Gastrointestinal Stromal TumorsPhase 3NCT00688766
Study Evaluating IPI-504 in Patients With Gastrointestinal Stromal Tumors (GIST) Following Failure of at Least Imatinib and Sunitinib
A Phase 3 study testing retaspimycin hydrochloride (IPI-504) in people with Gastrointestinal Stromal Tumors, run by Infinity Pharmaceuticals, Inc..
Trial summary
IPI-504-06 is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of IPI-504 as compared to placebo in patients with metastatic and/or unresectable GIST following failure of at least imatinib and sunitinib. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gastrointestinal Stromal Tumors
- What is being tested?
- retaspimycin hydrochloride (IPI-504)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 47 participants
- Where
- No sites listed yet
- Study dates
- Aug 2008to May 2009
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- At least 18 years of age at the time of study randomization.
- Histologically confirmed metastatic and/or unresectable GIST.
- Measurable disease on CT or MRI as defined by RECIST.
- Documented radiographic progression or intolerance to imatinib and sunitinib.
- Clinical failure of the most recent prior therapy for GIST. Note: There is no limit to the number of prior therapies a patient may have received.
- Eastern Cooperative Oncology Group (ECOG) performance status: 0 or 1.
Show all 14Show fewer
- Hemoglobin ≥ 8.0 g/dL (80 g/L).
- Absolute Neutrophil Count ≥ 1500/µL (1.5 x 109/L).
- Platelets ≥ 100,000 /µL (100 x 109/L).
- ALT and AST ≤ 2.5 x upper limit of normal (ULN), or ≤ 5.0 x ULN if considered secondary to liver metastases.
- Alkaline phosphatase ≤ 2.5 x ULN, or ≤ 5.0 x ULN if considered secondary to liver metastases.
- Serum bilirubin ≤ 1.5 x ULN.
- PT and PTT ≤ 1.5 x ULN unless the patient is receiving warfarin. If the patient is receiving warfarin, the INR must be within therapeutic range.
- Serum creatinine ≤ 1.5 x ULN.
Who cannot join
- Previous administration of other known heat shock protein 90 (Hsp90) inhibitors.
- Surgery, radiotherapy, or lesion ablative procedure to the only area of measurable disease.
- Initiation or discontinuation of concurrent medication that is a potent CYP3A inhibitor less than 2 weeks prior to administration of IPI-504 or placebo.
- History of any of the following within the last 6 months: cardiac disease such as acute coronary syndrome or unstable angina, symptomatic congestive heart failure, uncontrolled hypertension, cirrhotic liver disease, cerebrovascular accident, or any other significant co-morbid condition or disease which, in the judgment of the investigator, would place the patient at undue risk or interfere with the study.
- Grade 3 or 4 hemorrhagic event within the last 6 months.
- Known human immunodeficiency virus positivity.
Show all 13Show fewer
- Sinus bradycardia (resting heart rate < 50 bpm) secondary to intrinsic conduction system disease.
- QTcF ≥ 470 milliseconds, or previous history of clinically significant QTc prolongation while taking other medications.
- History of prior malignancies within the past 3 years other than non-melanomatous skin cancers that have been controlled, prostate cancer that has been treated and has not recurred, non-muscle-invasive bladder cancer, and carcinoma in situ of the cervix.
- Active or recent history (within 3 months) of keratitis or keratoconjunctivitis confirmed by ophthalmology or optometry exam.
- Presence of Left Bundle Branch Block, Right Bundle Branch Block plus left anterior hemiblock, bifascicular block, or 3rd degree heart block. This does not include patients with a history of these events with adequate control by pacemaker.
- Known CNS metastases.
- Women who are pregnant or lactating.
About this study
Official title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Evaluating the Efficacy and Safety of IPI-504 in Patients With Metastatic and/or Unresectable GIST Following Failure of at Least Imatinib and Sunitinib
IPI-504-06 is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of IPI-504 as compared to placebo in patients with metastatic and/or unresectable GIST following failure of at least imatinib and sunitinib.
Approximately 195 patients will be randomized using a 2:1 ratio to receive either IPI-504 (N=130) or placebo (N=65). Upon unblinding, patients receiving either IPI-504 or placebo may receive IPI-504 in the open-label portion of the study if defined inclusion criteria are met.
