ST-001 nanoFenretinide in Relapsed/ Refractory Small Cell Lung Cancer

Phase 1

Not yet recruiting

• Conditions: Small Cell Lung Cancer
• Interventions: Drug: Fenretinide

• Registration Number: NCT06922539
• Lead Sponsor: SciTech Development, Inc.

Brief Summary

This study evaluates a fenretinide phospholipid suspension for the treatment of small cell lung cancer (SCLC).

Detailed Description

Fenretinide has been shown to be a relatively safe and effective anticancer therapy; however, low fenretinide bioavailability and dose limiting toxicities due to excipients used in previous formulations has impeded its therapeutic utility. The product formulation in the current study (ST-001) is a phospholipid suspension of nanoparticle sized fenretinide. The current study is a Phase 1 trial in in relapsed/refractory small cell lung cancer to determine the safety profile, pharmacology, and maximum tolerated dose (MTD) of ST-001.

Study Timeline

• Status Verified: 2025-03
• Study First Submitted: 2025-03-22
• Study First Submitted QC: 2025-04-03
• Last Update Submitted: 2025-04-09
• Study First Posted: 2025-04-10
• Last Update Posted: 2025-04-13
• Study Start: 2025-06-01
• Primary Completion: 2027-01-01
• Study Completion: 2028-06-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 44

Inclusion Criteria

• Small cell lung cancer (SCLC).
• Patients must all have at least one measurable disease site using RECIST version 1.1 criteria.
• Patients must have had prior treatment with radiation therapy or with platinum-based chemotherapy ± immunotherapy with no limit on the number of prior systemic treatment regimens.
• Relapsed/refractory disease of any stage if incurable in nature, is eligible for enrollment.
• Minimum of 4 weeks must have elapsed since last systemic treatment or radiation therapy treatment (or 6 weeks for any nitrosourea-containing regimens), and patients must have recovered from all toxicity of last treatment and cleared the pharmacological agent(s) used previously.
• ECOG performance status 0-1 (Karnofsky ≥60%).
• Life expectancy greater than 6 months.
• Patients must have normal organ and marrow function.
• Triglyceride blood level (fasting) <300mg/dL at time of enrollment (normal: <150mg/dL; borderline high = 150-199mg/dL; high = 200-499mg/dL; very high = 500mg/dL or higher).
• Women of non-child bearing potential, that is women who have been menopausal or surgically sterile for more than 1 year, are eligible for enrolment in the study.
• Informed consent of the patient or a legal authorized representative (LAR) must be obtained prior to any study related procedures.

Exclusion Criteria

• Mixed SCLC/NSCLC tumors are not eligible. Pregnant or breastfeeding women cannot take part in this study. Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.
• Patients who are receiving any other investigational agents. SCLC patients with history of CNS metastasis may be included if CNS disease is asymptomatic and controlled without progression at least 4 weeks after treatment with radiotherapy, and patient is either no longer taking corticosteroids or on a stable dose of corticosteroids.
• History of allergic reactions or sensitivity to retinoids or to any excipients of ST-001.
• Patients who require concurrent treatment with drugs that are strong CYP3A inducers are excluded from the trial.
• Patients who require concurrent treatment with drugs that are strong to moderate CYP3A inhibitors are excluded from the trial.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure (NY heart classification III/IV), unstable angina pectoris, cardiac arrhythmia, QTc interval >450 milliseconds for men and >460 milliseconds for women on baseline triplicate ECG, or psychiatric illness/social situations that would limit compliance with study requirements.
• HIV-positive patients on combination antiretroviral therapy are ineligible. Patients with any active hepatitis infections. Presence of nyctalopia (night blindness), or hemeralopia (defective vision in a bright light, 'day blindness') at enrollment, or any other retinal, ophthalmological condition (e.g.: retinitis pigmentosa, choroidoretinitis and xerophthalmia), and glaucoma.
• History of solid tumor malignancy other than the diseases under study, diagnosed within the last three (3) years of study enrollment, excluding adequately treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer, in situ breast cancer, in situ prostate cancer (patients must have shown no evidence of active disease for 2 years prior to enrollment).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Standard Phase 1a	Fenretinide	During the Standard Phase 1a, three patients are enrolled into each dose level cohort and the three patients must be evaluated for cycle 1 toxicity before the decision can be made to open the next higher dose level.
Expanded Phase 1b	Fenretinide	Expansion of the dose-finding Phase 1a to determine the safety and maximum tolerated dose (MTD) of the investigative drug product (ST-001).

Primary Outcome Measures

Name	Time	Method
To determine the MTD of ST-001 (12.5mg/mL) for IV infusion in patients with SCLC	From enrollment to end of treatment is 3 weeks	To determine the maximum tolerated dose (MTD) and dose limiting toxicity (DLT) of ST-001 12.5 mg/mL for IV infusion (ST-001) in patients with Relapsed/Refractory SCLC when ST-001 is administered via 4-hour IV infusion daily for five consecutive days, q3weeks. MTD reflects the highest dose of drug that did not cause a Dose-Limiting Toxicity (DLT) in \>33% of participants. DLTs are defined as any treatment-related Common Terminology Criteria for Adverse Events Version 5.0 (CTCAE 5.0) Grade 3 or 4 adverse events.

Secondary Outcome Measures

Name	Time	Method
To describe the toxicity profile of ST-001 in patients with SCLC	From enrollment to end of treatment is 3 weeks	Determine the number of participants with treatment-related adverse events as assessed by CTCAE v5.0
Objective Response Rate (ORR)	From enrollment to end of treatment is 3 weeks	ORR = (Number of patients with complete or partial response) / (Total number of evaluable patients) x 100. Minimum duration of individual patient participation is 3 weeks (one cycle of therapy) to be evaluable for response.
Progression-free survival (PFS)	From enrollment to end of treatment is 3 weeks	PFS is defined as the time from start of treatment to disease progression (increase in tumor size, new metastases) or death.
Cmax	From enrollment to end of treatment is 3 weeks	Peak plasma fenretinide concentration (in ng/mL)
Tmax	From enrollment to end of treatment is 3 weeks	The time it takes for fenretinide to reach the maximum concentration (Cmax) after drug administration (in hours)
Vd	From enrollment to end of treatment is 3 weeks	Volume of distribution; fenretinide's propensity to either remain in the plasma or redistribute to other tissue compartments (in liters)
t½	From enrollment to end of treatment is 3 weeks	Fenretinide elimination and half-life; the time it takes for the amount of a drug's active substance in your body to reduce by half (hours)
To observe and record anti-tumor activity of ST-001 in patients with SCLC	From enrollment to end of treatment is 3 weeks	Preclinical studies with fenretinide suggest potential efficacy against SCLC. Patients will be carefully monitored for tumor response and symptom relief in addition to safety and tolerability. Objective Response Rate (ORR) and progression-free survival (PFS) will be used as tumor response endpoints in this study.
To describe the pharmacokinetics of fenretinide when ST-001 is administered by daily infusion for 5 consecutive days every 3 weeks.	From enrollment to end of treatment is 3 weeks	The endpoint for the pharmacokinetic studies is to assess fenretinide blood plasma levels as a function of administered dose and to determine the following PK parameters: maximum fenretinide concentration (Cmax), time of Cmax (Tmax), volume of distribution (Vd), clearance (CL), elimination and fenretinide half-life (t½).