Clinical trials: Lymphatic Malformation
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Hemophilia A or B
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
A study to evaluate the safety of Hympavzi under the actual use in patients with congenital hemophilia who do not have inhibitors.
- Trial name
- HIZ (NCT07161687)
- Testing
- Marstacimab
- Where
- Japan
- Volunteers
- 50 people
- Sponsor
- Pfizer
Hemophilia A
BAX 826 Dose-Escalation Safety Study
1. To assess tolerability and safety of BAX 826 after a single infusion in previously treated patients (PTPs) with severe hemophilia A 2. To determine the pharmacokinetic (PK) parameters of BAX 826 compared to ADVATE 3.
- Trial name
- NCT02716194
- Testing
- BAX 826, Octocog alfa
- Where
- 28 sites in Bulgaria, Germany and 7 more
- Volunteers
- 40 people
- Sponsor
- Baxalta now part of Shire
Congenital Bleeding Disorder, Haemophilia B
A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B
This trial is conducted in Europe and the United States of America. The aim of this trial is to compare the pharmacokinetics (the exposure of the trial drug in the body) of nonacog beta pegol (N9-GP) and ALPROLIX® in patients…
- Trial name
- paradigm™7 (NCT03075670)
- Testing
- N9-GP, ALPROLIX®
- Where
- 12 sites in United States, Germany and 1 more
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.
- Trial name
- NCT01757405
- Testing
- Recombinant Factor VIIa BI (rFVIIa BI)
- Where
- 16 sites in United States, Japan and 7 more
- Volunteers
- 40 people
- Sponsor
- Baxalta now part of Shire
Niemann-Pick Disease, Niemann-Pick Disease, Type C
Biomarker for Niemann Pick Type C Disease (BioNPC)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Niemann Pick Type C disease from Blood (plasma)
- Trial name
- BioNPC (NCT01306604)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hereditary Hemorrhagic Telangiectasia (HHT)
Phase 1b Study of TX002100 in Participants With Hematologic Support-Dependent Hereditary Hemorrhagic Telangiectasia (HHT)
The primary purpose of this study is to assess the safety and tolerability of TX002100 in participants with hereditary hemorrhagic telangiectasia (HHT).
- Trial name
- NCT07863609
- Testing
- TX002100
- Volunteers
- 20 people
- Sponsor
- Tectonic Operating Company, Inc.
Hemophilia B
RIXUBIS Drug Use-Result Survey (Japan)
The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS. 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT02937831
- Testing
- RIXUBIS
- Where
- 5 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Baxalta now part of Shire
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
G6PD, G6PD Deficiency
Comparative Evaluation of the FINDER Instrument and FINDER G6PD Cartridge in Adults and Neonates
A prospective clinical study to compare the clinical performance of the FINDER™ Instrument and the G6PD assay to an FDA cleared instrument/reagent combination in adult and neonate populations.
- Trial name
- NCT04146246
- Testing
- G6PD assay
- Where
- 3 sites in United States
- Volunteers
- 119 people, healthy volunteers welcome
- Sponsor
- Baebies, Inc.
Cerebral Cavernous Malformation
The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in participants with symptomatic…
- Trial name
- SYCAMORE (NCT05085561)
- Testing
- REC-994
- Where
- 15 sites in United States
- Volunteers
- 62 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Β-thalassemia
Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.
This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia.
- Trial name
- NCT06655662
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- 3 sites in China
- Volunteers
- 8 people
- Sponsor
- Shenzhen Hemogen
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
Haemophilia B
A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B
The study investigates how well the medicine called nonacog beta pegol (N9-GP) works in Chinese people with haemophilia B. Participants will be treated with N9-GP.
- Trial name
- Paradigm9 (NCT05365217)
- Testing
- Nonacog beta pegol
- Where
- 15 sites in China
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Hemophilia A
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
Primary Objective: To evaluate the long-term safety of BIVV001 in previously treated subjects with hemophilia A Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-ed (NCT04644575)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 85 sites in United States, Argentina and 21 more
- Volunteers
- 261 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
- Trial name
- NCT06145373
- Testing
- Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 3 sites in United States, Taiwan
- Volunteers
- 20 people
- Sponsor
- Sanofi
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited