Clinical trials: Amino Acids
Mild Cognitive Impairment
A Retrospective Analysis of Cerefolin NAC® in Cognitively Impaired Patients
This retrospective analysis of de-identified data from 700 charts of consecutive patients from this patient population is to investigate the prevalence of hyperhomocysteinemia and associated metabolic abnormalities in this…
- Trial name
- NCT01594541
- Where
- United States
- Volunteers
- 700 people
- Sponsor
- Pamlab, Inc.
Urea Cycle Disorders, Organic Acidemias
At Home Ammonia Monitoring of Inborn Errors of Ammonia Metabolism
The goal of this observational study is to learn if people with certain ammonia metabolism disorders will measure their ammonia levels at home.
- Conditions
- Urea Cycle Disorders, Organic Acidemias, Fatty Acid Oxidation Disorder, Ammonia; Metabolic Disorder
- Trial name
- NCT06953505
- Testing
- Ammonia Study Device
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Sequitur Health Corp.
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Schizophrenia
Adaptive Phase II Study to Evaluate the Safety & Efficacy of NaBen®
The purpose of this study is to determine if NaBen® is a safe and effective add-on treatment for schizophrenia in adolescents.
- Trial name
- NCT01908192
- Testing
- NaBen®
- Where
- 23 sites in United States, Taiwan
- Volunteers
- 60 people
- Sponsor
- SyneuRx International (Taiwan) Corp
Ornithine Transcarbamylase Deficiency
Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency
The objectives of the study are to characterize urea production rates in patients with OTC, characterize the association of rate of ureagenesis and disease severity in OTC patients, characterize the association of rate of…
- Trial name
- NCT04717453
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Methylmalonic Acidemia, Propionic Acidemia
Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
The JUMP (Journey to Understand MMA and PA) Study is being conducted by HemoShear Therapeutics and AllStripes, a rare disease online research platform.
- Trial name
- JUMP (NCT05438485)
- Testing
- Registry
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 11 people
- Sponsor
- HemoShear Therapeutics
Tyrosinemia, AKU
Evaluation of TYR Sphere in France
10 participants aged 3 years and over with tyrosinaemia or alkaptonuria will be prescribed Tyr sphere following an assessment of their individual needs by their dietitian.
- Trial name
- TYR sphere Fra (NCT04761588)
- Testing
- Tyr sphere
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Vitaflo International, Ltd
Cow's Milk Allergy
Free Amino Acid-based Formula to Treat Children Cow's Milk Protein Allergy
It is a study to assess the tolerance to a new commercially available infant formula in children affected by Cow's Milk Allergy.
- Trial name
- NCT02536482
- Testing
- Amix
- Volunteers
- 15 people
- Sponsor
- Edetec Indústria Alimentícia S.A.
Phenylketonuria
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
- Trial name
- EPIPHENY (NCT06302348)
- Testing
- Sepiapterin
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 56 people
- Sponsor
- PTC Therapeutics
Ornithine Transcarbamylase Deficiency
Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g.
- Trial name
- NCT04442347
- Testing
- ARCT-810
- Where
- 8 sites in United States
- Volunteers
- 16 people
- Sponsor
- Arcturus Therapeutics, Inc.
Cytomegalovirus Retinitis, HIV Infections
A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )
To determine the clinically safe and effective dose of intravitreal ISIS 2922 alone and as an additive antiviral therapy to ganciclovir in AIDS patients with cytomegalovirus (CMV) retinitis.
- Trial name
- NCT00002156
- Testing
- Fomivirsen sodium, Ganciclovir
- Where
- 18 sites in United States
- Volunteers
- 194 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Cancer Patients With Cachexia
Evaluation of the Effects of a Mixture of Amino Acids (Amixea) on Lean Body Mass and Muscle Strengh of Patients With Unresectable Advanced Non-small Cell Lung Cancer
The primary objectives of this study are to evaluate the effects of an amino acid mixture (Amixea) on the body composition as assessed by DXA, and on QMVC as assessed by strain gauge, in advanced NSCLC patients, compared to…
- Trial name
- NCT02877966
- Testing
- Amixea
- Where
- 3 sites in Australia
- Volunteers
- healthy volunteers welcome
- Sponsor
- Helsinn Healthcare SA
Phenylketonuria (PKU)
A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
The primary objective of this clinical trial is to evaluate the safety and tolerability of single and multiple administrations of PL54 in patients aged 18-55 years.
- Trial name
- NCT07672756
- Testing
- PL54
- Where
- China
- Volunteers
- 48 people
- Sponsor
- Chongqing Peg-Bio Biopharm Co., Ltd.
Healthy Volunteers
Bioavailability of Different Collagen-based Treatments After Oral Intake
The goal of this clinical trial is to investigate the postprandial absorption of collagen and elucidate the absorption rate and bioavailability after oral intake of collagen-based products.
- Trial name
- NCT05722158
- Testing
- Dietary supplementation with the collagen-based product CP1., Dietary supplementation with the collagen-based product CP2., Dietary supplementation with the collagen-based product CP3.
- Where
- Spain
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Bionos Biotech S.L.
Ornithine Transcarbamylase (OTC) Deficiency
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
- Trial name
- CAPtivate (NCT03636438)
- Testing
- No Intervention
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 11 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
ARG1 Deficiency
Evaluation of the Safety of Loargys Arginine Test System in Loargys-treated Patients
Arginase 1 deficiency (ARG1-D) is a rare condition in which the body cannot properly break down a substance called arginine. This leads to high levels of arginine and related substances in the blood, which can cause serious…
- Trial name
- CLEAR (NCT07573059)
- Testing
- Loargys Arginine Test System
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Immedica Pharma US Inc
Phenylketonuria, Healthy
Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria
This Phase 1/2a, first-in-human, oral single and multiple dose-escalation, randomized, double-blinded, placebo-controlled study is evaluating SYNB1618 in healthy volunteers (HV) and subjects diagnosed with phenylketonuria (PKU)…
- Trial name
- PKU (NCT03516487)
- Testing
- SYNB1618
- Where
- 4 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Synlogic
Ornithine Transcarbamylase Deficiency
Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency
This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single escalating doses of MRT5201 administered intravenously to subjects with OTC Deficiency (OTCD).
- Trial name
- STEP-OTC (NCT03767270)
- Testing
- MRT5201
- Sponsor
- Translate Bio, Inc.
Phenylketonuria
Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
The purpose of this study is to assess the safety and tolerability of injections of rAvPAL-PEG in subjects with PKU.
- Trial name
- NCT00634660
- Testing
- rAvPAL-PEG
- Where
- 8 sites in United States
- Volunteers
- 25 people
- Sponsor
- BioMarin Pharmaceutical
Phenylketonurias
Evaluation of PKU Sphere Liquid
PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.
- Trial name
- NCT05096988
- Testing
- PKU sphere liquid
- Where
- 3 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Vitaflo International, Ltd