Clinical trials: Newborn Diseases
Hemophilia A With Inhibitor
A Pharmacokinetic and Clotting Activity Study of FVIII-PEGLip
The purpose of this study is to demonstrate that PEGylated liposomes (PEGLip) can shield FVIII from the immune system and inhibitors, and therefore provide a prophylactic FVIII replacement therapy for patients with inhibitors to…
- Trial name
- NCT04592692
- Testing
- PEGylated Liposome (PEGLip)
- Where
- 5 sites in Russia
- Volunteers
- 20 people
- Sponsor
- Ascension Healthcare Development Limited
Phenylketonurias
PheCheck Feasibility Study
The goal of this feasibility study clinical trial is to compare the accuracy of PheCheck™ for the rapid quantitative detection of phenylalanine (Phe) with the gold standard (HPLC amino acid analyzer), in patients with PKU.
- Trial name
- NCT05998109
- Testing
- PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Lumos Diagnostics
Cystic Fibrosis, Bronchiectasis
Dose Escalation Study of ALX-009 in Healthy Men and Cystic Fibrosis (CF) and Non-CF Bronchiectasis Patients
This is a Phase 1, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and pharmacokinetics of a single ascending doses (SAD) and multiple ascending doses (MAD) of Hypothiocyanite (OSCN-)…
- Trial name
- NCT02598999
- Testing
- ALX-009, OSCN-, bLF
- Where
- France
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Alaxia SAS
Pelizaeus-Merzbacher Disease, PMD
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
- Trial name
- NCT01391637
- Testing
- HuCNS-SC transplant in the lead-in phase
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- StemCells, Inc.
Preterm Labor, Preterm Birth
MIRACLE of LIFE Study
The goal of this observational study is to develop and validate cell-free RNA-based biomarkers for predicting a variety of adverse pregnancy outcomes in a pregnant person population. The main question it aims to answer are: 1.
- Conditions
- Preterm Labor, Preterm Birth, Preterm Birth Complication, Preterm Premature Rupture of Membrane
- Trial name
- MoL (NCT06074601)
- Testing
- Mirvie Predictive Test for Adverse Pregnancy Outcomes
- Where
- 10 sites in United States
- Volunteers
- 10,000 people
- Sponsor
- Mirvie
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn More About How Much of the Study Treatment Elinzanetant (or BAY3427080) Gets Absorbed, How Safe it is and How it Affects the Body in Healthy Female and Male Participants
Researchers are looking for a better way to treat men and women with vasomotor symptoms, a condition of having hot flashes caused by hormonal changes.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05351892
- Testing
- Elinzanetant (BAY3427080)
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Bayer
Cystic Fibrosis
Safety, Tolerability and Pharmacokinetics of Tiotropium in Cystic Fibrosis Patients
Study to obtain information about the safety and tolerability of tiotropium bromide administered via the Respimat® inhalation device in pediatric (≤11 y.o.) and adolescent/adult (≥12 y.o.) cystic fibrosis (CF) patients after…
- Trial name
- NCT02172534
- Testing
- Tiotropium bromide low, Tiotropium bromide medium, Tiotropium bromide high
- Volunteers
- 113 people
- Sponsor
- Boehringer Ingelheim
Haemophilia A
Adverse Event Data Collection From the EUHASS Registry on Turoctocog Alfa Pegol
This non-interventional study concerns a safety data collection based on adverse event data from a third-party registry (European Haemophilia Safety Surveillance System, EUHASS) that includes information about adverse events from…
- Trial name
- NCT04682145
- Testing
- Turoctocog alfa pegol
- Where
- Denmark
- Volunteers
- 50 people
- Sponsor
- Novo Nordisk A/S
Cystic Fibrosis
A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del
This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for…
- Trial name
- NCT04105972
- Testing
- ELX/TEZ/IVA, TEZ/IVA, IVA
- Where
- 35 sites in Australia, Belgium and 2 more
- Volunteers
- 176 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Retinitis Pigmentosa, Leber Congenital Amaurosis
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101).
- Trial name
- NCT05203939
- Testing
- OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose
- Where
- 7 sites in United States
- Volunteers
- 22 people
- Sponsor
- Ocugen
Primary Immune Deficiency, Secondary Immune Deficiency
Study of Immune Deficiency Patients Treated With Subcutaneous Immunoglobulin (IgPro20, Hizentra®) on Weekly and Biweekly Schedules
The purpose of the study is to assess the impact of immunoglobulin treatment by subcutaneous injections every 2 weeks (biweekly) on a cohort of patients with primary and secondary immune deficiencies.
- Trial name
- NCT02711228
- Testing
- Subcutaneous Immune Globulin (Human) (Hizentra)
- Where
- 2 sites in Canada
- Volunteers
- 26 people
- Sponsor
- CSL Behring
Hemophilia A
Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
The aim of the HEM-POWR study is to understand better how Damoctocog alfa pegol (Jivi) is used to treat people with Hemophilia A in day-to-day life, how well the treatment is tolerated and how satisfied patients and physicians…
- Trial name
- HEM-POWR (NCT03932201)
- Testing
- Damoctocog alfa pegol (Jivi, Bay94-9027)
- Where
- 29 sites in United States, Belgium and 18 more
- Volunteers
- 371 people
- Sponsor
- Bayer
Prader-Willi Syndrome
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.
- Trial name
- NCT04697381
- Testing
- somatropin - GH naïve pediatric cohort, somatropin - GH treated cohort, somatropin - adult cohort
- Where
- 5 sites in Japan
- Volunteers
- 33 people
- Sponsor
- Pfizer
X-linked Hypophosphatemia (XLH)
Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia
The purpose of this study is to better understand how X-linked hypophosphatemia (XLH) affects the body and daily life. Phosphate levels are critical in managing XLH, we aim to study how these levels change in patients taking…
- Trial name
- IBIS XLH (NCT07607600)
- Testing
- No treatment given
- Where
- 23 sites in United States, Australia and 6 more
- Sponsor
- Novo Nordisk A/S
Streptococcus Agalactiae
Feasibility Study for Identifying Anti Capsular Antibody Protection Against Invasive Group B Streptococcus (GBS) Disease in Newborns of 0-6 Days Age (Early Onset Disease [EOD]) as Well as Among Infants of 7-90 Days Age (Late Onset Disease [LOD])
The purpose of this study is to evaluate antibody levels against Group B streptococcus in mothers and the risk of developing invasive Group B streptococcus disease in newborns of less than 6 days age as well as infants of age…
- Trial name
- NCT01888471
- Testing
- Blood sample, Vaginal swab sample
- Where
- South Africa
- Volunteers
- 3,033 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Atopic Dermatitis (AD)
A Phase 3 Trial of OPA-15406 Foam in Children and Infants With Atopic Dermatitis
To investigate the superiority of 0.3% OPA-15406 foam to the vehicle in children with atopic dermatitis (AD)
- Trial name
- NCT07184645
- Testing
- OPA-15406, Vehicle
- Where
- Japan
- Volunteers
- 198 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Gout
Food Effect Study of Febuxostat XR in Healthy Participants
The purpose of this study is to assess the effect of food on the bioavailability of febuxostat after a single oral dose of 80 mg febuxostat XR and to evaluate the pharmacokinetics (PK) of febuxostat after single oral doses of 40…
- Trial name
- NCT02374164
- Testing
- Febuxostat XR
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Takeda
Fragile X Syndrome
Study of SPG601 in Adult Men With Fragile X Syndrome
This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
- Trial name
- NCT06413537
- Testing
- SPG601
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Spinogenix
Hemophilia A
Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry & Jivi: A Nested Study Within an Existing Registry
This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives.
- Trial name
- NCT03603275
- Testing
- New FVIII products
- Where
- 27 sites in United States
- Volunteers
- 3 people
- Sponsor
- Bayer
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