Trial · NCT04623216
Sabatolimab as a Treatment for Patients With Acute Myeloid Leukemia and Presence of Measurable Residual Disease After Allogeneic Stem Cell Transplantation.
NCT04623216, Phase 1, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2025-06-03
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Sabatolimab as a Treatment for Patients With Acute Myeloid Leukemia and Presence of Measurable Residual Disease After Allogeneic Stem Cell Transplantation. Trials, record NCT04623216. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04623216
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Sabatolimab as a Treatment for Patients With Acute Myeloid Leukemia and Presence of Measurable Residual Disease After Allogeneic Stem Cell Transplantation.
- NCT number
- NCT04623216
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 24 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Acute Myeloid Leukemia
- Interventions
- Sabatolimab, Azacitidine
- First posted
- 2020-11-10
- Last updated
- 2025-10-16
- Other registries' IDs
- eudract:2020-000869-17
- Results first posted
- 2025-06-03
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only) | From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days | Assessment of tolerability of sabatolimab in adults and adolescents in the post allogenic stem cell transplantation setting. This was determined by the number of participants with at least one event - All grades. A dose-limiting toxicity (DLT) is defined as an adverse event or abnormal laboratory value considered by the Investigator to be at least possibly related to sabatolimab as a single contributor that occurs during the DLT observation period and meets the severity criteria as per protocol. |
| Primary | Percentage of Adult Subjects With Absence of Hematologic Relapse Per Investigator Assessment (Safety Run-in and Expansion) | From Cycle 1 Day 1 to end of Cycle 6; Cycle = 28 Days | The percentage of adult participants for whom no evidence of hematologic relapse (no evidence of bone marrow blasts ≥5%, no evidence of reappearance of blasts in the blood; no evidence of development of extramedullary disease) has been documented after 6 cycles of study treatment or earlier discontinuation at the recommended dose of sabatolimab 800 mg. |
| Primary | Rate of Dose Limiting Toxicities (Safety Confirmation in Adolescent Cohort Only) | From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days | Assessment of tolerability of sabatolimab in adolescent participants in the post allogeneic stem cell transplantation setting. This was determined by the number of participants with at least one event - All grades. A dose-limiting toxicity (DLT) is defined as an adverse event or abnormal laboratory value considered by the Investigator to be at least possibly related to sabatolimab as a single contributor that occurs during the DLT observation period and meets the severity criteria as per protocol. |
| Secondary | Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD) | From start of treatment to up to 36 months from last patient first treatment. | Assessment of the treatment emergent grade III or IV aGvHD. Acute GvHD: Grade IV acute GvHD, Stage ≥3 lower GI acute GvHD (consistent with Grade III acute GvHD) or Stage ≥3 liver acute GvHD (consistent with Grade III GvHD). |
| Secondary | Incidence of Moderate to Severe Chronic GVHD (cGvHD) | From start of treatment to up to 36 months from last patient first treatment. | Assessment of the treatment emergent moderate or severe cGvHD. Chronic GvHD: Moderate chronic GvHD of the lungs, Severe chronic GvHD. |
| Secondary | Peak of Serum Concentration (Cmax) Sabatolimab | Cycle 1 Day 5 (end of infusion) and Cycle 3 Day 1 or Day 5 (end of infusion) and Cycle 24 Day 1 (end of infusion); Cycle =28 Days | Cmax is the maximal serum concentration of sabatolimab. |
| Secondary | Trough Serum Concentration of (Cmin) Sabatolimab | Adult cohorts: Pre-dose on Day 1 (safety run-in) or Day 5 (expansion) of Cycle 1, 3, 6 and 24 (safety run-in only); Adolescent cohort: Pre-dose on Day 1 of Cycle 1, 2, 3 and 6; Cycle = 28 Days | Cmin is the concentration of sabatolimab prior to next dosing or after end of treatment. |
| Secondary | Graft Versus Host Disease (GvHD)-Free/Relapse-free Survival (GRFS) | From start of treatment to up to 36 months from last patient first treatment | Time from start of treatment to the date of first documented occurrence or worsening of treatment emergent grade III or IV aGvHD or moderate to severe cGvHD requiring initiation of systemic treatment, morphologic/hematologic relapse, or death due to any cause, whichever occurs first |
