Clinical trials: Protein Metabolism Disorder
- Condition Protein Metabolism Disorder
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Hypophosphatasia
Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT02496689
- Testing
- asfotase alfa
- Where
- 5 sites in United States, France
- Sponsor
- Alexion Pharmaceuticals, Inc.
PKU, Phenylketonurias
Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute
The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU.
- Conditions
- PKU, Phenylketonurias, Inborn Errors of Metabolism, Metabolic Disease
- Trial name
- NCT04309331
- Testing
- Motion
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- Dr. Schär AG / SPA
Phenylketonuria (PKU)
PKU EASY Microtabs Plus - Acceptability and Tolerance
PEMP-AT is a prospective, single-arm, open-label, 1-week acceptability study to evaluate PKU EASY Microtabs Plus for the dietary management of participants with phenylketonuria (PKU).
- Trial name
- PEMP-AT (NCT06694662)
- Testing
- PKU EASY Microtabs Plus
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Galen Limited
Phenylketonurias, PKU
Evaluation of PKU Sphere in Italy
20 participants with PKU will build-up their dietary intake of PKU sphere over 2-16 weeks, depending on their level of metabolic control whilst doing so.
- Trial name
- NCT04679467
- Testing
- PKU sphere
- Where
- 4 sites in Italy
- Volunteers
- 13 people
- Sponsor
- Vitaflo International, Ltd
Hypophosphatasia
Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
This Clinical Trial is being conducted to study Hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT00894075
- Testing
- ENB-0040
- Sponsor
- Alexion Pharmaceuticals, Inc.
Phenylketonuria (PKU)
PANDA: PKU Amino Acid Evaluation
Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine…
- Trial name
- PANDA (NCT04086511)
- Where
- 2 sites in Belgium, United Kingdom
- Volunteers
- 37 people
- Sponsor
- Nutricia Research
Phenylketonuria
GMP Drink for PKU Study
This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).
- Trial name
- NCT02915510
- Testing
- Glycomacropeptide-based protein substitute
- Where
- 5 sites in United Kingdom
- Volunteers
- 40 people
- Sponsor
- Nutricia UK Ltd
PKU, Phenylketonurias
Market Research - Acceptability Study for a New PKU Protein Substitute
The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU.
- Conditions
- PKU, Phenylketonurias, Inborn Errors of Metabolism, Metabolic Disease
- Trial name
- NCT04318509
- Testing
- GMPOWER
- Where
- 2 sites in United Kingdom
- Volunteers
- 8 people
- Sponsor
- Dr. Schär AG / SPA
Cow's Milk Protein Allergy, Eosinophilic Gastrointestinal Disorders
A Study to Monitor the Use of an Amino Acid-Based Infant Formula
To assess the frequency and nature of adverse events in infants fed a free amino acid based infant formula.
- Conditions
- Cow's Milk Protein Allergy, Eosinophilic Gastrointestinal Disorders, Malabsorption/Maldigestion, Short Bowel Syndrome
- Trial name
- EAGLE (NCT02953223)
- Testing
- Free amino acid infant formula
- Where
- 29 sites in United States
- Volunteers
- 144 people
- Sponsor
- Société des Produits Nestlé (SPN)
RDS of Prematurity, Surfactant Protein B Deficiency
Administration of Surfactant Through an Instillation Device Infasurf® (Calfactant) in Neonates- A Pilot Study
A pilot study to explore and determine feasibility and safety of the administration of surfactant through a new supraglottic device in patients from 500 to 5000 grams with RDS.
- Conditions
- RDS of Prematurity, Surfactant Protein B Deficiency, Chronic Lung Disease
- Trial name
- NCT04056741
- Testing
- surfactant airway device
- Where
- 4 sites in United States
- Volunteers
- 55 people
- Sponsor
- ONY
Hypophosphatasia
Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01176266
- Testing
- asfotase alfa
- Where
- 23 sites in United States, Australia and 10 more
- Volunteers
- 69 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Phenylketonurias
Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria
Thirty eligible PKU patients (≥ 8 years), identified as tablet protein substitute users, will be recruited. Patients will firstly observe a 7-day baseline period, in which participants will continue with their existing diet…
- Trial name
- NCT03333720
- Testing
- Phenylalanine-free protein substitute tablets
- Sponsor
- Nutricia UK Ltd
Tyrosinemia, Type I, Tyrosinemia, Type II
Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies
The purpose of this prospective, observational study is to evaluate the tolerability and acceptability of Zero minis, a range of protein substitute tablets for use in the dietary management of children with either TYROSINAEMIA…
- Conditions
- Tyrosinemia, Type I, Tyrosinemia, Type II, Tyrosinemia, Type III, Alkaptonuria
- Trial name
- ZeroMinisMR (NCT06298292)
- Testing
- Zero minis (range of protein substitutes in tablet form)
- Volunteers
- 15 people
- Sponsor
- metaX Institut fuer Diatetik GmbH
Phenylketonurias
Evaluation of Low Phenylalanine Formulas
Ajinomoto Cambrooke has developed a PKU protein substitute that is a proprietary blend of purified Glycomacropeptide (GMP) and essential amino acids, under the brand name Glytactin®.
- Trial name
- NCT06332105
- Testing
- Glytactin, L-AA
- Where
- 2 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Ajinomoto Co., Inc.
Cerebral Adrenoleukodystrophy (CALD)
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major…
- Trial name
- Stargazer (NCT06224413)
- Testing
- No Intervention
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- Genetix Biotherapeutics Inc.
PKU
Evaluation of PKU UP
PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU).
- Trial name
- PKU UP (NCT05995717)
- Testing
- PKU UP
- Where
- 7 sites in United Kingdom
- Volunteers
- 16 people
- Sponsor
- Vitaflo International, Ltd
Phenylketonuria (PKU)
PKU Microtablets Case Studies
Phenylalanine (Phe) free protein substitutes are typically presented in ready to drink liquid or powder format and are made up with water to a set volume.
- Trial name
- NCT07671859
- Testing
- PKU Microtablet protein substitute
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Nutricia UK Ltd
Anemia, Iron-deficiency
Improvement of Fatigue Symptoms in the Iron Deficiency Anemia With Iron Succinylate Therapy
The treatment of anemia depends on its cause. Patients with underlying iron-deficient anemia should be treated or referred to a specialist (eg gynecologist, gastroenterologist) for treatment.deficiency anemia are global health…
- Conditions
- Anemia, Iron-deficiency, Iron Deficiency Anemia, Fatigue
- Trial name
- IFAISTOS (NCT03873584)
- Testing
- Fatigue
- Sponsor
- Elpen Pharmaceutical Co. Inc.
Hypophosphatasia
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042).
- Trial name
- NCT01205152
- Testing
- asfotase alfa
- Where
- 8 sites in United States, United Arab Emirates and 1 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.
- Trial name
- NCT00952484
- Testing
- asfotase alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.