AmyloidEarly Phase 1STARRNCT04984330
Selinexor for Treatment of Light Chain Amyloidosis With Relapsed/Refractory Disease
A Early Phase 1 study testing Selinexor and Dexamethasone in people with Amyloid and Amyloidosis, run by Weill Medical College of Cornell University at 1 site in United States.
Trial summary
The purpose of this study is to test the safety and efficacy of Selinexor and Dexamethasone and see what effects it has on AL amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloid, Amyloidosis, AL Amyloidosis
- What is being tested?
- Selinexor and Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2021to Jun 2024
- Phase
- Early Phase 1
- 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
- Diagnosis of primary AL amyloidosis
- Relapsed and/or refractory AL amyloidosis
- Measurable disease
- Male or female patients 18 years or older
- Able to give voluntary written consent
- Eastern Cooperative Oncology Group performance status and/or other performance status 0, 1, or 2.
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- Absolute neutrophil count (ANC) ≥ 1,000/mm3 and platelet count ≥ 75,000/mm3.
- Total bilirubin ≤ 1.5 × the upper limit of the normal range (ULN).
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 × ULN.
- Calculated creatinine clearance ≥ 30 mL/min
Who cannot join
- Non-AL amyloidosis
- Clinically overt myeloma
- Prior exposure to Selinexor
- Clinically significant cardiac disease
- Severe obstructive airway disease
- Female patients who are lactating or have a positive serum pregnancy test during the screening period
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- Planned high-dose chemotherapy and autologous stem cell transplantation within 6, 28-day treatment cycles after starting on treatment
- Failure to have fully recovered (ie, ≤ Grade 1 toxicity) from the reversible effects of prior chemotherapy.
- Major surgery within 14 days before enrollment.
- Radiotherapy within 14 days before enrollment.
- Infection requiring systemic intravenous antibiotic therapy or other serious infection within 14 days before study enrollment. Systemic treatment, within 14 days before the first dose, with strong CYP3A inducers (rifampin, rifapentine, rifabutin, carbamazepine, phenytoin, phenobarbital, see Appendix 11.7), or use of Ginkgo biloba or St. John's wort.
- Positive for human immunodeficiency virus (HIV), hepatitis B, and hepatitis C
- Serious medical or psychiatric illness
- GI disease or GI procedure that could interfere with the oral absorption or tolerance including difficulty swallowing
- Diagnosed or treated for another malignancy within 2 years before study enrollment or previously diagnosed with another malignancy and have any evidence of residual disease. Patients with nonmelanoma skin cancer or carcinoma in situ of any type are not excluded if they have undergone complete resection.
- Participation in another clinical trials involving investigational agents within 30 days of starting this trial
- Peripheral neuropathy (grade 2 with pain or grade 3 or higher).
Trial locations and contacts
1 site in 1 country.
United States1 site
Weill Cornell Medicine - Multiple Myeloma Center
New York, New York, 10065
About this study
The purpose of this study is to test the safety and efficacy of Selinexor and Dexamethasone and see what effects it has on AL amyloidosis.
- Conditions
- Amyloid, Amyloidosis, AL Amyloidosis
- Being tested
- Selinexor, Dexamethasone
- Medical subject headings
- Alzheimer Disease, Amyloidosis, Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SelinexorDrug
Selinexor will be given orally at a dose of 60mg once weekly on the first day of the week (day 1, 8, 15, and 22) for Cycle 1 and up to Cycle 12.
DexamethasoneDrug
Dexamethasone will be given orally at a dose of 20mg, if tolerated, or at a reduced dose if required, 30 to 60 minutes prior to selinexor for first 2 days of each week only (days 1, 2, 8, 9, 15, 16, 22, and 23 of each cycle).