| Secondary | Relapse-free Survival (RFS) | From start of treatment to up to 36 months from last patient first treatment | Time from start of treatment to the date of first documented hematologic relapse or death due to any cause, whichever occurs first. |
| Secondary | Percentage of Participants With Measurable Residual Disease (MRD) Positive at Baseline Who Become MRD Negative | From start of treatment until end of Cycle 6 (Cycle = 28 Days) | Percentage of participants with centrally confirmed MRD+ status at baseline converting to MRD- within the first 6 cycles of study treatment. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only) | From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days | Dose Determining Set included all participants from the FAS enrolled in the safety run-in part who met the minimum exposure criterion described and had sufficient safety evaluations or experienced a dose limiting toxicity (DLT) between Cycle 1 Day 1 (first dose of sabatolimab) and the end of Cycle 2 (both inclusive). | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Percentage of Adult Subjects With Absence of Hematologic Relapse Per Investigator Assessment (Safety Run-in and Expansion) | From Cycle 1 Day 1 to end of Cycle 6; Cycle = 28 Days | All adult participants treated with sabatolimab 800 mg monotherapy. | Percentage of participants | NUMBER |
| PRIMARY | Rate of Dose Limiting Toxicities (Safety Confirmation in Adolescent Cohort Only) | From Cycle 1 Day 1 to end of Cycle 2; Cycle =28 Days | Dose Determining Set included all participants from the FAS enrolled in the adolescent cohort who met the minimum exposure criterion described and had sufficient safety evaluations or experienced a dose limiting toxicity (DLT) between Cycle 1 Day 1 (first dose of sabatolimab) and the end of Cycle 2 (both inclusive). | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD) | From start of treatment to up to 36 months from last patient first treatment. | Safety Set includes all participants from the FAS. | Participants | NUMBER |
| SECONDARY | Incidence of Moderate to Severe Chronic GVHD (cGvHD) | From start of treatment to up to 36 months from last patient first treatment. | Safety Set includes all participants from the FAS. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Peak of Serum Concentration (Cmax) Sabatolimab | Cycle 1 Day 5 (end of infusion) and Cycle 3 Day 1 or Day 5 (end of infusion) and Cycle 24 Day 1 (end of infusion); Cycle =28 Days | Pharmacokinetics (PK) analysis set included all participants from the Safety Set who provided at least one evaluable sabatolimab PK concentration at the considered timepoint. | ug/ml | GEOMETRIC_MEAN |
| SECONDARY | Trough Serum Concentration of (Cmin) Sabatolimab | Adult cohorts: Pre-dose on Day 1 (safety run-in) or Day 5 (expansion) of Cycle 1, 3, 6 and 24 (safety run-in only); Adolescent cohort: Pre-dose on Day 1 of Cycle 1, 2, 3 and 6; Cycle = 28 Days | Pharmacokinetics (PK) analysis set included all participants from the Safety Set who provided at least one evaluable sabatolimab PK concentration at the considered timepoint. | µg/ml | GEOMETRIC_MEAN |
| SECONDARY | Graft Versus Host Disease (GvHD)-Free/Relapse-free Survival (GRFS) | From start of treatment to up to 36 months from last patient first treatment | Full Analysis Set (FAS) included all participants who received at least one dose of any component of the study treatment (i.e. at least one dose of sabatolimab or at least one dose of azacitidine). | Months | MEDIAN |
| SECONDARY | Relapse-free Survival (RFS) | From start of treatment to up to 36 months from last patient first treatment | FAS included all participants who received at least one dose of any component of the study treatment (i.e. at least one dose of sabatolimab or at least one dose of azacitidine). | Months | MEDIAN |
| SECONDARY | Percentage of Participants With Measurable Residual Disease (MRD) Positive at Baseline Who Become MRD Negative | From start of treatment until end of Cycle 6 (Cycle = 28 Days) | FAS included all participants who received at least one dose of any component of the study treatment (i.e. at least one dose of sabatolimab or at least one dose of azacitidine). | Percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group43
