NCT04094675 is a Phase 2 clinical trial of Sirolimus in PTEN Gene Mutation, sponsored by Ohio State University.

Who is sponsoring NCT04094675?

NCT04094675 is sponsored by Ohio State University.

What drugs are being tested in NCT04094675?

Interventions in NCT04094675: Sirolimus.

What condition does NCT04094675 study?

NCT04094675 studies PTEN Gene Mutation, PTEN Hamartoma Tumor Syndrome, PTEN Hamartoma Syndrome, Cowden Syndrome, Bannayan Syndrome, Bannayan Zonana Syndrome, Polyposis.

Is NCT04094675 still recruiting?

NCT04094675 is currently completed. Last updated 10 June 2025.

Last reviewed 2025-06-10 · How we verify

NCT04094675

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Sirolimus for Cowden Syndrome With Colon Polyposis

Completed Phase 2 Last updated 10 June 2025

What this trial tests

Phase 2 trial testing Sirolimus in PTEN Gene Mutation in 5 participants. Completed in 5 June 2025.

Timeline

16 September 2019

Primary endpoint
1 February 2025

5 June 2025

Quick facts

Lead sponsor	Ohio State University
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	16 September 2019
Primary completion	1 February 2025
Estimated completion	5 June 2025
Sites	1 location across United States

Drugs / interventions tested

Sirolimus (sirolimus) — full drug profile →

Conditions studied

PTEN Gene Mutation — all drugs for PTEN Gene Mutation →
PTEN Hamartoma Tumor Syndrome — all drugs for PTEN Hamartoma Tumor Syndrome →
PTEN Hamartoma Syndrome — all drugs for PTEN Hamartoma Syndrome →
Cowden Syndrome — all drugs for Cowden Syndrome →

Sponsor

Ohio State University

Who can join

Adults 18 to 80, any sex, with PTEN Gene Mutation or PTEN Hamartoma Tumor Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Colon polyposis (the presence of multiple colon polyps) is very common with Cowden syndrome, as over 60% of patients have 50 or more polyps. In a previous clinical trial, some participants had reduction in the number of colon polyps with the use of the medication sirolimus for a very short time period. This study is investigating sirolimus and its effect on the number of colon polyps in patients with Cowden syndrome and polyposis over a 1 year period.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

Long-term treatment of cancer-prone germline PTEN mutant mice with low-dose rapamycin extends lifespan and delays tumour development.
Tibarewal P, Rathbone V, Constantinou G, Pearce W, et al · · 2022 · cited 17× · PMID 36073856 · DOI 10.1002/path.6009
Rapamycin in Cerebral Cavernous Malformations: What Doses to Test in Mice and Humans.
Hagan MJ, Shenkar R, Srinath A, Romanos SG, et al · · 2022 · cited 13× · PMID 35592432 · DOI 10.1021/acsptsci.2c00006
mTOR pathway diseases: challenges and opportunities from bench to bedside and the mTOR node.
Mantoan Ritter L, Annear NMP, Baple EL, Ben-Chaabane LY, et al · · 2025 · cited 5× · PMID 40426219 · DOI 10.1186/s13023-025-03740-1
Sirolimus for Colon Polyposis in PTEN Hamartoma Tumor Syndrome.
Stanich PP, Stanich PP, Gofar K, Roberts ME, et al · · 2025 · PMID 40478255 · DOI 10.14309/ctg.0000000000000871

Verify or expand the search:

Other trials of Sirolimus

Trials testing the same drug.

NCT07542314 — Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a · Phase 4 · not yet recruiting
NCT06876142 — Combination Therapy (Mirdametinib and Sirolimus) for RAS Mutated Relapsed Refractory Multiple Myeloma · Phase 1, PHASE2 · suspended
NCT07052929 — Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators · Phase 1, PHASE2 · recruiting
NCT04370795 — Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-bas · Phase 1, PHASE2 · withdrawn
NCT07162038 — Phase I Trial Integrating HLA-Haploidentical Anti-CD19 CAR-T Cells With Post-Transplantation Cyclophosphamide-Based HLA- · Phase 1 · recruiting

Other recruiting trials for PTEN Gene Mutation

Currently open trials in the same condition.

NCT05420064 — An Intervention to Increase Genetic Testing in Families Who May Share a Gene Mutation Related to Cancer Risk and An Inte · NA · recruiting

Other Ohio State University trials

Trials by the same sponsor.

NCT06542458 — Under-Represented Communities Diagnosed With SCD or MCI Through Tele-Cog · not yet recruiting
NCT07217548 — Solving Stigma Through POV Simulation: Enhancing Pharmacist Empathy-based Practices With Sickle Cell Disease Patients · NA · enrolling by invitation
NCT07215221 — Implementation of TBI-RECOVER in Substance Use Treatment · NA · not yet recruiting
NCT07278427 — Reducing Parental Substance Use and Enhancing Family Resilience Among Rural Families Through Ohio START · recruiting
NCT07490444 — Metric-Optimized Spectacle Prescriptions for Children With Down Syndrome · Phase 2 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04094675 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Ohio State University
Last refreshed: 10 June 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04094675.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing