Trial Outcomes & Findings for A Study to Evaluate the Safety of Fenretinide in Healthy Volunteers (NCT NCT06181760)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

25 participants

Primary outcome timeframe

First Intervention (Day 1 to Day 8)

Results posted on

2025-01-27

Participant Flow

25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort were only 4 out of the 6 subjects were available. Overall 25 subjects were enrolled, but there were 4 subjects (3 subjects on ISLA101 and 1 subject on placebo) that also participated in the fed cohort.

Participant milestones

Participant milestones
Measure	Fenretinide 300 mg/m^2 Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions only	Fenretinide 600 mg/m^2 Single oral dose of fenretinide, 600 mg/m\^2 under fasted. Subjects completed a wash out period of at least 3 days. Subjects returned and took a Single oral dose of fenretinide, 600 mg/m\^2 and fed conditions	Fenretinide 900 mg/m^2 Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions only	Placebo Single oral dose of placebo capsules Placebo: Single oral dose of matching placebo capsules under fasted conditions Subjects completed a washout period of at least 3 days. Subjects returned to complete the second intervention period taking a single oral dose of matching placebo capsules under fed conditions
First Intervention (Fasted) STARTED	6	6	6	7
First Intervention (Fasted) COMPLETED	6	6	6	6
First Intervention (Fasted) NOT COMPLETED	0	0	0	1
Second Intervention (Fed) STARTED	0	3	0	1
Second Intervention (Fed) COMPLETED	0	3	0	1
Second Intervention (Fed) NOT COMPLETED	0	0	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Fenretinide 300 mg/m^2 Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions only	Fenretinide 600 mg/m^2 Single oral dose of fenretinide, 600 mg/m\^2 under fasted. Subjects completed a wash out period of at least 3 days. Subjects returned and took a Single oral dose of fenretinide, 600 mg/m\^2 and fed conditions	Fenretinide 900 mg/m^2 Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions only	Placebo Single oral dose of placebo capsules Placebo: Single oral dose of matching placebo capsules under fasted conditions Subjects completed a washout period of at least 3 days. Subjects returned to complete the second intervention period taking a single oral dose of matching placebo capsules under fed conditions
First Intervention (Fasted) Withdrawal by Subject	0	0	0	1

Baseline Characteristics

A Study to Evaluate the Safety of Fenretinide in Healthy Volunteers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fenretinide 300 mg/m^2 n=6 Participants Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions only	Fenretinide 600 mg/m^2 n=6 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted and fed conditions	Fenretinide 900 mg/m^2 n=6 Participants Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions only	Placebo n=7 Participants Single oral dose of placebo capsules under fasted and fed conditions	Total n=25 Participants Total of all reporting groups
Age, Continuous	32.57 years STANDARD_DEVIATION 10.443 • n=5 Participants	40.23 years STANDARD_DEVIATION 9.438 • n=7 Participants	34.33 years STANDARD_DEVIATION 13.049 • n=5 Participants	30.66 years STANDARD_DEVIATION 10.588 • n=4 Participants	34.3 years STANDARD_DEVIATION 10.872 • n=21 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants	7 Participants n=4 Participants	23 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants	2 Participants n=4 Participants	9 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	4 Participants n=4 Participants	12 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants	3 Participants n=21 Participants
Region of Enrollment Australia	6 participants n=5 Participants	6 participants n=7 Participants	6 participants n=5 Participants	7 participants n=4 Participants	25 participants n=21 Participants
Body Mass Index	24.9 kg/m^2 STANDARD_DEVIATION 4.35 • n=5 Participants	26.3 kg/m^2 STANDARD_DEVIATION 4.40 • n=7 Participants	22.78 kg/m^2 STANDARD_DEVIATION 2.79 • n=5 Participants	24.31 kg/m^2 STANDARD_DEVIATION 3.12 • n=4 Participants	24.56 kg/m^2 STANDARD_DEVIATION 3.69 • n=21 Participants

PRIMARY outcome

Timeframe: First Intervention (Day 1 to Day 8)

Population: 25 total subjects were enrolled in the study.

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 n=6 Participants Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=6 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 n=6 Participants Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo n=7 Participants Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Number and % of Subjects Experiencing Adverse Events Following a Single Oral Dose of Fenretinide Under Fasted Conditions	1 Participants	2 Participants	3 Participants	2 Participants	—	—

PRIMARY outcome

Timeframe: First Intervention (Day 1 to Day 8)

Population: 25 total subjects were enrolled in the study.

25 total subjects were enrolled in the study.

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 n=6 Participants Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=6 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 n=6 Participants Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo n=7 Participants Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Number and % of Subjects Experiencing Serious Adverse Events Following a Single Oral Dose of Fenretinide Under Fasted Conditions	0 Participants	0 Participants	0 Participants	0 Participants	—	—

PRIMARY outcome

Timeframe: Second Intervention (Day 9 to Day 17)

Population: 25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort and only 4 out of the 6 subjects were available (i.e., 3 subjects on ISLA101 and 1 subject on placebo).

Subjects in the 600 mg/m\^2 came back to complete the fed cohort, as this was the only group to return based on the recommendations by the Safety Review Committee. Only 4 out of the 6 subjects returned for this cohort (i.e., 3 subjects on ISLA101 and 1 subject on placebo).

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=3 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo n=1 Participants Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Number and % of Subjects Experiencing Adverse Events Following a Single Oral Dose of Fenretinide Under Fed Conditions	0 Participants	0 Participants	0 Participants	0 Participants	—	—

PRIMARY outcome

Timeframe: Second Intervention (Day 9 to 17)

Population: 25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort and only 4 out of the 6 subjects were available (i.e., 3 subjects on ISLA101 and 1 subject on placebo).

