Who is sponsoring NCT01736475?

NCT01736475 is sponsored by Baxalta now part of Shire.

What drugs are being tested in NCT01736475?

Interventions in NCT01736475: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII, PEGylated Recombinant Factor VIII, PEGylated Recombinant Factor VIII.

What condition does NCT01736475 study?

NCT01736475 studies Hemophilia A.

Is NCT01736475 still recruiting?

NCT01736475 is currently completed. Last updated 20 May 2021.

Are results published for NCT01736475?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2021-05-20 · How we verify

NCT01736475

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

Completed Phase 2, PHASE3 Results posted Last updated 20 May 2021

What this trial tests

Phase 2, PHASE3 trial testing Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method in Hemophilia A in 159 participants. Completed in 17 July 2014.

Timeline

31 January 2013

Primary endpoint
17 July 2014

17 July 2014

Quick facts

Lead sponsor	Baxalta now part of Shire
Phase	Phase 2, PHASE3
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	prevention
Enrollment	159
Start date	31 January 2013
Primary completion	17 July 2014
Estimated completion	17 July 2014
Sites	72 locations across Japan, Malaysia, Taiwan, Poland, South Korea, Netherlands, Sweden, Lithuania

Drugs / interventions tested

Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method — full drug profile →
PEGylated Recombinant Factor VIII — full drug profile →
PEGylated Recombinant Factor VIII — full drug profile →
PEGylated Recombinant Factor VIII — full drug profile →

Conditions studied

Hemophilia A — all drugs for Hemophilia A →

Sponsor

Baxalta now part of Shire — full company profile →

Who can join

Adults 12 to 65, male only, 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.

Annualized Bleeding Rate (ABR) Primary · 9 months

Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy.

Group	Value	95% CI
Prophylaxis	4.3	3.4 – 5.5
On-demand	43.4	25.2 – 74.8

Rate of Success of BAX 855 for Treatment of Bleeding Episodes Secondary · At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm.

Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. EXCELLENT: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. GOOD: Definite pain reli

Group	Value	95% CI
Participants With a Bleeding Episode	0.96	0.91 – 0.98

Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes Secondary · From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

Group	Value	95% CI
Prophylaxis	1.37	± 0.80
On-demand	1.21	± 0.35

Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes Secondary · From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)\*(12/365.2425)

No bleed

Group	Value	95% CI
Prophylaxis	45
On-demand	0

>6 Months

Group	Value	95% CI
Prophylaxis	5
On-demand	0

6 Months

Group	Value	95% CI
Prophylaxis	20
On-demand	0

5 Months

Group	Value	95% CI
Prophylaxis	3
On-demand	0

4 Months

Group	Value	95% CI
Prophylaxis	0
On-demand	0

3 Months

Group	Value	95% CI
Prophylaxis	11
On-demand	0

2 Months

Group	Value	95% CI
Prophylaxis	16
On-demand	0

≤1 Month

Group	Value	95% CI
Prophylaxis	20
On-demand	17

Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion Secondary · Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96h

Per Prophylactic Infusion (N= 5941 Infusions)

Group	Value	95% CI
All Study Participants	44.51	± 4.556

Per PK Infusion (N= 50 Infusions)

Group	Value	95% CI
All Study Participants	45.48	± 2.592

Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis Secondary · Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution.

Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.

Per Treatment of BE (N= 592 bleeds)

Group	Value	95% CI
All Study Participants	37.44	± 28.105

Per BE for Maintenance of Hemostasis (N=34 bleeds)

Group	Value	95% CI
All Study Participants	39.29	± 34.206

Percentage of Participants With Adverse Events Secondary · From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

Adverse Events (AEs) and Serious Adverse Events (SAEs)

SAE, Moderate, Unrelated

Group	Value	95% CI
All Study Participants	0.7

SAE, Severe, Unrelated

Group	Value	95% CI
All Study Participants	2.9

nSAE, Mild, Unrelated

Group	Value	95% CI
All Study Participants	40.1

nSAE, Mild, Related

Group	Value	95% CI
All Study Participants	3.6

nSAE, Moderate, Unrelated

Group	Value	95% CI
All Study Participants	19.0

nSAE, Moderate, Related

Group	Value	95% CI
All Study Participants	1.5

nSAE, Unknown Severity, Unrelated

Group	Value	95% CI
All Study Participants	0.7

nSAE, Severe, Unrelated

Group	Value	95% CI
All Study Participants	1.5

Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination Secondary · From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].

Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary

Inhibitory Antibodies to FVIII (N= 112, 14)

Group	Value	95% CI
Prophylaxis	0
On-demand	0

IgG: Binding Antibodies FVIII (N= 117, 15)

Group	Value	95% CI
Prophylaxis	0
On-demand	0

IgM: Binding Antibodies FVIII (N= 117, 15)

Group	Value	95% CI
Prophylaxis	0
On-demand	0

IgG: Binding Antibodies PEG (N= 117, 15)

Group	Value	95% CI
Prophylaxis	0
On-demand	0

IgM: Binding Antibodies PEG (N= 117, 15)

Group	Value	95% CI
Prophylaxis	0
On-demand	0

IgG: Binding Antibodies PEG-FVIII (N= 117, 15)

Group	Value	95% CI
Prophylaxis	0
On-demand	1

IgM: Binding Antibodies PEG-FVIII (N= 117, 15)

Group	Value	95% CI
Prophylaxis	0
On-demand	0

CHO-Protein Antibodies (N= 117, 15)

Group	Value	95% CI
Prophylaxis	0
On-demand	0

Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study Secondary · Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm].

The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms.

Bleed Severity Total Score

Group	Value	95% CI
Prophylaxis	-4.17	± 17.05
On-demand	-4.24	± 15.71

Pain Severity Total Score

Group	Value	95% CI
Prophylaxis	-1.22	± 12.50
On-demand	-0.17	± 11.88

Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study Secondary · Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm]

Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL.

Physical Functioning (N= 97, 12)

Group	Value	95% CI
Prophylaxis	0.49	± 5.27
On-demand	-2.46	± 4.29

Role-physical (N= 97, 12)

Group	Value	95% CI
Prophylaxis	1.31	± 7.36
On-demand	-3.67	± 9.01

Bodily Pain (N= 97, 12)

Group	Value	95% CI
Prophylaxis	2.08	± 8.19
On-demand	0.60	± 4.44

General Health (N= 96, 12)

Group	Value	95% CI
Prophylaxis	0.40	± 6.43
On-demand	-0.28	± 9.04

Vitality (N= 96, 12)

Group	Value	95% CI
Prophylaxis	-0.38	± 7.43
On-demand	0.26	± 9.36

Social Functioning (N= 97, 12)

Group	Value	95% CI
Prophylaxis	0.90	± 7.54
On-demand	-3.18	± 6.35

Role Emotional (N= 97, 12)

Group	Value	95% CI
Prophylaxis	-0.20	± 8.46
On-demand	0.65	± 7.92

Mental Health (N= 96, 12)

Group	Value	95% CI
Prophylaxis	0.09	± 7.26
On-demand	-3.29	± 7.95

Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay) Secondary · Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

Terminal half-life calculated as log\_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).

PK-1: ADVATE

Group	Value	95% CI
Pharmacokinetic Analysis Participants	10.40	± 2.244

PK-2: BAX 855

Group	Value	95% CI
Pharmacokinetic Analysis Participants	14.30	± 3.838

PK-3: BAX 855, After ≥50 Exposure Days (N=22)

Group	Value	95% CI
Pharmacokinetic Analysis Participants	16.02	± 4.922

Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay) Secondary · Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).

The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysi

PK-1: ADVATE

Group	Value	95% CI
Pharmacokinetic Analysis Participants	12.86	± 3.044

PK-2: BAX 855

Group	Value	95% CI
Pharmacokinetic Analysis Participants	19.56	± 5.315

PK-3: BAX 855, After ≥50 Exposure Days (N=22)

Group	Value	95% CI
Pharmacokinetic Analysis Participants	20.65	± 4.821

Adverse events — posted to ClinicalTrials.gov

Time frame: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

All Study Participants

Serious: 5/137 (4%)

Deaths: —

Serious adverse events (5 terms)

