Clinical trials: Lymphatic Malformation
Haemophilia
Effectiveness of the uPatient Platform in Prophylactic Treatment in Hemophilia
Research project whose main objective is to evaluate the use of a technology platform (uPatient) to register the prophylactic treatment of patients with hemophilia treated in different Hematology and Hemotherapy services of…
- Trial name
- uPatient (NCT02581735)
- Testing
- Patients with haemophilia
- Volunteers
- 40 people
- Sponsor
- Real Fundación Victoria Eugenia
Sickle Cell Disease (SCD), Invasive Bacterial Infections
Epidemiology of Invasive Bacterial Infections in Children With Sickle Cell Disease in France Between 2020 and 2025
Children with sickle cell disease are at high risk of invasive bacterial infections, which may lead to serious complications. Preventive measures, including antibiotic prophylaxis and vaccination, have changed the epidemiology of…
- Trial name
- DREPIBAC (NCT07639008)
- Where
- 31 sites in France
- Volunteers
- 350 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Diabetes, Chronic Kidney Diseases
Benchtop NMR Spectroscopy for Assessment of Clinical Human Pathologies (BRANCH-P STUDY)
This research programme seeks to combine the resources of NHS primary care, with the leading spectroscopic work in low-magnetic fields of the Wilson Group (Nottingham Trent University) to demonstrate the potential for benchtop…
- Conditions
- Diabetes, Chronic Kidney Diseases, Cancer, Bowel Disease
- Trial name
- BRANCH-P (NCT05473325)
- Where
- United Kingdom
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Willows Health
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to…
- Trial name
- NCT00768287
- Testing
- IB1001, nonacog alfa
- Where
- 23 sites in United States, France and 5 more
- Volunteers
- 77 people
- Sponsor
- Medexus Pharma, Inc.
Hemophilia A
A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels.
- Trial name
- PROTECT-VIII (NCT01580293)
- Testing
- BAY94-9027
- Where
- 59 sites in United States, Austria and 18 more
- Volunteers
- 145 people
- Sponsor
- Bayer
Chronic Myeloproliferative Disorders, Leukemia
12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: This phase I trial is studying the side effects and best dose of…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00004058
- Testing
- tetradecanoylphorbol acetate
- Where
- United States
- Sponsor
- University of Medicine and Dentistry of New Jersey
Anemia, Sickle Cell, Blood Disease
Cerebrovascular Involvement in Sickle Cell Disease - Comprehensive Sickle Cell Center
To continue studies on the two major neurological complications of sickle cell disease (SCD): namely, stroke and chronic encephalopathy.
- Conditions
- Anemia, Sickle Cell, Blood Disease, Cerebrovascular Accident
- Trial name
- NCT00005326
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fetal Conditions, Maternal; Procedure
A Prospective Study on the Role of Karl Storz Curved and Straight Fetoscopes (11508AAK and 11506AAK) for Fetoscopic Intrauterine Procedures
In this research study, the investigators want to learn more about the role of new innovative surgical devices, the Karl Storz Curved and Straight Fetoscopes for in-utero surgery.
- Conditions
- Fetal Conditions, Maternal; Procedure, Pregnancy Related, Twin to Twin Transfusion Syndrome
- Trial name
- NCT06056635
- Testing
- Karl Storz Curved Scope, Karl Storz Straight Scope
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Boston Children's Hospital
Hemophilia
Physiotherapy to Improve Balance and Walking Capacity in Hemophilia
Introduction: Hemarthrosis is the most common type of bleeding with 70-80% in patient with hemophilia (PwH) and the majority of hemarthrosis occurs in the knee and ankle joints.
- Trial name
- NCT07339410
- Testing
- Physiotherapy
- Where
- Turkey (Türkiye)
- Volunteers
- 35 people
- Sponsor
- Yuzuncu Yil University
PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations
A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)
This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, zovegalisib (RLY-2608), in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations…
- Conditions
- PIK3CA-Related Overgrowth Spectrum (PROS), Lymphatic Malformations, Vascular Malformations, PIK3CA Mutation
- Trial name
- NCT06789913
- Testing
- RLY-2608
- Where
- 40 sites in United States, Australia and 7 more
- Volunteers
- 277 people
- Sponsor
- Relay Therapeutics, Inc.
Leukemia, Myelodysplastic Syndromes
Yttrium Y 90 Anti-CD45 Monoclonal Antibody AHN-12 in Treating Patients With Advanced Leukemia
RATIONALE: Monoclonal antibodies can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Trial name
- NCT00618696
- Testing
- anti-CD45 monoclonal antibody AHN-12, yttrium Y 90 anti-CD45 monoclonal antibody AHN-12
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Plasmacytoma, POEMS Syndrome
Ixazomib Citrate, Lenalidomide, and Dexamethasone in Treating Patients With POEMS Syndrome
This phase II trial studies how well ixazomib citrate, lenalidomide, and dexamethasone work in treating patients with polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome.
- Trial name
- NCT02921893
- Testing
- Dexamethasone, Ixazomib Citrate, Lenalidomide
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Mayo Clinic
Factor X Deficiency
Safety & Efficacy of BPL's High Purity FACTOR X in Treatment of Factor X Deficient Subjects Undergoing Surgery
To primary efficacy variable is to assess the presence or absence of excessive blood loss during and after surgery. The secondary efficacy endpoints are as follows: 1.
- Trial name
- Ten03 (NCT01086852)
- Testing
- FACTOR X
- Where
- 7 sites in United States, Spain and 2 more
- Volunteers
- 4 people
- Sponsor
- Bio Products Laboratory
Sickle Cell Anemia
Prasugrel Versus Placebo in Adult Sickle Cell Disease
The purpose of this trial is to assess the safety of Prasugrel in adult patients with sickle cell disease (SCD) by monitoring the rate and severity of hemorrhagic events requiring medical intervention compared to placebo for 30…
- Trial name
- NCT01167023
- Testing
- Prasugrel
- Where
- 14 sites in United States
- Volunteers
- 62 people
- Sponsor
- Eli Lilly and Company
Haemophilia A
An Observational Research Study of the Health of Joints in People With Haemophilia Taking the Medicine Esperoct
This study will collect information on the long term health of joints in people with haemophilia A who have started treatment with Esperoct within twelve months prior to participation to the study.
- Trial name
- NCT05621746
- Testing
- Esperoct
- Where
- Canada
- Volunteers
- 123 people
- Sponsor
- Novo Nordisk A/S
Anemia, Sickle Cell, Blood Disease
Hemostasis in Sickle Cell Disease--Infancy to Adulthood
To assess in older children and adults with sickle cell disease (SCD) whether intrinsic activation (relevant to the origin of pain and acute inflammation) occurs only during vasocclusive crisis (VOC).
- Trial name
- NCT00005703
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hemophilia A, Hemophilia B
Validation and Reliability of the CHO-KLAT in Turkish
The aim of this study is to investigate the Turkish validity and reliability of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool version2.0.
- Trial name
- NCT05439642
- Testing
- Evaluation of health related quality of life
- Where
- 4 sites in Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- Yuzuncu Yil University
Telangiectasia, Hereditary Hemorrhagic
Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
This study will examine genes involved in the vascular dysplasia Hereditary haemorrhagic telangiectasia i(HHT)
- Trial name
- NCT00230620
- Where
- United Kingdom
- Volunteers
- 1,000 people
- Sponsor
- Imperial College London
Sickle Cell Anemia Crisis
Does IV Acetaminophen Reduce Opioid Requirement in Pediatric Patients With Acute Sickle Cell Crises?
The purpose of this study is to determine whether IV acetaminophen can decrease the need for subsequent opioid administration in the acute management of sickle cell crisis pain in the pediatric emergency room.
- Trial name
- NCT03541980
- Testing
- Acetaminophen, Normal saline
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Newark Beth Israel Medical Center
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
The primary purpose of Study INZ701-104 (the ENERGY study) is to assess the safety and tolerability of INZ-701 in infants with ENPP1 Deficiency or with ABCC6 Deficiency.
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency
- Trial name
- ENERGY (NCT05734196)
- Testing
- INZ-701
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 9 people
- Sponsor
- Inozyme Pharma