A Phase 1 interventional study of MEN1112 in Recurrent Adult Acute Myeloid Leukemia and Acute Myeloid Leukemia, in Relapse, sponsored by Menarini Group. Terminated at 29 sites in 5 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-08-12.
Sponsored by Menarini Group · Phase 1, Interventional, and Treatment
The purpose of this study is to assess the safety of MEN1112, given as intravenous infusion, in patients with relapsed or refractory AML. Pharmacokinetics, clinical activity and potential immunogenicity of MEN1112 will be evaluated as well.
This trial is designed as an open label, non randomised, dose escalation and cohort expansion, first administration to human study to be conducted in approximately 20 European sites. The study is aiming to identify the Dose Limiting Toxicity (DLT) and Maximum Tolerated Dose (MTD), to assess the pharmacokinetics and to determine the clinical activity and potential immunogenicity of MEN1112, administered as IV infusion for two 21-day cycles.
Approximately 100 male and female ≥ 18 years-old patients, with a documented diagnosis of relapsed or refractory AML (not M3 FAB subtype), will be treated in the study, which consists of two steps.
Step 1 is the dose escalation phase according to a 3+3 patients cohort design. Incremental mg/Kg doses will be tested. Briefly, MEN1112 doses are to be administered to 3 patients; if no DLT is observed in a cohort of 3 DLT evaluable patients at a given dose level, the next cohort of 3 new patients will be treated with the next higher dose. In case of DLT occurrence by one of the three patients at any dose, the cohort will be expanded to 6 DLT evaluable patients at the same dose level. If two or more patients at a given dose level exhibit DLT, the dose escalation phase will be concluded as the MTD will be identified as one dose level below the one at which ≥ 2 DLT out of 6 treated patients occur.
Step 2 is the cohort expansion phase which will include patients treated at the MTD or the maximum dose level judged to be tolerable.
In each study Step, patients will be given two induction cycles of MEN1112 followed by a four-week End of Treatment period and a Follow-up period. In Step 1 and Step 2, DLT and MTD will be assessed when MEN1112 is given as a 'one shot' infusion (first group of patients) for all doses as well as a 'ramp up' administration to be infused in 3 days for the first two doses in Cycle 1 (second group of patients).
Along the study period, adverse events, changes in hematology/serum biochemistry parameters and bone marrow treatment response will represent the major clinical findings to be monitored on regular basis. The individual experimental clinical phase will last up to 6 months (except for female patients of childbearing potential that will undergo monthly pregnancy test until 6 months from the last study drug administration) encompassing approx. 40 planned visits at site, including Screening,Treatment, End of Treatment, Follow-up period and the End of Study visit.
3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
This study's enrollment of 71 is above the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →Menarini Group is the lead sponsor of 28 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Dose escalation will occur using a standard 3+3 dose escalation approach, beginning in dose level I with dose cohorts and rules for escalation
Drug: MEN1112
Intravenous infusion of MEN1112 pro/Kg body weight dose will be administered for two 21-day cycles; MEN1112 dose is administered as' one shot infusion' (first group of patients) and as a dose to be infused in 3 days for the first two doses in Cycle 1 (second group of patients). Two treatment cycles will be followed by a 4-week End of Treatment Period and a Follow-up period. The individual treatment/observation period is six months (except for female patients of childbearing potential that will undergo monthly pregnancy test until 6 months from the last study drug administration).
Dose Limiting Toxicity (DLT)
Identification of DLT defined as an adverse event occurring during the first treatment cycle, judged to be related to MEN1112 and meeting any of the following criteria: * Grade 3 non-haematological toxicity lasting more than 7 days * Grade ≥ 4 non-haematological toxicity.
Time frame: over 3 weeks after the first dose
Maximum Tolerated Dose (MTD)
Identification of MTD defined as one dose level below the Maximum Administered Dose (i.e. one dose level below the one at which ≥ 2 DLTs out of 6 treated patients occur).
