Clinical trials: Lymphatic Abnormalities
Congenital Bleeding Disorder, Haemophilia A
Observational Study Describing the Usual Clinical Practice Use of NovoSeven® in the Home Treatment of Joint Bleeds in Patients With Haemophilia A or B and Inhibitors
This trial is conducted in Africa and Asia. The aim of this study is to evaluate the efficacy of home treatment of joint bleeds (haemarthrosis) with NovoSeven® (activated recombinant human factor VII) in patients with haemophilia…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01234545
- Testing
- activated recombinant human factor VII
- Where
- 5 sites in Algeria, Morocco and 3 more
- Volunteers
- 35 people
- Sponsor
- Novo Nordisk A/S
HDN - Hemolytic Disease of the Newborn
Neonatal Cord Blood Screening for HDN
To evaluate the diagnostic efficiency of antibodies screening in cord blood for detection of HDN. To help finding the antigen negative blood in a timely manner and reduce the morbidities and mortalities of HDN
- Trial name
- NCT05617612
- Volunteers
- 400 people
- Sponsor
- Assiut University
Hemophilia A
Emotions in the Communication and Relationship Styles of Parents With Hemophilic Children.
Severe hemophilia is a rare, inherited, X-linked disorder. Currently, standard therapy involves ev infusion of drugs from a very young age.
- Trial name
- NCT05754619
- Where
- Italy
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sickle Cell Disease
Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease
Background: Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood.
- Trial name
- NCT02225132
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hemophilia A
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital…
- Trial name
- ZEBRHA 2 (NCT07416604)
- Testing
- NXT007, Emicizumab
- Where
- 59 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Hoffmann-La Roche
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Sickle Cell Disease
Disseminating NIH Evidence Based Sickle Cell Recommendations in North Carolina
This project will improve the efficiency and quality of healthcare for persons with sickle cell disease, an under-served and at risk population by implementing a co-management model of care.
- Trial name
- NCT03253211
- Where
- United States
- Volunteers
- 4,392 people
- Sponsor
- Duke University
Hemophilia A With Inhibitor
Hemophilia Inhibitor Genetics Study (HIGS)
Several non-genetic and genetic factors that could influence the risk of inhibitor development in hemophilia A have been discussed but not fully explored. The aim of the HIGS is to identify these genetic factors.
- Trial name
- NCT00166387
- Testing
- Blood draw
- Where
- Sweden
- Volunteers
- 1,137 people
- Sponsor
- Skane University Hospital
Molar Incisor Hypomineralization, Oral Health
Prevalence, Etiology and Effects on Oral Health and Life Quality of Molar Incisor Hypomineralization
Molar incisor hypomineralization (MIH) can lead to many clinical conditions and affect oral health-related quality of life (OHRQoL).
- Conditions
- Molar Incisor Hypomineralization, Oral Health, Child, Only, Pediatric ALL
- Trial name
- NCT05779332
- Testing
- survey application
- Where
- Turkey (Türkiye)
- Volunteers
- 655 people, healthy volunteers welcome
- Sponsor
- Recep Tayyip Erdogan University Training and Research Hospital
Sickle Cell Anemia, Sickle Cell Disease
Novel Use Of Hydroxyurea in an African Region With Malaria
Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in…
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Malaria
- Trial name
- NOHARM (NCT01976416)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 208 people
- Sponsor
- Indiana University
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Fludarabine, Cyclophosphamide, and Total-Body Irradiation Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients Who Are Undergoing a Donor Umbilical Cord Blood Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00255684
- Testing
- graft-versus-tumor induction therapy, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Rochester
Niemann-Pick Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who…
- Trial name
- NCT02912793
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 5 sites in Israel, Sweden and 1 more
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
Sickle Cell Disease
Uganda Sickle Surveillance Study (US-3)
It is estimated that over 250,000 babies are born with sickle cell disease (SCD) annually in sub-Saharan Africa, and only 10% - 50% of them survive beyond five years of age.
- Trial name
- NCT06301893
- Where
- 4 sites in Uganda
- Volunteers
- 1,000,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Donor T Cells After Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This pilot phase II trial studies how well giving donor T cells after donor stem cell transplant works in treating patients with hematologic malignancies.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT01839916
- Testing
- therapeutic allogeneic lymphocytes, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- University of Chicago
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 people
- Sponsor
- Mandos LLC
Vaso-occlusive Pain Episodes
Arginine Therapy in Sickle Cell Disease-VOC Clinical Trial
Vaso-occlusion contributes significantly to morbidity in sickle cell disease (SCD). Vaso-occlusive painful episodes (VOE) are common and debilitating, causing the majority of emergency department visits.
- Trial name
- NCT01796678
- Testing
- Arginine
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Diabetic Retinopathy, Sickle Cell Trait
Diabetic Retinopathy and Sickle Trait
To more clearly ascertain the relationship between ocular manifestations of sickle cell disease and diabetes, specifically; whether the presence of sickle cell trait exacerbates the disease progression of diabetic retinopathy.
- Trial name
- NCT00720317
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical University of South Carolina
Anemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the…
- Trial name
- NCT05664737
- Testing
- Luspatercept
- Where
- 31 sites in China, Greece and 8 more
- Volunteers
- 189 people
- Sponsor
- Bristol-Myers Squibb
Hemophilia A
Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
- Trial name
- NCT04396639
- Testing
- Moroctocog-alfa (AF-CC)
- Where
- 5 sites in India
- Volunteers
- 50 people
- Sponsor
- Pfizer
Heterotaxy Syndrome
Defining Immunodeficiency in Heterotaxy Syndrome: Pilot Study Data
The investigators aim with this study is to investigate the mechanisms of immune deficiency in patients with heterotaxy syndrome through the use of novel biomarkers and a prospective questionnaire survey documenting the burden of…
- Trial name
- NCT01929967
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Boston Children's Hospital
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