Immune ThrombocytopeniaPhase 3NCT05046327
Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With ITP of ≤6 Months
A Phase 3 study testing Avatrombopag 20 mg Oral Tablet in people with Immune Thrombocytopenia, run by Sobi, Inc..
Trial summary
This study will evaluate the safety and efficacy of avatrombopag in subjects with a confirmed diagnosis of primary ITP (≤6 months duration) over 26 weeks of treatment, and also evaluate the incidence of ITP remission. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- Avatrombopag 20 mg Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 14 Oct 2021to 19 Feb 2025
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female subjects ≥18 years of age at Screening.
- Subject must be able to provide informed consent.
- Subject has a confirmed diagnosis of primary ITP according to the International Consensus Report on the Investigation and Management of Primary ITP within the previous 6 months prior to Visit 1 and has had a previous response to a first line treatment (corticosteroids, IVIg, or anti-D), in the opinion of the Investigator.
- Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit.
- Females of childbearing potential must have a negative pregnancy test at Screening and Baseline.
- Female subjects of childbearing potential who are sexually active and male subjects who are sexually active must agree to use effective methods of contraception.
- Subject is willing and able to comply with all aspects of the protocol.
Who cannot join
- Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
- Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary).
- Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
- History of myelodysplastic syndrome (MDS) or other hematologic malignancies.
- Current history of significant cardiac arrhythmias or decompensated congestive heart failure.
- History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
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- Previous use of eltrombopag, romiplostim, recombinant human TPO or other platelet-producing agents.
- Surgical resection of the spleen.
- Previous use of mycophenolate mofetil (MMF), rituximab (or other B-cell lymphocyte depleting agents), mercaptopurine (6-MP) or alkylating agents.
- Concurrent malignant disease, other than non-melanoma skin cancer or cervical cancer in-situ.
- Known allergy to avatrombopag or any of its excipients.
- Subject is unable to take oral medication or has a malabsorption syndrome or any other uncontrolled gastrointestinal condition.
- Enrollment in another clinical study with any investigational drug or device within 30 days of Day 1/Visit 2 (or 5 half-lives, whichever is longer); however, participation in observational studies is permitted.
- Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator.
- Considered unable or unwilling to comply with the study protocol requirements.
About this study
Official title: A Multicenter, Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With Immune Thrombocytopenia (ITP) of ≤6 Months Duration
This study will evaluate the safety and efficacy of avatrombopag in subjects with a confirmed diagnosis of primary ITP (≤6 months duration) over 26 weeks of treatment, and also evaluate the incidence of ITP remission.
Read the detailed description
This phase 3b, multi-center, open-label study will enroll approximately 75 adult subjects with a confirmed diagnoses of primary ITP (≤6 months duration) who have had a previous response to a first line treatment. The study will consist of a 26-week treatment period to evaluate the safety and efficacy of avatrombopag. Subjects with platelet counts ≥50×10⁹/L at Week 26 may enter a dose-tapering period in which the dose of avatrombopag will be decreased for up to 16 weeks until avatrombopag treatment is discontinued and the platelet count is maintained ≥50×10⁹/L. Once avatrombopag treatment has been discontinued, the subjects will enter a remission evaluation period of up to 24 weeks to evaluate whether they have entered a state of remission.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Avatrombopag 20 mg Oral Tablet
- Keywords
- ITP
- 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.
Avatrombopag 20 mg Oral TabletDrug
Also called Doptelet
Avatrombopag administered at a frequency to maintain a target platelet count between ≥50 and ≤150×10⁹/L
| Study group | Role | What they receive |
|---|---|---|
| 20 mg Avatrombopag daily | Experimental | Avatrombopag Receives: Drug: Avatrombopag 20 mg Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative Number of Weeks of Platelet Response
Cumulative number of weeks of platelet response in which the platelet count is ≥50×10⁹/L during 6 months of treatment in the absence of rescue therapy.
Measured: 6 Months of Active Treatment
Secondary outcomes (3)
Durable Platelet Response
Durable platelet response as defined by the incidence of subjects who have at least 6 out of 8 weekly platelet counts ≥50×10⁹/L during the last 8 weeks of treatment.
