2 and older, any sex, with Immunosuppresion or Thrombocytopenia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Hematologic ResponsePrimary· 3 months
Hematologic response is defined as subjects having blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in Severe Aplastic Anemia, equivalent to 2 of the following values obtained on 2 serial blood count measurements at least one week apart at landmark time points (3, 6 and 12 months)
* Absolute neutrophil count \> 500/ μL
* Platelet count \> 20,000/ μL
* Reticulocyte count \> 60,000/ μL
Improvement in counts that are dependent upon exogenously administered growth factors or transfusion will not be considered as fulfilling response criteria.
Group
Value
95% CI
Horse ATG/CsA Taper
38
Rabbit ATG/CsA
20
Alemtuzumab
3
Hematologic ResponsePrimary· 6 months
Hematologic response is defined as subjects having blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in Severe Aplastic Anemia, equivalent to 2 of the following values obtained on 2 serial blood count measurements at least one week apart at landmark time points (3, 6 and 12 months)
* Absolute neutrophil count \> 500/ μL
* Platelet count \> 20,000/ μL
* Reticulocyte count \> 60,000/ μL
Improvement in counts that are dependent upon exogenously administered growth factors or transfusion will not be considered as fulfilling response criteria.
Group
Value
95% CI
Horse ATG/CsA Taper
41
Rabbit ATG/CsA
22
Alemtuzumab
3
Hematologic ResponsePrimary· 12 months
Hematologic response is defined as subjects having blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in Severe Aplastic Anemia, equivalent to 2 of the following values obtained on 2 serial blood count measurements at least one week apart at landmark time points (3, 6 and 12 months)
* Absolute neutrophil count \> 500/ μL
* Platelet count \> 20,000/ μL
* Reticulocyte count \> 60,000/ μL
Improvement in counts that are dependent upon exogenously administered growth factors or transfusion will not be considered as fulfilling response criteria.
Group
Value
95% CI
Horse ATG/CsA Taper
40
Rabbit ATG/CsA
18
Alemtuzumab
2
Adverse events — posted to ClinicalTrials.gov
Time frame: 5 years.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Horse ATG/CsA Taper
Serious: 7/60 (12%)
Deaths: 11/60
Rabbit ATG/CsA
Serious: 11/60 (18%)
Deaths: 18/60
Alemtuzumab
Serious: 6/16 (38%)
Deaths: 7/16
Serious adverse events (13 terms)
Reaction
System
Horse ATG/CsA Taper
Rabbit ATG/CsA
Alemtuzumab
Infection (documented clinically or microbiologically with grade 3 or 4 neutropenia)
Infections and infestations
—
—
—
Serum sickness
Immune system disorders
—
—
—
CNS hemorrhage/bleeding
Blood and lymphatic system disorders
—
—
—
Edema: limb
Blood and lymphatic system disorders
—
—
—
Cardiac troponin I
Cardiac disorders
—
—
—
Hypertension
Cardiac disorders
—
—
—
Supraventricular arrhythmia
Cardiac disorders
—
—
—
Serum sickness/Infection
Immune system disorders
—
—
—
Infection without neutropenia
Infections and infestations
—
—
—
Creatinine (grade 2)
Metabolism and nutrition disorders
—
—
—
Hypokalemia
Metabolism and nutrition disorders
—
—
—
Arthralgia (joint pain)
Musculoskeletal and connective tissue disorders
—
—
—
Seizures
Nervous system disorders
—
—
—
Other adverse events (169 terms — click to expand)
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow. Allogeneic bone marrow transplantation offers the opportunity for cure in 70% of patients, but most patients are not suitable candidates for hematopoietic stem cell transplantation (HSCT) due to advanced age or lack of a histocompatible donor. For these patients, comparable long term survival is attainable with immunosuppressive treatment with anti-thymocyte globulin (ATG) and cyclosporine (CsA). However, of those patients treated with horse ATG(h-ATG)/CsA, one quarter to one third will not respond, and about 50% of responders relapse. Auto-reactive T cells may be resistant to the effect of ATG/CsA (non-responders), while in others residual auto-reactive T cells expand post-treatment, leading to hematopoietic stem cell destruction and recurrent pancytopenia (relapse). As long term survival is correlated to response rates and robustness of hematopoietic recovery, novel immunosuppressive regimens that can achieve hematologic response and decrease relapse rates are needed.
This trial will compare the effectiveness of three immunosuppressive regimens as first line therapies in patients with SAA with early hematologic response as the primary endpoint, as well as assess the role of extended CsA treatment after h-ATG in reducing numbers of late events of relapse and clonal evolution. Randomization is employed to obtain an equal distribution of subject to each arm; comparisons of early hematologic responses will be made among the rates observed among the three concurrent arms (rabbit-ATG \[r-ATG\] versus standard h-ATG; alemtuzumab vs standard h-ATG). For long course CSA, comparison of primary end points will be to well established historic relapse rate of 38% at 2-3 years and a cumulative rate of clonal evolution of 15%.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03597594 — Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Comb
· Phase 1, PHASE2
· active not recruiting
NCT02790515 — Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical
· Phase 2
· active not recruiting
NCT07516379 — GRAfT 2.0. A Multimodal Prospective Approach to Define the Mechanisms and Clinical Features of Acute and Chronic Rejecti
· not yet recruiting
NCT06948097 — Syk Inhibition in MItigating Lung Allograft Rejection (SIMILAR): A Trial to Evaluate the Safety and Tolerability of Fost
· Phase 1
· not yet recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Heart, Lung, and Blood Institute (NHLBI)
Last refreshed: 8 June 2017
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00260689.