AL AmyloidosisPhase 1/2NCT06569147
Elranatamab in Patients With Relapsed or Refractory AL Amyloidosis
A Phase 1/2 study testing Elranatamab in people with AL Amyloidosis, run by Dana-Farber Cancer Institute at 5 sites in United States.
Trial summary
This study will evaluate the safety, tolerability and efficacy of elranatamab in patients with relapsed or refractory AL amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AL Amyloidosis
- What is being tested?
- Elranatamab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 5 sitesUnited States
- Study dates
- 1 Nov 2024to 1 Jul 2029
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Previously diagnosed with AL amyloidosis based on IMWG criteria who have relapsed or refractory disease after treatment with at least one prior line of therapy (minimum 2 cycles).
- Participants must have progression of light chain disease, defined as dFLC >20mg/L.
- For Phase 2 only, measurable hematologic disease, satisfying one of the following criteria: Difference between involved and uninvolved free light chain (FLC) over 40 mg/L; Abnormal level of FLC with an abnormal κ/λ ratio (except in participants with CKD stage 3 or higher where a rise of lambda FLC to an abnormal level and of at least 50% over the nadir with a normal κ/λ ratio is acceptable); A serum M spike measuring ≥ 0.5 g/dL
- Age ≥ 18 years
- ECOG performance status ≤2 or Karnofsky ≥60%
- Participants must meet the following organ and marrow function as defined below: Absolute leukocyte count ≥3,000/mcL , Absolute neutrophil count ≥1,000/mcL, Absolute platelet count ≥75,000/mcL , Direct bilirubin ≤1.5 × institutional upper limit of normal (ULN) AST(SGOT)/ALT(SGPT) ≤3 × institutional ULN, Creatinine: Calculated clearance ≥30 mL/min using Cockcault-Groft equation
Show all 14Show fewer
- Participants who received belantamab mafodotin are eligible if discontinued due to intolerance or adverse event.
- For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
- Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.
- AL Amyloidosis Cardiac stage I, II or IIIa disease based on the 2013 European Modification of the 2004 Standard Mayo Clinic Staging in participants with advanced cardiac involvement (Dispenzieri et al., 2004; Wechalekar et al., 2013).
- The effects of elranatamab on the developing human fetus are unknown. Based on the mechanism of action, elranatamab may cause fetal harm when administered to a pregnant woman and therefore should not be used during pregnancy. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation and until 90 days since the last dose of elranatamab. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 90 days after completion of elranatamab administration.
- Ability to understand and the willingness to sign a written informed consent document.
- Willingness to undergo study procedures, including bone marrow biopsies as detailed in the schedule of events.
- Participants should have received prior treatment with Daratumumab + CyBorD.
Who cannot join
- Prior BCMA-targeting bispecific antibodies or BCMA-targeting CAR-T therapy.
- Participants refractory to belantamab mafodotin OR participants that have received belantamab as the immediate past line of therapy.
- Participants who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities > Grade 1) with the exception of alopecia.
- Participants who are receiving any other investigational agents for this condition.
- Participants with Stage IIIB Amyloidosis as defined by the 2004 Mayo Clinic Criteria (see above).
- History of allergic reactions to elranatamab.
Show all 23Show fewer
- Participants with an active malignancy (including lymphoma) with the following exceptions: adequately treated basal cell carcinoma, squamous cell carcinoma, or in situ cervical cancer; adequately treated stage I cancer from which the patient is currently in remission and has been for over 2 years; low-risk prostate cancer with a Gleason score < 7 and prostate specific antigen < 10ng/mL; other localized, indolent and/or low risk cancer may be permitted
- Women who are pregnant, breastfeeding, or planning to become pregnant while enrolled in this study or 4 months following discontinuation of elranatamab, whichever is longer. Pregnant women are excluded from this study because elranatamab is an agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with elranatamab, breastfeeding should be discontinued if the mother is treated with elranatamab.
