Peutz-Jeghers SyndromeNCT06722534
Celecoxib for Prevention of Progression in Peutz-Jeghers Syndrome
A study testing Celecoxib 400mg in people with Peutz-Jeghers Syndrome and Celecoxib, run by Air Force Military Medical University, China at 1 site in China.
Trial summary
The Peutz-Jeghers Syndrome (PJS) is a rare autosomal dominant syndrome characterized by mucocutaneous pigmentations, multiple gastrointestinal hamartomatous polyps, and an elevated risk of developing malignancies. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Peutz-Jeghers Syndrome, Celecoxib, Small Bowel Polyp
- What is being tested?
- Celecoxib 400mg
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 1 siteChina
- Study dates
- 1 Feb 2025to 1 Jan 2029
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with PJS ≥ 8 years of age
- Diagnostic criteria for PJS: meeting any of the following criteria or presence of an STK11 gene variant:
- Two or more histologically confirmed PJS hamartomatous polyps;
- Any number of PJS polyps detected in an individual with a family history of PJS in close relative(s);
- Characteristic mucocutaneous pigmentation in an individual with a family history of PJS in close relative(s);
- Any number of PJS polyps in an individual with characteristic mucocutaneous pigmentation.
Who cannot join
- Allergy to NSAIDs;
- Long-term use of any dose of NSAIDs, including aspirin or celecoxib, within 6 months prior to enrollment (willing to undergo a 3-month washout period to restore eligibility);
- Imaging indicate small intestinal polyps ≥ 3 cm in diameter, intestinal intussusception, intestinal obstruction or intestinal tumor at the time of enrollment;
- Surgical treatment for small intestinal polyps within 2 years prior to enrollment;
- Anticipated small bowel resection due to severe polyps within 6 months of enrollment;
- Receiving other medications for gastrointestinal polyps;
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- Peptic ulcer within 3 months prior to enrollment;
- Unstable cardiorespiratory condition;
- Serious renal, hepatic or haematological dysfunction (creatinine >1.5 × ULN; ALT >1.5 × ULN, AST >1.5 × ULN, ALP >1.5 × ULN, TBIL >2 × ULN; haemoglobin <10 g/dL, platelet count <100,000/mL, white blood cells <3000/mL) or other systemic diseases are unsuitable for participation in this study;
- Pregnancy or breastfeeding;
- Unwilling or unable to sign the informed consent form
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Xijing Hospital of Digestive Diseases, Air Force Military Medical University
Xi'an, Shaanxi, 710032
Hui Luo, 86-29-84771536, huiluowork@163.com
Qinrong Wang
About this study
Official title: Celecoxib for Prevention of Progression in Peutz-Jeghers Syndrome: A Double-blind, Randomized, Placebo-controlled Trial
The Peutz-Jeghers Syndrome (PJS) is a rare autosomal dominant syndrome characterized by mucocutaneous pigmentations, multiple gastrointestinal hamartomatous polyps, and an elevated risk of developing malignancies. Patients with PJS often experience recurrent gastrointestinal polyps that gradually increase in number and size, requiring repeated treatments. As the disease progresses, most patients are forced to undergo multiple surgical or endoscopic treatments. Small bowel polyps develop in 60-90% of patients with PJS, and intussusception occurs in 65% of these patients. Currently, on-demand surgery or scheduled endoscopic polypectomy is the standard of care for the management of small bowel polyps, and among patients who have undergone an initial surgery, reoperation is performed in up to 40% within 5 years. In addition, 8-40% of patients develop small bowel polyp-related complications even with multiple endoscopic treatments. However, surgery and endoscopic treatments are associated with complications, including short bowel syndrome, intestinal adhesions, bowel perforation and bleeding, and health-related quality of life. These problems often lead to decreased patient compliance and even treatment resistance, which increases the risk of disease progression. Because surgical and endoscopic treatment do not completely eliminate the potential for future polyps or extraintestinal neoplasms, there is an unmet medical need for the identification and use of pharmacologic agents to delay endoscopic or surgical interventions.
Cyclooxygenase (COX) is overexpressed in hamartomatous polyp tissue from PJS individuals, which may provide an avenue for possible effective chemoprevention of polyp formation and growth in PJS. Celecoxib, a COX-2 inhibitor, has been shown to reduce polyp burden by 54% in PJS model mice. In addition, the study evaluated the treatment effect of celecoxib on six patients with PJS, two of whom experienced a reduction in gastric polyp burden after six months. These findings provide preliminary evidence that celecoxib may delay the progression of PJS as a potential pharmacological prophylaxis.
