Ovarian CancerPhase 1PTC-001NCT03206645
Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy
A Phase 1 study testing Unesbulin in people with Ovarian Cancer, run by University of Oklahoma at 1 site in United States.
Trial summary
This is an open-label Phase Ib dose-escalation study to determine the safety, tolerability, and pharmacokinetics of Unesbulin when combined with and following conventional chemotherapy and as maintenance therapy as a capsule (weight based dosing) and a tablet (fixed dosing) in women with epithelial ovarian, fallopian… Read more about this study
- Who is it for?
- Ages 18 to 99 years, women only
- Conditions the study is for
- Ovarian Cancer
- What is being tested?
- Unesbulin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Aug 2017to 31 Mar 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histologically documented epithelial ovarian, primary peritoneal or fallopian tube carcinoma or high grade serous adenocarcinoma, endometrioid adenocarcinoma, undifferentiated carcinoma, clear cell adenocarcinoma, mixed epithelial carcinoma,or adenocarcinoma not otherwise specified (N.O.S.)
- Newly diagnosed and be previously untreated
- Adequate bone marrow function, renal function, and hepatic function
- Baseline neuropathy (sensory and motor) less than or equal to Grade 1
- Free of active infection requiring parenteral antibiotics or a serious uncontrolled medical illness or disorder within four weeks of study entry.
- Any hormonal therapy directed at the malignant tumor must be discontinued at least one week prior to registration. Continuation of hormone replacement therapy is permitted.
- Have a performance status score of 0, 1, or 2 by Eastern Cooperative Group (ECOG) criteria.
- Patients of childbearing potential must have a negative pregnancy test prior to the study entry and be practicing an effective form of contraception. If applicable, patients must discontinue breastfeeding prior to study entry.
Who cannot join
- Prior treatment with Unesbulin or standard of care drugs (cis- or carboplatin or paclitaxel.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to Unesbulin or other agents used in this study.
- Have a clinical requirement for ongoing systemic immunosuppressive therapy such as chronic steroid use not related to chemotherapy administration.
- Receiving treatment for active autoimmune disease including systemic lupus erythematosus, multiple sclerosis, inflammatory bowel disease and rheumatoid arthritis.
- History of other invasive malignancies, with the exception of non-melanoma skin cancer and other specific malignancies within the last three years. Patients are also excluded if their previous cancer treatment contraindicates this protocol therapy. Patients with concomitant endometrial cancer diagnosed at the time of their ovarian cancer are allowed to participate if the endometrial cancer is FIGO stage IB or less.
- Prior radiotherapy to any portion of the abdominal cavity or pelvis. Prior radiation for localized cancer of the breast, head and neck, or skin is permitted if completed more than three years prior to registration and the patient remains free of recurrent or metastatic disease.
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- Prior chemotherapy for any abdominal or pelvic tumor. Patients may have received prior adjuvant chemotherapy for localized breast cancer, provided that it was completed three years prior to registration, and that the patient remains free of recurrent or metastatic disease.
- Known active hepatitis, ongoing systemic bacterial, fungal, or viral infection; known human immunodeficiency virus (HIV) infection or acquired-immunodeficiency syndrome (AIDS)-related illness.
- Have concurrent severe medical problems unrelated to the malignancy that would significantly limit full compliance with the study or expose the patient to extreme risk or decreased life expectancy.
- Have childbearing potential but not practicing adequate contraception, or pregnant, or breastfeeding.
- History or active CNS disease, including primary brain tumor, seizures not controlled with standard medical therapy, any brain metastases, or history of cerebrovascular accident (CVA, stroke), transient ischemic attack (TIA) or subarachnoid hemorrhage within six months prior to study treatment.
- Concomitant therapy with any of the following: other non-study cytotoxic chemotherapy; other investigational therapies.
- Prior bone marrow/hematopoietic stem cell transplantation
- Use of an investigational drug within 4 weeks of dosing in the current study
- History of major surgical procedure within 28 days prior to start of study treatment
- Presence of history of moderate to severe pulmonary dysfunction (e.g., moderate/severe chronic obstructive pulmonary disease, post-pneumoectomy, shortness of breath with limited exertion).
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
About this study
Official title: Phase 1B Trial With Unesbulin in High-Grade Serous Ovarian Cancer: a Targeted Approach Toward Chemoresistant Stem-Like Cancer Cells
This is an open-label Phase Ib dose-escalation study to determine the safety, tolerability, and pharmacokinetics of Unesbulin when combined with and following conventional chemotherapy and as maintenance therapy as a capsule (weight based dosing) and a tablet (fixed dosing) in women with epithelial ovarian, fallopian tube or primary peritoneal cancer which is previously untreated.
