Clinical Trials Logo

Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05720234
Other study ID # AVA&IST-001
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date November 10, 2022
Est. completion date November 30, 2024

Study information

Verified date November 2022
Source Institute of Hematology & Blood Diseases Hospital
Contact Xin Zhao, M.D
Phone 8613702041366
Email zhaoxin@ihcams.ac.cn
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).


Description:

This is a single center, single arm, phase II clinical study. Fifty-three patients will be enrolled. Treatment protocol is as follows: 1) Anti-human thymocyte porcine immunoglobulin (P-ATG 20mg/kg/d) or rabbit anti human thymocyte globulin (R-ATG 3.0mg/kg/d) was administered intravenously for 5 days; 2) Cyclosporine (CSA) is given at 3-5 mg/kg.d in divided doses for at least 6 months. The trough concentration is maintained at 150-250 ng/ml. 3) Avatrombopag is given orally at 60 mg once a day for patients with body weight ≥ 50 kg, and 40 mg orally once a day for patients with body weight<50 kg, for a total of 12 weeks.


Recruitment information / eligibility

Status Recruiting
Enrollment 53
Est. completion date November 30, 2024
Est. primary completion date November 30, 2023
Accepts healthy volunteers No
Gender All
Age group 12 Years to 60 Years
Eligibility Inclusion Criteria: 1. Patients with newly diagnosed severe aplastic anemia. 2. Men and women aged between 12 and 60. 3. Subjects must complete all screening assessments as outlined in the test protocol. 4. Able to swallow or administer orally. 5. Before the start of the research procedure, the patient or guardian should fully understand the research procedure and purpose and sign the informed consent form. If the patient's signature is not conducive to the treatment of the disease, the patient's immediate family should sign the informed consent form. Exclusion Criteria: 1. Congenital bone marrow failure (eg. Fanconi anemia). 2. Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detection found somatic cloning abnormalities; Simple -Y abnormality can be included in this study;) . 3. ATG or middle/high-dose cyclophosphamide was used in the past. 4. Previous treatment with cyclosporine or tacrolimus > 6 months. 5. The total course of treatment with TPO receptor agonists (including thrombopoietin, eltrombopag,hetrombopag and avatrombopag) was more than 1 month. 6. Serious infectious diseases (tuberculosis without effective control, pulmonary aspergillosis, viral infections). 7. AIDS patients. 8. Pregnant or breastfeeding, fertile but unwilling to take effective contraceptive measures. 9. Patients with malignant tumors who are not suitable for ATG treatment. 10. A newly diagnosed history of cardio/cerebral vascular thrombosis within 12 months. 11. Those who are assessed as unsuitable for inclusion by the investigator.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
avatrombopag
Patients with body weight =50kg were given 60mg/day and patients with body weight < 50kg were given 40mg/day for 12 weeks.

Locations

Country Name City State
China Institute of Hematology & Blood Diseases Hospital Tianjin Tianjin

Sponsors (1)

Lead Sponsor Collaborator
Institute of Hematology & Blood Diseases Hospital

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary Treatment response Percentage of patients who achieves complete response(CR) at 12 weeks. From the start of study treatment (Day1) to end of week 12.
Secondary Treatment response Percentage of patients achieving hematologic response (OR) at 12 weeks. From the start of study treatment (Day1) to end of week 12.
Secondary Treatment response Percentage of patients achieving hematologic response and complete response (OR and CR) at 24 weeks. From the start of study treatment (Day1) to end of week 24.
Secondary Supportive treatment The time of red blood cell or platelet recovery to transfusion independence. From the start study treatment (Day1) up to transfusion independence.
Secondary Incidence of Treatment-Emergent Adverse Events by CTCAE Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 From the start study treatment (Day1) up to week 12.
Secondary Dose-effect relationship Correlation between avatrombopag's serum concentration with total and complete hematological response rate. From the start study treatment(Day1) up to week 24.
Secondary Change of CD34+ cell Change of CD34+ cells' proportion in bone marrow before and after avatrombopag treatment at week 12 and 24. From the start study treatment(Day1) up to end of week 12 and 24.
See also
  Status Clinical Trial Phase
Recruiting NCT02828592 - Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Phase 2
Completed NCT02833805 - NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Phase 2
Terminated NCT01319851 - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation N/A
Completed NCT00004143 - Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes Phase 2
Recruiting NCT05012111 - Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Recruiting NCT03836690 - Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation Phase 1
Recruiting NCT06039436 - Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT
Enrolling by invitation NCT05049668 - RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
Recruiting NCT01351545 - A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Recruiting NCT01472055 - Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation Phase 2
Completed NCT01703169 - Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia Phase 2
Withdrawn NCT01129323 - Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia N/A
Completed NCT00516152 - Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT Phase 2
Recruiting NCT06069180 - The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA Phase 4
Recruiting NCT03579875 - Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Phase 2
Recruiting NCT04304820 - Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) Phase 2
Terminated NCT00358657 - Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders Phase 2
Completed NCT02998645 - Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia Phase 2
Active, not recruiting NCT03825744 - Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia Phase 3
Not yet recruiting NCT06279494 - Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors Phase 1/Phase 2