Peripheral Nerve Sheath TumorPhase 2NCT04917042
Tazemetostat in Malignant Peripheral Nerve Sheath Tumors
A Phase 2 study testing Tazemetostat in people with Peripheral Nerve Sheath Tumor, run by University of Florida at 1 site in United States.
Trial summary
This phase 2, open label, single arm study will investigate the use of tazemetostat in patients with recurrent/refractory and/or metastatic malignant peripheral nerve sheath tumors. Read more about this study
- Who is it for?
- Ages 12 to 99 years
- Conditions the study is for
- Peripheral Nerve Sheath Tumor
- What is being tested?
- Tazemetostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Aug 2021to Dec 2026
- 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 12 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- A histologic confirmation of recurrent/refractory and/or metastatic malignant peripheral nerve sheath tumor with Response Evaluation Criteria in Solid Tumors (RECIST) measurable disease
- Patients ≥ 12 years of age at the time of enrollment
- Performance status: 12-15 years old: Lansky > 50; 16-17 years old: Karnofsky > 50; ≥ 18 years old: Eastern Cooperative Group (ECOG) score 0-2
- Subjects must have adequately recovered from the acute toxic effects of all prior anti-cancer therapy per enrolling physician and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment.
- Anti-cancer agents known to be Myelosuppressive: ≥ 28 days after the last dose of agent.
- Anti-cancer agents not known to be myelosuppressive: > 7 days after the last dose of agent.
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- Antibodies: > 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to Grade < 1.
- Systemic Corticosteroids: if related to prior therapy > 14 days must have elapsed, or on stable dose for treatment of CNS disease.
- Hematopoietic growth factors: > 14 days after the last dose of a long-acting growth factor.
- Interleukins, Interferons, and Cytokines (other than hematopoietic growth factors): > 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors).
- Radiation therapy (XRT)/External Beam Irradiation including Protons: > 14 days after local XRT; > 150 days after traumatic brain injury, craniospinal XRT or if radiation to > 50% of the pelvis; > 42 days if other substantial bone marow radiation.
- Radiopharmaceutical therapy: > 42 days after systemically administered radiopharmaceutical therapy.
- Major surgery > 14 days prior, with evidence of wound healing and no active surgical complications
- Subjects must not have had prior exposure to Tazemetostat or other inhibitor(s) of EZH2
- Adequate laboratory values of organ function, defined as:
- Peripheral absolute neutrophil count (ANC) > 1000/mm3.
- Platelet count > 100,000/mm2 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).
- Hemoglobin > 8.0 g/dL at baseline (may receive RBC transfusions).
- Creatinine clearance or radioisotope GFR > 70 ml/min/1.73 m2, or serum creatinine based on age/gender
- Total bilirubin < 1.5 ULN or direct bilirubin < 1 x ULN.
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 2.5 x ULN; if liver metastases present, then AST and ALT must be < 5 x ULN.
- Serum albumin > 2 g/dL.
- Coagulation INR < 1.5, while on anti-coagulation INR < 2.5.
- Nervous system disorders (CTCAE v5.0) resulting from prior therapy must be < Grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible.
- Subjects must not have more than one active malignancy at the time of enrollment
- Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures. All subjects and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional standard practice.
- Use of contraception:
- Women of childbearing potential (WOCBP) who are heterosexually active must be using two highly effective methods of contraception to avoid pregnancy throughout the study and for at least 6 months after the last dose of study drug to minimize the risk of pregnancy as Tazemetostat might counteract the effects of hormonal contraceptives. Birth control methods that can be used while in this study include: established use of oral, injected or implanted hormonal birth control or placement of an intrauterine device [IUD] or intrauterine system [IUS]. They or their partner must also use a second method, (e.g., condom with spermicidal foam/gel/film/cream/suppository or occlusive cap [diaphragm or cervical/vault caps] with spermicidal foam/gel/film/cream/suppository. If their male partner is vasectomized, they do not need to use any of the birth control methods listed above. The type of birth control they use must be discussed with the study doctor before beginning the study. The study doctor must approve the method you use before they can enter the study. Prior to study enrollment, women of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factors for an unintentional pregnancy.
- Males with female partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms,vasectomy) throughout the study and should avoid donating sperm, for 90 days following the last dose of study drug.
Who cannot join
- Subjects who are currently taking the following concomitant medications:
- Anti-cancer Agents: Subjects who are currently receiving other anti-cancer agents are not eligible.
- CYP3A4 Agents: Subjects who are currently receiving drugs that are strong inducers or strong inhibitors of CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 are prohibited from 14 days prior to the first dose of Tazemetostat to the end of the study. Note: Dexamethasone for CNS tumors or metastases, on a stable dose, is allowed.
