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Refractory Aplastic AnemiaNCT05518331

The Safety and Efficacy Study of Avatrombopag Switch in TPO-RA Refractory AA

A study testing avatrombopag in people with Refractory Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

This study was a single-arm, multicenter, phase Π clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at… Read more about this study

Who is it for?
Ages 14 years and older
Conditions the study is for
Refractory Aplastic Anemia
What is being tested?
avatrombopag
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
39 participants
Where
1 siteChina
Study dates
1 Jun 2022to 1 Jan 2026
Phase
Interventional
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 14 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Inclusion Criteria: Subjects eligible for inclusion in this study must meet all of the following criteria:

  1. Patients with confirmed TDNSAA/SAA/VSAA aplastic anemia who received standard IST therapy for at least 6 months, combined with Haitrombopag (15mg/d) or Eltrombopag (>50mg/d) for at least 3 Patients who have not obtained a hematological response (NR) for months and are not suitable or unwilling to undergo HSCT
  2. Age > 14 years old, male or female.
  3. Subjects must complete all screening assessments listed in the trial protocol.
  4. ECOG score ≤ 2 points.
  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: Subjects meeting any of the following criteria were excluded from this study:

  1. Patients with severe infectious diseases (uncured tuberculosis, pulmonary aspergillosis, various bacterial and viral infections) and active bleeding that cannot be controlled after standard treatment.
  2. Patients with AIDS, active viral hepatitis B, and hepatitis C RNA nucleic acid test positive.
  3. Those who are pregnant or breastfeeding, have fertility but are unwilling to take effective contraceptive measures.
  4. Congenital hematopoietic failure diseases (such as Fanconi anemia).
  5. Patients with cytogenetic clonal changes (excluding germline mutations and acquired chromosome clones of +8, 20q- and -y).
  6. Combined with malignant tumor within 3 years.
  7. Combined with other systemic diseases that cannot be controlled.
  8. Significant abnormalities in cardiopulmonary function.
  9. Abnormal liver and kidney function: creatinine level > 1.5 times the upper limit of normal, transaminase and bilirubin level > 2 times the upper limit of normal, and those who cannot be enrolled in the group as judged by the clinician.
  10. Those who are considered unsuitable for enrollment by the investigator.

Trial locations and contacts

1 site in 1 country.

Fengkui Zhang, Doctor contact

8602223909229

fkzhang@ihcams.ac.cn

Liping Jing, Doctor contact

8602223909223

jingliping@ihcams.ac.an

China1 site, 1 recruiting

  • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 300020

    Fengkui Zhang, Doctor, 8602223909229, fkzhang@ihcams.ac.cn

    Liping Jing, Doctor, 8602223909223, jingliping@ihcams.ac.cn

    Recruiting

Other trials in China

About this study

Official title: Avatrombopag in TPO-RA Refractory Aplastic Anemia Patients Safety and Efficacy Study --Single-arm, Multicenter, Open, Phase Π Clinical Study

This study was a single-arm, multicenter, phase Π clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight <80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight <80 kg; 60 mg daily for weight >80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug

Read the detailed description

This study was a single-arm, multicenter, phase Π clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight <80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight <80 kg; 60 mg daily for weight >80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug.Selection of study population Severe aplastic anemia patients with poor efficacy of IST combined with TPO-RA Patients should be judged for inclusion and exclusion criteria. Number of subjects: 35 effective cases, 39 patients should be included according to the dropout rate of 10%.

Being tested
avatrombopag
Keywords
Avatrombopag, TPO-RA, refractory aplastic anemia
Medical subject headings
Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • avatrombopagDrug

    Also called CSA

    Avatrombopag, 40-60 mg (body weight < 80 kg, 40 mg per day; body weight > 80 kg, 60 mg per day) orally once daily for at least 3 months and followed up for 3 months to determine hematological response and evaluate the drug security.