- Have any other medical, social or psychological factors that could affect the participant's safety or ability to consent personally or comply with study procedures.
- Participants meeting criteria for active MM based on presence of CRAB criteria (a ratio of involved versus uninvolved FLC over 100 is allowed in the absence of CRAB criteria).
- Participants with active clinically significant autoimmune diseases.
- Participants seropositive for the human immunodeficiency virus (HIV).
- Severe, uncontrolled orthostatic hypotension resulting in syncopal/pre-syncopal events despite optimized medical management (e.g., midodrine, pyridostigmine) and in the absence of volume depletion.
- Plan for autologous stem cell transplant during the first 6 months of protocol therapy.
- History of acute coronary syndrome or uncontrolled ventricular arrhythmias within 3 months prior to screening.
- Evidence of LV systolic dysfunction as defined by LVEF is < 30% by echocardiogram at Screening per site cardiology interpretation.
- Presence of severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area < 1.0 cm2) or severe congenital heart disease.
- Have history of sustained ventricular tachycardia or aborted ventricular fibrillation or a history of atrioventricular nodal or sinoatrial nodal dysfunction if a permanent pacemaker (PPM) or implantable cardioverter-defibrillator (ICD) is not placed.
- QT corrected by Fridericia (QTcF) is > 550 msec on Screening ECG unless they have a PPM/ICD implanted.
- Screening EKG showing acute myocardial ischemia or active conduction system abnormalities with the exception of any of the following: First degree atrioventricular block; Second degree atrioventricular block Type 1 (Mobitz Type 1/Wenckebach type); Right or left bundle branch block (e.g., Left Bundle Branch Block, Right Bundle Branch Block, Left Anterior Fascicular Block, or Left Posterior Fascicular Block); Atrial fibrillation with a controlled ventricular rate; Bifascicular block assessed as benign by the Investigator
- Major surgery that required general anesthesia within 4 weeks of randomization or is planning major surgery during the study.
- NYHA class IV symptoms or participants with acute decompensation of congestive heart failure.
- Transplant eligible participants who have not undergone transplant are not eligible.
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Giada Bianchi, MD, 857-215-3652, Giada_bianchi@dfci.harvard.edu
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Giada Bianchi, MD, 857-215-3652
- Recruiting
Tufts Medical Center
Boston, Massachusetts, 44195
Giada Bianchi, MD, 857-215-3652, Giada_bianchi@dfci.harvard.edu
- Recruiting
Cleaveland Clinic
Cleveland, Ohio, 44195
Giada Bianchi, MD, 857-215-3652, Giada_bianchi@dfci.harvard.edu
- Recruiting
Yale University
New Haven, Connecticut, 06520
Giada Bianchi, MD, 857-215-3652, Giada_bianchi@dfci.harvard.edu
About this study
Official title: A Phase I/II, Open Label, Study to Evaluate Safety, Tolerability and Efficacy of Elranatamab in Patients With Relapsed or Refractory AL Amyloidosis
This study will evaluate the safety, tolerability and efficacy of elranatamab in patients with relapsed or refractory AL amyloidosis.
Read the detailed description
This is a Phase I/II, multi-center, open-label study designed to evaluate the safety, tolerability and efficacy of elranatamab in patients with relapsed or refractory light chain (AL) amyloidosis. Phase 1 will enroll 4-20 participants, beginning enrollment at Dose Level 0. If two or more participants at Level 0 experience DLT, dose will be decreased to Level -1. Each participant will complete the 28-day dose-limiting toxicity (DLT) evaluation period prior to dosing the next patient. Assuming a recommended phase 2 dose (RP2D) is found, Phase 2 will enroll an additional 29 participants for a total of 49 participants.
Dose level 0 consists of: Cycle 1: 12 mg (D1)/32 mg (D4)/ 76 mg (D8, 15, 22); Cycle 2: 76 mg on D1, D8, D15 and D22. If < VGPR after Cycle 2: Cycles 3-6: 76 mg on D1, 8, 15 and 22. If ≥ VGPR after Cycle 2: Cycles 3-6: 76 mg on D1 and 15
Dose Level -1 consists of: Cycle 1: 12 mg (D1)/32 mg (D4)/ 76 mg (D8, D22), Cycles 2-6: 76 mg D1 and D15
- Conditions
- AL Amyloidosis
- Being tested
- Elranatamab
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ElranatamabDrug
Also called Elrexfio
Elranatamab administered subcutaneously for 6 cycles of treatment with 28 days in a treatment cycle
| Study group | Role | What they receive |
|---|---|---|
| Elranatamab Dose Schedule | Experimental | Phase I: Phase I will enroll up to 20 participants. All participants in Phase I will be hospitalized during Cycle 1 dosing. The first 5 participants will be hospitalized for 9 days, and subsequent participants will be hospitalized for 5 days. Elranatamab will be administered subcutaneously at a dose of 12 mg on C1D1, 32 mg on C1D4, and 76 mg on C1D8. Subsequent treatment doses will be 76 mg thereafter. If two or more participants at Level 0 experience DLT, dose will be decreased to Level -1. If 2 or more participants at Level -1 experience DLT, the trial will be discontinued.
Phase II: All participants in Phase 2 will receive the RP2D determined in Phase 1. Phase 2 will enroll an additional 29 participants. If RP2D is determined to be Dose Level 0, the treatment schedule will be the same as described in Dose Level 0. If RP2D is determined to be Dose Level -1, the treatment schedule will be the same as described in Dose Level -1.Treatment duration is 6 cycles of 28 days per cycle. Receives: Drug: Elranatamab |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine RP2D
The recommended phase 2 dose determined in phase 1
Measured: up to 26 months
To evaluate objective response rate (ORR)
Defined as the proportion of patients with CR, VGPR, or PR, and will be provided as unconfirmed and confirmed ORR at the end of six cycles of treatment. Confirmed responses are those that persist on repeat imaging study at least 28 days after the initial documentation of response.
Measured: up to 60 months
Secondary outcomes (8)
To assess safety and tolerability of elranatamab in patients with relapse or refractory AL amyloidosis
Number of participants with treatment-related adverse events as assessed by CTCAE version 5.0
Measured: up to 72 months
To assess best overall response (BOR)
BOR will be analyzed as the number of patients (percentage) with each response, divided by the number of patients treated.
Measured: up to 72 months
To assess duration of response (DoR)
DoR will be analyzed using Kaplan-Meier method and is defined as the time from the first documentation of CR, VGPR, or PR to the first documentation of objective progression or death due to any cause. .
Measured: up to 72 months
To assess progression-free survival (PFS)
Progression-free Survival (PFS) will be analyzed using Kaplan-Meier method and is defined as the time from Cycle 1 Day 1 to first documentation of objective progression (PD) or death due to any cause, whichever comes first.
Measured: up to 72 months
To assess overall survival (OS)
OS is defined as the time from the date of Cycle 1 Day 1 to date of death due to any cause.
Measured: up to 72 months
Incidence and severity of cytokine release syndrome (CRS) and immune effector cell- associated neurotoxicity syndrome (ICANS)
Number of cases of CRS and ICANs and Grade of CRS and ICANs cases
Measured: up to 72 months
Incidence of treatment emergent adverse events
Number of treatment- emergent AEs. An AE is treatment-emergent if the onset occurs on Cycle 1 Day 1 through 90 days after the last dose of investigational product.
Measured: up to 72 months
To assess major organ deterioration (MOD) progression free survival (PFS) for the duration of the study
PFS is defined as the time from Cycle 1 Day 1 to first documentation of objective progression (PD) or death due to any cause, whichever comes first.
