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Immune ThrombocytopeniaPhase 2NCT04217148

The Combination of ATRA and High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia

A Phase 2 study testing Dexamethasone and ATRA in people with Immune Thrombocytopenia, run by Peking University People's Hospital at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Randomized, open-label, multicenter study to compare the efficacy and safety of ATRA plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP). Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Immune Thrombocytopenia
What is being tested?
Dexamethasone and ATRA
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
132 participants
Where
1 siteChina
Study dates
1 Jan 2020to 30 Jun 2020
Phase
Phase 2
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 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Confirmed newly-diagnosed, treatment-naive ITP;
  • Platelet counts <30×109/L ;
  • Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
  • Willing and able to sign written informed consent.

Who cannot join

  • Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
  • Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
  • Current HIV infection or hepatitis B virus or hepatitis C virus infections;
  • Active infection;
  • Maligancy;
  • Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
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  • Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy
  • Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
  • Patients who are deemed unsuitable for the study by the investigator.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Peking University Insititute of Hematology, Peking University People's Hospital

    Beijing, Beijing Municipality, 100044

Other trials in China

About this study

Official title: The Combination of Oral All-trans Retinoic Acid and High-dose Dexamethasone vs High-dose Dexamethasone as First-line Treatment in Adult Immune Thrombocytopenia: A Multicenter, Randomized, Open-label Trial

Randomized, open-label, multicenter study to compare the efficacy and safety of ATRA plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

Read the detailed description

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 132 adults with ITP in China. Patients were randomized to ATRA+ high-dose dexamethasone and high-dose dexamethasone monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Being tested
Dexamethasone, ATRA
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.

  • DexamethasoneDrug

    Also called HD-DXM, High-dose Dexamethasone

    Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated in the case of lack of response by day 10)

  • ATRADrug

    Also called All-trans retinoic acid

    ATRA, po,10mg bid, for 12 weeks

Study groups in NCT04217148
Study groupRoleWhat they receive
ATRA and HD-DXMExperimentalDexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10) and ATRA 10mg bid po, 12 consecutive weeks
Receives: Drug: Dexamethasone; Drug: ATRA
HD-DXMActive comparatorDexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 10)
Receives: Drug: Dexamethasone

What is being measured

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

  1. Sustained response

    The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.

    Measured: 6 month

Secondary outcomes (7)

  1. complete response (CR)

    complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.

    Measured: day 14

  2. Response (R)

    Response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.

    Measured: day 14

  3. Number of patients with bleeding

    Number of patients with bleeding complication ( WHO bleeding score).

    Measured: 6 month

  4. Number of patients with adverse events

    Number of patients with adverse events.

    Measured: 6 month

  5. Time to response

    The time from starting treatment to time of achievement of CR or R

    Measured: 6 month

  6. Duration of response (DOR)

    Duration of response at 6-month follow up.

    Measured: 6 month

  7. Loss of response

    Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R)

    Measured: 6 month

Timeline

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

  1. First posted3 Jan 2020
  2. Study start1 Jan 2020
  3. Primary completion30 Jun 2020
  4. Study completion30 Jun 2020

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
132 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04217148
Sponsor's protocol ID
ITP-PKU007
Status last verified
Sep 2021
First submitted
30 Dec 2019
Last updated
28 Sep 2021
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Qualified researchers can request access to data, including deidentified individual participant data, and the study protocol from this clinical trial. These data will be available beginning 3 months and ending 36 months following publication. All requests must include a description of the research proposal and be sent to the corresponding author (zhangxh@bjmu.edu.cn). Data requestors will need to sign a data access agreement to obtain access.

Publications

  • Huang QS, Liu Y, Wang JB, Peng J, Hou M, Liu H, Feng R, Wang JW, Xu LP, Wang Y, Huang XJ, Zhang XH. All-trans retinoic acid plus high-dose dexamethasone as first-line treatment for patients with newly diagnosed immune thrombocytopenia: a multicentre, open-label, randomised, controlled, phase 2 trial. Lancet Haematol. 2021 Oct;8(10):e688-e699. doi: 10.1016/S2352-3026(21)00240-4.

    PubMed 34560012

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Dexamethasone and ATRA in people with Immune Thrombocytopenia, run by Peking University People's Hospital 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 4 inclusion and 9 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: finished, as of the sponsor's last update on 28 Sep 2021. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Sustained response (time frame: 6 month). 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 NCT04217148, which also lists 1 linked publication.

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 28 Sep 2021.

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