A Phase 2 interventional study of AMG531 in Aplastic Anemia, sponsored by Kyowa Kirin Korea Co., Ltd.. Completed at 1 site in Korea, Republic of. Open to participants aged 19 Years and older. Per ClinicalTrials.gov, last updated 2024-04-30.
Sponsored by Kyowa Kirin Korea Co., Ltd. (part of Kyowa Kirin Co., Ltd.) · Phase 2, Interventional, and Treatment
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia and also to assess the pharmacokinetics of this product. Its efficacy and safety during the extension period beyond one year will also be evaluated.
371 studies on the registry are indexed under Anemia, Aplastic; 98 are open to participants now.
This study's enrollment of 35 is close to the median of 37 across 281 interventional studies indexed under Anemia, Aplastic.
Browse Anemia, Aplastic studies →Kyowa Kirin Korea Co., Ltd. is the lead sponsor of 6 studies on the registry; 2 are open to participants now.
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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 weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: AMG531
Drug: AMG531
Drug: AMG531
Drug: AMG531
Subcutaneous, weekly injection
Proportion of Subjects Achieving a Platelet Response at Week 9
The proportion of subjects achieving a platelet response at Week 9, and two-sided 95% confidence interval will be calculated. Platelet response is defined as 1) achieving absolute platelet increase of ≥ 20x10\^9/L above baseline or 2) increase to ≥ 10x10\^9/L and by at least 100% from baseline.
Time frame: At week 9
Proportion of Subjects Achieving a Platelet Response
The proportion of subjects with a platelet response in any time during the initial dose evaluation period, Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104 and Week 1 through Week 156.
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Platelet Transfusion Independency
The proportion of subjects achieving platelet transfusion independency in any time during the initial dose evaluation period , Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104, and Week1 through Week 156 will be calculated in the same manner as the primary variables (The number of subjects discontinued or subjects who have missing data will not be calculated). Platelet transfusion independence is defined as achieving transfusion free period of at least 8 consecutive weeks (56 days).
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Erythroid Response and/or Neutrophil Response
The proportion of subjects with erythroid response, neutrophil response and erythroid and/or neutrophil response in any time during the initial dose evaluation period, Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104, and Week1 through Week 156 will be calculated in the same manner as the primary variables (The number of subjects discontinued or subjects who have missing data will not be calculated).
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Duration of Platelet Response and Time to Platelet Response
For subjects with a platelet response in any time, time to the initial platelet response and duration of platelet response will be summarized Time to the initial platelet response will be calculated as sampling date (response achieved) minus first dose date of study drug plus 1. Duration of platelet response will be calculated as the maximum of the duration of platelet response for each subject, each duration of response calculated as the date when response is disappeared minus the date when response is achieved. The censoring date is defined as the date of the last Platelet examination or EOS whichever archives first.
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Proportion of Subjects Achieving Tri-lineage Responses
Tri-lineage response is defined in those achieving platelet response, erythroid response, and neutrophil response all together. The proportion of subjects with tri-lineage response will be calculated in the same manner as the primary variables. Time to tri-lineage response will be summarized in the same manner as time to platelet response. Time to tri-lineage response is defined as duration of the time from Day 1 to the date of platelet response, erythroid response or neutrophil response whichever achieves last in Visit.
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 157
Duration of Study Drug Discontinuation While Maintaining Stable Response
The longest duration of study drug discontinuation in subjects while maintaining a stable response is summarized
Time frame: Initial dose evaluation period (Week 9), Week 12, Week 16, Week 24, Week 52, Week 104, and Week 156
Subjects were enrolled from 14 April 2014 through 28 November 2014 from two clinical centers (Seoul St. Mary's Hospital and Samsung Medical Center, Seoul, South Korea).
| Milestone | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Started | 7 | 9 | 9 | 10 |
| Completed | 2 | 1 | 4 | 3 |
| Not completed | 5 | 8 | 5 | 7 |
| Withdrew: Adverse event | 0 | 0 | 0 | 1 |
| Withdrew: Eligibility criteria | 0 | 1 | 0 | 0 |
| Withdrew: No responder | 4 | 4 | 4 | 3 |
| Withdrew: Unable to conduct observations or assess | 0 | 1 | 0 | 0 |
| Withdrew: Withdrawal by subject | 1 | 2 | 1 | 3 |
The proportion of subjects achieving a platelet response at Week 9, and two-sided 95% confidence interval will be calculated. Platelet response is defined as 1) achieving absolute platelet increase of ≥ 20x10\^9/L above baseline or 2) increase to ≥ 10x10\^9/L and by at least 100% from baseline.
| Participants | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Proportion of Subjects Achieving a Platelet Response at Week 9 | 0 | 0 | 3 | 7 |
The proportion of subjects with a platelet response in any time during the initial dose evaluation period, Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104 and Week 1 through Week 156.
| Participants | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Initial dose evaluation period | 3 | 3 | 6 | 10 |
| Week 1 - Week 12 | 4 | 4 | 7 | 10 |
| Week 1 - Week 16 | 4 | 4 | 7 | 10 |
| Week 1 - Week 24 | 6 | 4 | 8 | 10 |
| Week 1 - Week 52 | 6 | 5 | 8 | 10 |
| Week 1 - Week 104 | 6 | 5 | 8 | 10 |
| Week 1 - Week 156 | 6 | 5 | 8 | 10 |
The proportion of subjects achieving platelet transfusion independency in any time during the initial dose evaluation period , Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104, and Week1 through Week 156 will be calculated in the same manner as the primary variables (The number of subjects discontinued or subjects who have missing data will not be calculated). Platelet transfusion independence is defined as achieving transfusion free period of at least 8 consecutive weeks (56 days).
| Participants | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| All subjects : Initial dose evaluation period | 0 | 2 | 0 | 2 |
| All subjects : Week 1 - Week 12 | 0 | 2 | 1 | 4 |
| All subjects : Week 1 - Week 16 | 0 | 2 | 2 | 4 |
| All subjects : Week 1 - Week 24 | 1 | 2 | 3 | 6 |
| All subjects : Week 1 - Week 52 | 2 | 2 | 3 | 6 |
| All subjects : Week 1 - Week 104 | 2 | 2 | 3 | 6 |
| All subjects : Week 1 - Week 156 | 2 | 2 | 3 | 6 |
| Subjects with TF : Initial dose evaluation period | 0 | 2 | 0 | 2 |
| Subjects with TF : Week 1 - Week 12 | 0 | 2 | 1 | 4 |
| Subjects with TF : Week 1 - Week 16 | 0 | 2 | 2 | 4 |
| Subjects with TF : Week 1 - Week 24 | 1 | 2 | 3 | 6 |
| Subjects with TF : Week 1 - Week 52 | 2 | 2 | 3 | 6 |
| Subjects with TF : Week 1 - Week 104 | 2 | 2 | 3 | 6 |
| Subjects with TF : Week 1 - Week 156 | 2 | 2 | 3 | 6 |
The proportion of subjects with erythroid response, neutrophil response and erythroid and/or neutrophil response in any time during the initial dose evaluation period, Week 1 through Week 12, Week 1 through Week 16, Week 1 through Week 24, Week 1 through Week 52, Week 1 through Week 104, and Week1 through Week 156 will be calculated in the same manner as the primary variables (The number of subjects discontinued or subjects who have missing data will not be calculated).