Read the detailed description
For all cohorts, the study drug Unesbulin will be orally administered prior to chemotherapy (paclitaxel and carboplatin), twice a week of each 21-day cycle. Chemotherapy will be administered on day 1 of each cycle by vein. Dose escalation levels for Unesbulin will occur per protocol and doses may be de-escalated for management of toxicity. Patients will receive a total of 3 cycles of Unesbulin and chemotherapy. Surgery will be performed within 6 weeks after the last dose of Cycle 3. An additional 3-7 cycles of Unesbulin and chemotherapy (carboplatin and paclitaxel) may be given as maintenance therapy for up to two years at the discretion of the treating physician.
- Conditions
- Ovarian Cancer
- Being tested
- Unesbulin
- Medical subject headings
- Ovarian Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UnesbulinDrug
Also called PTC596
Unesbulin will be given in combination with carboplatin and paclitaxel for up to 7 cycles depending on which cohort the patient is in.
| Study group | Role | What they receive |
|---|---|---|
| Carboplatin/Paclitaxel + PTC596 | Experimental | Carboplatin AUC 6mg/L IV on day 1; Paclitaxel 175mg/m2 IV on day 1; Unesbulin PO, twice a week, on day 1, 4, 8, 11, 15 and 18 per 21-day cycle for the first 3 cycles. Receives: Drug: Unesbulin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose and dose limiting toxicities
To determine the maximum tolerated dose (MTD) and dose limiting toxicities (DLTs) of Unesbulin in combination with and following conventional chemotherapy and as maintenance as a capsule (weight-based dosing) and a tablet (fixed dosing). Standard chemotherapy will be comprised of carboplatin and weekly paclitaxel. The DLTs will be based on adverse events that occur during administration of the first 2 cycles of combination Unesbulin and chemotherapy (a cycle = 21 days).
Measured: 42 days
Number of patients able to tolerate drug combination
To examine the tolerability of the combination at the MTD of Unesbulinassessed in combination with standard neoadjuvant chemotherapy.
Measured: 3 years
Phase 2 Dose
To determine the recommended phase II dose (RP2D) of Unesbulin in combination with standard neoadjuvant chemotherapy.
Measured: 3 years
Secondary outcomes (7)
Evaluate clinical response
Evaluate clinical response by measuring serum CA-125. If CA-125 is initially above the upper normal limit, it must normalize for patients to be considered in complete clinical response.
Measured: 3 years
Evaluate pharmacokinetic Area Under the Curve (AUC)
To evaluate pharmacokinetic parameters of Unesbulin in patients with stage III or IV epithelial ovarian, primary peritoneal or fallopian tube cancer receiving neoadjuvant chemotherapy with carboplatin/paclitaxel
Measured: 3 months
Evaluate pharmacokinetic Maximum Concentration observed (Cmax)
To evaluate pharmacokinetic parameters of Unesbulin in patients with stage III or IV epithelial ovarian, primary peritoneal or fallopian tube cancer receiving neoadjuvant chemotherapy with carboplatin/paclitaxel
Measured: 3 months
Evaluate pharmacokinetic Time of Maximum Concentration observed (Tmax)
To evaluate pharmacokinetic parameters of Unesbulin in patients with stage III or IV epithelial ovarian, primary peritoneal or fallopian tube cancer receiving neoadjuvant chemotherapy with carboplatin/paclitaxel
Measured: 3 months
Evaluate pharmacokinetic half life (t1/2)
To evaluate pharmacokinetic parameters of Unesbulin in patients with stage III or IV epithelial ovarian, primary peritoneal or fallopian tube cancer receiving neoadjuvant chemotherapy with carboplatin/paclitaxel
Measured: 3 months
Progression free survival
The duration of time (one year) that patients are alive and their cancer is progression-free.
Measured: one year
Progression free survival
The duration of time (two years) that patients are alive and their cancer is progression-free.
Measured: two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2017
- Study start28 Aug 2017
- Primary completion11 Aug 2021
- Study completion31 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ovarian CancerStudies of the same condition, open and under way.
- More trials run by University of OklahomaThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This trial will utilize a traditional 3+ 3 design with dose escalation.
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Oklahoma
- Sponsor type
- University, hospital or other
- Collaborators
- PTC Therapeutics
- Responsible party
- Sponsor
- Investigators
- Kathleen Moore, MD, Study chair, University of Oklahoma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03206645
- Sponsor's protocol ID
- OU-SCC-PTC-001
- Acronym
- PTC-001
- Status last verified
- Apr 2024
- First submitted
- 1 Jun 2017
- Last updated
- 23 Apr 2024
- 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 23 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1B Trial With Unesbulin in High-Grade Serous Ovarian Cancer: a Targeted Approach Toward Chemoresistant Stem-Like Cancer Cells
- ClinicalTrials.gov ID
- NCT03206645
- Acronym / sponsor ID
- PTC-001 / OU-SCC-PTC-001
- Conditions
- Ovarian Cancer
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- University of OklahomaUniversity, hospital or other
- Collaborators
- PTC Therapeutics
- Study start
- 28 Aug 2017 (actual)
- Primary completion
- 11 Aug 2021 (actual)
- Study completion
- 31 Mar 2024 (actual)
- First posted
- 2 Jul 2017 (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.
