Gastrointestinal Stromal Tumor of RectumPhase 3PIRKERNCT05970900
Preoperative Imatinib Mesylate Combined With Rectal-sparing Surgery in Patients With c-KIT Gene-mutant Rectal GIST
A Phase 3 study testing Imatinib Mesylate and Local resection in people with Gastrointestinal Stromal Tumor of Rectum, run by Fujian Medical University Union Hospital at 1 site in China.
Trial summary
Prior to the implementation of preoperative imatinib mesylate therapy, a considerable percentage (ranging from 34.5% to 67.5%) of individuals diagnosed with rectal gastrointestinal stromal tumors (GIST) underwent abdominoperineal resection (APR), a surgical procedure that involved the removal of the anus and… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Gastrointestinal Stromal Tumor of Rectum
- What is being tested?
- Imatinib Mesylate and Local resection
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteChina
- Study dates
- 1 Oct 2023to 1 Oct 2029
- Phase
- Phase 3
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Over the age of 18.
- Newly pathology-diagnosed rectal GIST
- Tumor > 2cm; local resection of R0 is not possible in the initial evaluation.
- The lower margin of the tumor is ≤ 5cm from the anal verge.
- C-KIT gene mutation.
- Male or non-pregnant female.
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- ECOG score 0-2.
- Did not receive targeted therapy before the start of the clinical trial.
- Sufficient organ functions are defined as follows:
- Total bilirubin < 1.5×ULN (upper limit of normal, ULN), serum AST (SGOT) and ALT (SGPT) < 2. 5 × ULN, creatinine < 1.5×ULN, neutrophil count > 1. 5 ×109 / L, platelet > 100 × 109 / L.
- The patient's informed consent has been obtained.
Who cannot join
- Pathology is non-rectal GIST.
- Under the age of 18.
- Patients with distant metastasis.
- The patient is not permitted to have additional primary malignant tumors within five years unless those tumors are currently deemed clinically insignificant and do not necessitate active intervention, such as basal cell skin cancer or cervical cancer in situ. The presence of any other malignant diseases is strictly prohibited.
- Individuals diagnosed with stage III or IV cardiac conditions, specifically congestive heart failure and myocardial infarction occurring within six months prior to the commencement of the study.
- The patient presents with severe and/or uncontrolled medical ailments, such as unmanaged diabetes, advanced chronic kidney disease, or active uncontrolled infection.
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- Co-administration of imatinib with warfarin or acetaminophen is contraindicated, necessitating the substitution of alternative medications (e.g., low molecular weight heparin in place of warfarin).
- Subjects undergoing radiotherapy, chemotherapy, and/or targeted therapy.
- Pregnant or lactating female patients.
- Cognitive or psychiatric disorders.
- Profound cardiac, hepatic, and renal dysfunction.
- Non-adherence by the patient or the researchers' assessment of the patient's inability to complete the entire trial.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Preoperative Imatinib Mesylate Combined With Rectal-sparing Surgery in Patients With c-KIT Gene-mutant Rectal GIST: an Open-label, Single-arm, Phase III Trial（PIRKER）
Prior to the implementation of preoperative imatinib mesylate therapy, a considerable percentage (ranging from 34.5% to 67.5%) of individuals diagnosed with rectal gastrointestinal stromal tumors (GIST) underwent abdominoperineal resection (APR), a surgical procedure that involved the removal of the anus and necessitated a permanent colostomy.
This study aims to investigate the safety and viability of an organ-preserving approach involving preoperative imatinib mesylate treatment in conjunction with local resection for rectal GIST, specifically targeting patients with c-KIT gene mutations.
Read the detailed description
Prior to the implementation of preoperative imatinib mesylate therapy, a considerable percentage (ranging from 34.5% to 67.5%) of individuals diagnosed with rectal gastrointestinal stromal tumors (GIST) underwent abdominoperineal resection (APR), a surgical procedure that involved the removal of the anus and necessitated a permanent colostomy.
Previous studies have established that preoperative administration of imatinib mesylate effectively diminishes the size of rectal gastrointestinal stromal tumors (GIST) and enhances the likelihood of sphincter preservation. After initiating preoperative imatinib mesylate treatment, the sphincter preservation rate has notably escalated from 4.2% to 33.0%-94.9%.
In theory, lymph node resection is not required for Gastrointestinal Stromal Tumors (GIST); the local excision of rectal GIST enables sphincter preservation and yields satisfactory anal function and quality of life (QoL). Various surgical techniques are utilized for local excision, including traditional transanal (TA) and transanal minimally invasive surgery (TAMIS) approaches.
This study aims to explore the safety and feasibility of an organ-preservation strategy of preoperative imatinib mesylate combined with local resection in rectal gastrointestinal stromal tumor (GIST), specifically for patients with c-KIT gene mutations.
