Hemophilia BPhase 4NCT04286412
Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B
A Phase 4 study testing Nonacog alfa in people with Hemophilia B, run by Pfizer at 5 sites in India.
Trial summary
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control… Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- Nonacog alfa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 5 sitesIndia
- Study dates
- 10 Feb 2020to 24 Sep 2020
- 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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male subjects ≥12 years to ≤65 years with a diagnosis of congenital moderately-severe to severe hemophilia B (FIX activity ≤2%).
- Documented history of at least 50 exposure days (EDs) to FIX-containing products.
- Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study. For minors under the age of legal consent in India, assent of the participating child needs to be documented for the age range 12 to 18 in addition to the parental informed consent.
Who cannot join
- Prior history of inhibitor to FIX or positive inhibitor testing (≥0.6 BU/mL) during Screening. Clinical signs or symptoms of decreased response to FIX.
- Known hypersensitivity to the active substance or any of the excipients.
- Known allergic reaction to hamster proteins.
- Presence of any bleeding disorder in addition to hemophilia B.
- Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation.
- Planned surgery within 6 months from the start of the study.
Show all 11Show fewer
- Unsuitable to participate in study for any other reason as assessed by the investigator; including any disorder, except for conditions associated with hemophilia B, which in the investigator's opinion might jeopardize subject's safety or compliance with the protocol.
- Subjects (or a legally acceptable representative) is not able to understand study documents and study procedure.
- Immunocompromised subjects due to human immunodeficiency virus (HIV) infection (defined as viral load above or equal to 100,000 copies/mL; and for HIV+ subjects: cluster of differentiation 4 positive (CD4+) lymphocyte count below or equal to 200/μL). HIV status and CD4+ lymphocyte count results may be obtained at screening or from available medical records; results must be not older than 6 months prior to screening.
- Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, subjects who have been previously enrolled into the study, or subjects who are Pfizer employees directly involved in the conduct of the study.
- Planned use of any non-study medication for treatment of hemophilia (eg, other factor replacement agents, bypassing agents, or non-factor treatments [such as anti-tissue factor pathway inhibitors]).
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
India5 sites
Christian Medical College and Hospital
Ludhiana, Punjab, 141008
K.J. Somaiya Hospital and Research Centre
Mumbai, Maharashtra, 400022
Sahyadri Clinical Research & Development Centre
Pune, Maharashtra, 411004
Sahyadri Super Specialty Hospital
Pune, 411004
Nirmal Hospital Pvt Ltd.
Surat, Gujarat, 395002
About this study
Official title: A SINGLE COUNTRY, MULTICENTER, OPEN-LABEL AND NON-RANDOMIZED CLINICAL TRIAL WITH NONACOG ALFA PROPHYLAXIS AND TREATMENT OF BLEEDING EPISODES IN PREVIOUSLY TREATED PATIENTS WITH MODERATELY-SEVERE TO SEVERE HEMOPHILIA B FOR A DURATION OF 8 WEEKS.
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.
- Conditions
- Hemophilia B
- Being tested
- Nonacog alfa
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nonacog alfaBiological
Also called BeneFIX
Nonacog alfa is indicated in India for the treatment and prophylaxis of bleeding in patients with hemophilia B (congenital factor IX deficiency).
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | At least twenty five eligible male subjects will be enrolled in the treatment arm to receive Nonacog alfa until 16 exposure days (EDs) or a period of up to 8 weeks on treatment had occurred (whichever occurs first). Receives: Biological: Nonacog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Developed Factor IX (FIX) Inhibitors
FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study.
Measured: At Visit 4 (any 1 day between Day 52 to Day 60)
Secondary outcomes (6)
Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs)
An adverse events (AE) is any untoward medical occurrence in clinical investigation participant administered a product or medical device; event need not necessarily to have a causal relationship with treatment or usage. SAEs: an AE resulting in any of following outcomes/deemed significant for any other reason: death; initial/prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. MIEs: any confirmed FIX inhibitor development (titer greater than or equal to 0.6 BU/mL confirmed by central laboratory testing), thrombotic events (any event associated with the formation of a blood clot including catheter-associated thrombi and thrombotic complications) or hypersensitivity reactions (hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgment).
Measured: Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. TEAEs are events between first dose of study drug and up to 116 days that were absent before treatment or that worsened relative to pretreatment state.
Measured: Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)
Mean Annualized Bleeding Rate (ABR)
ABR: number of bleeding episodes per year. ABR for each participant = number of bleeds during treatment interval duration (TID)/(TID/365.25). Number of bleeds for each participant for ABR included all new bleeds (with unique start date and time) requiring treatment with nonacog alfa during TID. TID=date of Visit 4 (3 to 10 days after last dose) - date of Visit 2 (Day 1) +1. For participants who had Visit 4 beyond 3 (+7) days after final dose due to COVID-19 pandemic, date of final dose was used in place of date of Visit 4. For discontinued participants, date of final dose/last study visit date (whichever occurred later) was used to replace Visit 4.
