Nirogacestat for Kaposi Sarcoma

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: AIDS Malignancy Consortium

Must be taking: Antiretroviral therapy

Must not be taking: Zidovudine, Protease inhibitors

Disqualifiers: Opportunistic infection, Cytotoxic therapy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: 19

Duration: 12 cycles (28 days each)

What You Need to Know Before You Apply

What is the purpose of this trial?

This phase II trial studies how well nirogacestat works in treating patients with skin Kaposi sarcoma (KS). Several anti-cancer drugs work well in treating KS, but there is no treatment that cures KS. Nirogacestat binds to a protein called gamma secretase, which blocks the activation of other proteins called Notch receptors. Blocking these proteins may help keep tumor cells from growing and may kill them. Nirogacestat is a type of gamma secretase inhibitor. Nirogacestat may be effective in shrinking the size of KS lesions and reducing the spread of lesions.

Who Is on the Research Team?

Lee Ratner

Principal Investigator

AIDS Malignancy Consortium

Are You a Good Fit for This Trial?

Inclusion Criteria

I am 18 years old or older.

My heart condition is stable and classified as NYHA Class II or better.

Life expectancy ≥ 3 months

Exclusion Criteria

I have not had local treatment to my KS lesion unless it has clearly gotten worse since then.

I need blood transfusions to keep my hemoglobin at a safe level.

Concurrent, acute, active opportunistic infection other than oral thrush or genital herpes within 14 days of enrollment

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive nirogacestat orally twice daily on days 1-28 of each cycle. Cycles repeat every 28 days for 12 cycles.

12 cycles (28 days each)

Monthly visits for 12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

5 years

Every 6 months

What Are the Treatments Tested in This Trial?

Interventions

• Nirogacestat

How Is the Trial Designed?

Group I: Treatment (nirogacestat) Experimental Treatment 5 Interventions

Patients receive nirogacestat PO BID on days 1-28 of each cycle. Cycles repeat every 28 days for 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo skin biopsy and chest X-ray during screening as well as blood sample collection throughout the study. Patients may also undergo an additional optional skin biopsy on study and CT throughout the study.

Who Is Running the Clinical Trial?

AIDS Malignancy Consortium

Lead Sponsor

Trials

64

Recruited

9,600+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+