- Conditions
- Gastrointestinal Stromal Tumor of Rectum
- Being tested
- Imatinib Mesylate, Local resection
- Keywords
- Gastrointestinal Stromal Tumor, Rectum, Imatinib mesylate, c-KIT gene, Local resection
- Medical subject headings
- Gastrointestinal Stromal Tumors, Piebaldism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Imatinib MesylateDrug
Also called Gleevec
- For patients with c-KIT exon 11 mutation, imatinib mesylate, 400mg, qd.
- For patients with c-KIT exon 9 mutation, imatinib mesylate, 600mg or 800mg, qd.
Local resectionProcedure or surgery
According to the characteristics of the location of the tumor, the surgeon decides the surgical approach based on the existing literature and the availability of surgical equipment, including:
- Local transanal resection (TA)
- Local resection transsacralapproach
- Local resection via perineal approach
- Local resection transvaginal approach
| Study group | Role | What they receive |
|---|---|---|
| Preoperative Imatinib + local excision | Experimental | Following the attainment of the maximum treatment response through imatinib mesylate administration, typically occurring within 6-12 months, as evidenced by two consecutive imaging evaluations, the tumor exhibited no further reduction in size, thus necessitating the selection of surgical intervention.
According to the characteristics of the location of the tumor, the surgeon decides the surgical approach based on the existing literature and the availability of surgical equipment, including:
1. Local transanal resection (TA)
2. Local resection transsacralapproach
3. Local resection via perineal approach
4. Local resection transvaginal approach Receives: Drug: Imatinib Mesylate; Procedure: Local resection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Organ preservation
Rectum intact, owing to no total mesorectal excision (TME), no locoregional regrowth unless amenable to limited, curative (R0) salvage surgery by local excision (LE) and no permanent stoma (including a never reversed protective stoma, or a stoma owing to toxicities and/or poor functional outcomes)
Measured: 18 months
Secondary outcomes (6)
3-year disease-free survival
The proportion of participants who remain disease-free at 3 years after surgery
Measured: 36 months
Local recurrence rate
The local recurrence rate is defined as the incidence detection of a tumor involving the bowel wall only that occurs after LE or TME
Measured: 36 months
Overall survival
The proportion of participants who remain survival at 3 years after surgery
Measured: 36 months
R0 resection rate
The R0 resection rate is defined as the rate of R0 resection
Measured: 18 months
Quality of life based on EORTC-QLQs-C30 and EORTC-QLQs-CR29
Quality of life accessed by EORTC-QLQs-C30 and EORTC-QLQs-CR29 questionnaire
Measured: Baseline, 3 months, 12 months, 24 months, and 36 months after surgery
Anorectal function
Anorectal function based on LARS score
Measured: Baseline, 3 months, 12 months, 24 months, and 36 months after surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2023
- Study start1 Oct 2023 (estimated)
- Primary completion1 Oct 2026 (estimated)
- Study completion1 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gastrointestinal Stromal Tumor of RectumStudies of the same condition, open and under way.
- More trials run by Fujian Medical University Union HospitalThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 23 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fujian Medical University Union Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, 蒋伟忠, Fujian Medical University Union Hospital
- Investigators
- Weizhong Jiang, MD, Principal investigator, Fujian Medical University Union Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05970900
- Sponsor's protocol ID
- 2023XHYG0025-01
- Acronym
- PIRKER
- Status last verified
- Jul 2023
- First submitted
- 23 Jul 2023
- Last updated
- 1 Aug 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Preoperative Imatinib Mesylate Combined With Rectal-sparing Surgery in Patients With c-KIT Gene-mutant Rectal GIST: an Open-label, Single-arm, Phase III Trial（PIRKER）
- ClinicalTrials.gov ID
- NCT05970900
- Acronym / sponsor ID
- PIRKER / 2023XHYG0025-01
- Conditions
- Gastrointestinal Stromal Tumor of Rectum
- Keywords
- Gastrointestinal Stromal Tumor, Rectum, Imatinib mesylate, c-KIT gene, Local resection
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 23 participants (estimated)
- Lead sponsor
- Fujian Medical University Union HospitalUniversity, hospital or other
- Study start
- 1 Oct 2023 (estimated)
- Primary completion
- 1 Oct 2026 (estimated)
- Study completion
- 1 Oct 2029 (estimated)
- First posted
- 1 Aug 2023 (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.
Prior to the implementation of preoperative imatinib mesylate therapy, a considerable percentage (ranging from 34.5% to 67.5%) of individuals diagnosed with rectal gastrointestinal stromal tumors (GIST) underwent abdominoperineal resection (APR), a surgical procedure that involved the removal of the anus and necessitated a permanent colostomy.