| Participants | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Erythroid Response : Initial dose evaluation period | 0 | 2 | 1 | 3 |
| Erythroid Response : Week 1 - Week 12 | 0 | 4 | 4 | 4 |
| Erythroid Response : Week 1 - Week 16 | 3 | 4 | 4 | 4 |
| Erythroid Response : Week 1 - Week 24 | 5 | 5 | 7 | 5 |
| Erythroid Response : Week 1 - Week 52 | 6 | 5 | 7 | 7 |
| Erythroid Response : Week 1 - Week 104 | 6 | 5 | 7 | 7 |
| Erythroid Response : Week 1 - Week 156 | 6 | 5 | 7 | 7 |
| Neutrophil Response : Initial dose evaluation period | 1 | 3 | 4 | 6 |
| Neutrophil Response : Week 1 - Week 12 | 1 | 3 | 5 | 6 |
| Neutrophil Response : Week 1 - Week 16 | 2 | 4 | 5 | 6 |
| Neutrophil Response : Week 1 - Week 24 | 4 | 4 | 5 | 7 |
| Neutrophil Response : Week 1 - Week 52 | 4 | 5 | 5 | 7 |
| Neutrophil Response : Week 1 - Week 104 | 4 | 5 | 5 | 7 |
| Neutrophil Response : Week 1 - Week 156 | 4 | 5 | 5 | 7 |
| Erythroid response and/or Neutrophil response : Initial dose evaluation period | 1 | 4 | 5 | 7 |
| Erythroid response and/or Neutrophil response : Week 1 - Week 12 | 1 | 5 | 7 | 7 |
| Erythroid response and/or Neutrophil response : Week 1 - Week 16 | 4 | 5 | 7 | 7 |
| Erythroid response and/or Neutrophil response : Week 1 - Week 24 | 7 | 6 | 8 | 8 |
| Erythroid response and/or Neutrophil response : Week 1 - Week 52 | 7 | 6 | 8 | 8 |
| Erythroid response and/or Neutrophil response : Week 1 - Week 104 | 7 | 6 | 8 | 8 |
| Erythroid response and/or Neutrophil response : Week 1 - Week 156 | 7 | 6 | 8 | 8 |
For subjects with a platelet response in any time, time to the initial platelet response and duration of platelet response will be summarized Time to the initial platelet response will be calculated as sampling date (response achieved) minus first dose date of study drug plus 1. Duration of platelet response will be calculated as the maximum of the duration of platelet response for each subject, each duration of response calculated as the date when response is disappeared minus the date when response is achieved. The censoring date is defined as the date of the last Platelet examination or EOS whichever archives first.
| days | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Duration of platelet response | 24.5 (14 to 506) | 21.0 (9 to 862) | 503.5 (7 to 959) | 131.0 (5 to 886) |
| Time to the initial platelet response | 59.0 (8 to 127) | 44.0 (15 to 219) | 53.5 (22 to 134) | 41.0 (36 to 50) |
Tri-lineage response is defined in those achieving platelet response, erythroid response, and neutrophil response all together. The proportion of subjects with tri-lineage response will be calculated in the same manner as the primary variables. Time to tri-lineage response will be summarized in the same manner as time to platelet response. Time to tri-lineage response is defined as duration of the time from Day 1 to the date of platelet response, erythroid response or neutrophil response whichever achieves last in Visit.
| Participants | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Week 9 | 0 | 0 | 0 | 0 |
| Week 13 | 0 | 0 | 0 | 0 |
| Week 17 | 0 | 1 | 0 | 0 |
| Week 25 | 1 | 1 | 1 | 1 |
| Week 27 | 0 | 1 | 2 | 0 |
| Week 53 | 1 | 1 | 2 | 1 |
| Week 105 | 1 | 1 | 2 | 1 |
| Week 157 | 1 | 1 | 3 | 0 |
The longest duration of study drug discontinuation in subjects while maintaining a stable response is summarized
| days | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Duration of Study Drug Discontinuation While Maintaining Stable Response | — | — | 273.0 (56 to 490) | 483.0 (483 to 483) |
Collected over Up to 3 years. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| AMG531 (Dose 1) | 0/7 (0%) | 0/7 (0%) | 3/7 (42.9%) |
| AMG531 (Dose 2) | 1/9 (11.1%) | 2/9 (22.2%) | 5/9 (55.6%) |
| AMG531 (Dose 3) | 0/9 (0%) | 2/9 (22.2%) | 7/9 (77.8%) |
| AMG531 (Dose 4) | 0/10 (0%) | 3/10 (30%) | 4/10 (40%) |
| Event | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| Febrile neutropeniaBlood and lymphatic system disorders | 0/7 | 1/9 | 0/9 | 0/10 |
| Macular fibrosisEye disorders | 0/7 | 0/9 | 1/9 | 0/10 |
| AppendicitisInfections and infestations | 0/7 | 0/9 | 1/9 | 0/10 |
| CellulitisInfections and infestations | 0/7 | 1/9 | 0/9 | 0/10 |
| Tendon injuryInjury, poisoning and procedural complications | 0/7 | 0/9 | 1/9 | 0/10 |
| CataractEye disorders | 0/7 | 0/9 | 0/9 | 1/10 |
| Retinal detachmentEye disorders | 0/7 | 0/9 | 0/9 | 1/10 |
| Inguinal herniaGastrointestinal disorders | 0/7 | 0/9 | 0/9 | 1/10 |
| Transfusion reactionInjury, poisoning and procedural complications | 0/7 | 0/9 | 0/9 | 1/10 |
| Event | AMG531 (Dose 1) | AMG531 (Dose 2) | AMG531 (Dose 3) | AMG531 (Dose 4) |
|---|---|---|---|---|
| FatigueGeneral disorders | 0/7 | 2/9 | 3/9 | 0/10 |
| Transfusion reactionInjury, poisoning and procedural complications | 2/7 | 0/9 | 0/9 | 1/10 |
| MyalgiaMusculoskeletal and connective tissue disorders | 1/7 | 2/9 | 2/9 | 0/10 |
| InsomniaPsychiatric disorders | 0/7 | 0/9 | 2/9 | 0/10 |
| Upper respiratory tract infectionInfections and infestations | 1/7 | 0/9 | 0/9 | 0/10 |
| Musculoskeletal painMusculoskeletal and connective tissue disorders | 1/7 | 0/9 | 0/9 | 0/10 |
| ConstipationGastrointestinal disorders | 0/7 | 0/9 | 1/9 | 1/10 |
| Abdominal discomfortGastrointestinal disorders | 0/7 | 0/9 | 1/9 | 0/10 |
| Abdominal painGastrointestinal disorders | 0/7 | 0/9 | 1/9 | 0/10 |
| Abdominal pain upperGastrointestinal disorders | 0/7 | 0/9 | 1/9 | 0/10 |
| Age, Continuous(years) | AMG531 (Dose 1) | AMG531 (Dose 3) | AMG531 (Dose 2) | AMG531 (Dose 4) | Total |
|---|---|---|---|---|---|
| Median | 45.0 (32 to 62) | 51.0 (33 to 60) | 50.0 (28 to 68) | 51.5 (33 to 76) | 49.0 (28 to 76) |
| Sex: Female, Male(Participants) | AMG531 (Dose 1) | AMG531 (Dose 3) | AMG531 (Dose 2) | AMG531 (Dose 4) | Total |
|---|---|---|---|---|---|
| Female | 2 | 6 | 4 | 6 | 18 |
| Male | 5 | 3 | 5 | 4 | 17 |
| Race and Ethnicity Not Collected(Participants) | AMG531 (Dose 1) | AMG531 (Dose 3) | AMG531 (Dose 2) | AMG531 (Dose 4) | Total |
|---|---|---|---|---|---|
| Count of participants | — | — | — | — | 0 |
| Baseline platelet count(cells 10^9/L) | AMG531 (Dose 1) | AMG531 (Dose 3) | AMG531 (Dose 2) | AMG531 (Dose 4) | Total |
|---|---|---|---|---|---|
| Mean | 7.5 ± 3.6 | 9.9 ± 8.5 | 10.7 ± 6.9 | 9.8 ± 5.1 | 9.6 ± 6.2 |
| Baseline hemoglobin concentration(g/dL) | AMG531 (Dose 1) | AMG531 (Dose 3) | AMG531 (Dose 2) | AMG531 (Dose 4) | Total |
|---|---|---|---|---|---|
| Mean | 6.73 ± 0.57 | 6.63 ± 0.71 | 5.95 ± 1.04 | 6.50 ± 2.14 | 6.48 ± 1.26 |
| Baseline neutrophil count(cells 10^9/L) | AMG531 (Dose 1) | AMG531 (Dose 3) | AMG531 (Dose 2) | AMG531 (Dose 4) | Total |
|---|---|---|---|---|---|
| Mean | 0.8902 ± 0.3606 | 0.9611 ± 0.6784 | 0.8756 ± 0.5000 | 0.9215 ± 0.3836 | 0.9136 ± 0.4797 |
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Studies of the drug tested here: Romiplostim