TriangleNCT03386513 · CITED IN SOURCE DOCUMENTS
Study of IMGN632 in Patients With Untreated BPDCN and Relapsed/Refractory BPDCN
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE1, PHASE2
- Status at capture
- ACTIVE NOT RECRUITING
- Registry last update
- 2026-09-04
This is an open-label, multi-center, Phase 1/2 study to determine the MTD and assess the safety, tolerability, PK, immunogenicity, and anti-leukemia activity of IMGN632 when administered as monotherapy to patients with CD123+ disease.
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
Composite Complete Remission/Response (CCR) Rate As Assessed by Investigator in Frontline De Novo Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Participants
Dose Escalation Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Dose Escalation Phase: Number of Participants With Dose-limiting Toxicities (DLTs)
Maximum Plasma Concentration (Cmax) of IMGN632 (Antibody Drug Conjugate [ADC]) and Total Antibody
Cmax of FGN849
Area Under the Curve From Time Zero to the Last Quantifiable Concentration (AUC0-last) of IMGN632 (ADC) and Total Antibody
AUC0-last of FGN849
Number of Participants With Anti-drug Antibodies (ADAs) at Any Post-baseline Visit
Dose Escalation Phase: Overall Response Rate (ORR), As Assessed by Investigator in Participants With AML
Dose Escalation Phase: CR+CRh Rate, As Assessed by Investigator in Participants With AML
Dose Escalation Phase: CR+CRh+CRi Rate, As Assessed by Investigator in Participants With AML
CCR Rate As Assessed by Investigator in Participants With Relapsed/Refractory (R/R) BPDCN
Duration of CCR (DOCR) As Assessed by Investigator in Frontline De Novo BPDCN Participants With CR or CRc
DOCR As Assessed by Investigator in R/R BPDCN Participants With CR or CRc
DOCR As Assessed by Investigator in Total Frontline BPDCN Participants With CR or CRc
Number of Participants With TEAEs in Participants Who Received IMGN632 As a Single Agent at the RP2D Level (0.045 mg/kg)
Rate of CR+CRc+CRh As Assessed by Investigator in Total R/R BPDCN Participants
Duration of CR+CRc+CRh As Assessed by Investigator in Total R/R BPDCN Participants
ORR As Assessed by Investigator in Total R/R BPDCN Participants
Duration of Overall Response As Assessed by Investigator in Total R/R BPDCN Participants
Overall Survival in Total R/R BPDCN Participants
CCR Rate As Assessed by Investigator in All Frontline BPDCN Participants With Post-Treatment Consolidative Stem Cell Transplant (SCT)
CCR Rate As Assessed by Investigator in All R/R BPDCN Participants With Post-Treatment Consolidative SCT
Percentage of Participants With Post-baseline Transfusion Independence in BPDCN Participants
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility Criteria
- Inclusion Criteria: 1. Disease Characteristics: a. Confirmation of CD123 positivity by flow cytometry or IHC. Participants who received prior CD123-targeting agents will be allowed as long as the blasts still have detectable CD123 expression. 2. Expansion inclusion: * Cohort 1 - Participants with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN) with 1-3 prior lines of therapy * Cohort 2 - Participants with relapsed AML * Cohort 3 - Participants with relapsed relapsed or refractory ALL (including any subtypes: B-cell, T-cell, Ph+ and Ph-) * Cohort 4 - Participants with relapsed or refractory other hematologic malignancies not included in the cohorts above (eg, high risk/very high-risk MDS, MPN, CMML, BP-CML). * Cohort 5 - Participants with relapsed relapsed or refractory (to nonintense therapies) CD123+ AML. * Cohort 6 - Participants with frontline de novo BPDCN at screening who have not received prior systemic therapy and participants with frontline BPDCN who have PCHM and have not received prior systemic therapy. Note: Participants in Cohort 6 may have received local therapy (radiotherapy, surgical excision, photodynamic therapy). Eligible participants must have a recurrence or progression in the field of local therapy OR disease outside the field of local therapy. Exclusion Criteria: 1. Participants who, in the judgment of their treating physician, have appropriate standard of care therapies will be excluded from Cohorts 1 through 5. 2. Frontline BPDCN participants with central nervous system (CNS) disease will be excluded. A lumbar puncture must be performed during the 28-day screening period, prior to drug administration. Relapsed or refractory BPDCN participants with a known history of CNS disease must have been treated locally, have at least 1 lumbar puncture with no evidence of CNS disease, and must be clinically stable prior to first dose. Concurrent therapy for CNS prophylaxis or continuation of therapy for controlled CNS disease is permitted with the approval of the Sponsor. 3. Participants with a history of veno-occlusive disease (sinusoidal obstruction syndrome) of the liver. 4. Participants with a history of Grade 4 capillary leak syndrome, or non-cardiac Grade 4 edema are ineligible, eg, related to tagraxofusp-erzs or other etiology. 5. Interval from prior cancer therapy: 1. For frontline BPDCN participants with prior local therapy (eg, radiotherapy), participants must not have received treatment within 14 days prior to drug administration on this study. 2. Relapsed or refractory BPDCN participants must not have received any anti-cancer therapy including chemotherapy, immunotherapy, radiotherapy, hormonal, biologic, or any investigational agents within 14 days prior to drug administration on this study. Participants must have recovered to baseline from all acute toxicity from this prior therapy. Note: the exception that participants who have received a checkpoint inhibitor must not have received that therapy within 28 days prior to drug administration on this study.
- healthy Volunteers
- false
- minimum Age
- 18 Years
- sex
- ALL
- std Ages
- ADULT
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- description
- Escalation: IMGN632 was administered by IV on 2 different schedules for participants with relapsed/refractory AML, ALL, or BPDCN. Expansion: IMGN632 was administered by IV: * Cohort 1: Relapsed or refractory BPDCN participants who have received 1-3 prior systemic therapies (incl. tagraxofusp-erzs and/or any other systemic therapy deemed appropriate for the treatment of BPDCN) * Cohort 2: Relapsed AML * Cohort 3: Relapsed or refractory ALL * Cohort 4: Other relapsed or refractory hematologic malignancies * Cohort 5: Relapsed or refractory AML at alternate dose or schedule * Cohort 6: Pivotal cohort for frontline BPDCN participants who have not received prior systemic therapy and participants with frontline BPDCN who have prior or concomitant hematologic malignancy (PCHM) and have not received prior systemic therapy.
- intervention Names
- Drug: IMGN632
- label
- Escalation and Expansion
- type
- EXPERIMENTAL
- interventions
- arm Group Labels
- Escalation and Expansion
- description
- CD123-targeted ADC
- name
- IMGN632
- type
- DRUG
Study design
- allocation
- NA
- intervention Model
- SINGLE_GROUP
- masking Info
- masking
- NONE
- primary Purpose
- TREATMENT
Enrollment
- count
- 179
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- CCR rate was defined as percentage of participants with complete remission/response (CR) and clinical complete remission (CRc). CR was defined as normalization of blast percentage (≤ 5%); normalization of neutrophil count (≥ 1000/microliter \[μL\]) and platelet count (≥ 100,000/μL); absence of leukemic blasts; 100% clearance of all skin lesions from baseline; no new lesions in participants without lesions at baseline; nodal masses regression to normal size on computed tomography (CT); and spleen and liver not palpable and nodules disappeared. CRc was defined as normalization of blast percentage (≤ 5%); normalization of neutrophil count (≥ 1000/μL) and platelet count (≥ 100,000/μL); absence of leukemic blasts; marked clearance of all skin lesions from baseline, residual hyperpigmentation or abnormality with BPDCN identified on biopsy (or no biopsy performed); nodal masses regression to normal size on CT; and spleen and liver not palpable and nodules disappeared.
- measure
- Composite Complete Remission/Response (CCR) Rate As Assessed by Investigator in Frontline De Novo Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Participants
- time Frame
- Up to approximately 81 months
- secondary Outcomes
- description
- An adverse event (AE) was defined as any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered study drug related. This included an exacerbation of a pre-existing condition. SAEs included death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. TEAEs were defined as any new AEs that begin or any pre-existing conditions that worsen in severity from the first dose of study drug through 30 days after the last dose or prior to the subsequent therapy, whichever occurs first.
