What side effects are associated with this type of intervention?

mTOR inhibitors, such as everolimus and sirolimus, commonly cause side effects including stomatitis, diarrhea, fatigue, peripheral edema, and rash. Severe adverse events can include infections, pneumonitis, and hyperglycemia. Chemotherapy agents like doxorubicin can cause myelosuppression, cardiotoxicity, nausea, and alopecia. Targeted therapies, such as bevacizumab, may lead to hypertension, proteinuria, and increased risk of bleeding and thromboembolic events.

What prior FDA approvals exist?

Several mTOR inhibitors have received FDA approval for the treatment of metastatic conditions. Everolimus, an mTOR inhibitor, has been approved for various cancers, including advanced renal cell carcinoma, progressive neuroendocrine tumors of pancreatic origin, and advanced hormone receptor-positive, HER2-negative breast cancer. Temsirolimus, another mTOR inhibitor, is approved for advanced renal cell carcinoma. These drugs work by inhibiting the mTOR pathway, which is crucial for cell growth and proliferation, thereby helping to control the progression of metastatic cancers.

What other types of research exist?

Promising novel alternatives to Nab-sirolimus for metastasis patients in 2024 include: 1) Cabozantinib, a multi-kinase inhibitor targeting VEGFR and MET, which has shown efficacy in various cancers including renal cell carcinoma. 2) Pembrolizumab, an anti-PD-1 immunotherapy, often used in combination with other agents like chemotherapy or targeted therapies. 3) Atezolizumab, an anti-PD-L1 immunotherapy, particularly in combination with cabozantinib for renal cell carcinoma. 4) Combination therapies involving nivolumab (anti-PD-1) and ipilimumab (anti-CTLA-4) for enhanced immune response. These alternatives have demonstrated significant clinical benefits in recent trials and are considered promising for metastatic cancer treatment.

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mTOR Inhibitor

Nab-sirolimus for Cancer

Phase 2

Waitlist Available

Research Sponsored by Aadi, LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 or Karnofsky Performance Status (KPS) ≥80 or Lansky play-performance scale for pediatric patients ≥80

Patients must have 1 or more measurable target lesions by CT scan or MRI (RECIST v1.1)

Must not have

Patients with primary brain tumors or PEComa

Patients with meningeal carcinomatosis, leptomeningeal carcinomatosis, spinal cord compression, untreated brain metastases or symptomatic or unstable brain metastases

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug on adults and adolescents with certain genetic changes in their tumors. These changes make their cancer hard to treat with standard methods. The drug works by blocking a pathway that helps the cancer grow, aiming to slow down or stop tumor growth. The drug has been studied for its effectiveness and safety in various types of cancer.

Who is the study for?

Adults and adolescents (12+) with malignant solid tumors that can't be surgically removed or have spread, and have specific genetic changes in TSC1/TSC2 genes. They should have tried all standard treatments without success or not be suitable for them. Participants need to meet certain health criteria like good organ function, stable vital signs, and agree to use contraception.

What is being tested?

The trial is testing nab-sirolimus on patients with advanced cancer who carry certain genetic alterations. It's an open-label study where everyone knows what treatment they're getting, aiming to see how effective this drug is against various types of solid tumors.

What are the potential side effects?

While the exact side effects are not listed here, drugs similar to nab-sirolimus may cause issues like mouth sores, skin problems, increased risk of infections due to a weakened immune system, high blood sugar levels, lung problems such as pneumonitis or pulmonary hypertension.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am very active or mostly active with slight limitations.

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I have at least one tumor that can be measured on a scan.

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I am 12 years old or older.

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My cancer has a specific genetic change in TSC1 or TSC2, found through advanced testing.

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My kidneys work well enough (creatinine clearance ≥30 mL/min).

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My cancer has spread and cannot be removed by surgery without severe complications.

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I've had no major surgery or cancer treatment in the last 4 weeks and have recovered from any serious side effects.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a primary brain tumor or PEComa.

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I have brain complications from cancer, such as untreated or unstable brain metastases.

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I have had interstitial lung disease, pneumonitis, or pulmonary hypertension.

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I have been treated with an mTOR inhibitor before.

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I don't have any serious illnesses that could get worse with the study treatment.

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I have severe lung problems.

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I do not have recent serious heart issues or uncontrolled heart rhythm problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Overall response rate (ORR)

Secondary study objectives

Disease control rate

Duration of response (DOR)

Incidence and severity of treatment-emergent and treatment-related adverse events (AEs)

+4 more

Side effects data

From 2022 Phase 1 & 2 trial • 60 Patients • NCT03439462

67%

Mucositis

58%

Fatigue

43%

Rash

38%

Diarrhea

37%

Myelosuppression (Thrombocytopenia)

35%

Nausea

30%

Myelossupression (Neutropenia)

28%

Hypertriglyceridemia

27%

Weight decreased

27%

Myelosuppression (Anemia)

25%

Decreased appetite

22%

Mucosal inflammation

18%

Lipase increased

18%

Hypokalemia

18%

Dermatitis

18%

Vomiting

17%

Dysgeusia

17%

Epistaxis

15%

Hyperglycemia

13%

Amylase increased

12%

Abdominal pain

12%

Hypercholesterolemia

10%

Headache

10%

Infection

10%

Hypophosphataemia

ALT

Dry mouth

Anal inflammation

AST

Hypoalbuminaemia

Candida infection

Proctalgia

Dehydration

Gastrooesophageal reflux disease

Nail disorder

Dry skin

Proteinuria

Alopecia

Anorexia

Dizziness

Oedema peripheral

Dry eye

Hypertension

Application site pain

Hypomagnesaemia

Taste disorder

Pneumonia

Colitis

Enterocolitis infectious

Rectal perforation

Stomatitis

100%

80%

60%

40%

20%

Study treatment Arm

Total

Cohort 1: Nab-Sirolimus 30 mg/m2 qw3/4

Cohort 2 Nab-Sirolimus 20 mg/m2 qw3/4

Cohort 3: Nab-Sirolimus 20 mg/m2 qw2/4

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Arm B: Pathogenic inactivating TSC2 alterationsExperimental Treatment1 Intervention

Patients with pathogenic inactivating TSC2 alterations.

Group II: Arm A: Pathogenic inactivating TSC1 alterationsExperimental Treatment1 Intervention

Patients with pathogenic inactivating TSC1 alterations.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

nab-sirolimus

2018

Completed Phase 2

~130

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for metastatic cancer include chemotherapy, targeted therapy, and immunotherapy. Chemotherapy works by killing rapidly dividing cells, including cancer cells, but can also affect healthy cells. Targeted therapies, such as mTOR inhibitors like nab-sirolimus, specifically block molecular pathways that cancer cells use to grow and divide, thereby reducing tumor growth with potentially fewer side effects. Immunotherapy boosts the body's immune system to recognize and destroy cancer cells. For metastasis patients, these treatments are crucial as they can slow disease progression, manage symptoms, and improve quality of life. mTOR inhibitors are particularly important as they target specific pathways involved in cancer cell proliferation and survival, offering a more tailored and potentially effective treatment option.