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Primary Immune ThrombocytopeniaPhase 4(ITP;rhTPO)NCT03771378

Efficacy and Safety of rhTPO and Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP)

A Phase 4 study testing rhTPO and eltrombopag in people with Primary Immune Thrombocytopenia, run by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology 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 The sponsor has not verified this record for over two years, so it may be out of date.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

Primary immune thrombocytopenia (ITP) is an acquired autoimmune hemorrhagic disease. In most cases, platelet count is negatively correlated with the severity of bleeding. Correcting low platelets is the main measure to prevent bleeding in patients with ITP. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Primary Immune Thrombocytopenia
What is being tested?
rhTPO and eltrombopag
Could I receive a placebo?
No, everyone receives an active treatment
How many people
96 participants
Where
1 siteChina
Study dates
16 Jan 2019to 15 May 2020
Phase
Phase 4
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 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • The patient signs an informed consent form.
  • Age from 18 to 75 years old
  • The patient's first diagnosis of ITP is at least 6 months before enrollment; the platelet count must <30×109/L before taking the study drug (48 hours before).
  • Patients who were diagnosed with ITP by bone marrow biopsy and other related examinations before enrollment(Bone marrow biopsy is valid for 30 days, including 30 days);
  • The patient has been treated with splenectomy for relapse or relapse; or the patient has not undergone splenectomy, but was ineffective or relapses after treatment with at least one first line drug. Past ITP therapy can include, but is not limited to, corticosteroids, immunoglobulins (IVIG or anti-D Immunoglobulin), azathioprine, danazol, cyclophosphamide and immunomodulators;
  • Previous salvage treatments included infusion of platelets, immunoglobulins, immunomodulators, and cyclophosphamide must be completed 2 weeks prior to enrollment or treatment. Corticosteroids must end at least 14 days before enrollment.
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  • Patients receiving immunosuppressive agents (including corticosteroids, azathioprine, danazol, cyclosporin A, mycophenolate mofetil) or proprietary Chinese medicines have maintained a stable therapeutic dose for at least the last month; patients who received rituximab should be discontinued half a year prior to enrollment; patients with spleen were enrolled six months after surgery;
  • No heart disease in the past 3 months, including NYHA grade III/IV charge
  • , heart failure, arrhythmia or myocardial infarction requiring medical treatment;
  • Laboratory tests for coagulation function showed that prothrombin time (PT/INR) and activated partial thromboplastin time (APTT) values did not exceed 20% of the normal reference range.No history of coagulation abnormalities except ITP;
  • White blood cell count, neutrophil absolute value, hemoglobin in the normal value.Except in the following cases: a) Platelet count <30×10^9/L within Day1 or Day1 within 48 hours; b) Hemoglobin: if anemia is clearly caused by ITP (thrombocytopenia caused blood loss), the lower limit of the subject's hemoglobin level below the normal value can be based on the investigator's judgment to decide the subject whether to be selected; c) absolute neutrophil count ≥ 1.5 × 109 / L can be enrolled;
  • The following clinical biochemical indicators must be within 20% of the normal range: creatinine, alanine aminotransferase, aspartate aminotransferase, total bilirubin and alkaline phosphatase. In addition, serum albumin is not lower than the lower limit of normal value by 10%;
  • Subjects took an approved method of contraception. Female subjects (or female partners of male subjects) must be infertile (hysterectomy, bilateral salpingectomy, bilateral tubal ligation or more than 1 year after menopause) or have fertility but before the first dose for 2 weeks, study-approved contraceptive methods were used throughout the study period to 28 days after the end of the study or discontinuation of the study.Male subjects with a fertile female partner must have undergone vasectomy or consent to effective contraception throughout the study period (2 weeks prior to the first dose, throughout the study period, until the end of the study or 28 days after the discontinuation of the study) method;
  • Women with fertility must have a negative serum pregnancy test within 24 hours prior to the first dose;
  • Subjects fully understand and are able to comply with the requirements of the research protocol and are willing to complete the study as planned.

Who cannot join

  • Subjects had a history of any arterial/venous thrombosis (including stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis, or pulmonary embolism) and had at least 2 of the following risk factors: hormone replacement therapy, oral contraception medicine (including estrogen), smoking, diabetes, hypercholesterolemia, drug-controlled hypertension, malignant tumors, hereditary coagulopathy;
  • Abnormalities other than ITP during the screening phase or any medical history or condition that the investigator considered unsuitable for participation in the study;
  • Patients with BMI ≥ 28;
  • Pregnant or lactating women;
  • A history of alcohol/drug abuse within 12 months prior to screening or first dose;
  • Previous treatment with a specific study drug other than rhTPO or other research treatments;
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  • The subject has previously received or is currently receiving treatment with exenatide or other thrombopoietin receptor agonists;
  • Throughout the study, medications that affected platelet function (including but not limited to aspirin, clopidogrel and/or non-steroidal anti-inflammatory drugs NSAIDs) or anticoagulant therapy were continued for >3 days;
  • Accept any herbal or nutritional supplements, excluding vitamin supplements and mineral supplements within 1 week prior to the start of the study;
  • There is a history of abnormal platelet aggregation that may affect the reliability of platelet count measurements;
  • Before the first dose administration, the bone marrow biopsy showed abnormality except for ITP within 4 weeks, and the investigator judged that the abnormality made the subject unsuitable for the study, or the bone marrow biopsy showed other primary disease which caused thrombocytopenia;
  • Evidence of all laboratory or clinical HIV infections, previous clinical history of hepatitis C,hepatitis B, or active hepatitis at screening. Laboratory tests during the screening period indicate hepatitis C infection or hepatitis B infection. (Defined as HBsAg test positive, in addition, if the HBsAg test is negative, but HBcAb is positive, regardless of the status of HBsAb, HBV DNA testing is required, if positive, subjects should be excluded);
  • Rescuing treatment is required before the first dose of the drug.

Trial locations and contacts

1 site in 1 country.

Hu Yu, M.D., Ph.D contact

86-13986183871 ext. 8613986183871

dr_huyu@126.com

Mei Heng, M.D., Ph.D contact

86-13886160811 ext. 8613986183871

mayheng@126.com

China1 site, 1 recruiting

  • Zhou Yiming

    Wuhan, Hubei, 430074

    zhou ming, +8613212794115, xumin_1015@163.com

    Xu MIN, +8613212794115, xumin_1015@163.com

    Recruiting

Other trials in China

About this study

Official title: Efficacy and Safety of rhTPO and Eltrombopag in the Treatment of Chinese Adults With Primary Immune Thrombocytopenia (ITP):a Multicenter, Double-blind, Randomized, Controlled Trial

Primary immune thrombocytopenia (ITP) is an acquired autoimmune hemorrhagic disease. In most cases, platelet count is negatively correlated with the severity of bleeding. Correcting low platelets is the main measure to prevent bleeding in patients with ITP. Both rhTPO and eltrombopag act on TPO receptors to increase platelets.Which one will have a better short-term (2 weeks) effect to improve platelets is the purpose of the investigator's research.

Read the detailed description

Primary immune thrombocytopenia (ITP) is an acquired autoimmune hemorrhagic disease. ITP patients have an increased risk of bleeding and the clinical symptoms are petechia, ecchymosis, organ hemorrhage and even life-threatening bleeding. The mortality rate in ITP is increased approximately 60% compared with age- and sex matched comparisons, bleeding is one of the leading causes of death, thus treating and preventing bleeding is particularly important for those patients. In most cases, platelet count is negatively correlated with the severity of bleeding. Correcting low platelets is the main measure to prevent bleeding in patients with ITP. Recombinant human thrombopoietin (rhTPO), a full-length glycated TPO, is a recombinant form of c-Mpl ligand that competes with endogenous TPO for binding to TPO receptors (TPO-R, c-Mp0), many clinical trials have shown that rhTPO can efficiently and safely stimulate platelet production, increase peripheral blood platelet count. The State Food and Drug Administration approved the drug as the second-line treatment for patients with ITP in 2005. Eltrombopag is an oral, synthetic non-peptide small molecule human thrombopoietin receptor agonist approved by the US FDA in November 2008 for the treatment of patients with chronic ITP, which were inefficiently by use of glucocorticoids, immunoglobulin or splenectomy. However, pharmacokinetic study of eltrombopag showed that Asians have twice the drug exposure rate than non-Asians, so eltrombopag may have different effects in different populations. Therefore, this trial will compare the efficacy and safety of intravenous rhTPO or oral use of eltrombopag after 14 days of treatment in Chinese ITP patients.