Early and frequent imaging timepoints (Weeks 2, 5, 8, 14 and every 6 weeks thereafter) are incorporated into this study to capture progression events and limit patient exposure to ineffective agents.
- Conditions
- Gastrointestinal Stromal Tumors
- Being tested
- retaspimycin hydrochloride (IPI-504), placebo, Best supportive care
- Keywords
- GIST, Metastatic and/or Unresectable Gastrointestinal Stromal
- Medical subject headings
- Gastrointestinal Stromal Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
retaspimycin hydrochloride (IPI-504)Drug
IPI-504 is a novel small molecule inhibitor of heat shock protein 90 (Hsp90). Patients will receive 400 mg/m2 of IPI-504 as a 30-minute IV infusion twice weekly for 2 weeks followed by 1 week off.
placeboDrug
Patients will receive a 30-minute IV infusion twice weekly for 2 weeks followed by 1 week off.
Best supportive careOther
Best supportive care will be according to institutional standard, but will not include administration of systemic cancer-specific therapies including chemotherapies, biologic therapies, investigational therapies, TKIs (e.g., imatinib, sunitinib, nilotinib, dasatinib), or local therapies such as surgery, radiotherapy, or lesion ablative therapies.
| Study group | Role | What they receive |
|---|---|---|
| IPI-504 | Experimental | retaspimycin hydrochloride (IPI-504) plus best supportive care Receives: Drug: retaspimycin hydrochloride (IPI-504); Other: Best supportive care |
| Placebo | Placebo comparator | Placebo plus best supportive care Receives: Drug: placebo; Other: Best supportive care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare the progression free survival (PFS) in both study arms
Measured: Multiple timepoints
Secondary outcomes (4)
Compare the disease control rate (DCR) in both arms
Measured: Multiple timepoints
Compare the time to progression (TTP) in both arms
Measured: Multiple timepoints
Compare the overall survival (OS) in both arms
Measured: Continuous
Evaluate the safety and tolerability of IPI-504 in this patient population
Measured: Signing of the informed consent to 30 days after discontinuation of drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jun 2008 (estimated)
- Study startAug 2008
- Primary completionMay 2009
- Study completionMay 2009
Why it stopped: Based on Independent Data Monitoring Committee (IDMC) recommendation.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gastrointestinal Stromal TumorsStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Infinity Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- MedImmune LLC, AstraZeneca
- Responsible party
- Sponsor
- Investigators
- Pedro Santabarbara, M.D., Study director, Infinity Pharmaceuticals, Inc.George Demetri, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00688766
- Sponsor's protocol ID
- IPI-504-06
- Status last verified
- Dec 2012
- First submitted
- 29 May 2008
- Last updated
- 11 Dec 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Evaluating the Efficacy and Safety of IPI-504 in Patients With Metastatic and/or Unresectable GIST Following Failure of at Least Imatinib and Sunitinib
- ClinicalTrials.gov ID
- NCT00688766
- Acronym / sponsor ID
- IPI-504-06
- Conditions
- Gastrointestinal Stromal Tumors
- Keywords
- GIST, Metastatic and/or Unresectable Gastrointestinal Stromal
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Based on Independent Data Monitoring Committee (IDMC) recommendation.
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Infinity Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- MedImmune LLC, AstraZeneca
- Study start
- Aug 2008
- Primary completion
- May 2009 (actual)
- Study completion
- May 2009 (actual)
- First posted
- 3 Jun 2008 (estimated)
- 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.
IPI-504-06 is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of IPI-504 as compared to placebo in patients with metastatic and/or unresectable GIST following failure of at least imatinib and sunitinib.
Approximately 195 patients will be randomized using a 2:1 ratio to receive either IPI-504 (N=130) or placebo (N=65). Upon unblinding, patients receiving either IPI-504 or placebo may receive IPI-504 in the open-label portion of the study if defined inclusion criteria are met.
Early and frequent imaging timepoints (Weeks 2, 5, 8, 14 and every 6 weeks thereafter) are incorporated into this study to capture progression events and limit patient exposure to ineffective agents.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IPI-504 | Experimental | Drug: retaspimycin hydrochloride (IPI-504), Other: Best supportive careretaspimycin hydrochloride (IPI-504) plus best supportive care |
| Placebo | Placebo comparator | Drug: placebo, Other: Best supportive carePlacebo plus best supportive care |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- At least 18 years of age at the time of study randomization.
