Clinical trials: Lymphatic Abnormalities
Sickle Cell Anemia
Evaluation of Hydroxyurea Plus L-arginine or Sildenafil to Treat Sickle Cell Anemia
Patients with sickle cell disease have abnormal hemoglobin (the protein in red blood cells that carries oxygen to the body). This abnormality causes red blood cells to take on a sickle shape, producing disease symptoms.
- Trial name
- NCT00056433
- Testing
- Hydroxyurea, L-Arginine, Sildenafil
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Sickle Cell Disease
Prevalence and Pathophysiology of Systemic Arterial Pressure Abnormalities in Childhood Sickle Cell Disease
It is usually found that the blood pressure of adults with sickle cell disease is lower than in non-sickle cell patients. On the other hand, three recent prospective studies in children with sickle cell disease show prevalence of…
- Trial name
- DrépaPA (NCT04911049)
- Testing
- Blood Pressure measurement
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
VWD - Von Willebrand's Disease
Effectiveness and Tolerability of Eqwilate in Real-life Conditions
MOdalities of use, effectiveNEss and TOlerability of Eqwilate® a balanced combInatiON of VWF and FVIII in von WillEbrand patients in real-life conditions: the ONE-TO-ONE study
- Trial name
- NCT04106908
- Testing
- Eqwilate
- Where
- 9 sites in France
- Volunteers
- 38 people
- Sponsor
- Octapharma
Graft Versus Host Disease, Leukemia
Selective Depletion of CD45RA+T Cells From Allogeneic Peripheral Blood Stem Cell Grafts for the Prevention of GVHD
RATIONALE: Allogeneic hematopoietic stem cell transplant (HSCT) is a treatment that can cure acute leukemia and myelodysplasia. After giving the patient chemotherapy and total body irradiation to stop the growth of cancer and…
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00914940
- Testing
- Fludarabine Phosphate, Tacrolimus, Thiotepa
- Where
- 2 sites in United States
- Volunteers
- 41 people
- Sponsor
- Fred Hutchinson Cancer Center
Ataxia Telangiectasia, Leukemia
Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia
Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability.
- Conditions
- Ataxia Telangiectasia, Leukemia, Lymphoma
- Trial name
- NCT04037189
- Where
- Israel
- Volunteers
- 250 people
- Sponsor
- Rabin Medical Center
Hemophilia, Knee Arthropathy
Hemophilic Knee Arthropathy Virtual Reality
This study intended to examine the effect of a physical rehabilitation program incorporating Kinect-based virtual reality on gait characteristics, functional ability, and physical activity level in adolescents with hemophilic…
- Trial name
- hemophilia (NCT05802368)
- Testing
- virtual reality group, exercise group
- Where
- Saudi Arabia
- Volunteers
- 56 people
- Sponsor
- Maged Basha
Glanzmann Thrombasthenia
Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)
This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder.
- Trial name
- STRENGTH (NCT07136857)
- Testing
- EPTACOG BETA
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Emory University
Leukemia
Drug Biomarkers in Cell Samples From Patients With Acute Myeloid Leukemia
RATIONALE: Studying cell samples from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer PURPOSE: This research study is studying drug…
- Trial name
- NCT01150058
- Testing
- western blotting, flow cytometry, laboratory biomarker analysis
- Volunteers
- 40 people
- Sponsor
- Children's Oncology Group
Acid Sphingomyelinase Deficiency (ASMD)
Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)
Acid sphingomyelinase Deficiency known as Neiman _PICK disease is a group of rare genetic diseases. This study includes analysis of clinical manifestations in patients with ASMD and investigations done for diagnosis of these…
- Trial name
- NCT07274826
- Where
- Egypt
- Volunteers
- 7 people
- Sponsor
- Sohag University
Sickle Cell Disease
Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders
The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic…
- Trial name
- NCT04416178
- Where
- United States
- Volunteers
- 352 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Severe Hemophilia A With an Inhibitor
The FEIBA NovoSeven Comparative Study
FENOC is a prospective, open-label, randomized, cross-over, multi-center study to investigate and compare the hemostatic effect and cost-efficacy of two different by-passing agents in the treatment of joint hemorrhages in…
- Trial name
- NCT00166309
- Testing
- FEIBA and NovoSeven
- Where
- Sweden
- Volunteers
- 60 people
- Sponsor
- Skane University Hospital
Chediak Higashi Syndrome, Chronic Granulomatous Disease
Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders
This study will try to determine what causes learning, behavioral and emotional problems in children with chronic granulomatous disease (GCD) and other phagocyte disorders.
- Conditions
- Chediak Higashi Syndrome, Chronic Granulomatous Disease, Job's Syndrome, Leukocyte Disorder
- Trial name
- NCT00005933
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST)
Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study
The purpose of this study was to allow continued use of nilotinib in patients who were on nilotinib treatment in a Novartis-sponsored, Oncology Clinical Development \& Medical Affairs (CD\&MA) study and were benefiting from the…
- Conditions
- Chronic Myelogenous Leukemia (CML), Metastatic Gastrointestinal Stromal Tumors (GIST), Acute Lymphoblastic Leukemia (ALL), Receptor Tyrosine Kinase (KIT) Mutated Melanoma
- Trial name
- NCT01735955
- Testing
- Nilotinib
- Where
- 33 sites in United States, Austria and 14 more
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Hyper-Immunoglobulin E. Syndrome (HEIS), Chronic Granulomatous Disease (CGD)
Skin Immunity Sample Collection Involving Blisters and Biopsies
Background: The way the body heals and protects itself from getting sick is called the immune response. Some people with weak immune systems get sick often or get rashes and skin infections.
- Trial name
- NCT03921515
- Testing
- Blister induction, Skin Biopsies
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Glucose-6-Phosphate Dehydrogenase Deficiency
Diet Challenge in G6PD Deficient Egyptian Children: A One- Year Prospective Single Center Study With Genotype - Phenotype Correlation
Glucose-6-phosphate dehydrogenase (G6PD) deficiency is prevalent and add a burden on families in Egypt and Middle East due to lifelong diet restriction, non-fava beans diet is main food for most families in the region and parents…
- Trial name
- NCT02498340
- Testing
- non- fava beans
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Ain Shams University
Cesarean Section, Infant Conditions
Intact-cord Stabilisation and Physiology-based Cord Clamping in Caesarean Sections
This is a feasibility study with historical control designed to evaluate whether delivery of the placenta prior to umbilical cord clamping at caesarean sections is a feasible, safe and acceptable way of facilitating intact-cord…
- Trial name
- INTACT (NCT05461950)
- Testing
- Extrauterine placental transfusion and physiology-based umbilical cord clamping, Extrauterine placental transfusion, intact cord stabilisation and physiology-based umbilical cord clamping, Delayed umbilical cord clamping
- Where
- Norway
- Volunteers
- 263 people
- Sponsor
- Helse Møre og Romsdal HF
Thalassemia
Thalassemia Knowledge in Young People of Indonesia
There is still an ongoing lack of awareness about thalassemia among Indonesians despite high disease prevalence, and this is associated with poor implementation of preventive and promotive measures.
- Trial name
- NCT04706585
- Where
- Indonesia
- Volunteers
- 1,010 people, healthy volunteers welcome
- Sponsor
- Fakultas Kedokteran Universitas Indonesia
Beta Thalassemia Intermedia
Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon
Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body.
- Trial name
- LB-04-THAL (NCT01642758)
- Testing
- Sodium 2,2 dimethylbutyrate
- Where
- Lebanon
- Volunteers
- 10 people
- Sponsor
- Boston University
Telangiectasia, Hereditary Hemorrhagic
Study of Histological Samples From Patients With Hereditary Haemorrhagic Telangiectasia
In this study the investigators will obtain histological samples from people with hereditary haemorrhagic telangiectasia (HHT, also known as Osler-Weber-Rendu Syndrome).
- Trial name
- NCT00733655
- Where
- United Kingdom
- Volunteers
- 6 people
- Sponsor
- Imperial College London
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