Clinical trials: Newborn Diseases
Non-squamous Metastatic Non-Small-Cell Lung Carcinoma
NIS to Examine the Effectiveness of TDC in Patients With Metastatic Non-squamous NSCLC and High-risk Genetic Alterations
This prospective, multicenter, non-interventional study (NIS) in Germany aims to collect real-life data of patients with non-squamous (NSQ) metastatic non-small cell lung cancer (mNSCLC) (incl.
- Trial name
- NAUTIC (NCT06494540)
- Where
- 30 sites in Germany
- Volunteers
- 600 people
- Sponsor
- AstraZeneca
Hereditary Angioedema, Pediatric
Berotralstat Treatment in Children With Hereditary Angioedema
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years of age for prophylactic…
- Trial name
- APeX-P (NCT05453968)
- Testing
- Berotralstat
- Where
- 15 sites in Austria, Canada and 8 more
- Volunteers
- 29 people
- Sponsor
- BioCryst Pharmaceuticals
Cystic Fibrosis, Steatorrhea
An Effectiveness, Safety, and Palatability Study of Pancrelipase Microtablets in Infants and Toddlers With Cystic Fibrosis and Fat Malabsorption
The purpose of this study is to evaluate the effectiveness and safety of PANCREASE MT (pancrelipase microtablets) to improve steatorrhea (excessive excretion of fat in feces) in infants and toddlers with cystic fibrosis who have…
- Trial name
- NCT00217204
- Testing
- Pancrelipase microtablets
- Volunteers
- 18 people
- Sponsor
- McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.
Hemophilia B
Registry For Patients Treated With BeneFix In Usual Care Setting In Germany
The purpose of this observational study is to describe the incidence of adverse events among patients treated with BeneFix® in usual health care settings in Germany.
- Trial name
- NCT00714415
- Testing
- BeneFIX
- Where
- 21 sites in Austria, Germany
- Volunteers
- 80 people
- Sponsor
- Pfizer
Mucopolysaccharidosis Type II (MPS II)
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system.
- Trial name
- NCT03566043
- Testing
- RGX-121
- Where
- 5 sites in United States, Brazil
- Volunteers
- 48 people
- Sponsor
- REGENXBIO Inc.
Hemophilia A
Study Evaluating Inhibitor Specificity in Hemophilia A
This investigation will utilize a systematic determination of anti-FVIII antibody specificity in PTPs (> 50 ED) with hemophilia A who have developed inhibitors in response to treatment with any FVIII product(s).
- Trial name
- NCT00151385
- Testing
- single blood draw
- Where
- 23 sites in United States, Belgium and 5 more
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory…
- Trial name
- CMT4J (NCT03810508)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Neurogene Inc.
Atopic Dermatitis, Dermatology
A Prospective, Observational Study of Clinician and Patient-reported Outcomes in Patients With Dermatological Conditions
The primary goal of the study is to collect standardized patient and clinician reported outcome measures for patients diagnosed with a range of dermatological conditions in an academic clinical practice setting.
- Conditions
- Atopic Dermatitis, Dermatology, Vitiligo, Alopecia Areata
- Trial name
- ClinPRO-DERM (NCT05084417)
- Testing
- N/A This is an observational study
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- OM1, Inc.
Familial Adenomatous Polyposis
A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
The purpose of this phase III study is to evaluate the safety and efficacy of the combination of eflornithine and sulindac compared to single agent sulindac or eflornithine in reducing the number of polyps in patients with…
- Trial name
- NCT01245816
- Testing
- Eflornithine plus Sulindac
- Sponsor
- Cancer Prevention Pharmaceuticals, Inc.
Ornithine Transcarbamylase Deficiency
Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.
- Trial name
- NCT04416126
- Testing
- ARCT-810
- Where
- New Zealand
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Arcturus Therapeutics, Inc.
Homozygous Familial Hypercholesterolemia
Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor
The primary objective of this study is to evaluate the safety and tolerability of 4 doses of lomitapide (AEGR-733; BMS-201038) given as an initial low dose and then escalated through an additional 3 dose levels over a 16-week…
- Trial name
- NCT01556906
- Testing
- Lomitapide
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Aegerion Pharmaceuticals, Inc.
Familial Lipoprotein Lipase Deficiency
Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects
This trial is designed to expand the currently available data on the safety and efficacy of alipogene tiparvovec treatment in lipoprotein lipase deficiency (LPLD) and to further the understanding of possible mechanisms of action…
- Trial name
- NCT00891306
- Testing
- Alipogene Tiparvovec (AMT-011), Human LPL [S447X], mycophenolate mofetil, cyclosporine
- Where
- 2 sites in Canada
- Volunteers
- 5 people
- Sponsor
- Amsterdam Molecular Therapeutics
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617847
- Testing
- WVE-120101
- Where
- 18 sites in Australia, Canada and 5 more
- Volunteers
- 27 people
- Sponsor
- Wave Life Sciences USA, Inc.
Dermatitis Atopic
A Study to Investigate Vaccine Responses in Subcutaneous Amlitelimab Treated Atopic Dermatitis Participants Aged 18 Years and Older Compared With Placebo
This is a Phase 2, multicenter, randomized, double-blind placebo controlled, 2-arm study to evaluate the effect of amlitelimab on vaccine antibody responses, and the safety of amlitelimab concurrently administered with non-live…
- Trial name
- HYDRO (NCT06015308)
- Testing
- Amlitelimab, Tdap vaccine, PPS vaccine
- Where
- 57 sites in United States, Canada
- Volunteers
- 224 people
- Sponsor
- Sanofi
Dermatitis, Atopic
Study of the Effectiveness and Safety of Desloratadine (Aerius) Syrup in Children With Allergic Skin Inflammation (P03475)
The purpose of this study was to test the effectiveness and safety of desloratadine (Aerius) syrup in children with allergic skin inflammation. Patients took desloratadine syrup once a day for 28 days.
- Trial name
- NCT00817076
- Testing
- desloratadine
- Volunteers
- 40 people
- Sponsor
- Organon and Co
Atopic Dermatitis
Topical YR001 Ointment in Adult With Mild to Moderate Atopic Dermatitis
This study is to evaluate the safety, tolerability, and pharmacokinetics of YR001 topical ointment in adult patients with mild to moderate atopic dermatitis
- Trial name
- NCT06309355
- Testing
- YR001 ointment
- Where
- 4 sites in United States
- Volunteers
- 35 people
- Sponsor
- Hangzhou Yirui Pharmaceutical Technology Co., Ltd
Atopic Dermatitis
Dupilumab Skin Barrier Function Study in Atopic Dermatitis
Primary Objective: Evaluate changes in skin barrier function with transepidermal water loss (TEWL) assessed after skin tape stripping (STS) in pre-defined lesional skin in participants with moderate to severe atopic dermatitis…
- Trial name
- BALISTAD (NCT04447417)
- Testing
- Dupilumab SAR231893
- Where
- 2 sites in United States, Canada
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Sanofi
Hemoglobinopathies, Myelodysplastic Syndromes
Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients
- Conditions
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome
- Trial name
- NCT00673608
- Testing
- deferasirox
- Where
- 4 sites in Australia
- Volunteers
- 118 people
- Sponsor
- Novartis
Alpha-Mannosidosis
Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis
The overall objective is to evaluate the long-term efficacy, safety and tolerability of repeated Lamazym i.v. treatment in patients 5-21 years of age with alpha-Mannosidosis
- Trial name
- NCT01681940
- Testing
- Lamazym
- Where
- 4 sites in Belgium, Denmark and 2 more
- Volunteers
- 10 people
- Sponsor
- Zymenex A/S
Hemophilia
Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery
This is an interventional, prospective, international, multicenter, single-arm, Phase 3, and sequential efficacy and safety study in adolescents and adults with congenital hemophilia A or B with inhibitors to factor VIII (FVIII)…
- Trial name
- SCOPE HIM (NCT05695391)
- Testing
- Coagulation Factor VIIa (Recombinant)
- Where
- 15 sites in United States, Malaysia and 4 more
- Volunteers
- 2 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
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