Efficacy and Safety of Avatrombopag in the Treatment of Thrombocytopenia After Haplo-HSCT

NCT06202625 | Recruiting Phase 2

• 1 other identifier: 2023PHD009-001
• Study Type: interventional
• Target: 142
• Locations: 1 country
• Sites: 14

Brief Summary

In this study, investigators aim to evaluate the efficacy of avatrombopag in thrombocytopenic patients after haploidentical hematopoietic stem cell transplantation (haplo-HSCT) through a prospective, multi-center, double-blinded, randomized placebo-controlled clinical trial.

Conditions

Thrombocytopenia; Stem Cell Transplant Complications

Keywords

Thrombocytopenia; hematopoietic stem cell transplantation; haploidentical

Outcome Measures

Primary Outcomes (1)

• the proportion of complete response(CR) on day 60 after haplo-HSCT

  the proportion of participants whose PLT≥50×10\^9/L on day 60 after haplo-HSCT independent of PLT transfusion for 7 consecutive days or above

  from randomization to day 60 after haplo-HSCT

Secondary Outcomes (10)

• the proportion of resonse(R)/remission on day 60 after haplo-HSCT

  from randomization to day 60 after haplo-HSCT

• the proportion of R/CR on day 30 after haplo-HSCT

  from randomization to day 30 after haplo-HSCT

• the proportion of CR/remission on day 90 after haplo-HSCT

  from randomization to day 90 after haplo-HSCT

• Time to R/CR/remission

  from randomization to day 60 after haplo-HSCT

• PLT transfusion dependence

  from randomization to day 60 after haplo-HSCT

Study Arms (2)

avatrombopag

EXPERIMENTAL

Avatrombopag 20 mg/d will be taken orally from +D7 after haplo-HSCT until reaching the adjustment indication or to +D60 after haplo-HSCT. Routine treatment is allowed. Adjustment indication: When PLT\<50×10\^9/L or PLT transfusion dependent on the +D30 after haplo-HSCT, increase the dosage to 40 mg/d; When the dosage has been increased to 40 mg/d, and PLT≥80×10\^9/L excluding the factor of PLT transfusion, decrease the dosage to 20 mg/d; When PLT≥80×10\^9/L for 7 consecutive days excluding the factor of PLT transfusion, or PLT≥300×10\^9/L excluding the factor of PLT transfusion, stop administration; When PLT\<50×10\^9/L or become PLT transfusion dependent after stopping administration, initiate administration again at the dosage 40 mg/d. PLT transfusion Indication: When PLT\<20×10\^9/L, and/or with the symptom or risk of bleeding.

Drug: avatrombopag

Placebo

PLACEBO COMPARATOR

Placebo 20 mg/d will be taken orally from +D7 after haplo-HSCT until reaching the adjustment indication or to +D60 after haplo-HSCT. Routine treatment is allowed. Adjustment indication: When PLT\<50×10\^9/L or PLT transfusion dependent on the +D30 after haplo-HSCT, increase the dosage to 40 mg/d; When the dosage has been increased to 40 mg/d, and PLT≥80×10\^9/L excluding the factor of PLT transfusion, decrease the dosage to 20 mg/d; When PLT≥80×10\^9/L for 7 consecutive days excluding the factor of PLT transfusion, or PLT≥300×10\^9/L excluding the factor of PLT transfusion, stop administration; When PLT\<50×10\^9/L or become PLT transfusion dependent after stopping administration, initiate administration again at the dosage 40 mg/d. PLT transfusion Indication: When PLT\<20×10\^9/L, and/or with the symptom or risk of bleeding.

Drug: Placebo

Interventions

avatrombopag DRUG

The avatrombopag 20mg/d will be orally taken from +D7 after haplo-HSCT until meeting the adjustment indication or to +D60 after haplo-HSCT; When PLT\<50×10\^9/L or PLT transfusion-dependent on the +D30 after haplo-HSCT, increase avatrombopag dosage to 40 mg/d; When PLT≥80×10\^9/L and without PLT transfusion within avatrombopag dosage at 40 mg/d, decrease avatrombopag dosage to 20 mg/d; When PLT≥80×10\^9/L for 7 consecutive days or PLT≥300×10\^9/L and without PLT transfusion, stop avatrombopag; When PLT\<50×10\^9/L or PLT transfusion-dependent after stopping avatrombopag , reuse avatrombopag at 40 mg/d.

Also known as: Doptelet

avatrombopag

Placebo DRUG

The placebo 20mg/d will be orally taken from +D7 after haplo-HSCT until meeting the adjustment indication or to +D60 after haplo-HSCT; When PLT\<50×10\^9/L or PLT transfusion-dependent on the +D30 after haplo-HSCT, increase placebo dosage to 40 mg/d; When PLT≥80×10\^9/L and without PLT transfusion within placebo dosage at 40 mg/d, decrease placebo dosage to 20 mg/d; When PLT≥80×10\^9/L for 7 consecutive days or PLT≥300×10\^9/L and without PLT transfusion, stop placebo; When PLT\<50×10\^9/L or PLT transfusion-dependent after stopping placebo, reuse placebo at 40 mg/d.

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, aged between 18-65 years;
• PLT\<20×10\^9/L or transfusion dependent on +D7 after haplo-HSCT;
• Agree to receive the treatment of avatrombopag after Haplo-HSCT and sign the informed consent form.

You may not qualify if:

• With active infection;
• ALT or AST\>3ULN, or total Bil\>2ULN
• Ccr\<50 mL/min;
• With the history of arteriovenous thrombosis;
• With history of cardiovascular disease (such as NYHA Class III/IV congestive heart failure, arrhythmia that increases the risk of thromboembolic events \[such as atrial fibrillation\] and angina), and subjects who have undergone coronary stent implantation, angioplasty, or coronary artery bypass grafting;
• With treatment of drugs to promote platelet production two weekes before enrollment, including but not limited to rhTPO and TPO-RA;
• HBsAg or anti-HCV or anti-HIV positive;
• Known to be allergic to avatrombopag and any of its excipients;
• With secondary or multiple HSCT;
• Females who were pregnant or breastfeeding or who had fertile ability but refuse to take effective contraceptive measures during and one month after this trial;
• With any other clinical trial of investigational product or device within 30 days prior to the baseline visit, except for observational study;
• Deemed unsuitable for enrollment by the investigator for any history of or concomitant medical condition.
• Concomitant medication:The rhIL-11, rhTPO or TPO-RA(such as eltrombopag, hetrombopag and romiplostim) and desitabine, etc. were not allowed for use during this trial.

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Xiangya Hospital of Central South University: collaborator
• Sichuan Provincial People's Hospital: collaborator
• First Affiliated Hospital of Harbin Medical University: collaborator
• The First Affiliated Hospital of Zhengzhou University: collaborator
• First Affiliated Hospital of Xinjiang Medical University: collaborator
• Shanxi Bethune Hospital: collaborator
• 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China: collaborator
• Tang-Du Hospital: collaborator
• The First Affiliated Hospital of Nanchang University: collaborator

Study Sites (14)

Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

RECRUITING

Guangzhou First People's Hospital, School of Medicine, South China University of Technology

Guangzhou, Guangdong, China

NOT YET RECRUITING

Peking University Shenzhen Hospital

Shenzhen, Guangdong, China

RECRUITING

The First Affiliated Hospital, Harbin Medical University

Harbin, Heilongjiang, China

NOT YET RECRUITING

The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, China

NOT YET RECRUITING

Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology

Wuhan, Hubei, China

NOT YET RECRUITING

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

Wuhan, Hubei, China

NOT YET RECRUITING

Xiangya Hospital, Central South University

Changsha, Hunan, 410008, China

NOT YET RECRUITING

The First Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, China

NOT YET RECRUITING

Shanxi Tumor Hospital Affiliated to Shanxi Medical University

Taiyuan, Shanxi, China

NOT YET RECRUITING

Tangdu Hospital, PLA Air Force Military Medical University

Xi’an, Shanxi, China

NOT YET RECRUITING

Xinqiao Hospital, Army Military Medical University

Chongqing, Sichuan, China

NOT YET RECRUITING

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Kunming, Yunnan, 650100, China

NOT YET RECRUITING

Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, China

NOT YET RECRUITING

MeSH Terms

Conditions

Thrombocytopenia

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Officials

• Xiaohui Zhang

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Haixia Fu

CONTACT

13581830157; fuhaixia_210@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking Univeristy Institute of Hematology

Study Record Dates

• First Submitted: December 14, 2023
• First Posted: January 11, 2024
• Study Start: May 13, 2024
• Primary Completion: October 30, 2024
• Study Completion: October 30, 2025
• Last Updated: October 1, 2024

Record last verified: 2024-09

Locations

CN (14)