Measured: Up to 88 Days
Mean Annualized Total Factor Consumption (TFC) Per Participant
The TFC per participant was the sum of the total amount (in international units [IU]) infused for each infusion for each participant. Annualized TFC of nonacog alfa was derived for each participant by using the following formula; Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as the number of days beginning on Visit 2 ("Day 1", provided an infusion was given) up to Visit 4 (any 1 day between Day 52 to Day 60).
Measured: Up to 88 Days
Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant
The total amount in international units (IU) per kilogram infused for each infusion recorded were summed to calculate the total factor consumption for each participant. Annualized TFC = (TFC / treatment interval duration)*365.25. Treatment interval duration was calculated as Date of Visit 4 - Date of Visit 2 +1. If Date of Visit 4 - Last dosing date was > 10 days then Date of Visit 4 was replaced with Last dosing date. If Date of Visit 4 was missing (due to discontinuation or any other reason), then Date of Visit 4 was replaced with last dosing date or last subject visit date, whichever was greater. International unit per kilogram= IU/kg.
Measured: Up to 88 days
Mean Number of Nonacog Alfa Infusions Used to Treat Each Bleed
Number of nonacog alfa infusions used to treat each bleed in participants was calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). On demand treatment was defined as treatment used to treat a bleeding episode.
Measured: Up to 88 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2020
- Study start10 Feb 2020
- Primary completion24 Sep 2020
- Study completion24 Sep 2020
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.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by PfizerThe 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
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04286412
- Sponsor's protocol ID
- B1821059
- Other IDs
- 2020-004430-38
- Status last verified
- Jun 2022
- First submitted
- 12 Feb 2020
- Last updated
- 1 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A SINGLE COUNTRY, MULTICENTER, OPEN-LABEL AND NON-RANDOMIZED CLINICAL TRIAL WITH NONACOG ALFA PROPHYLAXIS AND TREATMENT OF BLEEDING EPISODES IN PREVIOUSLY TREATED PATIENTS WITH MODERATELY-SEVERE TO SEVERE HEMOPHILIA B FOR A DURATION OF 8 WEEKS.
- ClinicalTrials.gov ID
- NCT04286412
- Acronym / sponsor ID
- B1821059
- Conditions
- Hemophilia B
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 10 Feb 2020 (actual)
- Primary completion
- 24 Sep 2020 (actual)
- Study completion
- 24 Sep 2020 (actual)
- First posted
- 27 Feb 2020 (actual)
- Results
- Posted 30 Jul 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Biological: Nonacog alfaAt least twenty five eligible male subjects will be enrolled in the treatment arm to receive Nonacog alfa until 16 exposure days (EDs) or a period of up to 8 weeks on treatment had occurred (whichever occurs first). |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Male subjects ≥12 years to ≤65 years with a diagnosis of congenital moderately-severe to severe hemophilia B (FIX activity ≤2%).
- Documented history of at least 50 exposure days (EDs) to FIX-containing products.
- Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative, parent(s)/legal guardian) has been informed of all pertinent aspects of the study. For minors under the age of legal consent in India, assent of the participating child needs to be documented for the age range 12 to 18 in addition to the parental informed consent.
Key exclusion criteria (11 listed)
- Prior history of inhibitor to FIX or positive inhibitor testing (≥0.6 BU/mL) during Screening. Clinical signs or symptoms of decreased response to FIX.
- Known hypersensitivity to the active substance or any of the excipients.
- Known allergic reaction to hamster proteins.
- Presence of any bleeding disorder in addition to hemophilia B.
- Participation in other studies involving investigational drug(s) (Phases 1-4) within 30 days before the current study begins and/or during study participation.
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 |
|---|---|---|
| Nonacog alfaAlso: BeneFIXNonacog alfa is indicated in India for the treatment and prophylaxis of bleeding in patients with hemophilia B (congenital factor IX deficiency). | Biological | Treatment Arm |
Primary outcome
Percentage of Participants Who Developed Factor IX (FIX) Inhibitors
Time frame: At Visit 4 (any 1 day between Day 52 to Day 60)
FIX inhibitor development was defined as an inhibitor titer >= 0.6 Bethesda units per milliliter (BU/mL) confirmed by central laboratory testing during the course of the study.
6 secondary outcomes
Number of Participants With Serious Adverse Events (SAEs) and Medically Important Events (MIEs)
Time frame: Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline up to 28 days after last dose of study drug (i.e, up to 116 days)
Mean Annualized Bleeding Rate (ABR)
Time frame: Up to 88 Days
Mean Annualized Total Factor Consumption (TFC) Per Participant
Time frame: Up to 88 Days
Mean Annualized Total Factor Consumption (TFC) by Weight Per Participant
Time frame: Up to 88 days
Mean Number of Nonacog Alfa Infusions Used to Treat Each Bleed
Time frame: Up to 88 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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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.
Pfizer CT.gov Call Center study director
Pfizer
5 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 Nonacog alfa in people with Hemophilia B, run by Pfizer at 5 sites in India.
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 3 inclusion and 11 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 1 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in India. 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 30 Jul 2021). 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?
Percentage of Participants Who Developed Factor IX (FIX) Inhibitors (time frame: At Visit 4 (any 1 day between Day 52 to Day 60)). 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 1 Jul 2022; AzTrials last refreshed its copy on 8 Nov 2026.