma, Thymus Neoplasms, Peripheral Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PT-112Drug
PT-112 will be administered intravenously in 28-day cycles, on Days 1 and 15 at a dose of 360 mg/m2 for cycle 1, and on day 1 at 250mg/m2 for each subsequent cycle,
| Study group | Role | What they receive |
|---|---|---|
| PT-112 | Experimental | PT-112 will be administered intravenously in 28-day cycles, on Days 1 and 15 at a dose of 360 mg/m2 for cycle 1, and on day 1 at 250mg/m2 for each subsequent cycle, until disease progression, development of intolerable adverse events, or until 8 years after an individual participant has been on study Receives: Drug: PT-112 |
What is being measured
The primary outcome is the main question the study is designed to answer.
overall response rate (ORR)
best overall response is the best response recorded per RECIST 1.1 criteria, from the start of the treatment until disease progression/recurrence
Measured: assessed every 8 weeks while on treatment and then every 3 months after that for a maximum of 8 years from the start of study
Secondary outcomes (5)
overall response rate (ORR) based on ITMIG modified RECIST (ITMIG)
best overall response is the best response recorded per ITMIG modified RECIST criteria
Measured: start of the treatment until disease progression/recurrence for a maximum of 8 years from the start of study
overall survival (OS)
time from the date of start of treatment until death from any cause
Measured: during treatment and then every 3 months for a maximum of 8 years from the start of study
progression-free survival (PFS)
time from date of start of treatment until time of disease relapse, disease progression, or death
Measured: assessed every 8 weeks while on treatment and then every 3 months for a maximum of 8 years from the start of study
duration of response (DOR)
time measurement criteria are met for CR or PR (whichever is first recorded) until the first date that recurrent or progressive disease is objectively documented
Measured: assessed every 8 weeks while on treatment and then every 3 months for a maximum of 8 years from the start of study
safety of PT-112
type, frequency, and grade of events will be collected and reported as assessed per CTCAE criteria, version 5
Measured: safety data routinely collected from initiation of study therapy through long term follow up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2021
- Study start6 Apr 2022
- Primary completion31 Dec 2027 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thymic Epithelial TumorStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- 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
- 53 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Arun Rajan, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05104736
- Sponsor's protocol ID
- 10000317
- Other IDs
- 000317-C
- Status last verified
- 13 Oct 2026
- First submitted
- 2 Nov 2021
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD recorded in the medical record will be shared with intramural investigators upon request. All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Open-Label Trial of PT-112 in Subjects With Thymoma and Thymic Carcinoma
- ClinicalTrials.gov ID
- NCT05104736
- Acronym / sponsor ID
- 10000317
- Conditions
- Thymic Epithelial Tumor, Recurrent Thymoma, Thymic Cancer
- Keywords
- metallo-pyrophosphate, immunogenic cell death, osteotropism, damage-associated molecular patterns, Peripheral Neuropathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 53 participants (estimated)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 6 Apr 2022 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 3 Nov 2021 (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.
Background:
There are no approved drugs to treat recurrent thymoma and thymic carcinoma. New therapies are needed for people with these cancers. Researchers want to see if the drug PT-112 can help. PT-112 kills cancer cells. It also helps the body s immune system fight cancer.
Objective:
To see if the study drug PT-112 can cause tumors to shrink.
Eligibility:
People ages 18 and older who have thymoma or thymic cancer and whose disease returned or progressed after treatment with at least one platinum-containing chemotherapy, or who have refused standard treatment.
Design:
Participants will be screened with:
Review of medical history and medications
Physical exam
Blood and urine tests
CT or MRI scans of parts of the body, including the brain
Participants will get PT-112 in 28-day cycles, on days 1 and 15 of of the first cycle and on day 1 of each cycle after that. They will get the drug by infusion through a catheter. The catheter is a small plastic tube put into a vein. On days they receive the drug, participants will have physical exams and blood and urine tests. They will have an ECG to test heart function on day 1 of each cycle.
Participants will have scans every 8 weeks.
Participants may choose to have tumor biopsies on day 1 of cycles 1 and 3. Biopsies may be guided by an ultrasound or CT scan.
Participants will continue treatment as long as they can handle the side effects and their disease does not get worse, for up to 8 years.
Participants will have follow-up visits 2 weeks and 4 weeks after they stop therapy. Then the study team will check on participants every 3 months until 8 years after the participant joined the study.
Detailed description
Background
Platinum-based chemotherapy is the standard of care for advanced unresectable thymic epithelial tumors (TETs). However, more than half of these participants experience disease recurrence and require second-line therapy.
There are no approved drugs for treatment of recurrent thymoma and thymic carcinoma and new therapeutic options are needed for participants who have disease progression on or after platinum-containing therapy.
PT-112, a first-in-class metallo-pyrophosphate conjugate, offers a unique set of properties of both cellular interaction and molecular antitumor mechanisms, including resistance to DNA repair pathways and induction of immunogenic cell death.
PT-112 has been clinically proven to be safe and tolerable and has demonstrated efficacy.
