NCT04985682

A Study of ADVATE in People With Hemophilia A in India

Completed Phase 4 Results posted Last updated 7 March 2024

What this trial tests

Phase 4 trial testing ADVATE in Hemophilia A in 50 participants. Completed in 10 February 2023.

Timeline

14 January 2022

Primary endpoint
10 February 2023

10 February 2023

Quick facts

Lead sponsor	Baxalta now part of Shire
Phase	Phase 4
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	50
Start date	14 January 2022
Primary completion	10 February 2023
Estimated completion	10 February 2023
Sites	5 locations across India

Drugs / interventions tested

• ADVATE (OCTOCOG ALFA) — full drug profile →

Conditions studied

• Hemophilia A — all drugs for Hemophilia A →

Sponsor

Baxalta now part of Shire — full company profile →

Who can join

Eligibility, any sex, with Hemophilia A. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE Primary · Baseline (Day 0) up to end of study (up to 12.9 months)

An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. An SAE was defined as any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of present hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect or was a medically important event. Number of participants with SAEs (including FVIII

Group	Value	95% CI
Hemophilia A Group	0

Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

An AE was any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this investigational product or medicinal product. Number of participants with non-serious AEs that were at least possibly related to ADVATE were reported.

Group	Value	95% CI
Hemophilia A Group	0

Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

Clinical laboratory parameters included hematology, clinical chemistry, viral serology, factor VIII (FVIII) antigen, FVIII activity, incremental recovery, and FVIII inhibitor. Clinical significance was judged as per Investigator's assessment.

Group	Value	95% CI
Hemophilia A Group	0

Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error was estimated using a generalized linear model (GLM). The total ABR is reported in this outcome measure.

Group	Value	95% CI
Hemophilia A Group	2.65	± 0.195

ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed sites (example, joint, soft tissue, muscle, other \[mouth, gums or nose\] are reported in this outcome measure.

Bleed Location: Joint

Group	Value	95% CI
Hemophilia A Group	2.33	± 0.203

Bleed Location: Soft Tissue

Group	Value	95% CI
Hemophilia A Group	0.04	± 1.000

Bleed Location: Muscle

Group	Value	95% CI
Hemophilia A Group	0.16	± 0.628

Bleed Location: Other (Mouth, Gums or Nose)

Group	Value	95% CI
Hemophilia A Group	0.12	± 0.813

ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

ABR was defined as number of bleeding episodes during the study period divided by total number of study period days multiplied by 365.25. The mean ABR and standard error were estimated using a GLM. The ABR by bleed cause (example, spontaneous, injury, and unknown) are reported in this outcome measure.

Bleed Type: Spontaneous

Group	Value	95% CI
Hemophilia A Group	1.86	± 0.211

Bleed Type: Injury

Group	Value	95% CI
Hemophilia A Group	0.20	± 0.692

Bleed Type: Unknown

Group	Value	95% CI
Hemophilia A Group	0.59	± 0.541

Total Number of ADVATE Infusions Required During Prophylactic Treatment Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

Group	Value	95% CI
Haemophilia A Group	65.8	± 22.92

Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

Group	Value	95% CI
Hemophilia A Group	2.5	± 0.87

Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

Group	Value	95% CI
Hemophilia A Group	11.0	± 3.76

Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

Body mass adjusted consumption international units per kilograms (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.

Group	Value	95% CI
Hemophilia A Group	1739.7	± 621.18

Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.

Group	Value	95% CI
Hemophilia A Group	67.1	± 24.12

Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment Secondary · Baseline (Day 0) up to end of study (up to 12.9 months)

Body mass adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion.

Group	Value	95% CI
Hemophilia A Group	291.7	± 104.80

Adverse events — posted to ClinicalTrials.gov

Time frame: Baseline (Day 0) up to end of study (up to 12.9 months). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Hemophilia A Group

Serious: 0/50 (0%)

Deaths: 0/50

Other adverse events (5 terms — click to expand)

Reaction	System	Hemophilia A Group
Pyrexia	General disorders	—
Skin laceration	Injury, poisoning and procedural complications	—
Varicella	Infections and infestations	—
Animal bite	Injury, poisoning and procedural complications	—
Joint injury	Injury, poisoning and procedural complications	—

Data from ClinicalTrials.gov NCT04985682 adverse events section.

Sponsor's own description

The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.

The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.