Clinical trials: Newborn
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay
This clinical trial uses a laboratory test called a high throughput sensitivity assay in planning treatment for patients with relapsed or refractory acute myeloid leukemia.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01872819
- Testing
- antitumor drug screening assay, chemotherapy, biological therapy
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Washington
Neonatal Sepsis, Preterm Infants
Effect of Vitamin D as Adjuvant Therapy in Preterm Infants With Neonatal Sepsis
The goal of this clinical trial is to learn the effect of vitamin D as an adjunctive therapy for preterm neonates with sepsis measured by the outcomes, which are sepsis score and C-reactive protein after 7 days.
- Trial name
- NCT07245277
- Testing
- Vitamin D
- Where
- 2 sites in Indonesia
- Volunteers
- 78 people
- Sponsor
- Universitas Padjadjaran
Thyroid Eye Disease, Graves' Orbitopathy
Treatment of Graves' Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis With Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study
The overall objective is to investigate the efficacy, tolerability, and safety of teprotumumab (a fully human monoclonal antibody [mAb] inhibitor of the insulin-like growth factor-1 receptor [IGF-1R]) administered once every 3…
- Trial name
- OPTIC (NCT03298867)
- Testing
- Teprotumumab
- Where
- 14 sites in United States, Germany and 1 more
- Volunteers
- 83 people
- Sponsor
- Amgen
Colorectal Cancer, Colon Cancer
CB-839 + Capecitabine in Solid Tumors and Fluoropyrimidine Resistant PIK3CA Mutant Colorectal Cancer
This study has two portions. The main goal of the Phase I portion of this research study is to see what doses of CB-839 and capecitabine can safely be given to patients without having too many side effects.
- Conditions
- Colorectal Cancer, Colon Cancer, Rectal Cancer, Solid Tumor
- Trial name
- NCT02861300
- Testing
- CB-839, Capecitabine
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- David Bajor, MD
Erythropoietic Protoporphyria
Phase III Confirmatory Study in Erythropoietic Protoporphyria
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Between 75 and 100 eligible patients will be enrolled.
- Trial name
- NCT01605136
- Testing
- Afamelanotide
- Where
- 7 sites in United States
- Volunteers
- 93 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Severe Hemophilia A
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant…
- Trial name
- NCT04046848
- Testing
- OCTA101
- Where
- Bulgaria
- Volunteers
- 36 people
- Sponsor
- Octapharma
ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant…
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- Trial name
- NCT05030831
- Testing
- INZ-701
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Inozyme Pharma
Vaso-occlusive Crisis, Reduction in Hospitalizations
Apixaban in Patients With Sickle Cell Disease
In patients with SCD, the use of low dose anticoagulation as an outpatient may lead to a significant decrease in morbidity and as a result, decrease healthcare utilization and costs.
- Conditions
- Vaso-occlusive Crisis, Reduction in Hospitalizations, Sickle Cell Disease
- Trial name
- NCT02179177
- Testing
- Apixaban
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Nirmish Shah
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
Electroencephalographic Neonatal Seizures
A Study to Test the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Newborns With Repeated Electroencephalographic Seizures
The purpose of the study is to evaluate the pharmacokinetics (PK) of brivaracetam (BRV) in neonates who have seizures that are not adequately controlled with previous antiepileptic drug (AED) treatment, and to identify the…
- Trial name
- NCT03325439
- Testing
- Brivaracetam (BRV) intravenous (iv), Brivaracetam (BRV) oral
- Where
- 12 sites in Belgium, Czechia and 5 more
- Volunteers
- 9 people
- Sponsor
- UCB Biopharma SRL
Complication of Renal Dialysis, End Stage Renal Disease
A Study Testing the Use of a Perivascular Sirolimus Formulation (Sirogen) in ESRD Patients Undergoing AV Fistula Surgery
The primary study objective is to evaluate the benefit of the Sirolimus eluting Collagen implant (SeCI; Sirogen), a single dose prophylactic treatment delivered intraoperatively at the time of surgical creation of an…
- Conditions
- Complication of Renal Dialysis, End Stage Renal Disease, End Stage Kidney Disease, ESRD
- Trial name
- ACCESS2 (NCT05425056)
- Testing
- Sirolimus
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 136 people
- Sponsor
- Vascular Therapies, Inc.
Leukemia, Lymphoma
Donor Peripheral Stem Cell Transplant, Fludarabine, and Busulfan in Treating Patients With Hematologic Cancers
Giving chemotherapy drugs, such as fludarabine and busulfan, before a donor peripheral stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00619645
- Testing
- busulfan, cyclosporine, fludarabine phosphate
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of California, Davis
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del…
- Trial name
- NCT03537651
- Testing
- TEZ/IVA, IVA
- Where
- 55 sites in United States, Australia and 9 more
- Volunteers
- 130 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Fragile X Syndrome
Trial of Minocycline to Treat Children With Fragile X Syndrome
This is a single center study at the UC Davis MIND Institute in patients age 3.5-16 years of age with fragile X syndrome (FXS), funded by a National Fragile X Foundation Grant.
- Trial name
- NCT01053156
- Testing
- minocycline hydrochloride
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- University of California, Davis
Arteriovenous Fistula
An Efficacy and Safety Study of SRM003 in the Treatment of Subjects Undergoing Creation of an Arteriovenous Fistula to Facilitate Hemodialysis Access
A study to evaluate the efficacy of SRM003 treatment versus participating sites' standard practice treatment in improving the rate of AVF maturation and use in subjects with end-stage renal disease undergoing surgery for creation…
- Trial name
- NCT01806545
- Testing
- SRM003, Participating Site's standard practice
- Where
- 47 sites in United States
- Volunteers
- 64 people
- Sponsor
- Shire
Leukemia, Myelodysplastic Syndromes
Donor Umbilical Cord Blood Natural Killer Cells, Aldesleukin and Umbilical Cord Blood Transplant in Patients With Refractory Hematologic Cancers.
RATIONALE: Giving chemotherapy, natural killer cells, aldesleukin, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of abnormal cells and cancer cells.
- Trial name
- NCT00354172
- Testing
- aldesleukin, filgrastim, natural killer cell (NK) therapy
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
- Trial name
- NCT02971839
- Testing
- VX-561, IVA
- Where
- 14 sites in United States
- Volunteers
- 11 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Gaucher Disease
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
This was a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are ≥18 and ≤50 years of age and post pubertal at Screening with a confirmed…
- Trial name
- NCT04145037
- Testing
- AVR-RD-02
- Where
- 5 sites in United States, Canada
- Volunteers
- 8 people
- Sponsor
- AVROBIO
Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with LGMD2B and FSHD.
- Trial name
- FSHD (NCT02579239)
- Testing
- ATYR1940
- Where
- 6 sites in United States, Denmark and 1 more
- Volunteers
- 18 people
- Sponsor
- aTyr Pharma, Inc.
Hemophilia A
Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery
This study will examine the efficacy and safety of ReFacto AF in patients with severe and moderately severe hemophilia A undergoing elective major surgery when administered by either bolus injections (BI) or continuous infusion…
- Trial name
- NCT00243659
- Testing
- ReFacto AF
- Where
- 17 sites in United States, Australia and 6 more
- Volunteers
- 30 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
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