Waldenstrom's MacroglobulinemiaPhase 2NCT02439138
Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia
A Phase 2 study testing GS-1101 in people with Waldenstrom's Macroglobulinemia, run by Dana-Farber Cancer Institute at 1 site in United States.
Trial summary
This research study is evaluating a drug called idelalisib (formerly known as GS-1101 or CAL-101) as a possible treatment for Waldenstrom's Macroglobulinemia (WM). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Waldenstrom's Macroglobulinemia
- What is being tested?
- GS-1101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2015to Apr 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- Clinicopathological diagnosis of Waldenstrom's Macroglobulinemia and meeting criteria for treatment using consensus panel criteria from the Second International Workshop on Waldenstrom's macroglobulinemia (Owen 2003; Kyle 2003).
- Measurable disease, defined as presence of serum immunoglobulin M (IgM) with a minimum IgM level of > 2 times the upper limit of normal of each institution is required.
- Have received at least one prior therapy for WM.
- Age ≥18 years.
- ECOG performance status <2 (see Appendix A.).
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- Participants must have normal organ and marrow function as defined below:
- Absolute neutrophil count > 1,000/mm3
- Platelets > 50,000/mm3
- Hemoglobin > 8 g/dL
- Total bilirubin ≤1.5 mg/dL or < 2 mg/dL if attributable to hepatic infiltration by neoplastic disease
- AST (SGOT) and ALT (SGPT) < 2.5 X institutional upper limit of normal
- Creatinine ≤ 2 mg/dL
- Not on any active therapy for other malignancies with the exception of topical therapies for basal cell or squamous cell cancers of the skin.
- Females of childbearing potential (FCBP) must agree to use two reliable forms of contraception simultaneously or have or will have complete abstinence from heterosexual intercourse during the following time periods related to this study:
- 1) while participating in the study; and 2) for at least 28 days after discontinuation from the study. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy. FCBP must be referred to a qualified provider of contraceptive methods if needed.
- Able to adhere to the study visit schedule and other protocol requirements.
- Ability to understand and the willingness to sign a written informed consent document
Who cannot join
- Any serious medical condition, laboratory abnormality, uncontrolled intercurrent illness, or psychiatric illness/social condition that would prevent the participant from signing the informed consent form
- Concurrent use of any other anti-cancer agents or treatments or any other study agents
- Prior exposure to idelalisib
- Prior or ongoing clinically significant illness, medical condition, surgical history, physical finding, ECG finding, or laboratory abnormality that, in the investigator's opinion, could affect the safety of the patient; alter the absorption, distribution, metabolism or excretion of Idelalisib; or impair the assessment of study results
- Grade > 2 toxicity (other than alopecia) continuing from prior anti-cancer therapy
- Known central nervous system lymphoma
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- Significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of screening
- New York Heart Association classification III or IV heart failure.
- Malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel, ulcerative colitis, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.
- Known history of Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), and/or Hepatitis C Virus (HCV) infection
- Lactating or pregnant women
- Inability to swallow capsules
- History of non-compliance to medical regimens
- Unwilling or unable to comply with the protocol
Trial locations and contacts
1 site in 1 country.
United States1 site
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
About this study
Official title: Phase II Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia
This research study is evaluating a drug called idelalisib (formerly known as GS-1101 or CAL-101) as a possible treatment for Waldenstrom's Macroglobulinemia (WM).
Read the detailed description
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug, idelalisib, to learn whether idelalisib works in treating a specific cancer. "Investigational" means that idelalisib is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if idelalisib is effective for treating different types of cancer. Idelalisib has already been approved in the US by the FDA to treat patients with relapsed chronic lymphocytic leukemia, follicular lymphoma and small lymphocytic lymphoma.
Idelalisib is a newly discovered drug that is being developed as an anti-cancer agent. This drug has been used in laboratory experiments and other research studies in B-cell malignancies and information from those other research studies suggests that idelalisib may help to target the tumor cells in B-cell malignancies, including WM. B cells are a type of white blood cell responsible for making antibodies.
In this research study, the investigators are testing the safety and efficacy of idelalisib as a treatment option for relapsed or refractory Waldenstrom's Macroglobulinemia.
- Conditions
- Waldenstrom's Macroglobulinemia
- Being tested
- GS-1101
- Keywords
- Waldenstrom's Macroglobulinemia
- Medical subject headings
- Waldenstrom Macroglobulinemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GS-1101Drug
Also called Idealisib, Zydelig, CAL-101
Oral twice daily for 6 months followed by once daily until disease progression or unacceptable toxicity.
| Study group | Role | What they receive |
|---|---|---|
| GS-1101 | Experimental | After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression. Receives: Drug: GS-1101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
ORR measured by decrease in serum IgM level by at least 25% from baseline.
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Secondary outcomes (7)
Percentage of Participants With Adverse Events
Assess the safety and tolerability of idelalisib
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Complete Response (CR)
CR measured by decrease in serum IgM levels to normal range, disappearnace of monoclonal protein by immunofixation, no evidence of bone marrow involvement, and resolution of any extramedullary disease by CT scan.