Investigators plan to conduct a multicenter, double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of celecoxib, and they will use a time-to-event analysis with a composite efficacy end point to determine whether celecoxib can delay disease progression or reduce the need for important endoscopic or surgical procedures in patients with PJS.
- Conditions
- Peutz-Jeghers Syndrome, Celecoxib, Small Bowel Polyp
- Being tested
- Celecoxib 400mg, Placebo
- Medical subject headings
- Peutz-Jeghers Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Celecoxib 400mgDrug
Participants in the interventional group receive 200 mg celecoxib twice daily for 6 months
PlaceboDrug
Participants in the control group receive identically appearing placebo twice daily for 6 months
| Study group | Role | What they receive |
|---|---|---|
| Celecoxib group | Experimental | Participants in the interventional group receive 200 mg celecoxib twice daily for 6 months Receives: Drug: Celecoxib 400mg |
| Placebo group | Active comparator | Participants in the control group receive identically appearing placebo twice daily for 6 months Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
The progression of small bowel disease (composite end point)
- 25% increase in the mean or median diameter of the 5 largest small bowel polyps in abdominal CT/MR (less than 5 polyps will be counted as actual number);
- Imaging suggestive small bowel obstruction or small bowel intussusception;
- Enteroscopy treatment is required due to the large size of the small bowel polyps or related symptoms;
- Surgical treatment is required for intestinal obstruction or intussusception when conservative treatment is ineffective, unresectable small bowel polyps by enteroscopy, or other serious complications associated with small bowel polyps.
Measured: 2 years
Secondary outcomes (9)
Drug-related adverse events (Severity assessed according to CTCAE 5.0)
Measured: 2 years
Burden of gastroduodenal and colonic polyps based on gastrointestinal endoscopy (mean or median diameter of 5 largest polyps)
Measured: 2 years
Other complications of PJS polyps
- Gastrointestinal bleeding (hemoglobin drop of 3 g)
- Malnutrition (albumin ≤ 35 g/L)
Measured: 2 years
European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30（EORTC QLQ-C30）
Changes from baseline by time point in EORTC QLQ-C30 scores
Measured: 2 years
European Organization for Research and Treatment of Cancer Quality of Life Questionnaire ⁃ Colorectal Cancer 29（EORTC QLQ ⁃CR29）
Changes from baseline by time point in EORTC QLQ-CR29 scores
Measured: 2 years
EuroQol Five Dimensions Questionnaire（EQ-5D）
Changes from baseline by time point in EQ-5D scores
Measured: 2 years
Number of enteroscopy or surgical treatments and related complications
Measured: 2 years
Incidence of gastrointestinal and other systemic tumors
Measured: 2 years
The mortality rate
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2024
- Study start1 Feb 2025
- Primary completion1 Jan 2029 (estimated)
- Study completion1 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Peutz-Jeghers SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants in the interventional group receive 200 mg celecoxib twice daily for 6 months
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Air Force Military Medical University, China
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yanglin Pan, Air Force Military Medical University, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06722534
- Sponsor's protocol ID
- KY20242368
- Status last verified
- May 2025
- First submitted
- 12 Nov 2024
- Last updated
- 30 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Other researchers can contact PI to get IPD.
Study at a glance
From the registry record, last updated by the sponsor on 30 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Celecoxib for Prevention of Progression in Peutz-Jeghers Syndrome: A Double-blind, Randomized, Placebo-controlled Trial
- ClinicalTrials.gov ID
- NCT06722534
- Acronym / sponsor ID
- KY20242368
- Conditions
- Peutz-Jeghers Syndrome, Celecoxib, Small Bowel Polyp
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Air Force Military Medical University, ChinaUniversity, hospital or other
- Study start
- 1 Feb 2025 (actual)
- Primary completion
- 1 Jan 2029 (estimated)
- Study completion
- 1 Jan 2029 (estimated)
- First posted
- 9 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Peutz-Jeghers Syndrome (PJS) is a rare autosomal dominant syndrome characterized by mucocutaneous pigmentations, multiple gastrointestinal hamartomatous polyps, and an elevated risk of developing malignancies. Patients with PJS often experience recurrent gastrointestinal polyps that gradually increase in number and size, requiring repeated treatments. As the disease progresses, most patients are forced to undergo multiple surgical or endoscopic treatments. Small bowel polyps develop in 60-90% of patients with PJS, and intussusception occurs in 65% of these patients. Currently, on-demand surgery or scheduled endoscopic polypectomy is the standard of care for the management of small bowel polyps, and among patients who have undergone an initial surgery, reoperation is performed in up to 40% within 5 years. In addition, 8-40% of patients develop small bowel polyp-related complications even with multiple endoscopic treatments. However, surgery and endoscopic treatments are associated with complications, including short bowel syndrome, intestinal adhesions, bowel perforation and bleeding, and health-related quality of life. These problems often lead to decreased patient compliance and even treatment resistance, which increases the risk of disease progression. Because surgical and endoscopic treatment do not completely eliminate the potential for future polyps or extraintestinal neoplasms, there is an unmet medical need for the identification and use of pharmacologic agents to delay endoscopic or surgical interventions.
