Clinical trials: Factor VIII Deficiency
Hemophilia
Are There Differences Between Carriers of Haemophilia A and B?
This study aims to develop a systematic genetic screening strategy for (potential) female carriers of haemophilia by identifying as many female carriers as possible within the families of haemophilia patients regularly followed…
- Trial name
- NCT05217992
- Testing
- Genetic assessment (hemophilia testing)
- Where
- Belgium
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Haemophilia A
A Study to Compare the Blood Levels of Mim8 in Healthy Men When Administered With the DV3407-C1 Pen Injector or a Syringe and Cartridge
Novo Nordisk is developing the study medicine Mim8 for the treatment of haemophilia A. The study aims to show similar levels of Mim8 in blood when using a new pen injector, called DV3407-C1 pen injector, and when using a syringe…
- Trial name
- NCT05681845
- Testing
- NNC0365-3769 (Mim8)
- Where
- Germany
- Volunteers
- 205 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Investigation of the Pharmacokinetics of NNC172-2021, at Two Different Dose Levels, in Healthy Japanese Subjects
This trial is conducted in Europe. The aim of this trial is to investigate the pharmacokinetics (how the trial drug is distributed in the body) of NNC172-2021 administered subcutaneously, at two different dose levels, in healthy…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B, Healthy
- Trial name
- NCT01555749
- Testing
- NNC172-2021
- Where
- United Kingdom
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Haemophilia A
A Study Looking at How Different Doses of Study Medicine (Inno8) Works in the Body of Healthy Men
This study will test how different doses of study medicine (Inno8) work in the healthy men. The purpose of this study is to prove safety of Inno8 in healthy men, which will support further development of Inno8 in people with…
- Trial name
- VOYAGER1 (NCT06649630)
- Testing
- NNC0442-0344 A
- Where
- 2 sites in Germany
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Evaluation of the Training Program for Community Pharmacists for Dispensing Emicizumab (HEMLIBRA®) in France
Haemophilia A (HA) is a rare constitutional haemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy.
- Trial name
- PASODOBLEDEMI1 (NCT05449197)
- Testing
- HEMOPHAR e-learning program
- Where
- France
- Volunteers
- 363 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC
Healthy Volunteers, Haemophilia A With or Without Inhibitors
A Research Study Investigating Mim8 in People With Haemophilia A
This study is investigating how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medication that will be used for prevention of bleeding episodes.
- Trial name
- FRONTIER1 (NCT04204408)
- Testing
- NNC0365-3769 (Mim8)
- Where
- 40 sites in United States, Austria and 10 more
- Volunteers
- 275 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Investigating the Bioequivalence of Eptacog Alfa A 6 mg and NovoSeven® in Healthy Male Subjects
This trial is conducted in Europe. The aim of the trial is to investigate the bioequivalence of eptacog alfa A 6 mg and NovoSeven® in healthy male subjects.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- NCT02084810
- Testing
- activated recombinant human factor VII, eptacog alfa (activated)
- Volunteers
- healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
A Multi-centre, Open Labelled, Multiple Dosing Trial Investigating Safety, Pharmacokinetics and Pharmacodynamics of NNC 0172-2021 Administered Subcutaneously to Healthy Male Subjects and Haemophilia Subjects
This trial is conducted in Europe. The aim of this trial is to investigate safety, pharmacokinetics (the exposure of the trial drug in the body) and pharmacodynamics (the effect of the investigated drug on the body) of NNC…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Healthy
- Trial name
- explorer™2 (NCT01631942)
- Testing
- NNC172-2021
- Where
- 6 sites in Austria, France and 3 more
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Haemophilia A
A Research Study Looking at How Different Doses of Study Medicine (Inno8) Work in the Body of People With Haemophilia A
This study will test how different doses of study medicine (Inno8) work in the bodies of people with haemophilia A. The purpose of the study is to see if Inno8 is safe to use for people with haemophilia A.
- Trial name
- VOYAGER2 (NCT07220564)
- Testing
- NNC0442-0344 A
- Where
- 33 sites in United States, Austria and 10 more
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hereditary Factor VIII Deficiency Disease With Inhibitor
A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A Patients With Inhibitor
Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are the only two drugs that are available to treat bleeds in haemophilia A patients with high titer inhibitors.
- Trial name
- BPATXAS (NCT01800435)
- Testing
- aPCC, aPCC + TXA, rFVIIa, rFVIIa + TXA
- Where
- Norway
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Oslo University Hospital
Haemophilia A
A Research Study Looking at How Food Intake Affects Inno8 in the Body of Healthy People
The purpose of this study is to test a new medicine called Inno8. The study will test how eating and drinking before and after taking Inno8 affects how well it is absorbed in the stomach. The study consists of four arms.
- Trial name
- NCT07238816
- Testing
- NNC0442-0344 A
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hemophilia a, Hemophilia B
The Safety and Tolerability of SerpinPC in Healthy Men and in Men with Severe Blood Disorders (haemophilia a and B)
The purpose of this study is to investigate the safety and activity in the body of a new drug called SerpinPC. The study will be split into 7 parts: Part 1a will be conducted in healthy male volunteers in the UK (up to 15) and…
- Trial name
- NCT04073498
- Testing
- SerpinPC
- Where
- 3 sites in Georgia, Moldova and 1 more
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- ApcinteX Ltd
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Vitamin D Deficiency, Sarcopenia
Vitamin D Deficiency and Body Composition in the Patients With Hemophilia
Hemophilia is associated with osteoporosis and increased prevalence of low bone mineral density (BMD). Many clinical risk factors have been reported, such as hemophilic arthropathy, reduced physical activity, decreased sun…
- Conditions
- Vitamin D Deficiency, Sarcopenia, Osteoporosis, Body Composition
- Trial name
- NCT05517070
- Where
- Taiwan
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Tsung-Ying Li
Hemophilia A, Hemophilia A, Severe
Hemophilia A Research Program
This study longitudinally observes the intergenerational (mother-child) continuum in hemophilia A from pregnancy through early childhood.
- Conditions
- Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency
- Trial name
- HARP (NCT07414511)
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- University of Washington
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
Safety and Tolerability of Intravenous Doses of Activated Recombinant Human Factor VII in Healthy Volunteers
This trial is conducted in Europe. The aim of this trial is to investigate the pharmacokinetics of three different single doses activated recombinant human factor VII in Caucasian and Japanese healthy subjects.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors, Healthy
- Trial name
- NCT01563471
- Testing
- activated recombinant human factor VII
- Where
- France
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Haemophilia B, Haemophilia A
Thrombin Generation Numerical Models Validation in Haemophilic Case
Personalized therapy in haemophilia has not been reached yet. Treatment is substitutive and its doses are only based on the levels of deficient factor VIII (for haemophilia A) or IX (for haemophilia B).
- Trial name
- NCT02300519
- Testing
- blood sampling
- Where
- France
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne