Acquired Hemophilia APhase 2NCT03700229
Study to Evaluate Rituximab and Bortezomib in Patients With Newly Diagnosed Acquired Hemophilia A
A Phase 2 study testing Bortezomib and Rituximab in people with Acquired Hemophilia A, run by Peking Union Medical College Hospital at 1 site in China.
Trial summary
The purpose of this prospective study is to determine the efficacy and safety of Rituximab plus Bortezomib in patients with newly diagnosed acquired hemophilia A. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acquired Hemophilia A
- What is being tested?
- Bortezomib and Rituximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteChina
- Study dates
- 10 Oct 2018to 15 Apr 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be ≥ 18 years;
- Understand and voluntarily sign an ICD prior to any study related assessments/procedures are conducted;
- Diagnosis of acquired hemophilia A；
- acute bleeding episodes(≥once).
Who cannot join
- Uncontrolled systemic infection;
- Allergy to rituximab;
- Positive for Lupus anticoagulant;
- Life expectancy < 3 months;
- Pregnant and breastfeeding women;
- Neuropathy>Grade 1;
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- Positive for Hepatitis B surface antigen or hepatitis C antibody or human immunodeficiency virus（HIV）antibody;
- Patients with poor compliance；
- Patient who is considered by the investigator not suitable for clinical study.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Huacong Cai, 01069158271, caihc@pumch.cn
Tienan Zhu, MD, 01069158271, zhutn@pumch.cn
About this study
Official title: An Open-label Phase II Study to Determine the Efficacy and Safety of Rituximab and Bortezomib in Patients With Newly Diagnosed Acquired Hemophilia A
The purpose of this prospective study is to determine the efficacy and safety of Rituximab plus Bortezomib in patients with newly diagnosed acquired hemophilia A.
Read the detailed description
This is a prospective, single-arm, open-label clinical trial to evaluate the efficacy and safety the regimen of Bortezomib with rituximab as first-line treatment to eradicate anti-factor VIII antibodies in newly diagnosed acquired hemophilia A.
All of enrolled patients in this study will be injected Bortezomib plus rituximab. This study will be performed for about 2 years and approximately 22 patients will be enrolled in our insititution.
After obtaining the written informed consent from the patients, the information of demographic and medical history will be collected and laboratory tests will be performed.
Patients who meet the inclusion/exclusion criteria will be received the regimens: Bortezomib (1.3mg/m2 d1,4,8,11) with rituximab (375mg/m2 for one dose). The information of adverse events will be collected. In case that the evaluation of treatment response might be performed in regular clinical practice, those data will also be collected as well.
- Conditions
- Acquired Hemophilia A
- Being tested
- Bortezomib, Rituximab
- Medical subject headings
- Factor 8 deficiency, acquired
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BortezomibDrug
Bortezomib intravenously 1.3mg/m2 d1,4,8,11 for 4 doses
RituximabDrug
rituximab intravenously 500 mg for one dose
| Study group | Role | What they receive |
|---|---|---|
| Bortezomib +Rituximab | Experimental | Bortezomib +Rituximab Receives: Drug: Bortezomib; Drug: Rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
The time to attain first complete remission (CR)
Complete remission defined as titer FVIII inhibitor lower than 0.6 Bethesda unit, factor VIII level> 50% and no bleeding events without bypass treatments for 24 hours
Measured: Last day of the treatment regimen (up to 3 months)
Secondary outcomes (3)
The time to durable treatment response
The time to durable treatment response was defined as the time from date of achieving CR until the date of relapse or date of death from any cause（whichever came first）
Measured: During 24 month
Adverse events
Include major bleeding, infection, nerve toxicity, and so on.
Measured: During 24 month
Overall survival
Overall survival was defined as the time from date of first diagnosis until the date of death from any cause，and death from any cause will be recorded.
Measured: During 24 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Oct 2018
- Study start10 Oct 2018
- Primary completion15 Oct 2019 (estimated)
- Study completion15 Apr 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Acquired Hemophilia AStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tienan Zhu, Peking Union Medical College Hospital
- Investigators
- Tienan Zhu, Principal investigator, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03700229
- Sponsor's protocol ID
- PUMCH-AHA-001
- Status last verified
- Nov 2018
- First submitted
- 4 Oct 2018
- Last updated
- 23 Nov 2018
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Collins P, Baudo F, Knoebl P, Levesque H, Nemes L, Pellegrini F, Marco P, Tengborn L, Huth-Kuhne A; EACH2 registry collaborators. Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2). Blood. 2012 Jul 5;120(1):47-55. doi: 10.1182/blood-2012-02-409185. Epub 2012 Apr 18.
PubMed 22517903Bras GP, Pinto RJ, Carvalho MM, Fernandes SP, Andrade JJ, Guimaraes JE. Bortezomib: Potential Key Role in the Treatment of Multiple Myeloma-Related Acquired Hemophilia A. Semin Thromb Hemost. 2017 Feb;43(1):109-112. doi: 10.1055/s-0036-1597648. Epub 2016 Dec 19. No abstract available.
PubMed 27992938Bonfanti C, Crestani S, Frattini F, Sissa C, Franchini M. Role of rituximab in the treatment of postpartum acquired haemophilia A: a systematic review of the literature. Blood Transfus. 2015 Jul;13(3):396-400. doi: 10.2450/2014.0242-14. Epub 2014 Dec 17. No abstract available.
PubMed 25545867Ratnasingam S, Walker PA, Tran H, Kaplan ZS, McFadyen JD, Tran H, Teh TC, Fleming S, Catalano JV, Chunilal SD, Johnston A, Opat SS, Shortt J. Bortezomib-based antibody depletion for refractory autoimmune hematological diseases. Blood Adv. 2016 Nov 22;1(1):31-35. doi: 10.1182/bloodadvances.2016001412. eCollection 2016 Nov 29.
PubMed 29296693
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Phase II Study to Determine the Efficacy and Safety of Rituximab and Bortezomib in Patients With Newly Diagnosed Acquired Hemophilia A
- ClinicalTrials.gov ID
- NCT03700229
- Acronym / sponsor ID
- PUMCH-AHA-001
- Conditions
- Acquired Hemophilia A
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 22 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 10 Oct 2018 (actual)
- Primary completion
- 15 Oct 2019 (estimated)
- Study completion
- 15 Apr 2020 (estimated)
- First posted
- 9 Oct 2018 (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.
The purpose of this prospective study is to determine the efficacy and safety of Rituximab plus Bortezomib in patients with newly diagnosed acquired hemophilia A.
Detailed description
This is a prospective, single-arm, open-label clinical trial to evaluate the efficacy and safety the regimen of Bortezomib with rituximab as first-line treatment to eradicate anti-factor VIII antibodies in newly diagnosed acquired hemophilia A.
All of enrolled patients in this study will be injected Bortezomib plus rituximab. This study will be performed for about 2 years and approximately 22 patients will be enrolled in our insititution.
After obtaining the written informed consent from the patients, the information of demographic and medical history will be collected and laboratory tests will be performed.
Patients who meet the inclusion/exclusion criteria will be received the regimens: Bortezomib (1.3mg/m2 d1,4,8,11) with rituximab (375mg/m2 for one dose). The information of adverse events will be collected. In case that the evaluation of treatment response might be performed in regular clinical practice, those data will also be collected as well.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bortezomib +Rituximab | Experimental | Drug: Bortezomib, Drug: RituximabBortezomib +Rituximab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Must be ≥ 18 years;
- Understand and voluntarily sign an ICD prior to any study related assessments/procedures are conducted;
- Diagnosis of acquired hemophilia A；
- acute bleeding episodes(≥once).
Key exclusion criteria (9 listed)
- Uncontrolled systemic infection;
- Allergy to rituximab;
- Positive for Lupus anticoagulant;
- Life expectancy < 3 months;
- Pregnant and breastfeeding women;
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 |
|---|---|---|
| BortezomibBortezomib intravenously 1.3mg/m2 d1,4,8,11 for 4 doses | Drug | Bortezomib +Rituximab |
| Rituximabrituximab intravenously 500 mg for one dose | Drug | Bortezomib +Rituximab |
Primary outcome
The time to attain first complete remission (CR)
Time frame: Last day of the treatment regimen (up to 3 months)
Complete remission defined as titer FVIII inhibitor lower than 0.6 Bethesda unit, factor VIII level> 50% and no bleeding events without bypass treatments for 24 hours
3 secondary outcomes
The time to durable treatment response
Time frame: During 24 month
Adverse events
Time frame: During 24 month
Overall survival
Time frame: During 24 month
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2018.
- Background: PubMed 22517903Collins P, Baudo F, Knoebl P, Levesque H, Nemes L, Pellegrini F, Marco P, Tengborn L, Huth-Kuhne A; EACH2 registry collaborators. Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2). Blood. 2012 Jul 5;120(1):47-55. doi: 10.1182/blood-2012-02-409185. Epub 2012 Apr 18.
- Background: PubMed 27992938Bras GP, Pinto RJ, Carvalho MM, Fernandes SP, Andrade JJ, Guimaraes JE. Bortezomib: Potential Key Role in the Treatment of Multiple Myeloma-Related Acquired Hemophilia A. Semin Thromb Hemost. 2017 Feb;43(1):109-112. doi: 10.1055/s-0036-1597648. Epub 2016 Dec 19. No abstract available.
- Background: PubMed 25545867Bonfanti C, Crestani S, Frattini F, Sissa C, Franchini M. Role of rituximab in the treatment of postpartum acquired haemophilia A: a systematic review of the literature. Blood Transfus. 2015 Jul;13(3):396-400. doi: 10.2450/2014.0242-14. Epub 2014 Dec 17. No abstract available.
- Results publication: PubMed 29296693Ratnasingam S, Walker PA, Tran H, Kaplan ZS, McFadyen JD, Tran H, Teh TC, Fleming S, Catalano JV, Chunilal SD, Johnston A, Opat SS, Shortt J. Bortezomib-based antibody depletion for refractory autoimmune hematological diseases. Blood Adv. 2016 Nov 22;1(1):31-35. doi: 10.1182/bloodadvances.2016001412. eCollection 2016 Nov 29.
- Individual participant dataSharing planned: Undecided
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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.
Tienan Zhu principal investigator
Peking Union Medical College Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bortezomib and Rituximab in people with Acquired Hemophilia A, run by Peking Union Medical College 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: status not confirmed, as of the sponsor's last update on 23 Nov 2018. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Huacong Cai (01069158271, caihc@pumch.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The time to attain first complete remission (CR) (time frame: Last day of the treatment regimen (up to 3 months)). 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 NCT03700229, which also lists 4 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 23 Nov 2018.