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only) | Sabatolimab 400mg Mono Adults | 0 | Participants | ||
| Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only) | Sabatolimab 800mg Mono Adults | 1 | Participants | ||
| Percentage of Adult Subjects With Absence of Hematologic Relapse Per Investigator Assessment (Safety Run-in and Expansion) | Sabatolimab 800mg Mono Adults | 36.4 | 95% Confidence Interval 10.9 to 69.2 | Percentage of participants | |
| Rate of Dose Limiting Toxicities (Safety Confirmation in Adolescent Cohort Only) | Sabatolimab 800mg Mono Adolescent | 0 | Participants | ||
| Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD) | Sabatolimab 400mg Mono Adults | 0 | Participants | ||
| Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD) | Sabatolimab 800mg Mono Adults | 0 | Participants | ||
| Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD) | Sabatolimab 800mg + Azacitidine Adults | 0 | Participants | ||
| Incidence of Grade III or IV Acute Graft Versus Host Disease (aGvHD) | Sabatolimab 800mg Mono Adolescent | 0 | Participants | ||
| Incidence of Moderate to Severe Chronic GVHD (cGvHD) | Sabatolimab 400mg Mono Adults | 0 | Participants | ||
| Incidence of Moderate to Severe Chronic GVHD (cGvHD) | Sabatolimab 800mg Mono Adults | 1 | Participants |
1 to 10 of 43Source ClinicalTrials.gov
See all 43 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sabatolimab 400mg Mono Adults | 10 | |
| Overall Study | STARTED | Sabatolimab 800mg Mono Adults | 11 | |
| Overall Study | STARTED | Sabatolimab 800mg + Azacitidine Adults | 2 | |
| Overall Study | STARTED | Sabatolimab 800mg Mono Adolescent | 1 | |
| Overall Study | Did Not Enter Post-treatment Follow-up (f/u) | Sabatolimab 400mg Mono Adults | 7 | |
| Overall Study | Did Not Enter Post-treatment Follow-up (f/u) | Sabatolimab 800mg Mono Adults | 9 | |
| Overall Study | Did Not Enter Post-treatment Follow-up (f/u) | Sabatolimab 800mg + Azacitidine Adults | 2 | |
| Overall Study | Did Not Enter Post-treatment Follow-up (f/u) | Sabatolimab 800mg Mono Adolescent | 1 | |
| Overall Study | Entered Post-treatment f/u, Discontinued | Sabatolimab 400mg Mono Adults | 3 | |
| Overall Study | Entered Post-treatment f/u, Discontinued | Sabatolimab 800mg Mono Adults | 2 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events320
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Sabatolimab 400mg Mono Adults | 1 | 10 | |
| other | Anaemia | Blood and lymphatic system disorders | Sabatolimab 800mg Mono Adults | 0 | 11 | |
| other | Anaemia | Blood and lymphatic system disorders | Sabatolimab 800mg + Azacitidine Adults | 0 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Sabatolimab 800mg Mono Adolescent | 0 | 1 | |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Sabatolimab 400mg Mono Adults | 1 | 10 | |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Sabatolimab 800mg Mono Adults | 0 | 11 | |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Sabatolimab 800mg + Azacitidine Adults | 0 | 2 | |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Sabatolimab 800mg Mono Adolescent | 0 | 1 | |
| other | Neutropenia | Blood and lymphatic system disorders | Sabatolimab 400mg Mono Adults | 2 | 10 | |
| other | Neutropenia | Blood and lymphatic system disorders | Sabatolimab 800mg Mono Adults | 1 | 11 |
1 to 10 of 320Source ClinicalTrials.gov
See all 320 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sabatolimab 400mg Mono Adults | 5 | 10 |
| Total, all-cause mortality | Sabatolimab 800mg Mono Adults | 2 | 11 |
| Total, all-cause mortality | Sabatolimab 800mg + Azacitidine Adults | 0 | 2 |
| Total, all-cause mortality | Sabatolimab 800mg Mono Adolescent | 0 | 1 |
| Total, other adverse events | Sabatolimab 400mg Mono Adults | 9 | 10 |
| Total, other adverse events | Sabatolimab 800mg Mono Adults | 9 | 11 |
| Total, other adverse events | Sabatolimab 800mg + Azacitidine Adults | 2 | 2 |
| Total, other adverse events | Sabatolimab 800mg Mono Adolescent | 1 | 1 |
| Total, serious adverse events | Sabatolimab 400mg Mono Adults | 2 | 10 |
| Total, serious adverse events | Sabatolimab 800mg Mono Adults | 3 | 11 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Sabatolimab 400mg Mono Adults vs Sabatolimab 800mg Mono Adults: Rate of Dose Limiting Toxicities (Safety Run-in in Adult Sabatolimab 400mg & 800mg Only) |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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