This study aims to investigate the safety and viability of an organ-preserving approach involving preoperative imatinib mesylate treatment in conjunction with local resection for rectal GIST, specifically targeting patients with c-KIT gene mutations.
Detailed description
Prior to the implementation of preoperative imatinib mesylate therapy, a considerable percentage (ranging from 34.5% to 67.5%) of individuals diagnosed with rectal gastrointestinal stromal tumors (GIST) underwent abdominoperineal resection (APR), a surgical procedure that involved the removal of the anus and necessitated a permanent colostomy.
Previous studies have established that preoperative administration of imatinib mesylate effectively diminishes the size of rectal gastrointestinal stromal tumors (GIST) and enhances the likelihood of sphincter preservation. After initiating preoperative imatinib mesylate treatment, the sphincter preservation rate has notably escalated from 4.2% to 33.0%-94.9%.
In theory, lymph node resection is not required for Gastrointestinal Stromal Tumors (GIST); the local excision of rectal GIST enables sphincter preservation and yields satisfactory anal function and quality of life (QoL). Various surgical techniques are utilized for local excision, including traditional transanal (TA) and transanal minimally invasive surgery (TAMIS) approaches.
This study aims to explore the safety and feasibility of an organ-preservation strategy of preoperative imatinib mesylate combined with local resection in rectal gastrointestinal stromal tumor (GIST), specifically for patients with c-KIT gene mutations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Preoperative Imatinib + local excision | Experimental | Drug: Imatinib Mesylate, Procedure: Local resectionFollowing the attainment of the maximum treatment response through imatinib mesylate administration, typically occurring within 6-12 months, as evidenced by two consecutive imaging evaluations, the tumor exhibited no further reduction in size, thus necessitating the selection of surgical intervention. According to the characteristics of the location of the tumor, the surgeon decides the surgical approach based on the existing literature and the availability of surgical equipment, including: 1. Local transanal resection (TA) 2. Local resection transsacralapproach 3. Local resection via perineal approach 4. Local resection transvaginal approach |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Over the age of 18.
- Newly pathology-diagnosed rectal GIST
- Tumor > 2cm; local resection of R0 is not possible in the initial evaluation.
- The lower margin of the tumor is ≤ 5cm from the anal verge.
- C-KIT gene mutation.
Key exclusion criteria (12 listed)
- Pathology is non-rectal GIST.
- Under the age of 18.
- Patients with distant metastasis.
- The patient is not permitted to have additional primary malignant tumors within five years unless those tumors are currently deemed clinically insignificant and do not necessitate active intervention, such as basal cell skin cancer or cervical cancer in situ. The presence of any other malignant diseases is strictly prohibited.
- Individuals diagnosed with stage III or IV cardiac conditions, specifically congestive heart failure and myocardial infarction occurring within six months prior to the commencement of the study.
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 |
|---|---|---|
| Imatinib MesylateAlso: Gleevec1. For patients with c-KIT exon 11 mutation, imatinib mesylate, 400mg, qd. 2. For patients with c-KIT exon 9 mutation, imatinib mesylate, 600mg or 800mg, qd. | Drug | Preoperative Imatinib + local excision |
| Local resectionAccording to the characteristics of the location of the tumor, the surgeon decides the surgical approach based on the existing literature and the availability of surgical equipment, including: 1. Local transanal resection (TA) 2. Local resection transsacralapproach 3. Local resection via perineal approach 4. Local resection transvaginal approach | Procedure or surgery | Preoperative Imatinib + local excision |
Primary outcome
Organ preservation
Time frame: 18 months
Rectum intact, owing to no total mesorectal excision (TME), no locoregional regrowth unless amenable to limited, curative (R0) salvage surgery by local excision (LE) and no permanent stoma (including a never reversed protective stoma, or a stoma owing to toxicities and/or poor functional outcomes)
6 secondary outcomes
3-year disease-free survival
Time frame: 36 months
Local recurrence rate
Time frame: 36 months
Overall survival
Time frame: 36 months
R0 resection rate
Time frame: 18 months
Quality of life based on EORTC-QLQs-C30 and EORTC-QLQs-CR29
Time frame: Baseline, 3 months, 12 months, 24 months, and 36 months after surgery
Anorectal function
Time frame: Baseline, 3 months, 12 months, 24 months, and 36 months after surgery
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 1 Aug 2023.
- Individual participant dataSharing planned: No
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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.
Weizhong Jiang, MD principal investigator
Fujian Medical University Union Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Imatinib Mesylate and Local resection in people with Gastrointestinal Stromal Tumor of Rectum, run by Fujian Medical University Union Hospital 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 11 inclusion and 12 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: opening soon, as of the sponsor's last update on 1 Aug 2023. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Weizhong Jiang, MD (+8613763828825, Jiangwz362100@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Organ preservation (time frame: 18 months). 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 1 Aug 2023.