Clinical trials: Congenital Deformity
Tuberous Sclerosis Complex, Seizures
An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)
This trial consists of 2 parts: a double-blinded phase and an open-label extension phase. The open-label extension phase only will be described in this record. All participants will receive the same dose of GWP42003-P.
- Trial name
- NCT02544750
- Testing
- GWP42003-P
- Where
- United States
- Volunteers
- 199 people
- Sponsor
- Jazz Pharmaceuticals
Fed
Bioequivalence Study of Olanzapine Orally Disintegrating Tablets, 5 mg Under Fed Condition
The purpose of this study is to compare the single oral dose bioequivalence of olanzapine 5 mg OD tablets with Zyprexa Zydis 5 mg tablets under fed conditions and to monitor adverse events and ensure safety of subjects.
- Trial name
- NCT01503450
- Testing
- Olanzapine OD Tablets 5 mg
- Where
- India
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
Retinitis Pigmentosa, Usher Syndrome Type 2
Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)
The purpose of this study is to evaluate the efficacy safety and tolerability of QR-421a administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene with…
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, Retinal Disease
- Trial name
- NCT05176717
- Testing
- QR-421a
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Laboratoires Thea
Osteogenesis Imperfecta
Safety, Pharmacokinetics and Pharmacodynamics of BPS804 in Osteogenesis Imperfecta
This is a randomized, open label intra-patient dose escalation study to evaluate safety and tolerability, pharmacokinetics, and pharmacodynamics of BPS804 in adults with osteogenesis imperfecta (OI).
- Trial name
- NCT01417091
- Testing
- BPS804
- Where
- 6 sites in United States, Belgium and 2 more
- Volunteers
- 10 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Pelizaeus-Merzbacher Disease
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
- Trial name
- NCT06150716
- Testing
- ION356
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
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
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