Actively Recruiting
Combination Gemcitabine and Nab-Sirolimus in Advanced Leiomyosarcomas or Soft-Tissue Sarcomas With TSC2 or TSC1 Mutations or Deletions
Led by M.D. Anderson Cancer Center · Updated on 2026-02-25
18
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
A
Aadi Bioscience
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying the combination of gemcitabine and nab-sirolimus in patients with advanced leiomyosarcomas or soft-tissue sarcomas that have specific genetic mutations called TSC2 or TSC1 loss-of-function mutations or deletions. This Phase I trial aims to find the highest dose of this drug combination that patients can tolerate safely. The study also looks at the side effects, tumor response, and survival outcomes, as well as exploring genetic and molecular markers related to treatment activity. Participants will receive gemcitabine and nab-sirolimus given intravenously. They will be assigned to different dose levels based on when they join the study. This trial focuses on patients with advanced or metastatic disease who meet specific genetic criteria. The study also includes an optional genomic research project to further understand rare tumors. During the study, participants will be closely monitored for safety and side effects throughout treatment, which lasts about one year on average. Researchers will use imaging tests to measure tumor size and check blood samples for tumor DNA changes. They will also assess molecular markers in tumor samples to understand the treatment's effects. Participants must agree to regular testing and follow-up visits during the study period.
CONDITIONS
Brief Title
A Phase I Trial of Combination Gemcitabine and Nab-Sirolimus in Advanced Leiomyosarcomas or Advanced Soft-Tissue Sarcomas With TSC2 or TSC1 Loss-of-function Mutations or Deletions
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically confirmed leiomyosarcoma or sarcoma with TSC2 or TSC1 loss-of-function mutations or deletions
- Metastatic or unresectable sarcoma without effective standard curative options
- One prior standard systemic therapy required for leiomyosarcoma patients
- Prior gemcitabine allowed if last dose was over 12 months ago without progression during treatment
- Measurable disease with at least one lesion ≥20 mm by chest x-ray or ≥10 mm by CT, MRI, or clinical exam
- Age 18 years or older
- ECOG performance status 0 to 2
- Life expectancy over 3 months
- Adequate organ and marrow function, including specific blood counts and liver and kidney tests
- Controlled hepatitis B or cured hepatitis C infections
- HIV patients with viral load under 400 copies/mL and stable treatment
- Treated brain metastases with no progression
- Cardiac function class 2B or better
- Agree to use contraception if of child-bearing potential
- Ability and willingness to sign informed consent
You will not qualify if you...
- Receiving or recently received other investigational agents within 3 weeks
- Allergic reactions to compounds similar to nab-sirolimus or gemcitabine
- Use of strong CYP3A4 inhibitors or inducers or certain CYP3A4 substrates before treatment
- Active concurrent malignancy
- Recent stroke or thromboembolic event within 6 months requiring anticoagulation, except stable deep venous thrombosis
- Active gastrointestinal bleeding
- Uncontrolled diabetes with HbA1c over 8%
- Severe or uncontrolled systemic disease or psychiatric condition limiting safe participation
- Pregnant or breastfeeding women
- Prior treatment with mTOR inhibitors for sarcoma
- Vulnerable populations will not be enrolled
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 1 year or until disease progression or unacceptable toxicity
Participants receive combination gemcitabine and nab-sirolimus by IV to treat advanced leiomyosarcomas or soft-tissue sarcomas with TSC2 or TSC1 mutations or deletions.
Repeated visits for drug administration and safety monitoring as per protocol
Duration - Up to 1 year after treatment completion
Participants are monitored for safety and adverse events after completing treatment.
Periodic follow-up visits for safety assessments
Trial Site Locations
Total: 1 location
1
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
E
Elise Nassif, MD
G
Gracy Zacharian, BSN,MSN,RN
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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