Clinical trials: Effect Neonatal
Fragile X Syndrome (FXS), Autism Spectrum Disorder (ASD)
A Study to Investigate the Effects and Safety of Codabakalner for the Treatment of FXS in Male Participants and Adult Participants With ADS
This is a Study to Investigate the Effects and Safety of Codabakalner for the Treatment of Fragile X Syndrome in Male Participants and Adult Participants with Autism Spectrum Disorders
- Trial name
- NCT07439510
- Testing
- Codabakalner
- Volunteers
- 120 people
- Sponsor
- Spinogenix
Dyslipidemias, Hypercholesterolemia
Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies
The study is a placebo-controlled, double-blind, randomized, phase 3 study in participants with heterozygous familial hypercholesterolemia (HeFH) and/or atherosclerotic cardiovascular disease (ASCVD) or multiple ASCVD risk…
- Conditions
- Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD
- Trial name
- TANDEM (NCT06005597)
- Testing
- Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe
- Where
- 45 sites in United States
- Volunteers
- 407 people
- Sponsor
- NewAmsterdam Pharma
Phenylketonurias
The Effects of CGMP in Children and Adults With PKU
Randomised controlled trial with a crossover design. For early and continuously treated patients with phenylketonuria (PKU) that are adherent.
- Trial name
- ELEMENT (NCT04076176)
- Testing
- CGMP protein substitute, L-amino acid protein substitute
- Where
- 3 sites in Denmark, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Vitaflo International, Ltd
Gout
A Study to Investigate the Clinical Effect and the Safety of PRX-115 Infused Intravenously at Different Dosing Regimens, With and Without Methotrexate, Versus Placebo in Adults Gout Patients (RELEASE)
This is a multicenter, randomized, double-blind, placebo-controlled phase II study assessing the efficacy, safety, and dosing regimen selection of multiple IV infusions of PRX-115 over 24 weeks, with or without MTX, versus the…
- Trial name
- RELEASE (NCT07280156)
- Testing
- PRX-115, Methotrexate (MTX)
- Where
- 35 sites in United States, Georgia and 1 more
- Volunteers
- 150 people
- Sponsor
- Protalix
Cystic Fibrosis
Study to Evaluate the Effect of KB001-A on Time-to-Need for Antibiotic Treatment
The purpose of this study is to confirm and extend the Phase 1-2 KB001 findings of an airway anti-inflammatory effect in CF individuals with chronic Pseudomonas aeruginosa (Pa) airway infection.
- Trial name
- KB001-A (NCT01695343)
- Testing
- KB001-A
- Where
- 67 sites in United States, Australia and 2 more
- Volunteers
- 182 people
- Sponsor
- Humanigen, Inc.
Healthy Volunteers
Evaluation Of The Potential Effect That The Administration Of Food Or Antacid Medication May Have In The Oral Absorption Of Dacomitinib (PF-00299804)
Evaluation of the potential effect that the administration of food or antacid medication may have in the oral absorption of dacomitinib relative to the administration of dacomitinib in absence of food or antacid medication
- Trial name
- NCT01702506
- Testing
- dacomitinib fasted, dacomitinib fed, dacomitinib+antacid
- Where
- Belgium
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Pfizer
Mucopolysaccharidosis (MPS), Hunter Syndrome
A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter…
- Trial name
- NCT05494593
- Testing
- ELAPRASE, Rituximab, Methotrexate
- Where
- 8 sites in United States
- Sponsor
- Takeda
Fetal Hypoxia, Fetal Distress
Trans-abdominal Fetal Pulse Oximetry
The Raydiant Oximetry Sensing System (Lumerah) is a non-invasive fetal pulse oximeter that measures fetal arterial oxygen saturation using safe, non-invasive, transabdominal near-infrared spectroscopy.
- Conditions
- Fetal Hypoxia, Fetal Distress, Fetal Acidemia, Fetal or Neonatal Effect of Complication of Labor
- Trial name
- NCT05147584
- Testing
- Dual fetal oxygen sensors
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Raydiant Oximetry, Inc.
Duchenne Muscular Dystrophy
Safety and Efficacy of P-188 NF in DMD Patients
This is an open-label study to evaluate the safety, tolerability and efficacy of daily, subcutaneous dosing with P-188 NF (Carmeseal-MD™) in non-ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03558958
- Testing
- P-188 NF
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Phrixus Pharmaceuticals, Inc.
Hereditary Tyrosinemia, Type I
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to compare the bioavailability of the Test Product, Nitisinone 10 mg Tablet, under fasting and fed conditions (food-effect).
- Trial name
- NCT02750332
- Testing
- Nitisinone
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Atopic Dermatitis
A Clinical Trial of BMX-010 in Adult Subjects With Atopic Dermatitis
Phase 2 clinical trial conducted in 2 parts: Part 1 - Pharmacokinetics and Part 2 - Randomized and Placebo Controlled (subject and clinical assessors will be blinded). Study Product will be applied to AD BID days 1-28.
- Trial name
- NCT05491447
- Testing
- BMX-010
- Where
- 19 sites in United States
- Volunteers
- 103 people
- Sponsor
- BioMimetix JV, LLC
Hereditary Angioedema
A Relative Bioavailability Study of Two Formulations of BCX7353
This is an open-label, randomized study to investigate the relative bioavailability of two formulations of BCX7353 and to determine if there is a food effect
- Trial name
- NCT03202784
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Fragile X Syndrome, Autism
Effects of CX516 on Functioning in Fragile X Syndrome and Autism
This study will investigate whether CX516 can improve attention, memory, language, or behavior in adults with Fragile X Syndrome and/or Autism. CX516 is an AMPAKINE® compound. AMPAKINE compounds enhance synaptic strength.
- Trial name
- NCT00054730
- Testing
- CX516 (Ampalex®)
- Where
- 2 sites in United States
- Sponsor
- RespireRx
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Skin Laxity
Bipolar RF Microneedling for Improved Laxity and Lift of the Submental and Neck Tissue and Effect on Elastin
This study is being conducted to evaluate the safety and efficacy of bipolar fractional radiofrequency treatment via use of the Profound System to achieve lift in lax submental (beneath the chin) and neck tissue
- Trial name
- NCT04245696
- Testing
- Dermal Handpiece / SubQ Handpiece
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Candela Corporation
To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks.
- Conditions
- To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell, Sickle Cell Anemia, Sickle Cell Disorders
- Trial name
- NCT04134299
- Testing
- AXA4010
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- Axcella Health, Inc
Amyloid Plaque, Beta-Amyloid
A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)
The purpose of this study is to assess the safety, tolerability, immunogenicity and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer's disease and in non-demented adults with Down syndrome.
- Conditions
- Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease
- Trial name
- NCT05462106
- Testing
- ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a
- Where
- 26 sites in United States, Spain and 1 more
- Volunteers
- 304 people
- Sponsor
- AC Immune SA
Alpha-1 Antitrypsin Deficiency
Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.
- Trial name
- NCT02900183
- Testing
- ARC-AAT Injection
- Where
- 4 sites in Canada, Ireland and 2 more
- Sponsor
- Arrowhead Pharmaceuticals
Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP)
A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
The purpose of this study is to evaluate the effect of givosiran on the pharmacokinetics of the 5-probe cocktail of midazolam, caffeine, losartan, omeprazole, and dextromethorphan, and their metabolites, in asymptomatic patients…
- Conditions
- Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP), Porphyria, Acute Intermittent, Acute Porphyria
- Trial name
- NCT03505853
- Testing
- Givosiran, 5-probe cocktail
- Where
- Sweden
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Duchenne Muscular Dystrophy
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT02329769
- Testing
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical