Peripheral Nerve TumorsPhase 2NCT02986919
Study of CPI-0610 in Patients With Malignant Peripheral Nerve Sheath Tumors
A Phase 2 study testing CPI-0610 in people with Peripheral Nerve Tumors, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
Establish safety and toxicity profile and preliminary response rate of CPI-0610 in MPNST patients and correlate response with pharmacodynamics markers and BET inhibition. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Peripheral Nerve Tumors
- What is being tested?
- CPI-0610
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 5 May 2017to 17 May 2018
- Phase
- Phase 2
- 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
- Age >18 years
- Must have histologically confirmed diagnosis of MPNST
- Must have measurable disease by CT scan or MRI
- Eastern Cooperative Oncology Group - ECOG performance status <2
- Adequate organ and marrow function as defined below:
- absolute neutrophil count greater than or equal to 1,000/mcL
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- platelets greater than or equal to 75,000/mcL
- total bilirubin <2X normal institutional limits
- AST(SGOT)/ALT(SPGT) greater than or equal to 2.5 X institutional upper limit of normal
- creatinine <2X institutional upper limit of normal
- Patients must have fully recovered from major surgery and from the acute toxic effects of prior chemotherapy and radiotherapy - residual grade 1 toxicity, e.g., grade 1 peripheral neuropathy and residual alopecia are allowed.
- Female patients who are pre-menopausal or have experienced menopause for less than 2 years must have a negative serum pregnancy test <72 hours before starting study treatment. Male and female patients with reproductive potential must agree to use appropriate contraceptive methods while on study and for 3 months after the last dose of CPI-0610. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
Who cannot join
- Current infection with HIV, hepatitis B or hepatitis C. Patients will have serologic testing performed during screening for HIV and hepatitis B and C. Any serologic results suggestive of an ongoing viral infection will be further investigated as necessary to clarify the patient's status.
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of CPI-0610, including any unresolved nausea, vomiting, or diarrhea that is CTCAE grade >1.
- Impaired cardiac function or clinically significant cardiac diseases, including any of the following:
- Acute myocardial infarction or angina pectoris <6 months prior to starting study drug
- Uncontrolled cardiac arrhythmia - patients with rate-controlled atrial fibrillation are not excluded.
- A past medical history of other clinically significant cardiovascular disease - e.g., uncontrolled hypertension, history of labile hypertension or history of poor compliance with an antihypertensive regimen.
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- Any other concurrent severe and/or uncontrolled concomitant medical condition that could compromise participation in the study - e.g., clinically significant pulmonary disease, clinically significant neurological disorder, active or uncontrolled infection.
- Systemic anti-cancer treatment or radiotherapy less than 2 weeks before the first dose of CPI-0610.
- Treatment with an investigational small molecule less than 2 weeks before the first dose of CPI-0610.
- Immunosuppressive treatment that cannot be discontinued prior to study entry and for the duration of the study. Immunosuppressive treatment should be discontinued for at least 1 week prior to start of the administration of CPI-0610. Oral prednisone at a dose of 10mg or less per day is allowed, as are other oral corticosteroids given at glucocorticoid-equivalent doses. Topical, nasal and inhaled corticosteroids are also allowed.
- Pregnant or lactating women.
- Women of child bearing potential and men with reproductive potential, if they are unwilling to use adequate contraception while on study therapy and for 3 months thereafter.
- Use of strong CYP inhibitors or drugs that carry a definite risk of Torsades de Pointes.
- Patients unwilling or unable to comply with the study protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
About this study
Official title: A Phase 2 Study of CPI-0610, a Small Molecule Inhibitor of Bromodomain and Extra-Terminal (BET) Proteins, in Patients With Malignant Peripheral Nerve Sheath Tumors
Establish safety and toxicity profile and preliminary response rate of CPI-0610 in MPNST patients and correlate response with pharmacodynamics markers and BET inhibition.
Read the detailed description
- Optional tumor biopsy will be obtained prior to Day 1 of CPI-0610 administration.
- CPI-0610 will be administered 200mg orally once a daily for 14 consecutive days followed by a 7-day break. The 14 days of CPI-0610 dosing and the 7-day break together constitute 1 cycle of treatment. The dose will not be adjusted for body weight or body surface area.
- Patients should be instructed to take their daily dose at approximately the same time of day. Each dose should be taken with a glass of water and consumed over as short a time as possible-e.g., within 5 minutes. Patients should be instructed to swallow the tablet whole and to not chew or cut them.
- Doses may be taken either with or without food.
- If vomiting occurs during the course of treatment, then no re-dosing of the patient is allowed before the next scheduled dose.
- If the patient forgets to take his/her daily morning dose, then he/she should take CPI-0610 within 6 hours after the missed dose. If more than 6 hours have passed, then that day's dose should be omitted, and the patient should resume treatment with the next scheduled dose.
- Repeat optional tumor biopsy will be obtained on day 8 of CPI-0610 treatment.
Toxicities and Dosing Delays/Dose Modifications During a cycle of treatment, CPI-0610 should continue to be administered as planned unless CTCAE grade 3-4 toxicities occur. In the case of CPI-0610-related neutropenia and/or thrombocytopenia, dosing as planned should continue as long as the ANC remains >0.5x109/L and the platelet count remains >25x109/L. Should either the ANC or platelet count fall below these values-become CTCAE grade 4-dosing with CPI-0610 should be interrupted. CPI-0610 dosing within the planned 14 days of treatment should not be resumed until the ANC is >0.75x109/L and the platelet count is >50x109/L. In addition, dosing within the cycle of treatment should not be resumed if the interruption has resulted in the omission of more than 2 of the planned 14 days of dosing. If it is possible to resume dosing within the planned 14 days of treatment, no attempt should be made to make up the missed doses of CPI-0610. If more than 2 of the planned 14 days of therapy have been omitted, then treatment should be resumed only with a new cycle of treatment, if the ANC>1x109/L and the platelet count >75x109/L. In addition, all other toxicities considered to be related to CPI-0610 must have resolved to CTCAE grade 1 or baseline. If the patient fails to meet the above-cited criteria for retreatment, then initiation of the next cycle of treatment should be delayed by one week. Following the additional week of no treatment, the next cycle may begin if the patient's ANC is>1x109/L and the platelet count is >75x109/L. In addition, all other toxicities considered to be related to CPI-0610 must have resolved to CTCAE grade 1 or baseline. If there are more than 28 days between the start of one cycle and the start of the next, the patient will no longer receive CPI-0610 therapy unless the patient's MPNST is stable or responding to therapy. Then consideration may be given to resuming treatment at a lower dose level to be discussed with Sponsor and Institutional Review Board (IRB). When a reduction in dose of CPI-0610 is required, no re-escalation of dose will be permitted.
Exceptions to the toxicity delay are CTCAE elevations in alkaline phosphatase and uric acid and <72 hours of grade 3 fatigue. When laboratory abnormalities form the basis of treatment decisions, they should be confirmed by repeated testing with a new blood sample or procedure. Optimal therapy for vomiting or diarrhea is based on physician preference with consideration of the prohibited medications listed in the appendix. G-CSF may be used to treat patients who have developed dose-limiting neutropenia, as per institutional guidelines, following discontinuation of CPI-0610 treatment. However, G-CSF may not be used during CPI-0610 administration or during the treatment break. Patients should not have dose reductions of CPI-0610 unless a grade 3-4 toxicity occurs and retreatment is not possible within the 28 day period. Then the patient is withdrawn from study unless the patient's MPNST has responded or stable on therapy. Then consideration may be given to resuming treatment at a lower dose level to be discussed with Sponsor and IRB. When a reduction in the dose of CPI-0610 is required, no re-escalation of dose will be permitted. Patients whose treatment is interrupted or permanently discontinued because of toxicities must be followed until the toxicity resolves or stabilizes.
- Conditions
- Peripheral Nerve Tumors
- Being tested
- CPI-0610
- Keywords
- Malignant peripheral Nerve Sheath Tumors (MPNST); Neurofibromatosis sarcoma
- Medical subject headings
- Peripheral Nervous System Neoplasms, Neurofibrosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPI-0610Drug
- Optional tumor biopsy will be obtained prior to Day 1 of CPI-0610 administration.
- CPI-0610 will be administered 200mg orally once a daily for 14 consecutive days followed by a 7-day break. The 14 days of CPI-0610 dosing and the 7-day break together constitute 1 cycle of treatment. The dose will not be adjusted for body weight or body surface area.
| Study group | Role | What they receive |
|---|---|---|
| CPI-0610 Treatment | Experimental | CPI-0610 will be administered 200mg orally once a daily for 14 consecutive days followed by a 7-day break. Receives: Drug: CPI-0610 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate of CPI-0610
Establish the response rate of CPI-0610 in MPNST patients
Measured: 21 day cycles for 84 days
Duration of CPI-0610
Establish the response duration of CPI-0610 in MPNST patients
Measured: 21 day cycles for 84 days
Adverse events associated with CPI-0610
Describe the adverse events associated with CPI-0610 at the RP2D
Measured: 21 day cycles for 84 days
Secondary outcomes (1)
Correlate tumor exposure to CPI-0610 with tumor BIM1 expression
Correlate tumor exposure to CPI-0610 with tumor BIM1 expression
Measured: 21 day cycles for 84 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2016 (estimated)
- Study start5 May 2017
- Primary completion17 May 2018
- Study completion17 May 2018
Why it stopped: Lack of enrollment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Peripheral Nerve TumorsStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02986919
- Sponsor's protocol ID
- STU 092016-074
- Status last verified
- Sep 2018
- First submitted
- 21 Nov 2016
- Last updated
- 11 Sep 2018
- 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 11 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of CPI-0610, a Small Molecule Inhibitor of Bromodomain and Extra-Terminal (BET) Proteins, in Patients With Malignant Peripheral Nerve Sheath Tumors
- ClinicalTrials.gov ID
- NCT02986919
- Acronym / sponsor ID
- STU 092016-074
- Conditions
- Peripheral Nerve Tumors
- Keywords
- Malignant peripheral Nerve Sheath Tumors (MPNST); Neurofibromatosis sarcoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of enrollment.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Study start
- 5 May 2017 (actual)
- Primary completion
- 17 May 2018 (actual)
- Study completion
- 17 May 2018 (actual)
- First posted
- 8 Dec 2016 (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.
Establish safety and toxicity profile and preliminary response rate of CPI-0610 in MPNST patients and correlate response with pharmacodynamics markers and BET inhibition.
Detailed description
- Optional tumor biopsy will be obtained prior to Day 1 of CPI-0610 administration.
- CPI-0610 will be administered 200mg orally once a daily for 14 consecutive days followed by a 7-day break. The 14 days of CPI-0610 dosing and the 7-day break together constitute 1 cycle of treatment. The dose will not be adjusted for body weight or body surface area.
- Patients should be instructed to take their daily dose at approximately the same time of day. Each dose should be taken with a glass of water and consumed over as short a time as possible-e.g., within 5 minutes. Patients should be instructed to swallow the tablet whole and to not chew or cut them.
- Doses may be taken either with or without food.
- If vomiting occurs during the course of treatment, then no re-dosing of the patient is allowed before the next scheduled dose.
- If the patient forgets to take his/her daily morning dose, then he/she should take CPI-0610 within 6 hours after the missed dose. If more than 6 hours have passed, then that day's dose should be omitted, and the patient should resume treatment with the next scheduled dose.
- Repeat optional tumor biopsy will be obtained on day 8 of CPI-0610 treatment.
Toxicities and Dosing Delays/Dose Modifications During a cycle of treatment, CPI-0610 should continue to be administered as planned unless CTCAE grade 3-4 toxicities occur. In the case of CPI-0610-related neutropenia and/or thrombocytopenia, dosing as planned should continue as long as the ANC remains >0.5x109/L and the platelet count remains >25x109/L. Should either the ANC or platelet count fall below these values-become CTCAE grade 4-dosing with CPI-0610 should be interrupted. CPI-0610 dosing within the planned 14 days of treatment should not be resumed until the ANC is >0.75x109/L and the platelet count is >50x109/L. In addition, dosing within the cycle of treatment should not be resumed if the interruption has resulted in the omission of more than 2 of the planned 14 days of dosing. If it is possible to resume dosing within the planned 14 days of treatment, no attempt should be made to make up the missed doses of CPI-0610. If more than 2 of the planned 14 days of therapy have been omitted, then treatment should be resumed only with a new cycle of treatment, if the ANC>1x109/L and the platelet count >75x109/L. In addition, all other toxicities considered to be related to CPI-0610 must have resolved to CTCAE grade 1 or baseline. If the patient fails to meet the above-cited criteria for retreatment, then initiation of the next cycle of treatment should be delayed by one week. Following the additional week of no treatment, the next cycle may begin if the patient's ANC is>1x109/L and the platelet count is >75x109/L. In addition, all other toxicities considered to be related to CPI-0610 must have resolved to CTCAE grade 1 or baseline. If there are more than 28 days between the start of one cycle and the start of the next, the patient will no longer receive CPI-0610 therapy unless the patient's MPNST is stable or responding to therapy. Then consideration may be given to resuming treatment at a lower dose level to be discussed with Sponsor and Institutional Review Board (IRB). When a reduction in dose of CPI-0610 is required, no re-escalation of dose will be permitted.
Exceptions to the toxicity delay are CTCAE elevations in alkaline phosphatase and uric acid and <72 hours of grade 3 fatigue. When laboratory abnormalities form the basis of treatment decisions, they should be confirmed by repeated testing with a new blood sample or procedure. Optimal therapy for vomiting or diarrhea is based on physician preference with consideration of the prohibited medications listed in the appendix. G-CSF may be used to treat patients who have developed dose-limiting neutropenia, as per institutional guidelines, following discontinuation of CPI-0610 treatment. However, G-CSF may not be used during CPI-0610 administration or during the treatment break. Patients should not have dose reductions of CPI-0610 unless a grade 3-4 toxicity occurs and retreatment is not possible within the 28 day period. Then the patient is withdrawn from study unless the patient's MPNST has responded or stable on therapy. Then consideration may be given to resuming treatment at a lower dose level to be discussed with Sponsor and IRB. When a reduction in the dose of CPI-0610 is required, no re-escalation of dose will be permitted. Patients whose treatment is interrupted or permanently discontinued because of toxicities must be followed until the toxicity resolves or stabilizes.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CPI-0610 Treatment | Experimental | Drug: CPI-0610CPI-0610 will be administered 200mg orally once a daily for 14 consecutive days followed by a 7-day break. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Age >18 years
- Must have histologically confirmed diagnosis of MPNST
- Must have measurable disease by CT scan or MRI
- Eastern Cooperative Oncology Group - ECOG performance status <2
- Adequate organ and marrow function as defined below:
Key exclusion criteria (14 listed)
- Current infection with HIV, hepatitis B or hepatitis C. Patients will have serologic testing performed during screening for HIV and hepatitis B and C. Any serologic results suggestive of an ongoing viral infection will be further investigated as necessary to clarify the patient's status.
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of CPI-0610, including any unresolved nausea, vomiting, or diarrhea that is CTCAE grade >1.
- Impaired cardiac function or clinically significant cardiac diseases, including any of the following:
- Acute myocardial infarction or angina pectoris <6 months prior to starting study drug
- Uncontrolled cardiac arrhythmia - patients with rate-controlled atrial fibrillation are not excluded.
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 |
|---|---|---|
| CPI-0610* Optional tumor biopsy will be obtained prior to Day 1 of CPI-0610 administration. * CPI-0610 will be administered 200mg orally once a daily for 14 consecutive days followed by a 7-day break. The 14 days of CPI-0610 dosing and the 7-day break together constitute 1 cycle of treatment. The dose will not be adjusted for body weight or body surface area. | Drug | CPI-0610 Treatment |
Primary outcome
Response rate of CPI-0610
Time frame: 21 day cycles for 84 days
Establish the response rate of CPI-0610 in MPNST patients
Duration of CPI-0610
Time frame: 21 day cycles for 84 days
Establish the response duration of CPI-0610 in MPNST patients
Adverse events associated with CPI-0610
Time frame: 21 day cycles for 84 days
Describe the adverse events associated with CPI-0610 at the RP2D
1 secondary outcome
Correlate tumor exposure to CPI-0610 with tumor BIM1 expression
Time frame: 21 day cycles for 84 days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2018.
- 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.
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 CPI-0610 in people with Peripheral Nerve Tumors, run by University of Texas Southwestern Medical Center 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 12 inclusion and 14 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: withdrawn, as of the sponsor's last update on 11 Sep 2018. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Response rate of CPI-0610 (time frame: 21 day cycles for 84 days). The study lists 3 primary outcomes in all. 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 11 Sep 2018.