Being tested
rhTPO, eltrombopag
Keywords
ITP, rhTPO, Eltrombopag, platelets
Medical subject headings
Purpura, Thrombocytopenic, Idiopathic

What participants receive

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

  • rhTPODrug

    After enrollment, all subjects receive TPO, the dose is 300 U / Kg, s.c. qd, blood routine examination is detected every 3 days during treatment. If the platelet count > 250 × 109 / L, the drug will stop until the platelet count ≤ 100 × 109 / L. Efficacy and safety will be evaluated on day 15. The evaluation criteria were based on the consensus of ITP. At the same time, all subjects will receive a mimetic (tablet), po, qd.

  • eltrombopagDrug

    After enrollment, all subjects receive eltrombopag, the dose is 25 mg/day, po, qd. blood routine examination is detected every 3 days during treatment. If the platelet count > 250 × 109 / L, the drug will stop until the platelet count ≤ 100 × 109 / L. Efficacy and safety will be evaluated on day 15. The evaluation criteria were based on the consensus of ITP. At the same time, all subjects will give a mimetic (injection), at a dose of 300 U/Kg, s.c. qd.

Study groups in NCT03771378
Study groupRoleWhat they receive
Effective of rhTPOExperimentalAfter enrollment, all subjects receive rhTPO, the dose is 300 U / Kg, s.c. qd, blood routine examination is detected every 3 days during treatment. If the platelet count > 250 × 109 / L, the drug will stop until the platelet count ≤ 100 × 109 / L. Efficacy and safety will be evaluated on day 15. The evaluation criteria were based on the consensus of ITP. At the same time, all subjects will receive a mimetic (tablet), po, qd.
Receives: Drug: rhTPO
Effective of eltrombopagActive comparatorAfter enrollment, all subjects receive eltrombopag, the dose is 25 mg/day, po, qd, blood routine examination is detected every 3 days during treatment. If the platelet count > 250 × 109 / L, the drug will stop until the platelet count ≤ 100 × 109 / L. Efficacy and safety will be evaluated on day 15. The evaluation criteria were based on the consensus of ITP. At the same time, all subjects will give a mimetic (injection), at a dose of 300 U/Kg, s.c. qd.
Receives: Drug: eltrombopag

What is being measured

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

  1. Treatment response

    Proportion of subjects with platelet counts ≥50×10^9/L after 14 days of treatment

    Measured: 14 days from treatment

Secondary outcomes (6)

  1. Remission rate

    Complete response(platelet count ≥100 X 109/L and absence of bleeding), effective(platelet count

    ≥30X109/L and at least 2-fold increase of the baseline count and absence of bleeding), ineffective(platelet count <30 X109/L or less than 2-fold increase of the baseline platelet count or bleeding) proportion of testers after 14 days of treatment

    Measured: 14 days from treatment

  2. Drug efficacy: Evaluation of effectiveness

    During treatment, the proportion of subjects, which the platelet count at least once reached ≥50×109/L.

    Measured: 14 days from treatment

  3. Evaluation of effectiveness: the proportion of subjects, which the platelet count increased at least 2 times compared with baseline

    During treatment, the proportion of subjects, which the platelet count increased at least 2 times compared with baseline.

    Measured: 14 days from treatment

  4. Using ITP-specific bleeding assessment tool (ITP-BAT) to accessvbleeding scoer

    Bleeding manifestations were grouped into three major domains: skin (S), visible mucosae (M), and organs (O), with gradation of severity (SMOG). Each bleeding manifestation is assessed at the time of examination. Severity is graded from 0 to 3 or 4, with grade 5 for any fatal bleeding.

    Bleeding reported by the patient without medical documentation is graded 1. Within each domain, the same grade is assigned to bleeding manifestations of similar clinical impact. The "worst bleeding manifestation since the last visit" (observation period) is graded (a suitable database collection form is provided), and the highest grade within each domain is recorded.bleeding score

    Measured: 1 month from treatment

  5. The incidence of adverse events

    During treatment, the proportion of subjects, which received at least once rescue.Adverse events were evaluated according to Common Terminology Criteria for Adverse Events,version3.0,published by US National Cancer Institute.Adverse events included liver injury, fever, headache, bleeding, abdominal pain, nausea, vomiting, etc

    Measured: 14 days from treatment

  6. The incidence of side effects of the drugs

    Assessing safety through the adverse events,such as liver damage, etc.