| Study group | Role | What they receive |
|---|---|---|
| selinexor/ dexamethasone (Sd) | Experimental | Selinexor • 60mg PO once weekly on days 1, 8, 15, 22 until disease progression or toxicity
Dexamethasone
• 20 mg PO administered 30-60 minutes prior to selinexor on days 1, 2, 8, 9, 15, 16, 22, 23 Receives: Drug: Selinexor; Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare number of dose limiting toxicity (DLT) occurence to measure safety and toxicity
Safety and toxicity will be determined by how many occurrence of dose limiting toxicity (DLT) occured during 12 months of treatment for each subject
Measured: approximately 12 months
Secondary outcomes (17)
Compare Hematologic Overall Response Rate (ORR)
Comparing proportion of subjects with Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), No Response (NR) and Progressive Disease (PD)
Measured: approximately 12 months
Hematologic Very Good Partial Response (VGPR) or better rate
Percentage of subjects that achieved Very Good Partial Response (VGPR) or better response
Measured: approximately 12 months
Hematologic Complete Response (CR) rate
Percentage of subjects that achieved Complete Response (CR)
Measured: approximately 12 months
Stringent dFLC response rate
Percentage of subjects that has stringent dFLC (dFLC is defined as the difference in involved amyloidogenic and uninvolved serum-free light chains).
Measured: approximately 12 months
Number of patients with peripheral blood mass spectrometry for monoclonal protein detection (MALDI-TOF)
For each subject, detection of monoclonal protein will be analyzed at screening, day 1 of each cycle (only when in ≥VGPR), at each end of treatment (EOT) visits, and day 1 of each post-treatment follow, up until PD or 24 months, whichever occurs first.
Measured: End of Study (approximately 3 years)
minimal residual disease (MRD) negative CR/VGPR rate
Percentage of subjects that is MRD negative when in complete response (CR) and Very Good Partial Response (VGPR)
Measured: approximately 12 months
Percentage of participants with organ response
Organ response for kidney and cardiac is defined as the proportion of baseline organ involved participants who achieve organ response in each corresponding organ. Organ response defined for cardiac: N-terminal brain pronatriuretic peptide (NT-proBNP) response (> 30% and > 300 nanogram per liter [ng/L] decrease in participants with baseline NT-proBNP >= 650 ng/L) or New York Heart Association (NYHA) class response (>= 2 class decrease in participants with baseline NYHA class 3 or 4); for kidney: decrease in proteinuria by >=30% or below 0.5 grams /24 hours without renal progression.
Measured: End of Study (approximately 3 years)
Median hematologic Progression Free Survival (PFS)
Median estimate of months that participants have progression free survival and median estimate is calculated using the Kaplan-Meier methodology
Measured: End of Study (approximately 3 years)
Time to first hematologic response
Measured in months between the date of enrollment and the first efficacy evaluation at which the participant has met the criteria for hematologic complete response.
Measured: approximately 12 months
Time to best hematologic response
Measured in months between the date of enrollment and the best hematologic response the subject achieved while on treatment
Measured: approximately 12 months
Time to hematologic progression
Measured in months between the date of enrollment and the first efficacy evaluation at which the participant has met the criteria for hematologic progression
Measured: End of Study (approximately 3 years)
Duration of hematologic response
Duration of hematologic response is defined as the time between the date of initial documentation of hematologic response (i.e. Complete Response, Very Good Partial Response, Partial Response, No Response and Progressive Disease) to the date of the next hematologic response
Measured: End of Study (approximately 3 years)
Time to next therapy
Measured in months from the date of enrollment to the start date of subsequent treatment for AL amyloidosis
Measured: End of Study (approximately 3 years)
Median organ Progression Free Survival (PFS)
Measured in months between the date of enrollment and the first efficacy evaluation at which the participant has met the criteria for hematologic complete response.