Primary Objectives
To determine the objective response rate (ORR) based on RECIST criteria v1.1 to PT-112 in participants with relapsed or refractory TETs
Eligibility
Participants >= age 18 years with histologically confirmed, unresectable thymoma or thymic carcinoma who have previously been treated with at least one platinum-containing chemotherapy regimen or must have refused cytotoxic chemotherapy.
Participants must have progressive and measurable disease
Adequate renal, hepatic and hematopoietic function
Design
This will be a single-arm, open-label study with two cohorts (cohort 1: thymoma; cohort 2: thymic carcinoma) to determine the clinical activity and safety of PT-112 in participants with relapsed or refractory TETs.
PT-112 will be administered intravenously at a dose of 360 mg/m^2 on days 1 and 15 of cycle 1, and at 250 mg/m^2 on day 1 of each subsequent cycle, until disease progression or development of intolerable adverse events. Each cycle is 28 days.
For participants who develop intolerable toxicity at a dose of 360 mg/m^2, dose reduction will be permitted to 300 mg/m^2 after resolution of adverse events to <= grade 1 or baseline. For participants who develop intolerable toxicity at a dose of 250 mg/m^2, up to two dose reductions will be permitted to 200 mg/m^2 or 150 mg/m^2 after resolution of adverse events to <= grade 1 or baseline.
Toxicity will be assessed every cycle by CTCAE, version 5.0.
Tumor response will be assessed after completion of every other cycle (8 weeks) using RECIST criteria, version 1.1. Additionally, for participants with pleural dissemination tumor response will be assessed using International Thymic Malignancy Interest Group (ITMIG) modified RECIST criteria.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PT-112 | Experimental | Drug: PT-112PT-112 will be administered intravenously in 28-day cycles, on Days 1 and 15 at a dose of 360 mg/m2 for cycle 1, and on day 1 at 250mg/m2 for each subsequent cycle, until disease progression, development of intolerable adverse events, or until 8 years after an individual participant has been on study |
Who may qualify
- Ages
- 18 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Participants must have histologically confirmed thymoma or thymic carcinoma.
- Participants should have received at least one prior line of platinum-based chemotherapy. For participants who have refused cytotoxic chemotherapy, a rationale for refusal to receive standard first-line therapy will be captured in the case report form and the medical record. Progressive disease must be documented prior to study entry and participants must have advanced, unresectable disease that is not amenable to surgical resection.
- Disease must be measurable with at least 1 unidimensional measurable lesion by RECIST 1.1.
- Participants must be aged >=18 years.
- ECOG performance status <=1.
Key exclusion criteria (19 listed)
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to PT-112. Since there is no definitive list of compounds of similar chemical or biologic composition to PT-112, the principal investigator if in doubt, will report known allergies to the pharmacist to make a determination as to whether it is safe to enroll a participant.
- Concurrent treatment with a non-permitted drug.
- Concurrent anticancer treatment within 14 days before initiation of study therapy (includes radiotherapy; however, palliative bone-directed radiotherapy is permitted).
- Major surgery within 14 days before enrollment (excluding prior diagnostic biopsy).
- Concurrent systemic therapy with immunosuppressive agents within 14 days (or 5 half-lives of a drug, whichever is shorter) before initiation of study therapy.
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 |
|---|---|---|
| PT-112PT-112 will be administered intravenously in 28-day cycles, on Days 1 and 15 at a dose of 360 mg/m2 for cycle 1, and on day 1 at 250mg/m2 for each subsequent cycle, | Drug | PT-112 |
Primary outcome
overall response rate (ORR)
Time frame: assessed every 8 weeks while on treatment and then every 3 months after that for a maximum of 8 years from the start of study
best overall response is the best response recorded per RECIST 1.1 criteria, from the start of the treatment until disease progression/recurrence
5 secondary outcomes
overall response rate (ORR) based on ITMIG modified RECIST (ITMIG)
Time frame: start of the treatment until disease progression/recurrence for a maximum of 8 years from the start of study
overall survival (OS)
Time frame: during treatment and then every 3 months for a maximum of 8 years from the start of study
progression-free survival (PFS)
Time frame: assessed every 8 weeks while on treatment and then every 3 months for a maximum of 8 years from the start of study
duration of response (DOR)
Time frame: assessed every 8 weeks while on treatment and then every 3 months for a maximum of 8 years from the start of study
safety of PT-112
Time frame: safety data routinely collected from initiation of study therapy through long term follow up
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 16 Oct 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All IPD recorded in the medical record will be shared with intramural investigators upon request. All IPD recorded in the medical record will be shared with intramural investigators upon request. In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
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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.
Arun Rajan, M.D. principal investigator
National Cancer Institute (NCI)
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 PT-112 in people with Thymic Epithelial Tumor and Recurrent Thymoma, run by National Cancer Institute (NCI) 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 18 inclusion and 19 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: looking for participants now, as of the sponsor's last update on 16 Oct 2026. 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. 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?
Contact Arun Rajan, M.D. ((240) 760-6236, rajana@mail.nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
overall response rate (ORR) (time frame: assessed every 8 weeks while on treatment and then every 3 months after that for a maximum of 8 years from the start of study). 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 16 Oct 2026.