25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort and only 4 out of the 6 subjects were available (i.e., 3 subjects on ISLA101 and 1 subject on placebo).

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=3 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo n=1 Participants Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Number and % of Subjects Experiencing Serious Adverse Events Following a Single Oral Dose of Fenretinide Under Fed Conditions	0 Participants	0 Participants	0 Participants	0 Participants	—	—

SECONDARY outcome

Timeframe: First Intervention (Day 1 to Day 8), washout period, Second Intervention (Day 9-17)

Population: The PK Population was comprised of all subjects in the safety population who have a pre-dose PK sample and at least one post-dose analyzable PK sample and at least 1 post-dose analyzable PK sample (quantifiable Plasma concentration). Subjects in the placebo group did not have Cmax analyzed as they did not receive study drug.

Secondary objective

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 n=6 Participants Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=6 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 n=6 Participants Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) n=3 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Assess the CMax - Observed Maximum Plasma Concentration Following a Single Oral Dose of Fenretinide	195.8 ng/mL Standard Deviation 92.35	524.8 ng/mL Standard Deviation 74.2	624.2 ng/mL Standard Deviation 142.2	—	1455 ng/mL Standard Deviation 521.6	—

SECONDARY outcome

Timeframe: First Intervention (Day 1 to Day 8), washout period, Second Intervention (Day 9-17)

Population: The PK Population was comprised of all subjects in the safety population who have a pre-dose PK sample and at least one post-dose analyzable PK sample and at least 1 post-dose analyzable PK sample (quantifiable Plasma concentration). Subjects in the placebo group did not have Tmax analyzed as they did not receive study drug.

25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort and only 4 out of the 6 subjects were available (i.e., 3 subjects on ISLA101 and 1 subject on placebo).

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 n=6 Participants Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=6 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 n=6 Participants Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) n=3 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Assess the TMax - Time to Reach Maximum Concentration Curve Following a Single Oral Dose of Fenretinide in the Fasted and Fed State	5.895 hours Interval 2.97 to 6.0	5.15 hours Interval 5.07 to 6.02	5.015 hours Interval 4.88 to 6.0	—	5.98 hours Interval 5.98 to 9.97	—

SECONDARY outcome

Timeframe: First Intervention (Day 1 to Day 8), washout period, Second Intervention (Day 9-17)

Population: The PK Population was comprised of all subjects in the safety population who have a pre-dose PK sample and at least one post-dose analyzable PK sample and at least 1 post-dose analyzable PK sample (quantifiable Plasma concentration). Subjects in the placebo group did not have AUC-∞ analyzed as they did not receive study drug.

25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort and only 4 out of the 6 subjects were available (i.e., 3 subjects on ISLA101 and 1 subject on placebo).

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 n=6 Participants Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=5 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 n=6 Participants Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) n=3 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Assess the AUC-∞ Area Under the Concentration Curve From Zero to Infinite Time Following a Single Oral Dose of Fenretinide in the Fasted and Fed State	3479 h*ng/mL Standard Deviation 1947	8209 h*ng/mL Standard Deviation 836.9	9621 h*ng/mL Standard Deviation 2145	—	25800 h*ng/mL Standard Deviation 8726	—

SECONDARY outcome

Timeframe: First Intervention (Day 1 to Day 8), washout period, Second Intervention (Day 9-17)

Population: The PK Population was comprised of all subjects in the safety population who have a pre-dose PK sample and at least one post-dose analyzable PK sample and at least 1 post-dose analyzable PK sample (quantifiable Plasma concentration). Subjects in the placebo group did not have AUC(last) analyzed as they did not receive study drug.

25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort and only 4 out of the 6 subjects were available (i.e., 3 subjects on ISLA101 and 1 subject on placebo).

Outcome measures

Outcome measures
Measure	Fenretinide 300 mg/m^2 n=6 Participants Single oral dose of fenretinide, 300 mg/m\^2 under fasted conditions	Fenretinide 600 mg/m^2 n=6 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fasted conditions	Fenretinide 900 mg/m^2 n=6 Participants Single oral dose of fenretinide, 900 mg/m\^2 under fasted conditions	Placebo Single oral dose of placebo capsules under fasted conditions	Fenretinide 600 mg/m^2 (Fed) n=3 Participants Single oral dose of fenretinide, 600 mg/m\^2 under fed conditions Study Days 9-17	Placebo (Fed) single oral doses of placebo under fed conditions Study Days 9-17
Assess the AUC(Last) - Area Under the Curve up to the Last Quantifiable Timepoint After a Single Oral Dose of Fenretinide in the Fasted and Fed State	3212 h*ng/mL Standard Deviation 1795	7894 h*ng/mL Standard Deviation 844.6	9089 h*ng/mL Standard Deviation 2039	—	24460 h*ng/mL Standard Deviation 8395	—

SECONDARY outcome

Timeframe: First Intervention (Day 1 to Day 8), washout period, Second Intervention (Day 9-17)

Population: The PK Population was comprised of all subjects in the safety population who have a pre-dose PK sample and at least one post-dose analyzable PK sample and at least 1 post-dose analyzable PK sample (quantifiable Plasma concentration). Subjects in the placebo group did not have half-life analyzed as they did not receive study drug.

25 total subjects were enrolled in the study. After all fasted cohorts completed (i.e., 300 mg/m\^2, 600 mg/m\^2, 900 mg/m\^2 as well as placebo) data was reviewed by the independent Safety Review Committee. The recommendation was to bring back the 600 mg/m\^2 group to complete the fed cohort and only 4 out of the 6 subjects were available (i.e., 3 subjects on ISLA101 and 1 subject on placebo).