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Very Good Partial Response (VGPR)
VGPR measured by decrease in serum IgM levels of at least 90% from baseline.
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Partial Response (PR)
PR measured by decrease in serum IgM levels of between 25% and 50% from baseline.
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Minimal Response
Minimal response measured by decrease in serum IgM levels of between 25% and 50%.
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Stable Disease
Stable disease measured by serum IgM levels <25% reduced from baseline.
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Progressive Disease
Progressive disease measured by an 25% increase in serum IgM level with an absolute increase of at least 500mg/dL from the lowest attained IgM on therapy.
Measured: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2015 (estimated)
- Study startOct 2015
- Primary completionApr 2016
- Study completionApr 2016
Why it stopped: Safety issues from trials in CLL
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Waldenstrom's MacroglobulinemiaStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Gilead Sciences
- Responsible party
- Principal investigator, Jorge J. Castillo, MD, Dana-Farber Cancer Institute
- Investigators
- Jorge J. Castillo, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02439138
- Sponsor's protocol ID
- 15-040
- Other IDs
- ISR IN-US-313-1609
- Status last verified
- Nov 2016
- First submitted
- 29 Apr 2015
- Last updated
- 11 Jan 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia
- ClinicalTrials.gov ID
- NCT02439138
- Acronym / sponsor ID
- 15-040
- Conditions
- Waldenstrom's Macroglobulinemia
- Keywords
- Waldenstrom's Macroglobulinemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Safety issues from trials in CLL
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Gilead Sciences
- Study start
- Oct 2015
- Primary completion
- Apr 2016 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 8 May 2015 (estimated)
- Results
- Posted 11 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is evaluating a drug called idelalisib (formerly known as GS-1101 or CAL-101) as a possible treatment for Waldenstrom's Macroglobulinemia (WM).
Detailed description
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug, idelalisib, to learn whether idelalisib works in treating a specific cancer. "Investigational" means that idelalisib is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if idelalisib is effective for treating different types of cancer. Idelalisib has already been approved in the US by the FDA to treat patients with relapsed chronic lymphocytic leukemia, follicular lymphoma and small lymphocytic lymphoma.
Idelalisib is a newly discovered drug that is being developed as an anti-cancer agent. This drug has been used in laboratory experiments and other research studies in B-cell malignancies and information from those other research studies suggests that idelalisib may help to target the tumor cells in B-cell malignancies, including WM. B cells are a type of white blood cell responsible for making antibodies.
In this research study, the investigators are testing the safety and efficacy of idelalisib as a treatment option for relapsed or refractory Waldenstrom's Macroglobulinemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GS-1101 | Experimental | Drug: GS-1101After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis. -- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- Clinicopathological diagnosis of Waldenstrom's Macroglobulinemia and meeting criteria for treatment using consensus panel criteria from the Second International Workshop on Waldenstrom's macroglobulinemia (Owen 2003; Kyle 2003).
- Measurable disease, defined as presence of serum immunoglobulin M (IgM) with a minimum IgM level of > 2 times the upper limit of normal of each institution is required.
- Have received at least one prior therapy for WM.
- Age ≥18 years.
Key exclusion criteria (14 listed)
- Any serious medical condition, laboratory abnormality, uncontrolled intercurrent illness, or psychiatric illness/social condition that would prevent the participant from signing the informed consent form
- Concurrent use of any other anti-cancer agents or treatments or any other study agents
- Prior exposure to idelalisib
- Prior or ongoing clinically significant illness, medical condition, surgical history, physical finding, ECG finding, or laboratory abnormality that, in the investigator's opinion, could affect the safety of the patient; alter the absorption, distribution, metabolism or excretion of Idelalisib; or impair the assessment of study results
- Grade > 2 toxicity (other than alopecia) continuing from prior anti-cancer therapy
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 |
|---|---|---|
| GS-1101Also: Idealisib, Zydelig, CAL-101Oral twice daily for 6 months followed by once daily until disease progression or unacceptable toxicity. | Drug | GS-1101 |
Primary outcome
Overall Response Rate (ORR)
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
ORR measured by decrease in serum IgM level by at least 25% from baseline.
7 secondary outcomes
Percentage of Participants With Adverse Events
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Complete Response (CR)
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Very Good Partial Response (VGPR)
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Partial Response (PR)
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Minimal Response
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Stable Disease
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Rate of Progressive Disease
Time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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. This study is not recruiting at the moment.
Jorge J. Castillo, MD principal investigator
Dana-Farber Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing GS-1101 in people with Waldenstrom's Macroglobulinemia, run by Dana-Farber Cancer Institute at 1 site in United States.
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 18 inclusion and 14 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 Jan 2017. 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 United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Jan 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Overall Response Rate (ORR) (time frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.). 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 Jan 2017.