Clinical trials: Nutritional Deficiencies
X-Linked Hypophosphatemia
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.
- Trial name
- NCT05181839
- Testing
- Burosumab
- Where
- 14 sites in France, Germany and 3 more
- Volunteers
- 25 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Amyloidosis
Pro-arrhythmic Potential of GSK3039294 in Healthy Subjects
GSK3039294 has been developed to offer an orally available alternative to parenteral GSK2315698 (miridesap) for plasma serum amyloid P component (SAP) depletion prior to and following use of anti-SAP Monoclonal Antibody (mAb) in…
- Trial name
- NCT03567499
- Testing
- GSK3039294
- Where
- United Kingdom
- Volunteers
- healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Amyotrophic Lateral Sclerosis
A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)
The study will generate data on safety, tolerability and pharmacokinetics after multiple daily doses of CK-2017357 in patients with ALS.
- Trial name
- NCT01378676
- Testing
- CK-2017357 (Part A), Riluzole 50 MG (Part B), CK-2017357 (Part B)
- Where
- 9 sites in United States
- Volunteers
- 49 people
- Sponsor
- Cytokinetics
Amyotrophic Lateral Sclerosis
Efficacy and Safety of Tazbentetol in ALS Participants
The objectives of this study are to examine the effects of tazbentetol on clinical measures of ALS, patient reported outcomes (PROs), long-term safety and tolerability.
- Trial name
- NCT07325591
- Testing
- Tazbentetol
- Volunteers
- 430 people
- Sponsor
- Spinogenix
Iron Deficiency Anemia
Treatment of (IDA) by (FPC) Delivered Via Infusion Pump in Patients Receiving Home Infusion Therapy
The main purpose is to determine whether FPC, administered via infusion, is safe and effective for the treatment of iron deficiency anemia (IDA) in patients receiving Home Infusion therapies (HI).
- Trial name
- NCT05110768
- Testing
- Triferic AVNU
- Volunteers
- 75 people
- Sponsor
- Rockwell Medical Technologies, Inc.
Transthyretin Amyloid Cardiomyopathy
Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
The primary objective of this study is to access the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the total…
- Trial name
- DepleTTR-CM (NCT06183931)
- Testing
- ALXN2220
- Where
- 213 sites in United States, Argentina and 25 more
- Volunteers
- 1,182 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
This observational registry collects existing medical and routine care data from people with amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and other motor system disorders.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy, Spinal Cord Diseases, Central Nervous System Diseases
- Trial name
- NCT07900672
- Testing
- Standard of Care (Investigator Choice)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Amyotrophic Lateral Sclerosis (ALS)
Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS)
The primary objective of this study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip once a day in patients with ALS based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24…
- Trial name
- NCT00330681
- Testing
- MCI-186
- Volunteers
- 206 people
- Sponsor
- Shionogi
Iron Deficiency Anemia, Impaired Renal Function
Evaluation of Efficacy and Safety of Ferric Carboxymaltose (FCM) in Patients With Iron Deficiency Anemia and Impaired Renal Function
The primary objective of this study is to examine the efficacy and safety (cardiovascular) of an investigational intravenous (IV) iron, ferric carboxymaltose (FCM), compared to IV iron sucrose (Venofer) in subjects who have iron…
- Trial name
- REPAIR-IDA (NCT00981045)
- Testing
- Ferric Carboxymaltose (FCM), Iron Sucrose (Venofer)
- Where
- United States
- Volunteers
- 2,561 people
- Sponsor
- American Regent, Inc.
Amyotrophic Lateral Sclerosis (ALS)
The Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis
A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects with Amyotrophic Lateral Sclerosis
- Trial name
- NCT07118319
- Testing
- Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)
- Where
- China
- Volunteers
- 12 people
- Sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
Amyotrophic Lateral Sclerosis
Early Feasibility Study of the MyoRegulator® for Treatment of ALS
This is a single-center, single-arm, open-label study aiming to assess the safety and feasibility of the MyoRegulator® device when used to treat individuals with amyotrophic lateral sclerosis (ALS).
