NCT03095703

Sirolimus and Familial Adenomatous Polyposis (FAP)

Completed Phase 2 Results posted Last updated 24 October 2024

What this trial tests

Phase 2 trial testing Sirolimus in Adenomatous Polyposis Coli in 4 participants. Completed in 10 December 2018.

Timeline

3 October 2017

Primary endpoint
10 December 2018

10 December 2018

Quick facts

Lead sponsor	Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	4
Start date	3 October 2017
Primary completion	10 December 2018
Estimated completion	10 December 2018
Sites	1 location across Netherlands

Drugs / interventions tested

• Sirolimus (sirolimus) — full drug profile →

Conditions studied

• Adenomatous Polyposis Coli — all drugs for Adenomatous Polyposis Coli →

Sponsor

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) — full company profile →

Who can join

18 and older, any sex, with Adenomatous Polyposis Coli. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Change in Marked Polyp Size Primary · 6 Months

Effect of sirolimus on the size of 5 marked polyps per patient based on video observations.

Decrease in marked polyp size

Group	Value	95% CI
Sirolimus	16

Same marked polyp size

Group	Value	95% CI
Sirolimus	4

Increase in marked polyp size

Group	Value	95% CI
Sirolimus	0

Median Number of Treatment-Related Adverse Events Per Participant Primary · 6 Months

Summary analysis of adverse events, clinical laboratory abnormalities and regular physical examination.

Group	Value	95% CI
Sirolimus	10	4 – 16

Median Difference in Number of Intestinal Polyps Secondary · 6 Months

Number of intestinal polyps was determined by two independent reviewers, blinded for the order of videos (before and after treatment). The median difference comparing baseline en after 6 months of treatment was reported.

Group	Value	95% CI
Sirolimus	25.75	6.5 – 50

Global Polyp Burden Secondary · 6 Months

The global polyp burden is estimated by the endoscopist and two independent reviewers. The second video in the pair could take the value of -2 (much better), -1 (better), 0 (same), 1 (worse) or 2 (much worse) relative to the first video. Mean scores are calculated for each subject and averaged for the three reviewers. If the assessment of the reviewers differs by more than 1 point from the assessment of the endoscopist, consensus is needed.

Group	Value	95% CI
Sirolimus	0
Sirolimus	3
Sirolimus	1
Sirolimus	0

Adverse events — posted to ClinicalTrials.gov

Time frame: 6 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Sirolimus

Serious: 1/4 (25%)

Deaths: 0/4

Serious adverse events (1 terms)

Reaction	System	Sirolimus
Desmoid tumor	Musculoskeletal and connective tissue disorders	—

Other adverse events (2 terms — click to expand)

Reaction	System	Sirolimus
Gastrointestinal disorders	Gastrointestinal disorders	—
Skin problems	Skin and subcutaneous tissue disorders	—

Most-reported serious reactions: Desmoid tumor.

Data from ClinicalTrials.gov NCT03095703 adverse events section.

Sponsor's own description

The aim of the study is to investigate the effect of sirolimus on the progression of intestinal adenomas in patients with FAP and to assess the safety of this treatment.

Publications & conference data

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

1. Sirolimus for the treatment of polyposis of the rectal remnant and ileal pouch in four patients with familial adenomatous polyposis: a pilot study.
   Roos VH, Meijer BJ, Kallenberg FGJ, Bastiaansen BAJ, et al · · 2020 · cited 15× · PMID 33376109 · DOI 10.1136/bmjgast-2020-000497
2. Advances in chemoprevention of familial adenomatous polyposis
   Li R, Cao R, Kang J, Li Y, et al ·