Clinical trials: Lymphatic Malformation
Chronic Hepatitis C Infection
Open-Labeled Study of PSI-7977 and RBV With and Without PEG-IFN in Treatment-Naïve Patients With HCV GT2 or GT3
This study is to assess the safety and tolerability of sofosbuvir (SOF) 400 mg with and without ribavirin (RBV) and/or with and without pegylated interferon alfa-2a (PEG) in subjects with genotype 1, 2 or 3 hepatitis C (HCV)…
- Trial name
- NCT01260350
- Testing
- SOF, RBV, PEG
- Where
- 2 sites in New Zealand
- Volunteers
- 292 people
- Sponsor
- Gilead Sciences
Hemophilia A, Hemophilia B
An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.
- Trial name
- JOIN-us (NCT05856266)
- Testing
- Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US), Haemophilia Joint Health Score (HJHS)
- Where
- 56 sites in Bulgaria, Croatia and 9 more
- Volunteers
- 10 people
- Sponsor
- Swedish Orphan Biovitrum
Sickle Cell Anemia
Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol
The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys.
- Trial name
- BABY HUG (NCT01783990)
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 150 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission
Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
RATIONALE: Giving chemotherapy before a donor umbilical cord blood transplant (UCBT) helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT01093586
- Testing
- double-unit umbilical cord blood transplantation, cytogenetic analysis, bone marrow aspiration
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Case Comprehensive Cancer Center
Sickle Cell Disease, Priapism
Sildenafil for Treatment of Priapism in Men With Sickle Cell Anemia
This research is being done to evaluate if the phosphodiesterase type 5 (PDE5) inhibitor sildenafil has an effect on the frequency of recurrent priapism and the quality of life of males with sickle cell disease (SCD).
- Trial name
- NCT00940901
- Testing
- sildenafil
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Johns Hopkins University
Anemia, Sickle Cell
Siplizumab for Sickle Cell Disease Transplant
The purpose of this study is to find out whether siplizumab is safe and effective for patients with SCD undergoing an allogeneic transplant and to prevent development of Graft versus Host Disease (GVHD) and graft failure.
- Trial name
- CD2 SCD (NCT06078696)
- Testing
- Siplizumab, Exchange Transfusion, Total Body Irradiation
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Columbia University
Sickle Cell Disease
Pain Management in Children and Young Adults With Sickle Cell Disease
This is a phase II double-blind placebo-controlled clinical trial evaluating the effect of gabapentin when added to standard pain management for patients with sickle cell disease experiencing acute pain crisis in the ambulatory…
- Trial name
- NCT01954927
- Testing
- Gabapentin
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- St. Jude Children's Research Hospital
Hemoglobin SC Disease
Treatment of Hemoglobin SC Disease With Hydroxyurea
Sickle cell disease (SCD), specifically hemoglobin SC disease (HbSC), is a subtype of sickle cell disease with typically higher hemoglobin and milder or later disease complications.
- Trial name
- SCYTHE (NCT02336373)
- Testing
- hydroxyurea
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Baylor College of Medicine
Pulmonary Hypertension, Sickle Cell Disease
Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension
The primary hypothesis of this study is that glutamine supplementation will improve the erythrocyte glutamine/glutamate ratio, a biomarker of oxidative stress, hemolysis and pulmonary hypertension (PH) in sickle cell disease…
- Conditions
- Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
- Trial name
- NCT01048905
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Hemophilia A, Hemophilia B
Understanding Hemophilia A and B Drug Dosage Administration Patterns
Study Design A prospective observational, cross-sectional epidemiological study in U.S. site-based clinical practice settings. 30 sites will enroll approximately 300 patients Participating patients - or their caregiver in the…
- Trial name
- NCT03248141
- Testing
- Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Pfizer
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Acute Myeloid Leukemia/Transient Myeloproliferative Disorder
Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia
RATIONALE: Giving chemotherapy, such as fludarabine phosphate, busulfan, and cyclophosphamide, and total-body radiation therapy before a donor peripheral stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Acute Myeloid Leukemia/Transient Myeloproliferative Disorder, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT00322101
- Testing
- total-body irradiation, allogeneic hematopoietic stem cell transplantation, cyclophosphamide
- Where
- 8 sites in United States, Germany
- Volunteers
- 25 people
- Sponsor
- Fred Hutchinson Cancer Center
Reversal of Coagulopathy
An Open-label, Randomized, Multicenter Phase IIIb Study to Assess the Efficacy, Safety and Tolerance of BERIPLEX® P/N (Kcentra) Compared With Plasma for Rapid Reversal of Coagulopathy Induced by Vitamin K Antagonists in Subjects Requiring an Urgent Surgical Procedure
The purpose of this study is to evaluate efficacy, safety and tolerance of Beriplex® P/N (Kcentra) compared with plasma in regard to rapid reversal of coagulopathy induced by vitamin K antagonists in subjects who require…
- Trial name
- BE1116_3003 (NCT00803101)
- Testing
- Beriplex® P/N (Kcentra), Fresh frozen plasma
- Where
- 30 sites in United States, Belarus and 4 more
- Volunteers
- 176 people
- Sponsor
- CSL Behring
Sickle Cell Disease
Regional Anesthesia for Sickle Cell Crisis Using Ultrasound in The Emergency Department: Phase I
The hypothesis of this study is: Femoral nerve blocks can feasibly be performed on patients with Sickle Cell Disease and painful crisis in the Emergency Department.
- Trial name
- RESCUED (NCT01256281)
- Testing
- Femoral Nerve Block
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Sickle Cell Disease
Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)
This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care.
- Trial name
- STRIDE2 (NCT02766465)
- Testing
- Busulfan, Fludarabine, r-ATG
- Where
- 35 sites in United States
- Volunteers
- 138 people
- Sponsor
- Medical College of Wisconsin
22Q11.2 Deletion Syndrome
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to <18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).
- Trial name
- INSPIRE (NCT05149898)
- Testing
- ZYN002
- Where
- 3 sites in United States, Australia
- Volunteers
- 20 people
- Sponsor
- Harmony Biosciences Management, Inc.
Hemophilia B
A Factor IX Gene Therapy Study (FIX-GT)
Severe haemophilia B (HB) is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing.
- Trial name
- FIX-GT (NCT03369444)
- Testing
- FLT180a
- Where
- 11 sites in United States, Ireland and 2 more
- Volunteers
- 10 people
- Sponsor
- University College, London
Beta Thalassemia Intermedia
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.
- Trial name
- NCT04059406
- Testing
- sapablursen
- Where
- 19 sites in Australia, Greece and 3 more
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Leukemia, Lymphoma
Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00054327
- Testing
- busulfan, cyclophosphamide, cytarabine
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Case Comprehensive Cancer Center
Jaundice, Neonatal, Hyperbilirubinemia, Neonatal
Stannsoporfin With Light Therapy for Newborn Babies With Jaundice
It is normal for red blood cells to die, even in newborn babies. The waste from that is called bilirubin. The liver clears bilirubin out of the body.
- Trial name
- JASMINE_204 (NCT01887327)
- Testing
- Phototherapy, Stannsoporfin
- Where
- 18 sites in United States
- Volunteers
- 91 people
- Sponsor
- InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company
Hemophilia A, Congenital
Alphanate in Immune Tolerance Induction Therapy
This is a multicenter, multinational, prospective, single-arm, nonrandomized, open-label study, planned in of approximately 25 male participants with congenital hemophilia A who will receive their first (primary) immune tolerance…
- Trial name
- NCT03095287
- Testing
- Alphanate
- Where
- 20 sites in United States, Canada and 4 more
- Volunteers
- 2 people
- Sponsor
- Grifols Therapeutics LLC