Time frame: over 3 weeks after the first dose
Treatment Emergent Signs and Symptoms (TESSs)
Number of patients with Treatment Emergent Signs and Symptoms (TESSs) by CTCAE severity grade \>3 and treatment related causality
Time frame: 6 months
MEN1112 Pharmacokinetic (PK) Parameter Cmax
Cmax is the maximum serum drug concentration. For Cohort 1 to 3c (administration as 'one shot' infusion) Cmax is measured at the end of the first intravenous infusion ( 1 to 6 hours; depending on the individual subjects being the infusion frequently interrupted) For Cohort 1.7, 2.0 and 3.0 (dose administration as ramp up, divided in three sub-doses), Cmax is measured at the end of each out of 3 intravenous infusions
Time frame: end of intravenous infusion
MEN1112 PK Parameter AUC (0-t)
AUC (0-t) is the area under the serum concentration-time curve from time 0 extrapolated to t time evaluated after the first dose
Time frame: Dose 1- cycle 1
MEN1112 PK Parameter AUC (0-∞)
AUC (0-∞) is the area under the serum concentration-time curve from time 0 extrapolated to infinite time
Time frame: dose 1 of cycle 1
MEN1112 PK Parameter t1/2
t1/2 is the drug elimination half-life It is calculated on the first dose for all cohorts
Time frame: dose 1 of cycle 1
Complete Remission (CR) Rate
CR rate at any time point, where CR is defined as: bone marrow blasts \<5%, absence of extramedullary disease, absolute neutrophil count \>1 x 109/L and platelet count \> 100 x 109/L. According to the protocol, the efficacy analysis population includes all patients completing the first treatment cycle and having a post-cycle peripheral blood lab test and bone marrow aspirate.
Time frame: 6 months
Best Response Rate
best observed response at any time point to include Complete Remission (CR is defined as bone marrow blasts \< 5%, absence of extramedullary disease, absolute neutrophil count \> 1 x 109 / L and platelet count \> 100 x 109 / L) Complete Remission with incomplete blood count recovery \[CRi defined as all criteria for CR except residual thrombocytopenia (platelets \<100 x 109/L), and/or neutropenia (absolute neutrophil count \<1 x 109/L)\] Partial remission (PR defined as all haematological criteria for CR with bone marrow blasts 5-25% and decrease of pre-treatment bone marrow blast percentage by at least 50%.
Time frame: 6 months
Overall Survival
Overall Survival (OS) is the time from the date of the first drug administration to the date of death from any cause. If the fatal event does not occur during the study, the overall survival time is censored at the date when the patient was last known to be alive. The overall survival (OS) is calculated on 29 patients (72.5%) out of 40 belonging to the efficacy population. Results are reported as mean (and range) days
Time frame: 6 months
Immunogenicity of MEN1112
Incidence of anti-MEN1112 auto-antibodies
Time frame: 64 days
| Milestone | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 |
|---|---|---|---|---|---|---|---|---|---|
| Started | 4 | 7 | 6 | 8 | 26 | 5 | 3 | 8 | 4 |
| Completed | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Not completed | 4 | 7 | 6 | 8 | 26 | 5 | 3 | 8 | 4 |
| Withdrew: Withdrawal by subject | 1 | 0 | 0 | 0 | 1 | 0 | 0 | 1 | 1 |
| Withdrew: Lack of efficacy | 2 | 1 | 2 | 4 | 13 | 2 | 2 | 2 | 1 |
| Withdrew: Reason not specified | 1 | 2 | 0 | 0 | 6 | 1 | 0 | 3 | 1 |
| Withdrew: Physician decision | 0 | 4 | 2 | 3 | 4 | 0 | 1 | 2 | 0 |
| Withdrew: Adverse event | 0 | 0 | 1 | 0 | 1 | 1 | 0 | 0 | 0 |
| Withdrew: Patient receiving other treatment for aml | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Dose limiting toxicity (dlt) | 0 | 0 | 0 | 1 | 1 | 1 | 0 | 0 | 1 |
Identification of DLT defined as an adverse event occurring during the first treatment cycle, judged to be related to MEN1112 and meeting any of the following criteria: * Grade 3 non-haematological toxicity lasting more than 7 days * Grade ≥ 4 non-haematological toxicity.