- measure
- Dose Escalation Phase: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
- time Frame
- Up to approximately 81 months
- description
- DLT was defined as all treatment-emergent adverse events (TEAEs) or abnormal laboratory values that met the protocol-defined DLT criteria, including those TEAEs and abnormal laboratory values that resulted in a failure to meet the criteria for re-treatment. A summary of all SAEs and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
- measure
- Dose Escalation Phase: Number of Participants With Dose-limiting Toxicities (DLTs)
- time Frame
- Up to approximately 81 months
- measure
- Maximum Plasma Concentration (Cmax) of IMGN632 (Antibody Drug Conjugate [ADC]) and Total Antibody
- time Frame
- Cycle 1 and Cycle 3 (each cycle length = 21 days)
- measure
- Cmax of FGN849
- time Frame
- Cycle 1 and Cycle 3 (each cycle length = 21 days)
- measure
- Area Under the Curve From Time Zero to the Last Quantifiable Concentration (AUC0-last) of IMGN632 (ADC) and Total Antibody
- time Frame
- Cycle 1 and Cycle 3 (each cycle length = 21 days)
- measure
- AUC0-last of FGN849
- time Frame
- Cycle 1 and Cycle 3 (each cycle length = 21 days)
- measure
- Number of Participants With Anti-drug Antibodies (ADAs) at Any Post-baseline Visit
- time Frame
- Up to approximately 81 months
- description
- ORR was defined as percentage of participants with CR without minimal residual disease (CRMRD-), CR, CR with partial hematologic recovery (CRh), CR with incomplete recovery (CRi), morphologic leukemia-free state (MLFS), or partial response (PR). CRMRD-: CR with negativity for a genetic marker. CR: Morphologic CR \<5% blasts; Absolute neutrophil count (ANC) \>1000/μL; Platelets ≥100,000/μL; Participant independent of transfusions; No residual evidence of active extramedullary disease; MRD+ or unknown. CRh: Met requirements for CR except ANC \>500/μL and platelets \>50,000/μL. CRi: Met requirements for CR except either ANC \<1000/μL or platelets \<100,000/μL. MLFS: Bone marrow \<5% blasts in an aspirate with spicules; No blasts with Auer rods or persistence of extramedullary disease; Marrow not "aplastic"; ≥200 cells should be enumerated or cellularity should be ≥10%. PR: Decrease of ≥50% in percentage of blasts to 5% to 25% in bone marrow aspirate (BMA) and normalization of blood counts.
- measure
- Dose Escalation Phase: Overall Response Rate (ORR), As Assessed by Investigator in Participants With AML
- time Frame
- Up to approximately 81 months
- description
- CR+CRh rate was defined as percentage of participants with CR, and CRh. CR: Morphologic CR \<5% blasts; Absolute neutrophil count (ANC) \>1000/μL; Platelets ≥100,000/μL; Participant independent of transfusions; No residual evidence of active extramedullary disease; MRD+ or unknown. CRh: Met requirements for CR except ANC \>500/μL and platelets \>50,000/μL.
- measure
- Dose Escalation Phase: CR+CRh Rate, As Assessed by Investigator in Participants With AML
- time Frame
- Up to approximately 81 months
- description
- CR+CRh+CRi rate was defined as percentage of participants with CR, CRh, or CRi. CR: Morphologic CR \<5% blasts; Absolute neutrophil count (ANC) \>1000/μL; Platelets ≥100,000/μL; Participant independent of transfusions; No residual evidence of active extramedullary disease; MRD+ or unknown. CRh: Met requirements for CR except ANC \>500/μL and platelets \>50,000/μL. CRi: Met requirements for CR except either ANC \<1000/μL or platelets \<100,000/μL.
- measure
- Dose Escalation Phase: CR+CRh+CRi Rate, As Assessed by Investigator in Participants With AML
- time Frame
- Up to approximately 81 months
- description
- CCR rate was defined as percentage of participants with CR and CRc. CR was defined as normalization of blast percentage (≤ 5%); normalization of neutrophil count (≥ 1000/μL) and platelet count (≥ 100,000/μL); absence of leukemic blasts; 100% clearance of all skin lesions from baseline; no new lesions in participants without lesions at baseline; nodal masses regression to normal size on CT; and spleen and liver not palpable and nodules disappeared. CRc was defined as normalization of blast percentage (≤ 5%); normalization of neutrophil count (≥ 1000/μL) and platelet count (≥ 100,000/μL); absence of leukemic blasts; marked clearance of all skin lesions from baseline, residual hyperpigmentation or abnormality with BPDCN identified on biopsy (or no biopsy performed); nodal masses regression to normal size on CT; and spleen and liver not palpable and nodules disappeared.
