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Encapsulated Rapamycin

Phase 2

Familial Adenomatous Polyposis | Small molecule | Other |Biodexa Pharmaceuticals plc|Last Updated: Jan 9, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDBiomarker
Total Trials1
Total Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04230499Trial of ERapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active SurveillancePHASE2 ACTIVE NOT_RECRUITING 30 — — Jan 18, 2021Mar 1, 2025Jan 9, 20254 United States
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Study Endpoints
Primary Endpoints
Frequency and severity of adverse events associated with low dose eRapa in FAP patients
All adverse events with start dates occurring any time after informed consent is obtained until 7 days (for non-serious adverse events) or 30 days (for serious adverse events) after the last day of study participation will be recorded.

Safety and tolerability of eRapa as determined by graded toxicity assessed throughout the trial per CTCAE v5.0.

Determine the Recommended Phase 2 Dose (RP2D)
After informed consent is obtained up to 30 days after the last day of study participation.

The Recommended Phase 2 Dose (RP2D) will be determined by examining and analyzing safety/ adverse events as reflected by Outcome 1, dose delays, dose reductions, withdrawal of treatment secondary to low-grade toxicities, and serum pharmacokinetic monitoring.

Efficacy of eRapa in delaying polyp progression in patients with FAP as measured by change in polyp burden over time.
Time for each patient is baseline to 6 months.

Percentage change from baseline in colorectal polyp burden as measure by endoscopy at 6 months.

Secondary Endpoints
Clinical effect of eRapa on polyp burden.
Following patients out to 12 months.
Clinical effect of eRapa on International Society for Gastrointestinal Hereditary Tumors Stage.
Following patients out to 6 and 12 months.
Clinical effect of eRapa on Spigelman Stage Score.
Following patients out to 6 and 12 months.
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALCohort 1 will receive 0.5mg of eRapa every other day.
Cohort 2EXPERIMENTALCohort 2 will receive 0.5mg of eRapa daily with 7 days on therapy, followed by 7 days off therapy.
Cohort 3EXPERIMENTALCohort 3 will receive 0.5 mg of eRapa daily.
Food EffectEXPERIMENTALUpon identification of the RP2D, after a 2 week washout (14 days), subjects will be randomized into a fed and fasted (2 weeks - 14 days) two period cross over, with an intervening 2 week washout (14 days) for a total of 2 months (8 weeks).
Interventions
NameTypeDescription
Encapsulated Rapamycin (eRapa)DRUGeRapa is encapsulated rapamycin. The rapamycin is encapsulated in order to deliver the rapamycin at a consistent and lower dosage. eRapa is a capsule, and is administered orally.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: 1. Sign and date an informed consent form. 2. Stated willingness to comply with all study procedures and availability for the duration of the study. 3. Male or female, age at least 18 years at the time of consent. 4. Phenotypic familial adenomatous polyposis (FAP) with disease i...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04230499studyFirstPostDate: changed