| Participants | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 |
|---|---|---|---|---|---|---|---|---|---|
| Dose Limiting Toxicity (DLT) | 0 | 0 | 1 | 1 | 2 | 2 | 0 | 0 | 1 |
Identification of MTD defined as one dose level below the Maximum Administered Dose (i.e. one dose level below the one at which ≥ 2 DLTs out of 6 treated patients occur).
| mg/kg | MEN1112 |
|---|---|
| Maximum Tolerated Dose (MTD) | 1.7 |
Number of patients with Treatment Emergent Signs and Symptoms (TESSs) by CTCAE severity grade \>3 and treatment related causality
| participants | Cohort 1 (0.1 mg/kg) | Cohort 2 (0.3 mg/kg) | Cohort 2b (0.6 mg/kg) | Cohort 3 (1.0 mg/kg) | Cohort 3b (1.7 mg/kg) | Cohort 3c (2.5 mg/kg) | Cohort 1.7 Ramp up | Cohort 2.0 Ramp up | Cohort 3.0 Ramp up |
|---|---|---|---|---|---|---|---|---|---|
| Patients with TESSs of CTCAE severity grade > or =3 | 3 | 6 | 6 | 8 | 26 | 5 | 3 | 8 | 4 |
| Patients with treatment related TESSs of CTCAE severity grade > or =3 | 2 | 1 | 4 | 5 | 11 | 5 | 1 | 2 | 2 |
Cmax is the maximum serum drug concentration. For Cohort 1 to 3c (administration as 'one shot' infusion) Cmax is measured at the end of the first intravenous infusion ( 1 to 6 hours; depending on the individual subjects being the infusion frequently interrupted) For Cohort 1.7, 2.0 and 3.0 (dose administration as ramp up, divided in three sub-doses), Cmax is measured at the end of each out of 3 intravenous infusions
| mcg/mL | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 (0.1 Out of 1.7 mg/kg) | Cohort 1.7 (0.6 Out of 1.7 mg/kg) | Cohort 1.7 (1.0 Out of 1.7 mg/kg) | Cohort 2.0 (0.1 mg Out of 2.0 mg/kg) | Cohort 2.0 (0.7 Out of 2.0 mg/kg) | Cohort 2 (1.2 Out of 2.0 mg) | Cohort 3.0 (0.2 Out of 3.0 mg/kg) | Cohort 3.0 (1.0 Out of 3.0 mg/kg) | Cohort 3.0 (1.8 Out of 3.0 mg/kg) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| MEN1112 Pharmacokinetic (PK) Parameter Cmax | 0.22 ± 0.32 | 1.86 ± 2.06 | 3.29 ± 1.87 | 9.29 ± 5.07 | 2.67 ± 15.73 | 31.59 ± 18.18 | 2.65 ± 4.49 | 11.8 ± 2.91 | 18.57 ± 8.54 | 0.97 ± 2.18 | 7.62 ± 1.59 | 17.53 ± 6.6 | 1.26 ± 0.96 | 14.83 ± 8.57 | 39.73 ± 20.44 |
AUC (0-t) is the area under the serum concentration-time curve from time 0 extrapolated to t time evaluated after the first dose
| h*mcg/mL | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 (0.1 Out of 1.7 mg/kg) | Cohort 1.7 (0.6 Out of 1.7 mg/kg) | Cohort 1.7 (1.0 Out of 1.7 mg/kg) | Cohort 2.0 (0.1 Out of 2.0 mg) | Cohort 2.0 (0.7 Out of 2 mg/kg Ramp up) | Cohort 2.0 (1.2 Out of 2.0 mg/kg) | Cohort 3.0 (0.2 Out of 3.0 mg/kg) | Cohort 3.0 (1.0 Out of 3.0 mg/kg) | Cohort 3.0 (1.8 Out of 3.0 mg/kg) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| MEN1112 PK Parameter AUC (0-t) | 0.47 ± 0.5 | 79.48 ± 80.69 | 74.11 ± 88.45 | 346.8 ± 288.82 | 891.09 ± 642.63 | 1791.32 ± 1525.94 | 62.53 ± 87.94 | 200.15 ± 79.17 | 940.48 ± 686.59 | 5.87 ± 10.55 | 115.1 ± 33.66 | 843.53 ± 518.65 | 18.67 ± 14.63 | 194.91 ± 103.12 | 2385.21 ± 1956.83 |