Study groups in NCT05518331
Study groupRoleWhat they receive
Avatrombopag in RAAExperimentalAfter the patients met the above-mentioned inclusion conditions and signed informed consent, they began to be included in this program. The main research objectives are to take avatrombopag conversion therapy for at least 3 months, to monitor hematological indicators, biochemical indicators and bone marrow related tests, to determine hematological responses, and to evaluate the safety of the drug. In the 6th and 12th months after treatment, comprehensive review of bone marrow and peripheral blood was performed to evaluate the recovery of hematopoiesis, determine the curative effect, evaluate adverse events, and whether there was clonal transformation. After the patients completed the main study observation, they were followed up for at least 3 months, that is, from the time the patients were enrolled, for a total of at least 6 months of follow-up.
Receives: Drug: avatrombopag

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The rate of HR in patients after switching to avatrombopag.

    Percentage of the total number of patients receiving treatment who received APAG

    Measured: 3 months

  2. Incidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading

    Incidence of Treatment-Emergent AE by CTCAE

    Measured: 3 months

  3. Percentage of patients with transformation

    Rate of patients with transformation to PNH or MDS,AML, or other disease

    Measured: 3 months

Secondary outcomes (6)

  1. The rate of HR in patients after switching to avatrombopag.

    Percentage of the total number of patients receiving treatment who received APAG

    Measured: 6months

  2. ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading

    Incidence of Treatment-Emergent AE by CTCAE

    Measured: 6months

  3. Percentage of patients with transformation

    Rate of patients with transformation to PNH or MDS,AML, or other disease

    Measured: 6months

  4. The rate of HR in patients after switching to avatrombopag.

    Percentage of the total number of patients receiving treatment who received APAG

    Measured: 12months

  5. ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading

    Incidence of Treatment-Emergent AE by CTCAE

    Measured: 12months

  6. Percentage of patients with transformation

    Rate of patients with transformation to PNH or MDS,AML, or other disease

    Measured: 12months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Aug 2022
  2. Study start1 Jun 2022
  3. Primary completion1 Jun 2025 (estimated)
  4. Study completion1 Jan 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
39 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05518331
Sponsor's protocol ID
IIT2021008-EC-1
Status last verified
Aug 2022
First submitted
11 Jun 2022
Last updated
26 Aug 2022
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: We can share the plan after completing the experiment

Publications

  • Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, Hillmen P, Ireland R, Kulasekararaj A, Mufti G, Snowden JA, Samarasinghe S, Wood A, Marsh JC; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207. doi: 10.1111/bjh.13853. Epub 2015 Nov 16. No abstract available.

    PubMed 26568159

  • Scheinberg P. Activity of eltrombopag in severe aplastic anemia. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):450-456. doi: 10.1182/asheducation-2018.1.450.

    PubMed 30504345

  • Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X, Desierto M, Leuva H, Bevans M, Wu C, Larochelle A, Calvo KR, Dunbar CE, Young NS. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017 Apr 20;376(16):1540-1550. doi: 10.1056/NEJMoa1613878.

    PubMed 28423296

  • Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X, Lozier J, Wu CO, Young NS, Dunbar CE. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. doi: 10.1056/NEJMoa1200931.

    PubMed 22762314

  • Peng G, He G, Chang H, Gao S, Liu X, Chen T, Li P, Han B, Miao M, Ge Z, Ge X, Li F, Li Y, Wang S, Wang Y, Shen Y, Zhang T, Zou J, Zhang F. A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy. Ther Adv Hematol. 2022 Mar 30;13:20406207221085197. doi: 10.1177/20406207221085197. eCollection 2022.

    PubMed 35371427

  • Ise M, Iizuka H, Kamoda Y, Hirao M, Kida M, Usuki K. Romiplostim is effective for eltrombopag-refractory aplastic anemia: results of a retrospective study. Int J Hematol. 2020 Dec;112(6):787-794. doi: 10.1007/s12185-020-02971-1. Epub 2020 Sep 2.

    PubMed 32876852

Questions about this trial

What is this clinical trial studying?

A study testing avatrombopag in people with Refractory Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 26 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Fengkui Zhang, Doctor (8602223909229, fkzhang@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The rate of HR in patients after switching to avatrombopag. (time frame: 3 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05518331, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2022.

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