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Also known as Encapsulated Rapamycin (eRapa), Encapsulated Rapamycin, eRapa (encapsulated rapamycin)
eRapa · 2 trials · 2 indications
* Death from any cause * Cancer/high-grade dysplasia * Major FAP-related surgery (e.g., colectomy, proctectomy, total proctocolectomy with ileal pouch anal anastomosis \[IPAA\], pouch resection, ileostomy, duodenectomy, or surgical ampullectomy) * Advancement of Spigelman stage (not related solely to increase in polyp number) * Meets criteria for surgery (consistent with United States \[US\] and European Union \[EU\] practice guidelines) (Yang, Gurudu et al. 2020, Zaffaroni, Mannucci et al.2024) * Retained rectum/sigmoid or pouch (≥10 polyps ≥3 mm in size at baseline) * Duodenum (Stage 3/4 and at least 1 polyp ≥10 mm removed in last 18 months)
Safety and tolerability of eRapa as determined by graded toxicity assessed throughout the trial per CTCAE v5.0.
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.
Percentage change from baseline in colorectal polyp burden as measure by endoscopy at 6 months.
| Arm | Type | Description |
|---|---|---|
| eRapa | EXPERIMENTAL | 0.5 mg eRapa once a day (QD) every other week |
| Placebo | PLACEBO_COMPARATOR | Placebo once a day (QD) every other week |
| Cohort 1 | EXPERIMENTAL | Cohort 1 will receive 0.5mg of eRapa every other day. |
| Cohort 2 | EXPERIMENTAL | Cohort 2 will receive 0.5mg of eRapa daily with 7 days on therapy, followed by 7 days off therapy. |
| Cohort 3 | EXPERIMENTAL | Cohort 3 will receive 0.5 mg of eRapa daily. |
| Food Effect | EXPERIMENTAL | Upon 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). |
| Name | Type | Description |
|---|---|---|
| eRapa (encapsulated rapamycin) | DRUG | 0.5 mg capsules for oral use; white opaque capsule filled with off-white powder; Trial intervention will be provided in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal. |
| Placebo | DRUG | Capsules in 28-count round high-density polyethylene bottles with a polypropylene child-resistant screw cap and foil induction seal. |
| Encapsulated Rapamycin (eRapa) | DRUG | eRapa 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. |
Inclusion Criteria: 1. Participant must be ≥18 years of age inclusive. 2. Participant must have documented FAP, confirmed by adenomatous polyposis coli genotype mutation testing. 3. Participant must have at least 1 of the following high-risk features: \>100 polyps but ≤500 polyps in the colon, or ≥...
eRapa is an investigational small molecule being developed for Familial Adenomatous Polyposis (FAP), a gastrointestinal condition. It is being studied to prevent progression of the disease in patients under active surveillance. eRapa has received Fast Track and Orphan Drug designations from the FDA for this indication.
eRapa is being developed by Biodexa Pharmaceuticals plc, which trades under the ticker BDRX. The company is advancing the asset through late-stage clinical development for Familial Adenomatous Polyposis, with two active trials currently listed, including a Phase 3 study.
eRapa is in Phase 3 clinical development for Familial Adenomatous Polyposis. The Phase 3 trial, NCT06950385, is currently recruiting, while an earlier Phase 2 trial, NCT04230499, is active but no longer recruiting. eRapa is investigational and has not been approved by the FDA.
eRapa is being evaluated in two clinical trials for Familial Adenomatous Polyposis. The Phase 3 trial NCT06950385 is recruiting 168 patients across the United States, Denmark, Germany, Netherlands, Puerto Rico, and Spain. The Phase 2 trial NCT04230499 has enrolled 30 patients in the United States and is active but not recruiting.
Yes, eRapa is the same as Encapsulated Rapamycin. The drug is also referred to as eRapa (encapsulated rapamycin) and Encapsulated Rapamycin (eRapa). These names all describe the same investigational small molecule being developed by Biodexa Pharmaceuticals for Familial Adenomatous Polyposis.
eRapa has received Fast Track and Orphan Drug designations from the FDA for Familial Adenomatous Polyposis. These designations are intended to support and expedite the development of therapies for serious conditions and rare diseases. eRapa remains investigational and is not FDA approved.