Measured: up to 72 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2024
- Study start1 Nov 2024
- Primary completion1 Jan 2029 (estimated)
- Study completion1 Jul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AL AmyloidosisStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 49 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Giada Bianchi, MD, Dana-Farber Cancer Institute
- Investigators
- Giada Bianchi, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06569147
- Sponsor's protocol ID
- 24-290
- Status last verified
- Aug 2026
- First submitted
- 16 Aug 2024
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Harvard Cancer Consortium encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Open Label, Study to Evaluate Safety, Tolerability and Efficacy of Elranatamab in Patients With Relapsed or Refractory AL Amyloidosis
- ClinicalTrials.gov ID
- NCT06569147
- Acronym / sponsor ID
- 24-290
- Conditions
- AL Amyloidosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 49 participants (estimated)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Study start
- 1 Nov 2024 (actual)
- Primary completion
- 1 Jan 2029 (estimated)
- Study completion
- 1 Jul 2029 (estimated)
- First posted
- 23 Aug 2024 (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.
This study will evaluate the safety, tolerability and efficacy of elranatamab in patients with relapsed or refractory AL amyloidosis.
Detailed description
This is a Phase I/II, multi-center, open-label study designed to evaluate the safety, tolerability and efficacy of elranatamab in patients with relapsed or refractory light chain (AL) amyloidosis. Phase 1 will enroll 4-20 participants, beginning enrollment at Dose Level 0. If two or more participants at Level 0 experience DLT, dose will be decreased to Level -1. Each participant will complete the 28-day dose-limiting toxicity (DLT) evaluation period prior to dosing the next patient. Assuming a recommended phase 2 dose (RP2D) is found, Phase 2 will enroll an additional 29 participants for a total of 49 participants.
Dose level 0 consists of: Cycle 1: 12 mg (D1)/32 mg (D4)/ 76 mg (D8, 15, 22); Cycle 2: 76 mg on D1, D8, D15 and D22. If < VGPR after Cycle 2: Cycles 3-6: 76 mg on D1, 8, 15 and 22. If ≥ VGPR after Cycle 2: Cycles 3-6: 76 mg on D1 and 15
Dose Level -1 consists of: Cycle 1: 12 mg (D1)/32 mg (D4)/ 76 mg (D8, D22), Cycles 2-6: 76 mg D1 and D15
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Elranatamab Dose Schedule | Experimental | Drug: ElranatamabPhase I: Phase I will enroll up to 20 participants. All participants in Phase I will be hospitalized during Cycle 1 dosing. The first 5 participants will be hospitalized for 9 days, and subsequent participants will be hospitalized for 5 days. Elranatamab will be administered subcutaneously at a dose of 12 mg on C1D1, 32 mg on C1D4, and 76 mg on C1D8. Subsequent treatment doses will be 76 mg thereafter. If two or more participants at Level 0 experience DLT, dose will be decreased to Level -1. If 2 or more participants at Level -1 experience DLT, the trial will be discontinued. Phase II: All participants in Phase 2 will receive the RP2D determined in Phase 1. Phase 2 will enroll an additional 29 participants. If RP2D is determined to be Dose Level 0, the treatment schedule will be the same as described in Dose Level 0. If RP2D is determined to be Dose Level -1, the treatment schedule will be the same as described in Dose Level -1.Treatment duration is 6 cycles of 28 days per cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Previously diagnosed with AL amyloidosis based on IMWG criteria who have relapsed or refractory disease after treatment with at least one prior line of therapy (minimum 2 cycles).
- Participants must have progression of light chain disease, defined as dFLC >20mg/L.
- For Phase 2 only, measurable hematologic disease, satisfying one of the following criteria: Difference between involved and uninvolved free light chain (FLC) over 40 mg/L; Abnormal level of FLC with an abnormal κ/λ ratio (except in participants with CKD stage 3 or higher where a rise of lambda FLC to an abnormal level and of at least 50% over the nadir with a normal κ/λ ratio is acceptable); A serum M spike measuring ≥ 0.5 g/dL
- Age ≥ 18 years
- ECOG performance status ≤2 or Karnofsky ≥60%
Key exclusion criteria (23 listed)
- Prior BCMA-targeting bispecific antibodies or BCMA-targeting CAR-T therapy.
