A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A

NCT04158934 · tracked via the Priya Life Science USA tracker

Sponsor

Baxalta now part of Shire

Phase

Observational

Started

2020-07-09

Last updated

2026-02-18

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

ADYNOVI/ADYNOVATE

ADYNOVI/ADYNOVATE: Participants will receive ADYNOVI/ADYNOVATE prescribed prophylactically by physicians based on their standard clinical practice and in accordance with the national SmPC.

Study summary

The main aim of this study is to check for long-term side effects from ADYNOVI/ADYNOVATE prophylaxis in participants with haemophilia A when used under standard clinical practice in the real-world clinical setting.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

Inclusion Criteria * Signed informed consent obtained from participant and/or legally authorised representative before any study related activities (any procedure related to recording of data according to the protocol). * Participant at any age with haemophilia A prescribed ADYNOVI/ADYNOVATE prophylaxis. * Negative factor VIII (FVIII) inhibitor test at study entry. * Decision to initiate treatment with commercially available ADYNOVI/ADYNOVATE has been made by the participant and/or legally authorised representative and the treating physician before and independently from the decision to include the participant in this study. Exclusion Criteria * Previous participation in this study. Participation is defined as signed informed consent. * Known or suspected hypersensitivity to ADYNOVI/ADYNOVATE or related products. * Mental incapacity, unwillingness or other barriers precluding adequate understanding or cooperation.

Primary outcome measure(s)

Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE) — Throughout the study period (approximately up to 10 years)
An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event. An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs and SAEs that are at least possibly related to study drug ADYNOVI/ADYNOVATE will be evaluated in this outcome.
Number of Participants With Adverse Events of Special Interest (AESI) — Throughout the study period (approximately up to 10 years)
Adverse events of special interest are as follows: thromboembolic events, hypersensitivity reactions, lack of efficacy and confirmed FVIII inhibitor development.
Number of Participants With Adverse Events (AE) Related to Impaired Renal Function — Throughout the study period (approximately up to 10 years)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to long-term effects of PEG accumulation impaired renal function will be evaluated in this outcome.
Number of Participants With Adverse Events (AE) Related to Impaired Hepatic Function — Throughout the study period (approximately up to 10 years)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to long-term effects of PEG accumulation impaired hepatic function will be evaluated in this outcome.
Number of Participants With Adverse Events (AE) Related to Impaired Neurologic Function — Throughout the study period (approximately up to 10 years)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. AEs (at least possibly related) that are potentially indicative of or related to long-term effects of PEG accumulation impaired neurologic function will be evaluated in this outcome.

Trial sites (36)

Facility	City	Region
UF Health Shands Hospital	Gainesville	Florida
SHAT of Oncohaematology Diseases	Sofia	Bulgaria
Clinical Hospital Sveti Duh	Zagreb	Croatia
University hospital centre Zagreb	Zagreb	Croatia
University hospital centre Zagreb	Zagreb	Croatia
Fakultni nemocnice v Motole	Prague	Czechia
Krajska zdravotni a.s. - Masarykova nemocnice v Usti nad Labem o.z.	Ústí nad Labem	Czechia
SRH Kurpfalzkrankenhaus Heidelberg GmbH	Heidelberg	Baden-Wurttemberg
Vivantes Klinikum im Friedrichshain	Berlin	Germany
Universitaetsklinikum Bonn AoeR	Bonn	Germany
Werlhof-Institut GmbH	Hanover	Germany
Heim Pal Orszagos Gyermekgyogyaszati Intezet	Budapest	Hungary
Eszak-Pesti Centrumkorhaz - Honvedkorhaz	Budapest	Hungary
Debreceni Egyetem	Debrecen	Hungary
Mohacsi Korhaz	Mohács	Hungary
Szabolcs-Szatmar-Bereg Varmegyei Oktatokorhaz	Nyíregyháza	Hungary
Pecsi Tudomanyegyetem Klinikai Kozpont	Pécs	Hungary
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico	Milan	Italy
Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza	Roma	Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Roma	Italy
University Medical Centre Groningen-UMCG	Groningen	Netherlands
Kyung Hee University Hospital at Gangdong	Seoul	South Korea
Ulsan University Hospital	Ulsan	South Korea
Hospital Universitari Son Espases	Palma de Mallorca	Balearic Islands
Hospital Universitari Vall d'Hebron	Barcelona	Spain
Hospital Universitario de Gran Canaria Dr. Negrin	Las Palmas	Spain
Hospital Universitario La Paz	Madrid	Spain
Hospital Regional Universitario de Malaga	Málaga	Spain
Sahlgrenska Universitetssjukhuset	Gothenburg	Sweden
MacKay Memorial Hospital_Taipei Branch	Taipei	Taiwan
Tri-Service General Hospital	Taipei	Taiwan
Chang Gung Memorial Hospital, Linkou	Taoyuan	Taiwan
King Chulalongkorn Memorial Hospital	Bangkok	Thailand
Phramongkutklao Hospital	Bangkok	Thailand
Siriraj Hospital	Bangkoknoi Bangkok	Thailand
Maharaj Nakorn Chiang Mai Hospital	Chiang Mai	Thailand

More Baxalta now part of Shire trials in the USA

Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B Active, not recruiting

Other trials for the same condition

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively Recruiting

Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A Active, not recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B Recruiting

A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A Recruiting

Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04158934 on ClinicalTrials.gov ↗ ← All trials in the USA