Immune ThrombocytopeniaPhase 4NCT04638829
Safety and Treatment Satisfaction in Adults With Chronic ITP After Switching to Avatrombopag From Eltrombopag or Romiplostim
A Phase 4 study testing Avatrombopag Oral Tablet in people with Immune Thrombocytopenia, run by Sobi, Inc. at 20 sites in United States.
Trial summary
Evaluate the safety and tolerability of avatrombopag given for 90 days after stopping treatment with eltrombopag or romiplostim. 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 Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 20 sitesUnited States
- Study dates
- 15 Mar 2021to 3 Jan 2024
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Subject has been undergoing treatment for primary ITP with eltrombopag or romiplostim for at least 90 days prior to the Screening Visit/Visit 1.
- Subject has had a previous response (at any time) to either eltrombopag or romiplostim, defined as at least 2 platelet counts ≥50×10⁹/L. Subject is willing and able to comply with all aspects of the protocol, including completing the self-administered Treatment Satisfaction Medication Questionnaire (TSQM).
Who cannot join
- Subject is currently receiving chemotherapy or radiation for any form of cancer.
- Subject with conditions that are likely to prevent them from accurately and reliably completing study assessments, including evidence of moderate to severe dementia, and/or severe and progressive medical illness, as determined by the Investigator.
- Any previous avatrombopag use.
- Previous participation in this study; a subject may not re-enroll after prior discontinuation or completion.
- Enrollment in another clinical study with any investigational drug or device within 30 days of Baseline (or 5 half-lives, whichever is longer); however, participation in observational studies within the previous 30 days is permitted.
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
United States20 sites
Sobi Site 101
Bethesda, Maryland, 20817
Sobi Site 121
Chapel Hill, North Carolina, 27514
Sobi Site 126
Chicago, Illinois, 60612
Sobi Site 128
Cleveland, Ohio, 44106
Sobi Site 113
Dallas, Texas, 75246
Sobi Site 116
Greenville, North Carolina, 27834
Sobi Site 103
Lincoln, Nebraska, 68506
Sobi Site 129
Miami, Florida, 33143
Sobi Site 124
New Orleans, Louisiana, 70112
Sobi Site 120
Ocala, Florida, 34474
Sobi Site 109
Peoria, Illinois, 61614
Sobi Site 127
Philadelphia, Pennsylvania, 19104
Sobi Site 104
Rock Hill, South Carolina, 29732
Sobi Site 108
Salt Lake City, Utah, 84132
Sobi Site 125
St. Petersburg, Florida, 33709
Sobi Site 118
Tampa, Florida, 33606
Sobi Site 110
Tucson, Arizona, 85745
Sobi Site 123
Washington D.C., District of Columbia, 20010
Sobi Site 119
Whittier, California, 90603
Sobi Site 102
York, Pennsylvania, 17403
About this study
Official title: Prospective, Multi-center, Open-label Study Measuring Safety and Treatment Satisfaction in Adult Subjects With Chronic Immune Thrombocytopenia (ITP) After Switching to Avatrombopag From Eltrombopag or Romiplostim
Evaluate the safety and tolerability of avatrombopag given for 90 days after stopping treatment with eltrombopag or romiplostim.
Read the detailed description
This Phase 4, prospective, multi-center, open-label study will evaluate safety, platelet count, and subject reported medication satisfaction in adult subjects with chronic ITP after switching to avatrombopag from eltrombopag or romiplostim. At least 100 subjects will be enrolled, 50 (±10) who have received eltrombopag and 50 (±10) who have received romiplostim for at least 90 days prior to study entry.
- Conditions
- Immune Thrombocytopenia
- Being tested
- Avatrombopag 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 Oral TabletDrug
Also called Doptelet
Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
| Study group | Role | What they receive |
|---|---|---|
| Avatrombopag | Other | Avatrombopag 20 mg oral tablet formulation for 90 days Receives: Drug: Avatrombopag Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability (Adverse Events)
Safety and Tolerability of Avatrombopag given for 90 days after stopping eltrombopag or romiplostim
The incidence and severity of adverse events (AEs), serious adverse events (SAE) and adverse events of special interest (AESIs) will be summarized for all enrolled subjects using counts and percentages. Treatment-emergent AEs and SAEs will be summarized overall, by system organ class, and by preferred term. AESIs will be summarized by event type (thromboembolic events and bleeding events). In addition, treatment-emergent AEs will be summarized by severity and by relationship to study drug.
Bleeding events reported during the study will be summarized by WHO grade.
