AmyloidosisPhase 2NCT02590588
Idelalisib for Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis
A Phase 2 study testing Idelalisib in people with Amyloidosis, run by John Mark Sloan at 1 site in United States.
Trial summary
The investigators expect to enroll 15 participants with relapsed or refractory IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg twice daily (may be increased to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloidosis
- What is being tested?
- Idelalisib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2016to 27 Mar 2017
- 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
- 3.1.1 IgM paraprotein identified on serum immunofixation electrophoresis OR light chain-restricted CD20+ lymphoplasmacytic population on biopsy of bone marrow or lymph node (identified by H\&E/immunohistochemistry or flow cytometry) OR positive myeloid differentiation primary response gene 88 (MYD88-L265P) OR CXCR4WHIM mutation (CXCR4 mutation - warts, hypogammaglobulinemia, infections, myelokathexis) on submitted samples
- 3.1.2 Biopsy-proven relapsed or refractory AL amyloidosis
- 3.1.3 Age ≥ 18 years
- 3.1.4 Eastern Cooperative Oncology Group (ECOG) performance status <2 (see Appendix A.)
- 3.1.5 Difference between serum free light chains (FLC) of >30 mg/L or quantifiable IgM paraprotein >0.5 g/L
- 3.1.6 Participants must have normal organ and marrow function as defined below:
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- Absolute neutrophil count > 1,000/mm3
- Platelets > 50,000/mm3
- 3.1.7 Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- 3.2.1 Previous treatment with idelalisib
- 3.2.2 Glomerular filtration rate (GFR) <15 ml/min
- 3.2.3 Cardiac biomarker Stage III disease as determined by B-type natriuretic peptide (BNP) >100 pg/mL and Troponin-I >0.1 ng/mL (Girnius 2014)
- 3.2.4 alanine-aminotransferase (ALT)/aspartate aminotransferase (AST) values >2.5x upper limit of normal, Bilirubin >1.5 upper limit of normal (ULN)
- 3.2.5 Central nervous system (CNS) malignancy or other active malignancy
- 3.2.6 Lactating or pregnant women
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- 3.2.7 Exposure to another investigational drug within 4 weeks prior to start of study treatment
- 3.2.8 Ongoing alcohol or drug addiction as determined by investigator
- 3.2.9 Amyloid-directed therapy within the past 28 days
- 3.2.10 History of Human Immunodeficiency Virus (HIV), active Hepatitis B Virus (HBV) (assessed by positive Hepatitis B polymerase chain reaction assay (PCR) or Hepatitis B Surface Antigen), and/or Hepatitis C Virus (HCV) infection
- 3.2.11 t(11,14) translocation identified on bone marrow cytogenetics or by Fluorescence in situ hybridization (FISH)
- 3.2.12 Known lytic bone lesions
- 3.2.13 Positive cytomegaly virus (CMV) Polymerase chain reaction (PCR)
- 3.2.14 Previously untreated AL amyloidosis (Newly diagnosed)
- 3.2.15 Unwilling or unable to comply with the protocol
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor, Idelalisib (GS-1101), in IgM-Associated AL Amyloid
The investigators expect to enroll 15 participants with relapsed or refractory IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg twice daily (may be increased to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Participants will be treated until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression.
Read the detailed description
This study includes the use of Idelalisib to treat previously treated patients with IgM-associated AL Amyloidosis at Boston Medical Center. Boston Medical Center is internationally recognized as a leader in amyloidosis research and patient care through the activities of the multidisciplinary Amyloid Center at Boston University. The problematic cell in most forms of AL amyloidosis shares similarities with multiple myeloma. However, in the small subset of AL Amyloidosis patients with an IgM paraprotein, the cells are more typically related to lymphoplasmacytic lymphoma or Waldenstrom's macroglobulinemia. Because clonal cluster of differentiation antigen 20 (CD20)+ lymphoplasmacytic cells are usually responsible for IgM paraproteins, treatment paradigms based on Waldenstrom's macroglobulinemia (WM) may be more appropriate than myeloma-based strategies. Idelalisib has been shown to be active and well tolerated in patients with relapsed/refractory non-Hodgkin lymphoma including chronic lymphocytic lymphoma, and lymphoplasmacytic lymphoma with or without Waldenström's macroglobulinemia (WM). The side effect profile of idelalisib merges well with the known predisposition to toxicity of amyloidosis patient.
The investigators expect to enroll 15 participants with IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg (1 tablet) twice daily (may be escalated to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Participants will be treated until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression.
- Conditions
- Amyloidosis
- Being tested
- Idelalisib
- Keywords
- IgM-associated AL amyloidosis
- Medical subject headings
- Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdelalisibDrug
Also called GS-1101
Idelalisib daily until unacceptable toxicity or disease progression.
| Study group | Role | What they receive |
|---|---|---|
| Idelalisib | Experimental | Idelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion. Receives: Drug: Idelalisib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response
Evaluate hematologic response according to standard criteria
Measured: 3 months
Secondary outcomes (4)
Progression Free Survival
Evaluate time to progression
Measured: 1 year
Organ Response
Number of patients with organ response using standard AL amyloidosis criteria.
