Clinical trials: Rare Bleeding Disorders, open to volunteers
Anemia, Bone Marrow Failure
European Rare Blood Disorders Platform (ENROL)
ENROL, the European Rare Blood Disorders Platform has been conceived in the core of ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare Hematological Diseases (RHDs).
- Conditions
- Anemia, Bone Marrow Failure, Bleeding Disorder, Iron Metabolism Disorders
- Trial name
- ENROL (NCT06250595)
- Testing
- Collection of clinical and laboratory data from EHR.
- Where
- Spain
- Volunteers
- 37,090 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Haemophilia
Defeat Rare Disease in Asia and Pacific (APAC) DRDA - Haemophilia
This study will help to better understand the course of disease in people with haemophilia (A or B [with or without inhibitors]) and the haemophilia care scenario in the selected countries and the experience so far in dealing…
- Trial name
- NCT07163260
- Testing
- No treatment given
- Where
- 4 sites in India, Malaysia and 2 more
- Volunteers
- 244 people
- Sponsor
- Novo Nordisk A/S
Myeloproliferative Disease, Polycythemia Vera
ENERGIA: Personalized Exercise Program for Fatigue in Patients With Myeloproliferative Neoplasms and Chronic Myeloid Leukemia
This study evaluates whether a personalized, supervised exercise program can improve fatigue and physical function in patients with myeloproliferative neoplasms (MPN) and chronic myeloid leukemia (CML).
- Conditions
- Myeloproliferative Disease, Polycythemia Vera, Thrombocythemia Essential, Myelofibrosis, Primary
- Trial name
- ENERGIA (NCT07518940)
- Testing
- Participants will take part in a 12-week exercise program that includes aerobic and resistance training, tailored to their individual fitness level and clinical condition.
- Where
- Italy
- Volunteers
- 25 people
- Sponsor
- Carmen Fava
Polycythemia Vera, ET (Essential Thrombocythemia)
MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)
The MPN PROGRESSion Registry is a multi-year, observational research study designed to improve understanding of myeloproliferative neoplasms (MPNs)-a group of rare, chronic blood cancers that include polycythemia vera (PV)…
- Conditions
- Polycythemia Vera, ET (Essential Thrombocythemia), Polycythemia Vera (PV), Essential Thrombocythemia (ET)
- Trial name
- NCT07362225
- Where
- 2 sites in United States
- Volunteers
- 5,000 people
- Sponsor
- MPN Research Foundation
Hematologic Disorder, Bleeding Disorder
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly.
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia
- Trial name
- NCT04398628
- Where
- 76 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- American Thrombosis and Hemostasis Network
Thrombotic Thrombocytopenic Purpura (TTP), Immune Thrombotic Thrombocytopenic Purpura
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic organ damage due to microvascular thrombosis.
- Conditions
- Thrombotic Thrombocytopenic Purpura (TTP), Immune Thrombotic Thrombocytopenic Purpura, Thrombotic Microangiopathies, Microangiopathy, Thromboic
- Trial name
- TWILIGHT (NCT07205861)
- Where
- France
- Volunteers
- 1,200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Factor X Deficiency
A Database For The Collection Of Clinical, Laboratory, And Treatment Data From Patients With Factor X Deficiency To Define The Natural History Of The Disease And Improve Patient Management
Factor X deficiency is one of the most severe rare bleeding disorders and may lead to serious spontaneous hemorrhages, particularly in patients with severe congenital forms.
- Trial name
- PRO-RBDD-FX (NCT07875478)
- Where
- 2 sites in Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Fondazione Luigi Villa