PEComa, MalignantNCT03817515
Expanded Access for ABI-009 in Patients With Advanced PEComa and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation
A study testing ABI-009 in people with PEComa, Malignant and TSC1, run by Aadi Bioscience, Inc..
Trial summary
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR Pathway Activation Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- PEComa, Malignant, TSC1, TSC2
- What is being tested?
- ABI-009
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 85 years
- All sexes
- Adult, Older adult
Who may be able to join
- A patient will be eligible for inclusion in this expanded access program only if all of the following criteria are met:
- a) Diagnosis of advanced (locally advanced and inoperable or metastatic) malignant PEComa confirmed by pathology and immunohistochemistry, whether or not previously exposed to an mTOR inhibitor, or b) diagnosis of any other malignancy with activation in any mTOR pathway component as identified by immunohistochemistry or an identified relevant rare genetic mutation in mTOR pathway genes, including but not limited to TSC1, TSC2, PIK3CA, PTEN, for which there are no FDA-approved treatments or no other comparable or satisfactory alternative therapies available whether or not they been previously exposed to a mTOR inhibitor.
- 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
- Acceptable blood chemistry levels at screening (obtained ≤14 days prior to enrollment (local laboratory) including:
- total bilirubin ≤1.5 x upper limit of normal (ULN)
- AST ≤2.5 x ULN (≤5 x ULN if attributable to liver metastases)
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- serum creatinine ≤1.5 x ULN
- Adequate biological parameters as demonstrated by the following blood counts at screening (obtained ≤14 days prior to enrollment, local laboratory):
- Absolute neutrophil count (ANC) ≥1.5 × 109/L;
- Platelet count ≥100,000/mm3 (100 × 109/L);
- Hemoglobin ≥8 g/dL.
- Fasting serum triglyceride <300 mg/dL; fasting serum total cholesterol <350 mg/dL.
- Male or non-pregnant and non-breast feeding female:
- Females of child-bearing potential must agree to use effective contraception without interruption from 28 days prior to starting IP and while on investigational medication and have a negative serum pregnancy test (β -hCG) result at screening and agree to ongoing pregnancy testing during the course of the expanded access protocol, and after the end of treatment. A second form of birth control is required even if she has had a tubal ligation.
- Male patients must practice abstinence or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the expanded access protocol. A second form of birth control is required even if he has undergone a successful vasectomy.
- Ability to understand and sign informed consent.
Who cannot join
- A patient will not be eligible for inclusion in this protocol if any of the following criteria apply:
- Uncontrolled serious medical or psychiatric illness. Patients with a "currently active" second malignancy other than non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer (staged pT2 with Gleason Score ≤ 6 and postoperative PSA <0.5 ng/mL), or other adequately treated carcinoma-in-situ are ineligible. Patients are not considered to have a "currently active" malignancy if they have completed therapy and are free of disease for ≥1 year.
- In the treating physician's opinion, the potential risks outweigh the potential benefits of therapy with ABI-009.
- Prior exposure to ABI-009.
About this study
Official title: Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients With Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR Pathway Activation
Read the detailed description
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR Pathway Activation
- Conditions
- PEComa, Malignant, TSC1, TSC2, mTOR Pathway Abberation
- Being tested
- ABI-009
- Medical subject headings
- Perivascular Epithelioid Cell Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABI-009Drug
Also called nab-sirolimus, nab-rapamycin, albumin-bound sirolimus, Sirolimus Albumin-bound Nanoparticles
Sirolimus Albumin-bound Nanoparticles
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PEComa, MalignantStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Aadi Bioscience, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03817515
- Sponsor's protocol ID
- PEX-002
- Status last verified
- Mar 2022
- First submitted
- 22 Jan 2019
- Last updated
- 16 Mar 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients With Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients With a Malignancy With Relevant Genetic Mutations or mTOR Pathway Activation
- ClinicalTrials.gov ID
- NCT03817515
- Acronym / sponsor ID
- PEX-002
- Conditions
- PEComa, Malignant, TSC1, TSC2, mTOR Pathway Abberation
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Aadi Bioscience, Inc.Industry (pharma, biotech, devices)
- First posted
- 25 Jan 2019 (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.
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR Pathway Activation
Detailed description
Expanded Access for an Intermediate-size Population for ABI-009 (Sirolimus Albumin-bound Nanoparticles for Injectable Suspension) in Patients with Advanced Perivascular Epithelioid Cell Tumors (PEComa) and Patients with a Malignancy with Relevant Genetic Mutations or mTOR Pathway Activation
Study design
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- A patient will be eligible for inclusion in this expanded access program only if all of the following criteria are met:
- a) Diagnosis of advanced (locally advanced and inoperable or metastatic) malignant PEComa confirmed by pathology and immunohistochemistry, whether or not previously exposed to an mTOR inhibitor, or b) diagnosis of any other malignancy with activation in any mTOR pathway component as identified by immunohistochemistry or an identified relevant rare genetic mutation in mTOR pathway genes, including but not limited to TSC1, TSC2, PIK3CA, PTEN, for which there are no FDA-approved treatments or no other comparable or satisfactory alternative therapies available whether or not they been previously exposed to a mTOR inhibitor.
- 18 years or older, with Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
- Acceptable blood chemistry levels at screening (obtained ≤14 days prior to enrollment (local laboratory) including:
- Adequate biological parameters as demonstrated by the following blood counts at screening (obtained ≤14 days prior to enrollment, local laboratory):
Key exclusion criteria (4 listed)
- A patient will not be eligible for inclusion in this protocol if any of the following criteria apply:
- Uncontrolled serious medical or psychiatric illness. Patients with a "currently active" second malignancy other than non-melanoma skin cancers, carcinoma in situ of the cervix, resected incidental prostate cancer (staged pT2 with Gleason Score ≤ 6 and postoperative PSA <0.5 ng/mL), or other adequately treated carcinoma-in-situ are ineligible. Patients are not considered to have a "currently active" malignancy if they have completed therapy and are free of disease for ≥1 year.
- In the treating physician's opinion, the potential risks outweigh the potential benefits of therapy with ABI-009.
- Prior exposure to ABI-009.
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 |
|---|---|---|
| ABI-009Also: nab-sirolimus, nab-rapamycin, albumin-bound sirolimus, Sirolimus Albumin-bound NanoparticlesSirolimus Albumin-bound Nanoparticles | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2022.
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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.
Questions about this trial
What is this clinical trial studying?
A study testing ABI-009 in people with PEComa, Malignant and TSC1, run by Aadi Bioscience, Inc..
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 16 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 16 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
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.
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 Mar 2022.