Measured: 3 months
Evaluate Safety and Tolerability of Agent
Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0
Measured: 3 months
Quality of Life
Evaluate quality of life according to Functional Assessment of Cancer Therapy Lymphoma Subscale (FACT-Lym) assessment tool
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2015 (estimated)
- Study startJan 2016
- Primary completion27 Mar 2017
- Study completion27 Mar 2017
Why it stopped: Poor accrual
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 AmyloidosisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- John Mark Sloan
- Sponsor type
- University, hospital or other
- Collaborators
- Gilead Sciences
- Responsible party
- Sponsor-investigator, John Mark Sloan, Boston Medical Center
- Investigators
- John "Mark" Sloan, MD, Principal investigator, Boston Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02590588
- Sponsor's protocol ID
- H-34318
- Status last verified
- Aug 2017
- First submitted
- 9 Sep 2015
- Last updated
- 21 Sep 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor, Idelalisib (GS-1101), in IgM-Associated AL Amyloid
- ClinicalTrials.gov ID
- NCT02590588
- Acronym / sponsor ID
- H-34318
- Conditions
- Amyloidosis
- Keywords
- IgM-associated AL amyloidosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor accrual
- Enrolment
- 1 participants (actual)
- Lead sponsor
- John Mark SloanUniversity, hospital or other
- Collaborators
- Gilead Sciences
- Study start
- Jan 2016 (actual)
- Primary completion
- 27 Mar 2017 (actual)
- Study completion
- 27 Mar 2017 (actual)
- First posted
- 29 Oct 2015 (estimated)
- Results
- Posted 10 May 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The investigators expect to enroll 15 participants with relapsed or refractory IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg twice daily (may be increased to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Participants will be treated until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression.
Detailed description
This study includes the use of Idelalisib to treat previously treated patients with IgM-associated AL Amyloidosis at Boston Medical Center. Boston Medical Center is internationally recognized as a leader in amyloidosis research and patient care through the activities of the multidisciplinary Amyloid Center at Boston University. The problematic cell in most forms of AL amyloidosis shares similarities with multiple myeloma. However, in the small subset of AL Amyloidosis patients with an IgM paraprotein, the cells are more typically related to lymphoplasmacytic lymphoma or Waldenstrom's macroglobulinemia. Because clonal cluster of differentiation antigen 20 (CD20)+ lymphoplasmacytic cells are usually responsible for IgM paraproteins, treatment paradigms based on Waldenstrom's macroglobulinemia (WM) may be more appropriate than myeloma-based strategies. Idelalisib has been shown to be active and well tolerated in patients with relapsed/refractory non-Hodgkin lymphoma including chronic lymphocytic lymphoma, and lymphoplasmacytic lymphoma with or without Waldenström's macroglobulinemia (WM). The side effect profile of idelalisib merges well with the known predisposition to toxicity of amyloidosis patient.
The investigators expect to enroll 15 participants with IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg (1 tablet) twice daily (may be escalated to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Participants will be treated until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Idelalisib | Experimental | Drug: IdelalisibIdelalisib 100 mg twice daily with possible escalation after 3 months to 150 mg twice daily at investigator discretion. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 3.1.1 IgM paraprotein identified on serum immunofixation electrophoresis OR light chain-restricted CD20+ lymphoplasmacytic population on biopsy of bone marrow or lymph node (identified by H\&E/immunohistochemistry or flow cytometry) OR positive myeloid differentiation primary response gene 88 (MYD88-L265P) OR CXCR4WHIM mutation (CXCR4 mutation - warts, hypogammaglobulinemia, infections, myelokathexis) on submitted samples
- 3.1.2 Biopsy-proven relapsed or refractory AL amyloidosis
- 3.1.3 Age ≥ 18 years
- 3.1.4 Eastern Cooperative Oncology Group (ECOG) performance status <2 (see Appendix A.)
- 3.1.5 Difference between serum free light chains (FLC) of >30 mg/L or quantifiable IgM paraprotein >0.5 g/L
Key exclusion criteria (15 listed)
- 3.2.1 Previous treatment with idelalisib
- 3.2.2 Glomerular filtration rate (GFR) <15 ml/min
- 3.2.3 Cardiac biomarker Stage III disease as determined by B-type natriuretic peptide (BNP) >100 pg/mL and Troponin-I >0.1 ng/mL (Girnius 2014)
- 3.2.4 alanine-aminotransferase (ALT)/aspartate aminotransferase (AST) values >2.5x upper limit of normal, Bilirubin >1.5 upper limit of normal (ULN)
- 3.2.5 Central nervous system (CNS) malignancy or other active malignancy
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 |
|---|---|---|
| IdelalisibAlso: GS-1101Idelalisib daily until unacceptable toxicity or disease progression. | Drug | Idelalisib |
Primary outcome
Overall Response
Time frame: 3 months
Evaluate hematologic response according to standard criteria
4 secondary outcomes
Progression Free Survival
Time frame: 1 year
Organ Response
Time frame: 3 months
Evaluate Safety and Tolerability of Agent
Time frame: 3 months
Quality of Life
Time frame: 3 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 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.
John "Mark" Sloan, MD principal investigator
Boston Medical Center
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 Idelalisib in people with Amyloidosis, run by John Mark Sloan 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 9 inclusion and 15 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 21 Sep 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 10 May 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 (time frame: 3 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 21 Sep 2017.