Measured: Screening through Day 90 or End of Study
Secondary outcomes (8)
Change From Baseline TSQM Convenience Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Convenience domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Measured: Day 90
Change From Baseline TSQM Side Effects Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Side Effects domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Measured: Day 90
Change From Baseline TSQM Effectiveness Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Effectiveness domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Measured: Day 90
Change From Baseline TSQM Global Satisfaction Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Global Satisfaction domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Measured: Day 90
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Measured: Day 15
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Measured: Day 30
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Measured: Day 60
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Measured: Day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2020
- Study start15 Mar 2021
- Primary completion3 Jan 2024
- Study completion3 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 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
- NCT04638829
- Sponsor's protocol ID
- AVA-ITP-401
- Status last verified
- Dec 2024
- First submitted
- 11 Nov 2020
- Last updated
- 16 Jan 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Tarantino MD, Mosalpuria K, Kolodny S, Zhang J, Vredenburg M, Jamieson BD. Safety, efficacy, and treatment satisfaction in adults with ITP who switched to avatrombopag from another TPO-RA. Blood Adv. 2025 Jun 10;9(11):2733-2743. doi: 10.1182/bloodadvances.2024015635.
PubMed 40101243
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Multi-center, Open-label Study Measuring Safety and Treatment Satisfaction in Adult Subjects With Chronic Immune Thrombocytopenia (ITP) After Switching to Avatrombopag From Eltrombopag or Romiplostim
- ClinicalTrials.gov ID
- NCT04638829
- Acronym / sponsor ID
- AVA-ITP-401
- Conditions
- Immune Thrombocytopenia
- Keywords
- ITP
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Sobi, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Mar 2021 (actual)
- Primary completion
- 3 Jan 2024 (actual)
- Study completion
- 3 Jan 2024 (actual)
- First posted
- 20 Nov 2020 (actual)
- Results
- Posted 16 Jan 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluate the safety and tolerability of avatrombopag given for 90 days after stopping treatment with eltrombopag or romiplostim.
Detailed description
This Phase 4, prospective, multi-center, open-label study will evaluate safety, platelet count, and subject reported medication satisfaction in adult subjects with chronic ITP after switching to avatrombopag from eltrombopag or romiplostim. At least 100 subjects will be enrolled, 50 (±10) who have received eltrombopag and 50 (±10) who have received romiplostim for at least 90 days prior to study entry.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Avatrombopag | Other | Drug: Avatrombopag Oral TabletAvatrombopag 20 mg oral tablet formulation for 90 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Subject has been undergoing treatment for primary ITP with eltrombopag or romiplostim for at least 90 days prior to the Screening Visit/Visit 1.
- Subject has had a previous response (at any time) to either eltrombopag or romiplostim, defined as at least 2 platelet counts ≥50×10⁹/L. Subject is willing and able to comply with all aspects of the protocol, including completing the self-administered Treatment Satisfaction Medication Questionnaire (TSQM).
Key exclusion criteria (5 listed)
- Subject is currently receiving chemotherapy or radiation for any form of cancer.
- Subject with conditions that are likely to prevent them from accurately and reliably completing study assessments, including evidence of moderate to severe dementia, and/or severe and progressive medical illness, as determined by the Investigator.
- Any previous avatrombopag use.
- Previous participation in this study; a subject may not re-enroll after prior discontinuation or completion.
- Enrollment in another clinical study with any investigational drug or device within 30 days of Baseline (or 5 half-lives, whichever is longer); however, participation in observational studies within the previous 30 days is permitted.
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 Oral TabletAlso: DopteletAvatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information | Drug | Avatrombopag |
Primary outcome
Safety and Tolerability (Adverse Events)
Time frame: Screening through Day 90 or End of Study
Safety and Tolerability of Avatrombopag given for 90 days after stopping eltrombopag or romiplostim The incidence and severity of adverse events (AEs), serious adverse events (SAE) and adverse events of special interest (AESIs) will be summarized for all enrolled subjects using counts and percentages. Treatment-emergent AEs and SAEs will be summarized overall, by system organ class, and by preferred term. AESIs will be summarized by event type (thromboembolic events and bleeding events). In addition, treatment-emergent AEs will be summarized by severity and by relationship to study drug. Bleeding events reported during the study will be summarized by WHO grade.
8 secondary outcomes
Change From Baseline TSQM Convenience Domain Score
Time frame: Day 90
Change From Baseline TSQM Side Effects Domain Score
Time frame: Day 90
Change From Baseline TSQM Effectiveness Domain Score
Time frame: Day 90
Change From Baseline TSQM Global Satisfaction Domain Score
Time frame: Day 90
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 15
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 30
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 60
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 90
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40101243Tarantino MD, Mosalpuria K, Kolodny S, Zhang J, Vredenburg M, Jamieson BD. Safety, efficacy, and treatment satisfaction in adults with ITP who switched to avatrombopag from another TPO-RA. Blood Adv. 2025 Jun 10;9(11):2733-2743. doi: 10.1182/bloodadvances.2024015635.
Related trials for Immune Thrombocytopenia
RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
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.
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Avatrombopag Oral Tablet in people with Immune Thrombocytopenia, run by Sobi, Inc. at 20 sites in United States.
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 2 inclusion and 5 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 16 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. 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?
Yes. Results are posted in the registry (first posted 16 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Safety and Tolerability (Adverse Events) (time frame: Screening through Day 90 or End of Study). 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 NCT04638829, 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 16 Jan 2025.