Measured: End of Study (approximately 3 years)
Time to organ response
Measured in months between the date of enrollment and the organ response
Measured: approximately 12 months
Time to organ progression
Measured in months between the date of organ response to organ progression
Measured: End of Study (approximately 3 years)
Duration of organ response
Measured in months between the date of initial documentation of organ response to organ progression
Measured: End of Study (approximately 3 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2021
- Study startDec 2021 (estimated)
- Primary completionJun 2023 (estimated)
- Study completionJun 2024 (estimated)
Why it stopped: Funding withdrawn
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AmyloidStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- Karyopharm Therapeutics Inc
- Responsible party
- Sponsor
- Investigators
- Cara Rosenbaum, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04984330
- Sponsor's protocol ID
- 21-01023225
- Acronym
- STARR
- Status last verified
- Nov 2022
- First submitted
- 29 Jul 2021
- Last updated
- 17 Nov 2022
- Results posted
- No
- 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 17 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Selinexor for Treatment of Light Chain Amyloidosis With Relapsed/Refractory Disease
- ClinicalTrials.gov ID
- NCT04984330
- Acronym / sponsor ID
- STARR / 21-01023225
- Conditions
- Amyloid, Amyloidosis, AL Amyloidosis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Funding withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Karyopharm Therapeutics Inc
- Study start
- Dec 2021 (estimated)
- Primary completion
- Jun 2023 (estimated)
- Study completion
- Jun 2024 (estimated)
- First posted
- 30 Jul 2021 (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 purpose of this study is to test the safety and efficacy of Selinexor and Dexamethasone and see what effects it has on AL amyloidosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| selinexor/ dexamethasone (Sd) | Experimental | Drug: Selinexor, Drug: DexamethasoneSelinexor • 60mg PO once weekly on days 1, 8, 15, 22 until disease progression or toxicity Dexamethasone • 20 mg PO administered 30-60 minutes prior to selinexor on days 1, 2, 8, 9, 15, 16, 22, 23 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Diagnosis of primary AL amyloidosis
- Relapsed and/or refractory AL amyloidosis
- Measurable disease
- Male or female patients 18 years or older
- Able to give voluntary written consent
Key exclusion criteria (17 listed)
- Non-AL amyloidosis
- Clinically overt myeloma
- Prior exposure to Selinexor
- Clinically significant cardiac disease
- Severe obstructive airway disease
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 |
|---|---|---|
| SelinexorSelinexor will be given orally at a dose of 60mg once weekly on the first day of the week (day 1, 8, 15, and 22) for Cycle 1 and up to Cycle 12. | Drug | selinexor/ dexamethasone (Sd) |
| DexamethasoneDexamethasone will be given orally at a dose of 20mg, if tolerated, or at a reduced dose if required, 30 to 60 minutes prior to selinexor for first 2 days of each week only (days 1, 2, 8, 9, 15, 16, 22, and 23 of each cycle). | Drug | selinexor/ dexamethasone (Sd) |
Primary outcome
Compare number of dose limiting toxicity (DLT) occurence to measure safety and toxicity
Time frame: approximately 12 months
Safety and toxicity will be determined by how many occurrence of dose limiting toxicity (DLT) occured during 12 months of treatment for each subject
17 secondary outcomes
Compare Hematologic Overall Response Rate (ORR)
Time frame: approximately 12 months
Hematologic Very Good Partial Response (VGPR) or better rate
Time frame: approximately 12 months
Hematologic Complete Response (CR) rate
Time frame: approximately 12 months
Stringent dFLC response rate
Time frame: approximately 12 months
Number of patients with peripheral blood mass spectrometry for monoclonal protein detection (MALDI-TOF)
Time frame: End of Study (approximately 3 years)
minimal residual disease (MRD) negative CR/VGPR rate
Time frame: approximately 12 months
Percentage of participants with organ response
Time frame: End of Study (approximately 3 years)
Median hematologic Progression Free Survival (PFS)
Time frame: End of Study (approximately 3 years)
Time to first hematologic response
Time frame: approximately 12 months
Time to best hematologic response
Time frame: approximately 12 months
Time to hematologic progression
Time frame: End of Study (approximately 3 years)
Duration of hematologic response
Time frame: End of Study (approximately 3 years)
Time to next therapy
Time frame: End of Study (approximately 3 years)
Median organ Progression Free Survival (PFS)
Time frame: End of Study (approximately 3 years)
Time to organ response
Time frame: approximately 12 months
Time to organ progression
Time frame: End of Study (approximately 3 years)
Duration of organ response
Time frame: End of Study (approximately 3 years)
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2022.
- 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.
Cara Rosenbaum, MD principal investigator
Weill Medical College of Cornell University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Selinexor and Dexamethasone in people with Amyloid and Amyloidosis, run by Weill Medical College of Cornell University 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 10 inclusion and 17 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: withdrawn, as of the sponsor's last update on 17 Nov 2022. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 withdrawn before enrolling participants, so there are no results.
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 number of dose limiting toxicity (DLT) occurence to measure safety and toxicity (time frame: approximately 12 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 17 Nov 2022.