- Trial name
- ALSEFS (NCT06165172)
- Testing
- MyoRegulator®
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- PathMaker Neurosystems Inc.
Disease Related Malnutrition
Tolerance, Palatability and Acceptability Evaluation of an Oral Nutrition Supplement
To evaluate the tolerance (gastrointestinal) and acceptability of a new Oral Nutrition Supplement in patients with or at risk of disease related malnutrition.
- Trial name
- NCT07452523
- Testing
- AYMES SWIFT oral nutritional supplement, AYMES SWIFT FIBRE, AYMES SWIFT PLANT
- Where
- United Kingdom
- Volunteers
- 40 people
- Sponsor
- Aymes International Limited
Malnutrition, Child
Safety and Efficacy Study to Compare Smoflipid and Intralipid 20% in Pediatric Patients of 3 Months to 16 Years of Age
Evaluate the safety and efficacy of Smoflipid compared to standard of care lipid emulsion Intralipid 20% administered via a central vein in pediatric patients 3 months to 16 years of age who require parenteral nutrition for at…
- Trial name
- NCT03563222
- Testing
- Smoflipid, Intralipid, 20%
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Fresenius Kabi
Iron-deficiency
Safety and Effectiveness of IHAT in Iron Deficient Pre-menopausal Woman
This is a double blind, randomised, placebo-controlled trial to evaluate orally-dosed IHAT (iron hydroxide adipate tartrate) at 2 different doses compared to placebo for increasing serum ferritin levels in iron-deficient but…
- Trial name
- NCT05950841
- Testing
- Low dose IHAT, High Dose IHAT, Carob flour
- Where
- Australia
- Volunteers
- 180 people
- Sponsor
- RDC Clinical Pty Ltd
Amyotrophic Lateral Sclerosis (ALS)
Generation of Cell Banks and Biomarker Isolation From Patients With Amyotrophic Lateral Sclerosis (ALS) (HCD-SLA)
The aim of this research is to establish different cell banks from patients with amyotrophic lateral sclerosis (ALS).
- Trial name
- HCD-SLA (NCT07099066)
- Testing
- sampling blood
- Volunteers
- 50 people
- Sponsor
- Human Cell Design
Frontotemporal Dementia, Behavioral Variant Frontotemporal Dementia
Cognitive, Behavioral, and Functional Change in Behavioral Variant Frontotemporal Dementia (bvFTD)
The objectives of the study are to; (1) estimate the change in disease -related cognitive decline over 1 year on a battery of cognitive tests administered to participants with early-stage symptomatic Behavioral Variant…
- Trial name
- FORWARD (NCT03088956)
- Testing
- Assessments
- Where
- 12 sites in United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Biogen
Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS)
PRISM Registry: Pseudobulbar Affect Registry Series
PBA is a neurologic condition that is estimated to impact over a million patients and their families in the United States. PBA occurs secondary to an otherwise unrelated neurologic disease or injury, and manifests as involuntary…
- Conditions
- Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Parkinson's Disease
- Trial name
- NCT01366027
- Where
- United States
- Volunteers
- 5,290 people
- Sponsor
- Avanir Pharmaceuticals
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Malnutrition
A Study of an Oral Nutritional Supplement in a Malnourished Population With GI Tolerance Impairment
The purpose is to observe the compliance to a high-calorie, high protein peptide-based nutritional supplement after 3 months in a malnourished population with impaired gastrointestinal tolerance living in nursing homes and as…
- Trial name
- NCT02698540
- Testing
- Nutritional Supplement
- Where
- 23 sites in Spain
- Volunteers
- 95 people
- Sponsor
- Abbott Nutrition
Iron Deficiency Anemia
A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
Study AMAG-FER-CKD-253 is an extension study of the combined AMAG-FER-CKD-251 (NCT01155375) and AMAG-FER-CKD-252 (NCT01155388) studies to evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with…
- Trial name
- NCT01264679
- Testing
- Ferumoxytol
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- AMAG Pharmaceuticals, Inc.