- Grapefruit, grapefruit juice, Seville oranges and food/drinks containing them should be avoided one week before the first dose of the study intervention
- Subjects who are acutely ill with an uncontrolled active infection on systemic anti-infective agents are not eligible.
- Subjects with a prior history of T-cell lymphoblastic lymphoma/T-cell acute lymphoblastic leukemia, myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL) or other myeloproliferative neoplasm (MPN).
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- Subjects who have any of the following underlying major cardiac issues or conditions:
- Known QTc prolongation or documentation
- Documented New York Heart Association (NYHA) Class III or IV congestive heart failure.
- Myocardial infarction within 6 months prior to registration.
- Unstable angina within 6 months prior to registration.
- Symptomatic arrhythmia.
- Subjects who in the opinion of the investigator may be high risk for treatment complications or unable to comply with the safety monitoring requirements of the study
- Heterosexually active males or females of reproductive potential may not participate unless they have agreed to use two highly effective contraceptive methods for the duration of study treatment as Tazemetostat might counteract the effects of hormonal contraceptives. Female subjects of childbearing potential should agree to remain abstinent or use adequate contraceptive methods for 6 months after the last dose of Tazemetostat. Male subjects should agree to remain abstinent or use adequate contraceptive methods, and agree to refrain from donating sperm, and for 90 days after the last dose of Tazemetostat.
- Females who are pregnant or breastfeeding will not be entered on this study because there is currently no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal.
- Administration of a vaccine containing live virus within 30 days prior to the first dose of trial treatment. Note: Most flu vaccines are killed viruses, with the exception of the intra-nasal vainer (Flu-Mist) which is an attenuated live virus and therefore prohibited for 30 days prior to first dose. Non-live versions of the COVID-19 vaccine are allowed.
- Prisoners or subjects who are involuntarily incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.
- Known hypersensitivity to tazmetostat or any component of the formulation of tazemetostat
- Inability to take oral medication OR have malabsorption syndrome or any other uncontrolled gastrointestinal condition (eg, nausea, diarrhea, vomiting) that might impair the bioavailability of tazmetostat
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase 2 Study Using Tazemetostat in Patients With Recurrent/Refractory and/or Metastatic Malignant Peripheral Nerve Sheath Tumors (MPNST)
This phase 2, open label, single arm study will investigate the use of tazemetostat in patients with recurrent/refractory and/or metastatic malignant peripheral nerve sheath tumors.
- Conditions
- Peripheral Nerve Sheath Tumor
- Being tested
- Tazemetostat
- Keywords
- metastatic malignant peripheral nerve sheath tumors, Enhancer of zeste homolog 2 (EZH2) inhibition, sarcoma, pediatric, tazemetostat
- Medical subject headings
- Nerve Sheath Neoplasms, Neurofibrosarcoma, Inhibition, Psychological, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TazemetostatDrug
Also called TAZVERIK
Subjects will receive 520 mg/m2/dose (subjects 12-17 years old) or 800 mg of tazemetostat orally twice daily in 28 day cycles. Subjects will receive treatment with tazemetostat up to 2 years or until disease progression or unacceptable toxicity occurs.
| Study group | Role | What they receive |
|---|---|---|
| tazemetostat | Experimental | Not described Receives: Drug: Tazemetostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate
Assess the objective response rate, defined as the proportion of subjects who achieve either a complete response or partial response based on radiographic evaluation of treatment response via RECIST1.1. A complete response is the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to < 10 mm. A partial response is at least a 30% decrease in the sum of diameter of target lesions, taking as reference the baseline sum diameters.
Measured: 143 days
Secondary outcomes (4)
Progression Free Survival
Determine the progression free survival, defined as the length of time from the date of start of treatment to the date of disease progression per RECIST 1.1 criteria. Per RECIST 1.1 criteria, disease progression is defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (including the baseline sum if that is the smallest). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of 1 or more new lesions is also considered progression.
Measured: 143 days
Time to Progression
Determine the time to progression, defined as the length of time from the start of treatment until disease progression by RECIST 1.1 criteria. Per RECIST 1.1 criteria, disease progression is defined as at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (including the baseline sum if that is the smallest). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of 1 or more new lesions is also considered progression.
Measured: 143 days
Clinical Benefit
Determine the clinical benefit using the Numbered Pain Rating Scale. For this scale, the subject rates their pain on a scale of 0 to 10. Zero means "no pain," and 10 means "the worst possible pain." For each patient, the mean pain rating scale score was computed by adding all the non-missing scores the patient had and then taking the average.