Measured: 8 Weeks of Treatment
Incidence of ITP remission
Incidence of ITP remission as defined by platelet count ≥50×10⁹/L for 24 consecutive weeks with no ITP treatments (concomitant or rescue).
Measured: 24 Consecutive Weeks
Incidence of subjects achieving a platelet count response
Incidence of subjects achieving a platelet count response (≥50×10⁹/L) during the active treatment period of the study.
Measured: 6 Months of Active Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2021
- Study start14 Oct 2021 (estimated)
- Primary completion6 Apr 2024 (estimated)
- Study completion19 Feb 2025 (estimated)
Why it stopped: Corporate Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sobi, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05046327
- Sponsor's protocol ID
- AVA-ITP-306
- Status last verified
- Oct 2021
- First submitted
- 8 Sep 2021
- Last updated
- 22 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With Immune Thrombocytopenia (ITP) of ≤6 Months Duration
- ClinicalTrials.gov ID
- NCT05046327
- Acronym / sponsor ID
- AVA-ITP-306
- Conditions
- Immune Thrombocytopenia
- Keywords
- ITP
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Corporate Decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Sobi, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Oct 2021 (estimated)
- Primary completion
- 6 Apr 2024 (estimated)
- Study completion
- 19 Feb 2025 (estimated)
- First posted
- 16 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the safety and efficacy of avatrombopag in subjects with a confirmed diagnosis of primary ITP (≤6 months duration) over 26 weeks of treatment, and also evaluate the incidence of ITP remission.
Detailed description
This phase 3b, multi-center, open-label study will enroll approximately 75 adult subjects with a confirmed diagnoses of primary ITP (≤6 months duration) who have had a previous response to a first line treatment. The study will consist of a 26-week treatment period to evaluate the safety and efficacy of avatrombopag. Subjects with platelet counts ≥50×10⁹/L at Week 26 may enter a dose-tapering period in which the dose of avatrombopag will be decreased for up to 16 weeks until avatrombopag treatment is discontinued and the platelet count is maintained ≥50×10⁹/L. Once avatrombopag treatment has been discontinued, the subjects will enter a remission evaluation period of up to 24 weeks to evaluate whether they have entered a state of remission.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 20 mg Avatrombopag daily | Experimental | Drug: Avatrombopag 20 mg Oral TabletAvatrombopag |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female subjects ≥18 years of age at Screening.
- Subject must be able to provide informed consent.
- Subject has a confirmed diagnosis of primary ITP according to the International Consensus Report on the Investigation and Management of Primary ITP within the previous 6 months prior to Visit 1 and has had a previous response to a first line treatment (corticosteroids, IVIg, or anti-D), in the opinion of the Investigator.
- Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit.
- Females of childbearing potential must have a negative pregnancy test at Screening and Baseline.
Key exclusion criteria (15 listed)
- Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
- Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary).
- Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
- History of myelodysplastic syndrome (MDS) or other hematologic malignancies.
- Current history of significant cardiac arrhythmias or decompensated congestive heart failure.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Avatrombopag 20 mg Oral TabletAlso: DopteletAvatrombopag administered at a frequency to maintain a target platelet count between ≥50 and ≤150×10⁹/L | Drug | 20 mg Avatrombopag daily |
Primary outcome
Cumulative Number of Weeks of Platelet Response
Time frame: 6 Months of Active Treatment
Cumulative number of weeks of platelet response in which the platelet count is ≥50×10⁹/L during 6 months of treatment in the absence of rescue therapy.
3 secondary outcomes
Durable Platelet Response
Time frame: 8 Weeks of Treatment
Incidence of ITP remission
Time frame: 24 Consecutive Weeks
Incidence of subjects achieving a platelet count response
Time frame: 6 Months of Active Treatment
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2021.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Avatrombopag 20 mg Oral Tablet in people with Immune Thrombocytopenia, run by Sobi, Inc..
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 7 inclusion and 15 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: withdrawn, as of the sponsor's last update on 22 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was withdrawn before enrolling participants, so there are no results.
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?
Cumulative Number of Weeks of Platelet Response (time frame: 6 Months of Active Treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 Oct 2021.