- Histologically confirmed metastatic and/or unresectable GIST.
- Measurable disease on CT or MRI as defined by RECIST.
- Documented radiographic progression or intolerance to imatinib and sunitinib.
- Clinical failure of the most recent prior therapy for GIST. Note: There is no limit to the number of prior therapies a patient may have received.
Key exclusion criteria (13 listed)
- Previous administration of other known heat shock protein 90 (Hsp90) inhibitors.
- Surgery, radiotherapy, or lesion ablative procedure to the only area of measurable disease.
- Initiation or discontinuation of concurrent medication that is a potent CYP3A inhibitor less than 2 weeks prior to administration of IPI-504 or placebo.
- History of any of the following within the last 6 months: cardiac disease such as acute coronary syndrome or unstable angina, symptomatic congestive heart failure, uncontrolled hypertension, cirrhotic liver disease, cerebrovascular accident, or any other significant co-morbid condition or disease which, in the judgment of the investigator, would place the patient at undue risk or interfere with the study.
- Grade 3 or 4 hemorrhagic event within the last 6 months.
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 |
|---|---|---|
| retaspimycin hydrochloride (IPI-504)IPI-504 is a novel small molecule inhibitor of heat shock protein 90 (Hsp90). Patients will receive 400 mg/m2 of IPI-504 as a 30-minute IV infusion twice weekly for 2 weeks followed by 1 week off. | Drug | IPI-504 |
| placeboPatients will receive a 30-minute IV infusion twice weekly for 2 weeks followed by 1 week off. | Drug | Placebo |
| Best supportive careBest supportive care will be according to institutional standard, but will not include administration of systemic cancer-specific therapies including chemotherapies, biologic therapies, investigational therapies, TKIs (e.g., imatinib, sunitinib, nilotinib, dasatinib), or local therapies such as surgery, radiotherapy, or lesion ablative therapies. | Other | IPI-504, Placebo |
Primary outcome
Compare the progression free survival (PFS) in both study arms
Time frame: Multiple timepoints
4 secondary outcomes
Compare the disease control rate (DCR) in both arms
Time frame: Multiple timepoints
Compare the time to progression (TTP) in both arms
Time frame: Multiple timepoints
Compare the overall survival (OS) in both arms
Time frame: Continuous
Evaluate the safety and tolerability of IPI-504 in this patient population
Time frame: Signing of the informed consent to 30 days after discontinuation of drug
Results and safety
The study was stopped early (reason given: Based on Independent Data Monitoring Committee (IDMC) recommendation.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2012.
- Gist Support International - Patient Advocacy GroupLink provided by the sponsor.
- The Liferaft Group - Patient Advocacy GroupLink provided by the sponsor.
Related trials for Gastrointestinal Stromal Tumors
RecruitingObservational study
A Patient-Derived 3D Bioprinted Colorectal Cancer-Stroma-Immune Triad Model Uncovers Microenvironmental Mechanisms of Chemoresistance and Immune Evasion
Colorectal Cancer
RecruitingPhase 2
A Study to Learn More About How Well Treatment With Sevabertinib (BAY 2927088) Tablets Works and How Safe it is in Participants Who Have a Solid Tumor With Mutations of the Human Epidermal Growth Factor Receptor 2 (HER2)
Advanced Solid Tumors, HER2 Mutation
RecruitingPhase 2
Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumor
RecruitingObservational study
Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
Gastric Cancer, Gastric Neoplasm
RecruitingPhase 1
A Study of Ziftomenib in Combination With Imatinib in Patients With Advanced Gastrointestinal Stromal Tumors (GIST)
Gastrointestinal Stromal Tumor (GIST), Gastrointestinal Stromal Cancer
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. This study is not recruiting at the moment.
Pedro Santabarbara, M.D. study director
Infinity Pharmaceuticals, Inc.
George Demetri, MD principal investigator
Dana-Farber Cancer Institute
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing retaspimycin hydrochloride (IPI-504) in people with Gastrointestinal Stromal Tumors, run by Infinity Pharmaceuticals, Inc..
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 14 inclusion and 13 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: stopped early, as of the sponsor's last update on 11 Dec 2012. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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?
The study was stopped early (reason given: Based on Independent Data Monitoring Committee (IDMC) recommendation.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Compare the progression free survival (PFS) in both study arms (time frame: Multiple timepoints). 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 11 Dec 2012.