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Encapsulated Rapamycin

Phase 2

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

Target and mechanism

ModalitySmall molecule

Also known as Encapsulated Rapamycin (eRapa)

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDBiomarker
Total Trials1
Total Enrollment30

FDA Designations

No designations recorded

Clinical trial landscape

Encapsulated Rapamycin · 1 trial · 1 indication

Phase 2 1
NCT04230499Trial of ERapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active SurveillanceFamilial Adenomatous Polyposis
ACTIVE NOT_RECRUITING30 Analytics
PHASE2ACTIVE NOT_RECRUITING
Trial of ERapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Surveillance
Familial Adenomatous PolyposisUnlock trial analytics

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 to 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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Frequently asked questions about Encapsulated Rapamycin

What is Encapsulated Rapamycin used for?

Encapsulated Rapamycin (eRapa) is an investigational small molecule being studied for Familial Adenomatous Polyposis (FAP), a gastrointestinal condition. It is currently in Phase 2 clinical development and is not yet approved by the FDA.

What does Encapsulated Rapamycin target?

Encapsulated Rapamycin (eRapa) is a small molecule that works by inhibiting the mTOR pathway, which is involved in cell growth and proliferation. By targeting this pathway, it aims to prevent the progression of polyps in patients with Familial Adenomatous Polyposis.

Who is developing Encapsulated Rapamycin?

Encapsulated Rapamycin (eRapa) is being developed by Biodexa Pharmaceuticals plc, a biopharmaceutical company. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Familial Adenomatous Polyposis.

What phase is Encapsulated Rapamycin in?

Encapsulated Rapamycin (eRapa) is currently in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. The ongoing Phase 2 trial is active but not recruiting participants.

What clinical trials is Encapsulated Rapamycin in?

Encapsulated Rapamycin (eRapa) is being evaluated in a Phase 2 clinical trial with the identifier NCT04230499. The trial is titled 'Trial of ERapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Surveillance' and is enrolling 30 participants in the United States.

Is Encapsulated Rapamycin the same as eRapa?

Yes, Encapsulated Rapamycin is also known as eRapa. The drug is being studied under the name eRapa in clinical trials for Familial Adenomatous Polyposis, and both names refer to the same investigational small molecule.