Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A

NCT ID: NCT06155955

Last Updated: 2024-03-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-22

Study Completion Date

2024-06-21

Brief Summary

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* To outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration
* To study pharmacokinetic, side effect of low dose Emicizumab

Detailed Description

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* To compare outcome between low dose Emicizumab and low dose prophylaxis with FVIII concentration using ABR, AJBR and HJHS, HaemoQoL
* To study pharmacokinetic, side effect and effectiveness of low dose Emicizumab

Conditions

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Severe Hemophilia A Without Inhibitor Joint Bleed

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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low dose emicizumab

2 mg per kilograms per dose in first month, loading every 2 weeks then every 4 weeks

Group Type EXPERIMENTAL

"Emicizumab", "HEMLIBRA®"

Intervention Type DRUG

low dose

low dose factor VIII prophylaxis

factor VIII prophylaxis

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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"Emicizumab", "HEMLIBRA®"

low dose

Intervention Type DRUG

Other Intervention Names

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emicizumab-kxwh

Eligibility Criteria

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Inclusion Criteria

* Severe or moderate Hemophilia A with baseline FVIII:C of \<3 IU/dl or severe bleeding phenotype
* Receiving low dose FVIII prophylaxis for at least 6 months

Exclusion Criteria

* Detectable FVIII inhibitor at screening
* Detectable FVIII inhibitor at screening Having other underlying diseases: Juvenile rheumatoid arthritis, metabolic bone diseases, or other conditions mimicking or causing joint diseases
Minimum Eligible Age

3 Years

Maximum Eligible Age

30 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

Yes

Sponsors

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Chulalongkorn University

OTHER

Sponsor Role lead

Responsible Party

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Nuchanun Kessakorn, MD

MD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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NUCHANUN KESSAKORN

Role: PRINCIPAL_INVESTIGATOR

King Chulalongkorn Memorial Hospital

Locations

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King Chulalongkorn Memorial hospital

Bangkok, , Thailand

Site Status RECRUITING

Countries

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Thailand

Central Contacts

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NUCHANUN KESSAKORN, MD

Role: CONTACT

662-5054639

Facility Contacts

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NUCHANUN KESSAKORN, MD

Role: primary

66825054639

Darintr Sosothikul, Diplomate

Role: backup

References

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Kessakorn N, Gosriwatana I, Sasipong N, Srichumpuang C, Moonla C, Sosothikul D. Low-Dose Emicizumab Versus Low-/Intermediate-Dose Factor VIII Secondary Prophylaxis for Noninhibitor Haemophilia A Patients With Severe Bleeding Phenotype. Haemophilia. 2025 Jan;31(1):122-131. doi: 10.1111/hae.15146. Epub 2024 Dec 31.

Reference Type DERIVED
PMID: 39737816 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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KingChulalongkorn

Identifier Type: -

Identifier Source: org_study_id

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