Measured: 143 days
Clinical Benefit Rate
Assess the clinical benefit rate, defined as the proportion of subjects with complete or partial response or stable disease (by RECIST 1.1 criteria) lasting at least 4 months.
Per RECiST 1.1 criteria, a complete response is the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to < 10 mm. A partial response is at least a 30% decrease in the sum of diameter of target lesions, taking as reference the baseline sum diameters. Stable disease is defined as having neither sufficient shrinkage for a partial response nor sufficient increase for progressive disease, taking as reference the smallest sum diameters while on study.
Measured: 143 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2021
- Study start25 Aug 2021
- Primary completion14 Feb 2025
- Study completionDec 2026 (estimated)
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.
- Other trials for Peripheral Nerve Sheath TumorStudies of the same condition, open and under way.
- More trials run by University of FloridaThe 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
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Epizyme, Inc.
- Responsible party
- Sponsor
- Investigators
- Joanne Lagmay, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04917042
- Sponsor's protocol ID
- UF-SC-001
- Other IDs
- IRB202100865, OCR40197
- Status last verified
- May 2026
- First submitted
- 1 Jun 2021
- Last updated
- 4 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study Using Tazemetostat in Patients With Recurrent/Refractory and/or Metastatic Malignant Peripheral Nerve Sheath Tumors (MPNST)
- ClinicalTrials.gov ID
- NCT04917042
- Acronym / sponsor ID
- UF-SC-001
- Conditions
- Peripheral Nerve Sheath Tumor
- Keywords
- metastatic malignant peripheral nerve sheath tumors, Enhancer of zeste homolog 2 (EZH2) inhibition, sarcoma, pediatric, tazemetostat
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Epizyme, Inc.
- Study start
- 25 Aug 2021 (actual)
- Primary completion
- 14 Feb 2025 (actual)
- Study completion
- Dec 2026 (estimated)
- First posted
- 8 Jun 2021 (actual)
- Results
- Posted 4 Feb 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase 2, open label, single arm study will investigate the use of tazemetostat in patients with recurrent/refractory and/or metastatic malignant peripheral nerve sheath tumors.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| tazemetostat | Experimental | Drug: Tazemetostat |
Who may qualify
- Ages
- 12 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (29 listed)
- A histologic confirmation of recurrent/refractory and/or metastatic malignant peripheral nerve sheath tumor with Response Evaluation Criteria in Solid Tumors (RECIST) measurable disease
- Patients ≥ 12 years of age at the time of enrollment
- Performance status: 12-15 years old: Lansky > 50; 16-17 years old: Karnofsky > 50; ≥ 18 years old: Eastern Cooperative Group (ECOG) score 0-2
- Subjects must have adequately recovered from the acute toxic effects of all prior anti-cancer therapy per enrolling physician and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment.
- Subjects must not have had prior exposure to Tazemetostat or other inhibitor(s) of EZH2
Key exclusion criteria (19 listed)
- Subjects who are currently taking the following concomitant medications:
- Subjects who are acutely ill with an uncontrolled active infection on systemic anti-infective agents are not eligible.
- Subjects with a prior history of T-cell lymphoblastic lymphoma/T-cell acute lymphoblastic leukemia, myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL) or other myeloproliferative neoplasm (MPN).
- Subjects who have any of the following underlying major cardiac issues or conditions:
- Subjects who in the opinion of the investigator may be high risk for treatment complications or unable to comply with the safety monitoring requirements of the study
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 |
|---|---|---|
| TazemetostatAlso: TAZVERIKSubjects will receive 520 mg/m2/dose (subjects 12-17 years old) or 800 mg of tazemetostat orally twice daily in 28 day cycles. Subjects will receive treatment with tazemetostat up to 2 years or until disease progression or unacceptable toxicity occurs. | Drug | tazemetostat |
Primary outcome
Objective Response Rate
Time frame: 143 days
Assess the objective response rate, defined as the proportion of subjects who achieve either a complete response or partial response based on radiographic evaluation of treatment response via RECIST1.1. A complete response is the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to < 10 mm. A partial response is at least a 30% decrease in the sum of diameter of target lesions, taking as reference the baseline sum diameters.
4 secondary outcomes
Progression Free Survival
Time frame: 143 days
Time to Progression
Time frame: 143 days
Clinical Benefit
Time frame: 143 days
Clinical Benefit Rate
Time frame: 143 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Joanne Lagmay, MD principal investigator
University of Florida
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 Tazemetostat in people with Peripheral Nerve Sheath Tumor, run by University of Florida 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 29 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: under way, not looking for participants, as of the sponsor's last update on 4 Jun 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?
Yes. Results are posted in the registry (first posted 4 Feb 2026). 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?
Objective Response Rate (time frame: 143 days). 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 4 Jun 2026.