HemophiliaPhase 4NCT04108260
The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients
A Phase 4 study testing Albutrepenonacog Alfa 1 UNT [IDELVION] in people with Hemophilia, run by National Taiwan University Hospital at 1 site in Taiwan.
Trial summary
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median annualized spontaneous bleeding rate (AsBR) of 0.00. Read more about this study
- Who is it for?
- Ages 20 years and older, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- Albutrepenonacog Alfa 1 UNT [IDELVION]
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteTaiwan
- Study dates
- 21 Apr 2020to Apr 2022
- 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 20 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male patients with age ≥ 20 years old
- Endogenous FIX activity ≤1 IU/dL,
- At least 50 exposure days (EDs) with FIX products,
- No detectable inhibitor to FIX or inhibitor history,
- Had a minimum of 2 nontrauma-induced bleeding episodes (any type or location) treated in the 6 months preceding study entry,
- The patient or patient's parents or legal authorized representative, as applicable, are capable to understand the study objectives and procedure, and sign the written consent,
- Accept that the supply of Idelvion might be stopped once the study is completed,
- Able to complete a diary during 12 months or 50 EDs, whichever comes first.
Who cannot join
- Currently participating in an interventional clinical trial,
- Known hypersensitivity to any FIX product or hamster protein,
- Known inhibitor to FIX or inhibitor history,
- With other comorbidities which are not suitable for this study, at investigator's discretion,
- Not able to compliant with the prophylactic treatment,
- Lacking previous treatment and bleeding records.
Trial locations and contacts
1 site in 1 country.
Taiwan1 site, 1 recruiting
- Recruiting
National Taiwan University Hospital
Taipei
SHENG-CHIEH CHOU, MD/PhD, 886223123456 ext. 70173, potatoaureus@gmail.com
About this study
Official title: The Effectiveness of Recombinant Fusion Protein Linking Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients Switching From Previous Factor IX Treatment
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median annualized spontaneous bleeding rate (AsBR) of 0.00. The majority (99%) of bleeding episodes were managed successfully with one or two infusions, and 94% of bleeds were controlled with only one infusion regardless of the cause or location.
The rationale for this study is to collect the effectiveness and safety data of severe hemophilia B patients treated with Idelvion following institutional standard of care. It is the aim of this study to extend the results of the clinical trial program to a broader hemophilia B population, and to compare with current alternative factor IX treatments in Taiwan.
- Conditions
- Hemophilia
- Being tested
- Albutrepenonacog Alfa 1 UNT [IDELVION]
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Albutrepenonacog Alfa 1 UNT [IDELVION]Drug
Clinical data will be collected from patients who suffered from hemophilia B, and switch from other FIX products to Idelvion as prophylaxis, prevention of bleeding (e.g. surgery) or treatment of bleeding.
| Study group | Role | What they receive |
|---|---|---|
| Single arm_Idelvion treated | Experimental | Not described Receives: Drug: Albutrepenonacog Alfa 1 UNT [IDELVION] |
What is being measured
The primary outcome is the main question the study is designed to answer.
AsBR
To evaluate AsBR of Idelvion in routine patient care
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2019
- Study start21 Apr 2020
- Primary completionNov 2021 (estimated)
- Study completionApr 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies of the same condition, open and under way.
- More trials run by National Taiwan University 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Taiwan University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04108260
- Sponsor's protocol ID
- 201907004MIPB
- Status last verified
- Oct 2020
- First submitted
- 26 Sep 2019
- Last updated
- 30 Oct 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effectiveness of Recombinant Fusion Protein Linking Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients Switching From Previous Factor IX Treatment
- ClinicalTrials.gov ID
- NCT04108260
- Acronym / sponsor ID
- 201907004MIPB
- Conditions
- Hemophilia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- National Taiwan University HospitalUniversity, hospital or other
- Study start
- 21 Apr 2020 (actual)
- Primary completion
- Nov 2021 (estimated)
- Study completion
- Apr 2022 (estimated)
- First posted
- 30 Sep 2019 (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 pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median annualized spontaneous bleeding rate (AsBR) of 0.00. The majority (99%) of bleeding episodes were managed successfully with one or two infusions, and 94% of bleeds were controlled with only one infusion regardless of the cause or location.
The rationale for this study is to collect the effectiveness and safety data of severe hemophilia B patients treated with Idelvion following institutional standard of care. It is the aim of this study to extend the results of the clinical trial program to a broader hemophilia B population, and to compare with current alternative factor IX treatments in Taiwan.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm_Idelvion treated | Experimental | Drug: Albutrepenonacog Alfa 1 UNT [IDELVION] |
Who may qualify
- Ages
- 20 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male patients with age ≥ 20 years old
- Endogenous FIX activity ≤1 IU/dL,
- At least 50 exposure days (EDs) with FIX products,
- No detectable inhibitor to FIX or inhibitor history,
- Had a minimum of 2 nontrauma-induced bleeding episodes (any type or location) treated in the 6 months preceding study entry,
Key exclusion criteria (6 listed)
- Currently participating in an interventional clinical trial,
- Known hypersensitivity to any FIX product or hamster protein,
- Known inhibitor to FIX or inhibitor history,
- With other comorbidities which are not suitable for this study, at investigator's discretion,
- Not able to compliant with the prophylactic treatment,
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 |
|---|---|---|
| Albutrepenonacog Alfa 1 UNT [IDELVION]Clinical data will be collected from patients who suffered from hemophilia B, and switch from other FIX products to Idelvion as prophylaxis, prevention of bleeding (e.g. surgery) or treatment of bleeding. | Drug | Single arm_Idelvion treated |
Primary outcome
AsBR
Time frame: 12 months
To evaluate AsBR of Idelvion in routine patient care
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2020.
- 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 Albutrepenonacog Alfa 1 UNT [IDELVION] in people with Hemophilia, run by National Taiwan University Hospital at 1 site in Taiwan.
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 8 inclusion and 6 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 30 Oct 2020. 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 Taiwan. 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 SHENG-CHIEH DR. CHOU, MD/PHD (886223123456 ext. 70173, potatoaureus@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
AsBR (time frame: 12 months). 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 30 Oct 2020.