Reaction	System	All Study Participants
HERPES ZOSTER INFECTION NEUROLOGICAL	Infections and infestations	—
HUMERUS FRACTURE	Injury, poisoning and procedural complications	—
MUSCLE HAEMORRHAGE	Musculoskeletal and connective tissue disorders	—
OSTEOARTHRITIS	Musculoskeletal and connective tissue disorders	—
NEUROENDOCRINE CARCINOMA	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—

Other adverse events (3 terms — click to expand)

Reaction	System	All Study Participants
Nasopharyngitis	Infections and infestations	—
Upper respiratory tract infection	Infections and infestations	—
Headache	Nervous system disorders	—

Most-reported serious reactions: HERPES ZOSTER INFECTION NEUROLOGICAL, HUMERUS FRACTURE, MUSCLE HAEMORRHAGE, OSTEOARTHRITIS, NEUROENDOCRINE CARCINOMA.

Data from ClinicalTrials.gov NCT01736475 adverse events section.

Sponsor's own description

To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A.
Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, et al · · 2015 · cited 192× · PMID 26157075 · DOI 10.1182/blood-2015-03-630897
Outcome of Clinical Trials with New Extended Half-Life FVIII/IX Concentrates.
Mancuso ME, Santagostino E. · · 2017 · cited 72× · PMID 28350322 · DOI 10.3390/jcm6040039
Rurioctocog alfa pegol PK-guided prophylaxis in hemophilia A: results from the phase 3 PROPEL study.
Klamroth R, Windyga J, Radulescu V, Collins PW, et al · · 2021 · cited 61× · PMID 33150384 · DOI 10.1182/blood.2020005673
Safety of PEGylated recombinant human full-length coagulation factor VIII (BAX 855) in the overall context of PEG and PEG conjugates.
Stidl R, Fuchs S, Bossard M, Siekmann J, et al · · 2016 · cited 48× · PMID 26219204 · DOI 10.1111/hae.12762
Potential role of a new PEGylated recombinant factor VIII for hemophilia A.
Wynn TT, Gumuscu B. · · 2016 · cited 17× · PMID 27382347 · DOI 10.2147/jbm.s82457
Clinical studies of extended-half-life recombinant FVIII products for prophylaxis in adults and children: A critical review from the physician's perspective.
Hermans C, Reding MT, Astermark J, Klamroth R, et al · · 2022 · cited 13× · PMID 35390452 · DOI 10.1016/j.critrevonc.2022.103678
A Retrospective Observational Study of Rurioctocog Alfa Pegol in Clinical Practice in the United States.
Aledort L, Milligan S, Watt M, Booth J. · · 2020 · cited 10× · PMID 32223610 · DOI 10.18553/jmcp.2020.26.4.492
Efficacy, safety, and immunogenicity of rurioctocog alfa pegol for prophylactic treatment in previously treated patients with severe hemophilia A: a systematic review and meta-analysis of clinical trials.
Witarto BS, Visuddho V, Witarto AP, Sutanto H, et al · · 2021 · cited 5× · PMID 35136579 · DOI 10.12688/f1000research.73884.3

Verify or expand the search:

Other trials of Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Trials testing the same drug.

NCT02210091 — BAX 855 Pediatric Study · Phase 3 · completed
NCT01599819 — BAX 855 Dose-Escalation Safety Study · Phase 1 · completed

Other recruiting trials for Hemophilia A

Currently open trials in the same condition.

NCT07416526 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A · Phase 3 · recruiting
NCT07416604 — A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A · Phase 3 · recruiting
NCT07523399 — Joint Health, Balance and Quality of Life in Adults With Hemophilia A · recruiting
NCT06833983 — To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A · Phase 3 · recruiting
NCT06579144 — Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A · Phase 1 · recruiting

Other Baxalta now part of Shire trials

Trials by the same sponsor.

NCT04985682 — A Study of ADVATE in People With Hemophilia A in India · Phase 4 · completed
NCT04578535 — A Study to Assess the Tolerability, Safety, and Pharmacokinetics of Subcutaneous Immune Globulin Infusion 10% (Human) Wi · Phase 1 · completed
NCT04346108 — A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunode · Phase 3 · completed
NCT04158934 — A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A · active not recruiting
NCT04394286 — A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects · Phase 1, PHASE2 · terminated

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT01736475 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Baxalta now part of Shire
Last refreshed: 20 May 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01736475.

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