Hemophilia A With InhibitorPhase 4HA-LD-ITINCT03598725
Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in China
A Phase 4 study testing Coagulation Factor VIII, Prednisone and Rituximab in people with Hemophilia A With Inhibitor, run by Beijing Children's Hospital at 1 site in China.
Trial summary
The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression. Read more about this study
- Who is it for?
- Ages 1 year to 14 years, men only
- Conditions the study is for
- Hemophilia A With Inhibitor
- What is being tested?
- Coagulation Factor VIII, Prednisone and Rituximab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2016to 1 Dec 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 1 year to 14 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Males
- from 1 to 14 years old
- severe or moderate hemophilia A;
- inhibitors positive before ITI started.
Who cannot join
- Females
- <1 or >14 years old
- hemophilia B or mild haemophilia A;
- inhibitor negative before ITI started.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Beijing Children's Hospital
Beijing, Beijing Municipality, 100045
Runhui Wu, master, 0086-010-59617621, runhuiwu@hotmail.com
About this study
Official title: Beijing Children's Hospital, Capital Medical University
The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression.
Read the detailed description
Poor risk(s) includes:①peak historical inhibitor titer≥200BU ②inhibitor titer≥10BU before ITI initiation ③peak inhibitor titer during ITI≥200BU ④time to titer decline to<10BU before ITI≥24 months ⑤age≥8 years at start of ITI ⑥ITI initiated ≥5 years after inhibitor diagnosis ⑦interruptions in ITI≥2 weeks in duration. The low-dose ITI strategy consist of FⅧ（25-50IU/kg）alone or combining with immunosuppression: prednisone and Rituximab when the inhibitor titer ≥40BU ml/ml before or during ITI.
- Conditions
- Hemophilia A With Inhibitor
- Being tested
- Coagulation Factor VIII, Prednisone, Rituximab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Coagulation Factor VIIIDrug
Domastic plasma derived factor FVIII (with intermidiate vWF) 50IU/kg every other day
PrednisoneDrug
2mg/kg every day for 4 weeks then typering in 3 months
RituximabDrug
375mg/Square meter for consecutive 4 months
| Study group | Role | What they receive |
|---|---|---|
| ITI strategy | Experimental | The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically. Receives: Drug: Coagulation Factor VIII; Drug: Prednisone; Drug: Rituximab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Success rate
Success rate
Measured: 2 years
Secondary outcomes (3)
Annualized Bleeding Rate
How many times for all types of bleeding
Measured: 2 year
Annualized Joint Bleeding Rate
How many times for joint bleeding
Measured: 2 year
Success time
How long to success
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2018
- Study start1 Jan 2016
- Primary completion1 Dec 2019 (estimated)
- Study completion1 Dec 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia A With InhibitorStudies 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
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Runhui WU, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03598725
- Sponsor's protocol ID
- BCH-ITI-20180123
- Acronym
- HA-LD-ITI
- Status last verified
- Jul 2018
- First submitted
- 23 Jan 2018
- Last updated
- 26 Jul 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Li Z, Tang Y, Chen Z, Liu G, Yao W, Li G, Cheng X, Peng Y, Cai S, Cui C, Ai D, Zhang J, Poon MC, Zhang W, Wu R. Outcomes of immune tolerance induction with rituximab to eradicate high-titer inhibitor of hemophilia A: depicted by exponential decay model and the gene expression profile of different outcomes by RNA-sequencing. J Thromb Haemost. 2025 Aug;23(8):2436-2448. doi: 10.1016/j.jtha.2025.04.015. Epub 2025 Apr 24.
PubMed 40286913
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Beijing Children's Hospital, Capital Medical University
- ClinicalTrials.gov ID
- NCT03598725
- Acronym / sponsor ID
- HA-LD-ITI / BCH-ITI-20180123
- Conditions
- Hemophilia A With Inhibitor
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Beijing Children's HospitalUniversity, hospital or other
- Study start
- 1 Jan 2016 (actual)
- Primary completion
- 1 Dec 2019 (estimated)
- Study completion
- 1 Dec 2020 (estimated)
- First posted
- 26 Jul 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 study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression.
Detailed description
Poor risk(s) includes:①peak historical inhibitor titer≥200BU ②inhibitor titer≥10BU before ITI initiation ③peak inhibitor titer during ITI≥200BU ④time to titer decline to<10BU before ITI≥24 months ⑤age≥8 years at start of ITI ⑥ITI initiated ≥5 years after inhibitor diagnosis ⑦interruptions in ITI≥2 weeks in duration. The low-dose ITI strategy consist of FⅧ（25-50IU/kg）alone or combining with immunosuppression: prednisone and Rituximab when the inhibitor titer ≥40BU ml/ml before or during ITI.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ITI strategy | Experimental | Drug: Coagulation Factor VIII, Drug: Prednisone, Drug: RituximabThe low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically. |
Who may qualify
- Ages
- 1 year to 14 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Males
- from 1 to 14 years old
- severe or moderate hemophilia A;
- inhibitors positive before ITI started.
Key exclusion criteria (4 listed)
- Females
- <1 or >14 years old
- hemophilia B or mild haemophilia A;
- inhibitor negative before ITI started.
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 |
|---|---|---|
| Coagulation Factor VIIIDomastic plasma derived factor FVIII (with intermidiate vWF) 50IU/kg every other day | Drug | ITI strategy |
| Prednisone2mg/kg every day for 4 weeks then typering in 3 months | Drug | ITI strategy |
| Rituximab375mg/Square meter for consecutive 4 months | Drug | ITI strategy |
Primary outcome
Success rate
Time frame: 2 years
Success rate
3 secondary outcomes
Annualized Bleeding Rate
Time frame: 2 year
Annualized Joint Bleeding Rate
Time frame: 2 year
Success time
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2018.
- Derived from this study: PubMed 40286913Li Z, Tang Y, Chen Z, Liu G, Yao W, Li G, Cheng X, Peng Y, Cai S, Cui C, Ai D, Zhang J, Poon MC, Zhang W, Wu R. Outcomes of immune tolerance induction with rituximab to eradicate high-titer inhibitor of hemophilia A: depicted by exponential decay model and the gene expression profile of different outcomes by RNA-sequencing. J Thromb Haemost. 2025 Aug;23(8):2436-2448. doi: 10.1016/j.jtha.2025.04.015. Epub 2025 Apr 24.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Coagulation Factor VIII, Prednisone and Rituximab in people with Hemophilia A With Inhibitor, run by Beijing Children'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 4 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 26 Jul 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 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 Xin Ni, master (010-59616643, nixin@bch.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Success rate (time frame: 2 years). 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 NCT03598725, 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 26 Jul 2018.