AUC (0-∞) is the area under the serum concentration-time curve from time 0 extrapolated to infinite time
| h*mcg/mL | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 |
|---|---|---|---|---|---|---|---|---|
| MEN1112 PK Parameter AUC (0-∞) | 99.89 ± 81.08 | 90.55 ± 91.67 | 370.69 ± 318.5 | 1321.55 ± 800.89 | 2220.48 ± 2009.55 | 1310.79 ± 1091.61 | 1516.51 ± 867.13 | 3894.85 ± 3352.56 |
t1/2 is the drug elimination half-life It is calculated on the first dose for all cohorts
| hour | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 |
|---|---|---|---|---|---|---|---|---|
| MEN1112 PK Parameter t1/2 | 18.63 ± 6.47 | 10.76 ± 9.22 | 21.21 ± 15.05 | 46.49 ± 34.25 | 38.34 ± 26.72 | 46.84 ± 35.2 | 72.51 ± 46.88 | 61.75 ± 30.75 |
CR rate at any time point, where CR is defined as: bone marrow blasts \<5%, absence of extramedullary disease, absolute neutrophil count \>1 x 109/L and platelet count \> 100 x 109/L. According to the protocol, the efficacy analysis population includes all patients completing the first treatment cycle and having a post-cycle peripheral blood lab test and bone marrow aspirate.
| Participants | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 |
|---|---|---|---|---|---|---|---|---|---|
| Complete Remission (CR) Rate | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
best observed response at any time point to include Complete Remission (CR is defined as bone marrow blasts \< 5%, absence of extramedullary disease, absolute neutrophil count \> 1 x 109 / L and platelet count \> 100 x 109 / L) Complete Remission with incomplete blood count recovery \[CRi defined as all criteria for CR except residual thrombocytopenia (platelets \<100 x 109/L), and/or neutropenia (absolute neutrophil count \<1 x 109/L)\] Partial remission (PR defined as all haematological criteria for CR with bone marrow blasts 5-25% and decrease of pre-treatment bone marrow blast percentage by at least 50%.
| Participants | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 |
|---|---|---|---|---|---|---|---|---|---|
| Best Response Rate | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
Overall Survival (OS) is the time from the date of the first drug administration to the date of death from any cause. If the fatal event does not occur during the study, the overall survival time is censored at the date when the patient was last known to be alive. The overall survival (OS) is calculated on 29 patients (72.5%) out of 40 belonging to the efficacy population. Results are reported as mean (and range) days
| day | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 |
|---|---|---|---|---|---|---|---|---|---|
| Overall Survival | 33 (33 to 33) | 56.5 (46.0 to 67) | 76.8 (30 to 123) | 47 (33 to 73) | 55.2 (24 to 129) | 92 (76 to 108) | 30 (30 to 30) | 52.5 (29 to 76) | 37 (28 to 46) |
Incidence of anti-MEN1112 auto-antibodies
No measurements were reported for this outcome.
Collected over Adverse Events were to be collected for 180 days (i.e. from Informed Consent signature to the End of Study Visit) or 70 days after last study drug administration whichever occurs last.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1 (0.1 mg/kg) | 3/4 (75%) | 3/4 (75%) | 4/4 (100%) |
| Cohort 2 (0.3 mg/kg) | 4/7 (57.1%) | 5/7 (71.4%) | 7/7 (100%) |
| Cohort 2b (0.6 mg/kg) | 5/6 (83.3%) | 6/6 (100%) | 6/6 (100%) |
| Cohort 3 (1.0 mg/kg) | 8/8 (100%) | 8/8 (100%) | 8/8 (100%) |
| Cohort 3b (1.7 mg/kg) | 17/26 (65.4%) | 25/26 (96.2%) | 26/26 (100%) |
| Cohort 3c (2.5 mg/kg) | 4/5 (80%) | 5/5 (100%) | 5/5 (100%) |
| Cohort 1.7 Ramp up | 2/3 (66.7%) | 2/3 (66.7%) | 3/3 (100%) |
| Cohort 2.0 Ramp up | 5/8 (62.5%) | 8/8 (100%) | 8/8 (100%) |
| Cohort 3.0 Ramp up | 3/4 (75%) | 4/4 (100%) | 4/4 (100%) |
| Event | Cohort 1 (0.1 mg/kg) | Cohort 2 (0.3 mg/kg) | Cohort 2b (0.6 mg/kg) | Cohort 3 (1.0 mg/kg) | Cohort 3b (1.7 mg/kg) | Cohort 3c (2.5 mg/kg) | Cohort 1.7 Ramp up | Cohort 2.0 Ramp up | Cohort 3.0 Ramp up |
|---|---|---|---|---|---|---|---|---|---|
| Acute myeloid leukaemiaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 2/4 | 0/7 | 2/6 | 2/8 | 1/26 | 0/5 | 2/3 | 4/8 | 0/4 |
| Febrile neutropeniaBlood and lymphatic system disorders | 1/4 | 3/7 | 2/6 | 3/8 | 14/26 | 1/5 | 1/3 | 2/8 | 0/4 |
| Alanine aminotransferase increasedInvestigations | 0/4 | 0/7 | 0/6 | 1/8 | 0/26 | 0/5 | 0/3 | 0/8 | 2/4 |
| PneumoniaInfections and infestations | 0/4 | 1/7 | 0/6 | 0/8 | 1/26 | 2/5 | 0/3 | 2/8 | 0/4 |
| Anal abscessInfections and infestations | 0/4 | 0/7 | 0/6 | 0/8 | 0/26 | 0/5 | 1/3 | 1/8 | 0/4 |
| Anorectal cellulitisInfections and infestations | 0/4 | 0/7 | 0/6 | 0/8 | 0/26 | 0/5 | 1/3 | 0/8 | 0/4 |
| Haemorrhage intracranialNervous system disorders | 0/4 | 1/7 | 2/6 | 0/8 | 0/26 | 0/5 | 0/3 | 0/8 | 0/4 |
| Disease progressionGeneral disorders | 0/4 | 2/7 | 1/6 | 0/8 | 5/26 | 1/5 | 0/3 | 0/8 | 1/4 |
| SepsisInfections and infestations | 1/4 | 2/7 | 0/6 | 1/8 | 5/26 | 0/5 | 0/3 | 0/8 | 0/4 |
| Escherichia bacteraemiaInfections and infestations | 1/4 | 0/7 | 0/6 | 0/8 | 0/26 | 0/5 | 0/3 | 0/8 | 0/4 |
| Event | Cohort 1 (0.1 mg/kg) | Cohort 2 (0.3 mg/kg) | Cohort 2b (0.6 mg/kg) | Cohort 3 (1.0 mg/kg) | Cohort 3b (1.7 mg/kg) | Cohort 3c (2.5 mg/kg) | Cohort 1.7 Ramp up | Cohort 2.0 Ramp up | Cohort 3.0 Ramp up |
|---|---|---|---|---|---|---|---|---|---|
| Disseminated intravascular coagulationBlood and lymphatic system disorders | 0/4 | 0/7 | 2/6 | 0/8 | 2/26 | 4/5 | 0/3 | 0/8 | 0/4 |
| FatigueGeneral disorders | 3/4 | 0/7 | 0/6 | 1/8 | 4/26 | 1/5 | 0/3 | 0/8 | 0/4 |