Cyclooxygenase (COX) is overexpressed in hamartomatous polyp tissue from PJS individuals, which may provide an avenue for possible effective chemoprevention of polyp formation and growth in PJS. Celecoxib, a COX-2 inhibitor, has been shown to reduce polyp burden by 54% in PJS model mice. In addition, the study evaluated the treatment effect of celecoxib on six patients with PJS, two of whom experienced a reduction in gastric polyp burden after six months. These findings provide preliminary evidence that celecoxib may delay the progression of PJS as a potential pharmacological prophylaxis.
Investigators plan to conduct a multicenter, double-blind, randomized, placebo-controlled trial to evaluate the efficacy and safety of celecoxib, and they will use a time-to-event analysis with a composite efficacy end point to determine whether celecoxib can delay disease progression or reduce the need for important endoscopic or surgical procedures in patients with PJS.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Participants in the interventional group receive 200 mg celecoxib twice daily for 6 months
| Arm | Type | Receives |
|---|---|---|
| Celecoxib group | Experimental | Drug: Celecoxib 400mgParticipants in the interventional group receive 200 mg celecoxib twice daily for 6 months |
| Placebo group | Active comparator | Drug: PlaceboParticipants in the control group receive identically appearing placebo twice daily for 6 months |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patients with PJS ≥ 8 years of age
- Diagnostic criteria for PJS: meeting any of the following criteria or presence of an STK11 gene variant:
- Two or more histologically confirmed PJS hamartomatous polyps;
- Any number of PJS polyps detected in an individual with a family history of PJS in close relative(s);
- Characteristic mucocutaneous pigmentation in an individual with a family history of PJS in close relative(s);
Key exclusion criteria (11 listed)
- Allergy to NSAIDs;
- Long-term use of any dose of NSAIDs, including aspirin or celecoxib, within 6 months prior to enrollment (willing to undergo a 3-month washout period to restore eligibility);
- Imaging indicate small intestinal polyps ≥ 3 cm in diameter, intestinal intussusception, intestinal obstruction or intestinal tumor at the time of enrollment;
- Surgical treatment for small intestinal polyps within 2 years prior to enrollment;
- Anticipated small bowel resection due to severe polyps within 6 months of enrollment;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Celecoxib 400mgParticipants in the interventional group receive 200 mg celecoxib twice daily for 6 months | Drug | Celecoxib group |
| PlaceboParticipants in the control group receive identically appearing placebo twice daily for 6 months | Drug | Placebo group |
Primary outcome
The progression of small bowel disease (composite end point)
Time frame: 2 years
1. 25% increase in the mean or median diameter of the 5 largest small bowel polyps in abdominal CT/MR (less than 5 polyps will be counted as actual number); 2. Imaging suggestive small bowel obstruction or small bowel intussusception; 3. Enteroscopy treatment is required due to the large size of the small bowel polyps or related symptoms; 4. Surgical treatment is required for intestinal obstruction or intussusception when conservative treatment is ineffective, unresectable small bowel polyps by enteroscopy, or other serious complications associated with small bowel polyps.
9 secondary outcomes
Drug-related adverse events (Severity assessed according to CTCAE 5.0)
Time frame: 2 years
Burden of gastroduodenal and colonic polyps based on gastrointestinal endoscopy (mean or median diameter of 5 largest polyps)
Time frame: 2 years
Other complications of PJS polyps
Time frame: 2 years
European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30（EORTC QLQ-C30）
Time frame: 2 years
European Organization for Research and Treatment of Cancer Quality of Life Questionnaire ⁃ Colorectal Cancer 29（EORTC QLQ ⁃CR29）
Time frame: 2 years
EuroQol Five Dimensions Questionnaire（EQ-5D）
Time frame: 2 years
Number of enteroscopy or surgical treatments and related complications
Time frame: 2 years
Incidence of gastrointestinal and other systemic tumors
Time frame: 2 years
The mortality rate
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 May 2025.
- Individual participant dataSharing planned: No. Other researchers can contact PI to get IPD.
Related trials for Peutz-Jeghers Syndrome
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Celecoxib 400mg in people with Peutz-Jeghers Syndrome and Celecoxib, run by Air Force Military Medical University, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Hui Luo Associate professor (86-29-84771536, huiluowork@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The progression of small bowel disease (composite end point) (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 May 2025.