Clinical trials: Amino Acid Metabolism
Urea Cycle Disorder
Manipulating the Gut Microbiome Study
The objective is to determine if acetohydroxamic acid (AHA) can prevent hydrolysis of urea by inhibiting the bacterial urease of gut flora of both healthy control adults as well as adults with urea cycle disorders
- Trial name
- NCT03181828
- Testing
- Acetohydroxamic Acid Oral Tablet, No treatment
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Nicholas Ah Mew
Phenylketonuria
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
This Phase 3 study will evaluate the safety, effectiveness, tolerability, and how the body processes JNT-517 in children, adolescents, and adults with phenylketonuria.
- Trial name
- NCT06971731
- Testing
- JNT-517
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 120 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Phenylketonuria
Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)
SYNB1934-CP-003 was designed as a 3-part, adaptive study consisting of a dose-escalating, open-label period (DEP; Part 1) of up to 15 weeks, followed by a 4-week, double-blind, placebo-controlled, randomized withdrawal period…
- Trial name
- SYNPHENY-3 (NCT05764239)
- Testing
- SYNB1934v1
- Where
- 21 sites in United States, Canada and 2 more
- Volunteers
- 35 people
- Sponsor
- Synlogic
Arginase I Deficiency, Hyperargininemia
A Study of AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency
The purpose of this study is to determine the safety, tolerability, and efficacy of long-term intravenous and subcutaneous administration of pegzilarginase for the treatment of pediatric and adult patients with ARG1-D who…
- Trial name
- NCT03378531
- Testing
- AEB1102
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 14 people
- Sponsor
- Immedica Pharma AB
Allogeneic Stem Cell Transplantation
Study Comparing Valganciclovir Versus Ganciclovir in Patients Following Allogeneic Stem Cell Transplantation
The objective of this study is to assess the efficacy and safety of oral valganciclovir versus intravenous ganciclovir in patients following allogenic stem cell transplantation.
- Trial name
- CONVINCE (NCT01185223)
- Testing
- Valganciclovir, Ganciclovir
- Where
- 17 sites in Austria, Germany and 1 more
- Volunteers
- 212 people
- Sponsor
- Pierrel Research Europe GmbH
Phenylketonuria
Sapropterin in Individuals With Phenylketonuria
Investigators at Washington University will examine the effects of sapropterin (Kuvan) on brain and cognition in individuals with phenylketonuria (PKU) using neuropsychological and neuroimaging procedures.
- Trial name
- NCT00730080
- Testing
- Sapropterin (Kuvan)
- Where
- United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Arginase I Deficiency, Hyperargininemia
A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency
This is an open-label, multicenter study to evaluate the safety of weekly SC administration of pegzilarginase over 12 months in subjects with ARG1-D.
- Trial name
- NCT05676853
- Testing
- Pegzilarginase
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- Aeglea Biotherapeutics
Hereditary Tyrosinemia, Type I
Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine
he purpose of this study is to determine whether Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg
- Trial name
- Orfadin (NCT04113772)
- Testing
- Nitisinone, Orfadin
- Where
- India
- Volunteers
- 4 people
- Sponsor
- Sutphin Drugs
Cytomegalovirus Infections, HIV Infections
A Randomized, Double-Blind Study of the Efficacy and Safety of Oral Ganciclovir for the Prevention of CMV Disease in People Infected With the Human Immunodeficiency Virus
To evaluate the efficacy of oral ganciclovir in preventing progression to cytomegalovirus (CMV) disease (e.g., CMV retinitis, gastrointestinal CMV disease) in CMV-infected people with HIV infection and CD4 lymphocyte counts <=…
- Trial name
- NCT00002095
- Testing
- Ganciclovir
- Where
- 19 sites in United States
- Volunteers
- 700 people
- Sponsor
- Hoffmann-La Roche
Metabolic Diseases
Glycemic Response of Co-ingesting Carbohydrate and Amino Acids
The study will investigate if consumption of amino acid mixtures results a change in glycemic response of carbohydrate (white rice) and insulinemic response at different doses and timing (before consumption of carbohydrate).
- Trial name
- NCT02455765
- Testing
- Rice and Amino Acid mixtures
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Singapore Institute of Food and Biotechnology Innovation
Multimorbidity, Cardiovascular Diseases
Chuvashia Inappropriate Prescribing Study
This is a cross-sectional study based on an outpatient registry. All medical therapy will be registered in order to assess inappropriate prescribing's, in accordance with the criteria PROMPT and BEERS, also in accordance with the…
- Trial name
- CHIP (NCT06845397)
- Where
- Russia
- Volunteers
- 300 people
- Sponsor
- National Medical Research Center for Therapy and Preventive Medicine
Phenylketonurias
Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.
The study aim is to follow up body growth(body weight by kg, length by cm, head circumference, abdominal circumference, and body mass index ) and mental development of infants on phenylalanine restricted diet in comparison with…
- Trial name
- NCT04302194
- Testing
- phenylalanine restricted diet
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Assiut University
Phenylketonuria, Mild Hyperphenylalaninemia
Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
Phenylketonuria (PKU) is a genetic disorder known to cause severe reduction in intelligence and deficits in cognitive function; it is associated with an elevated level of Phenylalanine (Phe) in blood.
- Trial name
- NCT01924026
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- The Hospital for Sick Children
Ocular Albinism (OA), Oculocutaneous Albinism (OCA)
The Effects of Lutein and Zeaxanthin Supplementation on Vision in Patients With Albinism
The LUVIA study is a randomized placebo-controlled trial designed to investigate the effects of lutein and zeaxanthin supplementation on macular pigment and visual function in ocular or oculocutaneous albinism.
- Trial name
- LUVIA (NCT02200263)
- Testing
- Lutein plus Zeaxanthin
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Johns Hopkins University
Phenylketonuria (PKU)
A Long-Term Study of JNT-517 in Participants With Phenylketonuria
The goal of this Phase 3, open-label study is to evaluate the long-term safety of JNT-517 in pediatric and adult participants with Phenylketonuria (PKU) after completion of either Study JNT517-101 (NCT05781399) or JNT517-201…
- Trial name
- NCT06628128
- Testing
- JNT-517
- Where
- 12 sites in United States, Australia
- Volunteers
- 240 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Propionic Acidemia
AAVrh10-PCCA Gene Therapy for Propionic Acidemia
Propionic acidemia is a genetic metabolic disorder characterized by metabolic acidosis, ketosis, vomiting, lethargy, cognitive impairment, and risk of death.
- Trial name
- NCT07643844
- Testing
- AAVrh10-PCCA low dose, AAVrh10-PCCA middle dose, AAVrh10-PCCA high dose
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Mayo Clinic
Diabetes Mellitus, Type 2, Delayed Wound Healing
Hyaluronic Acid Treatment of the Post-extraction Tooth Socket Healing in Subjects With Diabetes Mellitus Type 2
The aim of the present split-mouth study was to investigate the effect of Hyaluronic Acid (HA) in improving the post-extraction tooth socket healing in subjects with diabetes mellitus type 2.
- Conditions
- Diabetes Mellitus, Type 2, Delayed Wound Healing, Tooth Extraction Status Nos
- Trial name
- NCT05896319
- Testing
- Hyaluronic acid gel
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- University of Turin, Italy
Phenylketonuria
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® (sapropterin dihydrochloride) in subjects aged 4 to 18 years who have phenylketonuria and with elevated…
- Trial name
- NCT01732471
- Testing
- Kuvan®
- Where
- Germany
- Volunteers
- 90 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Inherited Epidermolysis Bullosa, Ichthyosis
FARD (RaDiCo Cohort) (RaDiCo-FARD)
The goal of this observational study is to conduct a prospective assessment of the individual Burden of 9 rare skin diseases to assess disability in the broadest sense of the term (psychological, social, economic and physical)…
- Conditions
- Inherited Epidermolysis Bullosa, Ichthyosis, Ectodermal Dysplasia, Incontinentia Pigmenti
- Trial name
- FARD (NCT05954416)
- Where
- 15 sites in France
- Volunteers
- 900 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Esophageal Cancer
Outside the Cage (OTC) Robotic Esophagectomy
The goal of this clinical trial is to evaluate the feasibility and safety of a novel non-intercostal robotic approach for minimally invasive esophagectomy (OTC MIE) in the treatment of esophageal cancer.
- Trial name
- NCT06735638
- Testing
- OTC MIE Esophagectomy
- Where
- Canada
- Volunteers
- 31 people
- Sponsor
- Centre hospitalier de l'Université de Montréal (CHUM)