    Measured: 1 month from treatment

Timeline

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

  1. First posted11 Dec 2018
  2. Study start16 Jan 2019
  3. Primary completion1 May 2020 (estimated)
  4. Study completion15 May 2020 (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 4
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
This study was a randomized, double-blind trial in which patients were divided into two groups, one receiving rhTPO injection and oral placebo tablet; the other group receiving oral eltrombopag and receiving a injection placebo.
Masking
Single blind
Who is masked
Investigator
Enrolment
96 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03771378
Sponsor's protocol ID
T18008-A
Acronym
(ITP;rhTPO)
Status last verified
Nov 2019
First submitted
7 Dec 2018
Last updated
15 Nov 2019
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided: It is not clear whether the data would involve patient privacy or whether patients would agree to the disclosure of the data.

Publications

  • Rodeghiero F, Stasi R, Gernsheimer T, Michel M, Provan D, Arnold DM, Bussel JB, Cines DB, Chong BH, Cooper N, Godeau B, Lechner K, Mazzucconi MG, McMillan R, Sanz MA, Imbach P, Blanchette V, Kuhne T, Ruggeri M, George JN. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009 Mar 12;113(11):2386-93. doi: 10.1182/blood-2008-07-162503. Epub 2008 Nov 12.

    PubMed 19005182

  • Hodak E, David M, Ingber A, Rotem A, Hazaz B, Shamai-Lubovitz O, Sandbank M. The clinical and histopathological spectrum of IgA-pemphigus--report of two cases. Clin Exp Dermatol. 1990 Nov;15(6):433-7. doi: 10.1111/j.1365-2230.1990.tb02138.x.

    PubMed 2279341

  • Zhang Y, Kolesar JM. Eltrombopag: an oral thrombopoietin receptor agonist for the treatment of idiopathic thrombocytopenic purpura. Clin Ther. 2011 Nov;33(11):1560-76. doi: 10.1016/j.clinthera.2011.10.004. Epub 2011 Nov 4.

    PubMed 22054810

  • Erickson-Miller CL, DeLorme E, Tian SS, Hopson CB, Stark K, Giampa L, Valoret EI, Duffy KJ, Luengo JL, Rosen J, Miller SG, Dillon SB, Lamb P. Discovery and characterization of a selective, nonpeptidyl thrombopoietin receptor agonist. Exp Hematol. 2005 Jan;33(1):85-93. doi: 10.1016/j.exphem.2004.09.006.

    PubMed 15661401

  • Williams DD, Peng B, Bailey CK, Wire MB, Deng Y, Park JW, Collins DA, Kapsi SG, Jenkins JM. Effects of food and antacids on the pharmacokinetics of eltrombopag in healthy adult subjects: two single-dose, open-label, randomized-sequence, crossover studies. Clin Ther. 2009 Apr;31(4):764-76. doi: 10.1016/j.clinthera.2009.04.010.

    PubMed 19446149

  • Cheng G. Eltrombopag, a thrombopoietin- receptor agonist in the treatment of adult chronic immune thrombocytopenia: a review of the efficacy and safety profile. Ther Adv Hematol. 2012 Jun;3(3):155-64. doi: 10.1177/2040620712442525.

    PubMed 23556122

  • Tomiyama Y, Miyakawa Y, Okamoto S, Katsutani S, Kimura A, Okoshi Y, Ninomiya H, Kosugi H, Nomura S, Ozaki K, Ikeda Y, Hattori T, Katsura K, Kanakura Y. A lower starting dose of eltrombopag is efficacious in Japanese patients with previously treated chronic immune thrombocytopenia. J Thromb Haemost. 2012 May;10(5):799-806. doi: 10.1111/j.1538-7836.2012.04695.x.

    PubMed 22409309

  • Mei H, Xu M, Yuan G, Zhu F, Guo J, Huang R, Qin J, Lv T, Qin F, Cai H, Yin P, Qin T, Hu Y. A multicentre double-blind, double-dummy, randomised study of recombinant human thrombopoietin versus eltrombopag in the treatment of immune thrombocytopenia in Chinese adult patients. Br J Haematol. 2021 Dec;195(5):781-789. doi: 10.1111/bjh.17808. Epub 2021 Sep 16.

    PubMed 34528239

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing rhTPO and eltrombopag in people with Primary Immune Thrombocytopenia, run by Union Hospital, Tongji Medical College, Huazhong University of Science and Technology 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?

The sponsor lists 15 inclusion and 13 exclusion criteria. 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 15 Nov 2019. 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.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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 Hu Yu, M.D., Ph.D (86-13986183871 ext. 8613986183871, dr_huyu@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Treatment response (time frame: 14 days from treatment). 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 NCT03771378, which also lists 8 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 15 Nov 2019.

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