- measure
- CCR Rate As Assessed by Investigator in Participants With Relapsed/Refractory (R/R) BPDCN
- time Frame
- Up to approximately 81 months
- description
- DOCR was defined from the first response (CR or CRc) to the time of relapse or death from any cause, whichever came first. CR was defined as normalization of blast percentage (≤ 5%); normalization of neutrophil count (≥ 1000/μL) and platelet count (≥ 100,000/μL); absence of leukemic blasts; 100% clearance of all skin lesions from baseline; no new lesions in participants without lesions at baseline; nodal masses regression to normal size on CT; and spleen and liver not palpable and nodules disappeared. CRc was defined as normalization of blast percentage (≤ 5%); normalization of neutrophil count (≥ 1000/μL) and platelet count (≥ 100,000/μL); absence of leukemic blasts; marked clearance of all skin lesions from baseline, residual hyperpigmentation or abnormality with BPDCN identified on biopsy (or no biopsy performed); nodal masses regression to normal size on CT; and spleen and liver not palpable and nodules disappeared. Median and 95% CI were calculated by Kaplan-Meier estimation.
- measure
- Duration of CCR (DOCR) As Assessed by Investigator in Frontline De Novo BPDCN Participants With CR or CRc
- time Frame
- Up to approximately 81 months
Full study description
- brief Summary
- This is an open-label, multi-center, Phase 1/2 study to determine the MTD and assess the safety, tolerability, PK, immunogenicity, and anti-leukemia activity of IMGN632 when administered as monotherapy to patients with CD123+ disease.
- detailed Description
- IMGN632 is administered by IV on Day 1 of each cycle, with cycles repeating every 21 days.
Source references
- references
- citation
- Pemmaraju N, Marconi G, Montesinos P, Lane AA, Mazzarella L, Sallman DA, Ulrickson ML, Schiller GJ, Erba HP, Wang ES, Walter RB, Deconinck E, Aribi A, Legrand O, Lebon D, Maisano V, Martinelli G, DeAngelo DJ, Derenzini E, Du Y, Lakshmikanthan S, Potluri J, Kantarjian HM, Daver NG. Pivekimab Sunirine in Blastic Plasmacytoid Dendritic Cell Neoplasm. J Clin Oncol. 2026 Apr;44(10):861-873. doi: 10.1200/JCO-25-02083. Epub 2026 Feb 11.
- pmid
- 41671533
- type
- DERIVED
- citation
- Daver NG, Montesinos P, DeAngelo DJ, Wang ES, Papadantonakis N, Todisco E, Sweet KL, Pemmaraju N, Lane AA, Torres-Minana L, Thompson JE, Konopleva MY, Sloss CM, Watkins K, Bedse G, Du Y, Malcolm KE, Zweidler-McKay PA, Kantarjian HM. Pivekimab sunirine (IMGN632), a novel CD123-targeting antibody-drug conjugate, in relapsed or refractory acute myeloid leukaemia: a phase 1/2 study. Lancet Oncol. 2024 Mar;25(3):388-399. doi: 10.1016/S1470-2045(23)00674-5.
- pmid
- 38423051
- type
- DERIVED
Source notices and limitations
Discovery and provenance
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- sted on <a href="https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm">Drugs@FDA</a>.</p><h2>Efficacy and Safety</h2><p>Efficacy was evaluated in CADENZA (NCT03386513), a multicenter, open-label, single-arm clinical trial that included adult patients with treatment-naïve BPDCN (N=33) or relapsed or refractory BPDCN (N=51), w
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- : 2.4, 23.2+) in 6 patients with BPDCN with PCHM who achieved CR/CRc. 14.2 Relapsed or Refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) CADENZA (NCT03386513) was a multicenter, open-label, single-arm, clinical trial that included 51 adult patients with relapsed or refractory BPDCN without evidence of active CNS dis
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Preserved source evidence · Independent clinical review pending · Not medical advice