- Participants refractory to belantamab mafodotin OR participants that have received belantamab as the immediate past line of therapy.
- Participants who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities > Grade 1) with the exception of alopecia.
- Participants who are receiving any other investigational agents for this condition.
- Participants with Stage IIIB Amyloidosis as defined by the 2004 Mayo Clinic Criteria (see above).
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 |
|---|---|---|
| ElranatamabAlso: ElrexfioElranatamab administered subcutaneously for 6 cycles of treatment with 28 days in a treatment cycle | Drug | Elranatamab Dose Schedule |
Primary outcome
To determine RP2D
Time frame: up to 26 months
The recommended phase 2 dose determined in phase 1
To evaluate objective response rate (ORR)
Time frame: up to 60 months
Defined as the proportion of patients with CR, VGPR, or PR, and will be provided as unconfirmed and confirmed ORR at the end of six cycles of treatment. Confirmed responses are those that persist on repeat imaging study at least 28 days after the initial documentation of response.
8 secondary outcomes
To assess safety and tolerability of elranatamab in patients with relapse or refractory AL amyloidosis
Time frame: up to 72 months
To assess best overall response (BOR)
Time frame: up to 72 months
To assess duration of response (DoR)
Time frame: up to 72 months
To assess progression-free survival (PFS)
Time frame: up to 72 months
To assess overall survival (OS)
Time frame: up to 72 months
Incidence and severity of cytokine release syndrome (CRS) and immune effector cell- associated neurotoxicity syndrome (ICANS)
Time frame: up to 72 months
Incidence of treatment emergent adverse events
Time frame: up to 72 months
To assess major organ deterioration (MOD) progression free survival (PFS) for the duration of the study
Time frame: up to 72 months
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 19 Aug 2026.
- Individual participant dataSharing planned: Yes. The Harvard Cancer Consortium encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: [contact information for Sponsor Investigator or designee]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Related trials for AL Amyloidosis
RecruitingPhase 1
Study of GC012F, CAR-T Therapy Targeting CD19 and BCMA in Chinese Participants With Relapsed or Refractory AL Amyloidosis
Relapsed/Refractory AL Amyloidosis
RecruitingObservational study
Essen Amyloidosis Registry
Amyloidosis; Systemic, Amyloidosis Cardiac
Active, not recruitingPhase 2
Siltuximab to Decrease Symptom Burden After Autologous Stem Cell Transplantation for Patients With Multiple Myeloma and AL Amyloidosis
Multiple Myeloma, AL Amyloidosis
RecruitingPhase 2
Study of HBI0101 (NXC-201) CAR-T Therapy in Multiple Myeloma and Light-Chain Amyloidosis
Multiple Myeloma, Refractory to Standard Treatment, Multiple Myeloma, Relapsed
RecruitingPhase 2
A Study to Evaluate the Efficacy and Safety of Belantamab Mafodotin in Combination With Cyclophosphamide, Bortezomib, and Dexamethasone in Adult Participants With Newly Diagnosed Amyloid Light Chain (AL) Amyloidosis
Amyloidosis
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.
Giada Bianchi principal investigator
Dana-Farber Cancer Institute
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Elranatamab in people with AL Amyloidosis, run by Dana-Farber Cancer Institute at 5 sites 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 14 inclusion and 23 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: looking for participants now, as of the sponsor's last update on 19 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
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 Giada Bianchi (6175254953, GBIANCHI1@BWH.HARVARD.EDU), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine RP2D (time frame: up to 26 months). The study lists 2 primary outcomes in all. 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 19 Aug 2026.