This is an open-label Phase Ib dose-escalation study to determine the safety, tolerability, and pharmacokinetics of Unesbulin when combined with and following conventional chemotherapy and as maintenance therapy as a capsule (weight based dosing) and a tablet (fixed dosing) in women with epithelial ovarian, fallopian tube or primary peritoneal cancer which is previously untreated.
Detailed description
For all cohorts, the study drug Unesbulin will be orally administered prior to chemotherapy (paclitaxel and carboplatin), twice a week of each 21-day cycle. Chemotherapy will be administered on day 1 of each cycle by vein. Dose escalation levels for Unesbulin will occur per protocol and doses may be de-escalated for management of toxicity. Patients will receive a total of 3 cycles of Unesbulin and chemotherapy. Surgery will be performed within 6 weeks after the last dose of Cycle 3. An additional 3-7 cycles of Unesbulin and chemotherapy (carboplatin and paclitaxel) may be given as maintenance therapy for up to two years at the discretion of the treating physician.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This trial will utilize a traditional 3+ 3 design with dose escalation.
| Arm | Type | Receives |
|---|---|---|
| Carboplatin/Paclitaxel + PTC596 | Experimental | Drug: UnesbulinCarboplatin AUC 6mg/L IV on day 1; Paclitaxel 175mg/m2 IV on day 1; Unesbulin PO, twice a week, on day 1, 4, 8, 11, 15 and 18 per 21-day cycle for the first 3 cycles. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Histologically documented epithelial ovarian, primary peritoneal or fallopian tube carcinoma or high grade serous adenocarcinoma, endometrioid adenocarcinoma, undifferentiated carcinoma, clear cell adenocarcinoma, mixed epithelial carcinoma,or adenocarcinoma not otherwise specified (N.O.S.)
- Newly diagnosed and be previously untreated
- Adequate bone marrow function, renal function, and hepatic function
- Baseline neuropathy (sensory and motor) less than or equal to Grade 1
- Free of active infection requiring parenteral antibiotics or a serious uncontrolled medical illness or disorder within four weeks of study entry.
Key exclusion criteria (16 listed)
- Prior treatment with Unesbulin or standard of care drugs (cis- or carboplatin or paclitaxel.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to Unesbulin or other agents used in this study.
- Have a clinical requirement for ongoing systemic immunosuppressive therapy such as chronic steroid use not related to chemotherapy administration.
- Receiving treatment for active autoimmune disease including systemic lupus erythematosus, multiple sclerosis, inflammatory bowel disease and rheumatoid arthritis.
- History of other invasive malignancies, with the exception of non-melanoma skin cancer and other specific malignancies within the last three years. Patients are also excluded if their previous cancer treatment contraindicates this protocol therapy. Patients with concomitant endometrial cancer diagnosed at the time of their ovarian cancer are allowed to participate if the endometrial cancer is FIGO stage IB or less.
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 |
|---|---|---|
| UnesbulinAlso: PTC596Unesbulin will be given in combination with carboplatin and paclitaxel for up to 7 cycles depending on which cohort the patient is in. | Drug | Carboplatin/Paclitaxel + PTC596 |
Primary outcome
Maximum tolerated dose and dose limiting toxicities
Time frame: 42 days
To determine the maximum tolerated dose (MTD) and dose limiting toxicities (DLTs) of Unesbulin in combination with and following conventional chemotherapy and as maintenance as a capsule (weight-based dosing) and a tablet (fixed dosing). Standard chemotherapy will be comprised of carboplatin and weekly paclitaxel. The DLTs will be based on adverse events that occur during administration of the first 2 cycles of combination Unesbulin and chemotherapy (a cycle = 21 days).
Number of patients able to tolerate drug combination
Time frame: 3 years
To examine the tolerability of the combination at the MTD of Unesbulinassessed in combination with standard neoadjuvant chemotherapy.
Phase 2 Dose
Time frame: 3 years
To determine the recommended phase II dose (RP2D) of Unesbulin in combination with standard neoadjuvant chemotherapy.
7 secondary outcomes
Evaluate clinical response
Time frame: 3 years
Evaluate pharmacokinetic Area Under the Curve (AUC)
Time frame: 3 months
Evaluate pharmacokinetic Maximum Concentration observed (Cmax)
Time frame: 3 months
Evaluate pharmacokinetic Time of Maximum Concentration observed (Tmax)
Time frame: 3 months
Evaluate pharmacokinetic half life (t1/2)
Time frame: 3 months
Progression free survival
Time frame: one year
Progression free survival
Time frame: two years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2024.
- 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.
Kathleen Moore, MD study chair
University of Oklahoma
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Unesbulin in people with Ovarian Cancer, run by University of Oklahoma 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 8 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Apr 2024. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose and dose limiting toxicities (time frame: 42 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 23 Apr 2024.