| Infusion related reactionInjury, poisoning and procedural complications | 3/4 | 3/7 | 2/6 | 2/8 | 15/26 | 3/5 | 0/3 | 1/8 | 0/4 |
| AnaemiaBlood and lymphatic system disorders | 0/4 | 1/7 | 4/6 | 2/8 | 11/26 | 0/5 | 2/3 | 1/8 | 1/4 |
| NeutropeniaBlood and lymphatic system disorders | 0/4 | 0/7 | 2/6 | 1/8 | 6/26 | 0/5 | 2/3 | 0/8 | 0/4 |
| Oxygen saturation decreasedInvestigations | 0/4 | 0/7 | 0/6 | 0/8 | 1/26 | 0/5 | 2/3 | 1/8 | 0/4 |
| HyperglycaemiaMetabolism and nutrition disorders | 0/4 | 0/7 | 4/6 | 2/8 | 8/26 | 0/5 | 1/3 | 0/8 | 0/4 |
| HypoalbuminaemiaMetabolism and nutrition disorders | 0/4 | 0/7 | 1/6 | 1/8 | 3/26 | 0/5 | 2/3 | 2/8 | 1/4 |
| HypokalaemiaMetabolism and nutrition disorders | 1/4 | 0/7 | 3/6 | 2/8 | 5/26 | 0/5 | 2/3 | 2/8 | 1/4 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 0/4 | 0/7 | 0/6 | 0/8 | 1/26 | 0/5 | 2/3 | 0/8 | 0/4 |
| Age, Categorical(Participants) | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 1 | 5 | 5 | 3 | 7 | 0 | 3 | 6 | 2 | 32 |
| >=65 years | 3 | 2 | 1 | 5 | 19 | 5 | 0 | 2 | 2 | 39 |
| Age, Continuous(years) | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Mean | 69 (64 to 78) | 55.6 (20 to 76) | 53.8 (33 to 72) | 68.6 (56 to 77) | 69.2 (47 to 85) | 77.6 (71 to 87) | 58.3 (54 to 63) | 49.1 (19 to 71) | 56.8 (35 to 79) | 63.6 (19 to 87) |
| Sex: Female, Male(Participants) | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Female | 1 | 2 | 2 | 4 | 12 | 4 | 1 | 4 | 2 | 32 |
| Male | 3 | 5 | 4 | 4 | 14 | 1 | 2 | 4 | 2 | 39 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 | 0 | 8 | 0 | 0 | 0 | 0 | 8 |
| Not Hispanic or Latino | 4 | 7 | 6 | 8 | 17 | 5 | 3 | 8 | 4 | 62 |
| Unknown or Not Reported | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 | 1 |
| Region of Enrollment(participants) | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Belgium | 4 | 0 | 2 | 1 | 6 | 0 | 2 | 2 | 1 | 18 |
| Italy | 0 | 2 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 3 |
| France | 0 | 0 | 0 | 1 | 2 | 0 | 0 | 0 | 3 | 6 |
| Germany | 0 | 0 | 0 | 1 | 0 | 5 | 0 | 2 | 0 | 8 |
| Spain | 0 | 5 | 4 | 4 | 18 | 0 | 1 | 4 | 0 | 36 |
| Weight(kilo) | Cohort 1 | Cohort 2 | Cohort 2b | Cohort 3 | Cohort 3b | Cohort 3c | Cohort 1.7 | Cohort 2.0 | Cohort 3.0 | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Mean | 82.4 (54 to 101) | 62.8 (45 to 78) | 67.9 (54 to 77) | 69.6 (44 to 86) | 74 (52 to 103) | 67.7 (63 to 76) | 80.6 (57 to 97) | 66.6 (43 to 89) | 69.6 (58 to 81) | 71.1 (43 to 103) |
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