Clinical trials: Nutritional Deficiencies
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)
This study will be conducted to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NTLA-2001 in participants with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) and…
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Trial name
- NCT04601051
- Testing
- NTLA-2001
- Where
- 4 sites in France, New Zealand and 2 more
- Volunteers
- 72 people
- Sponsor
- Intellia Therapeutics
Malnutrition
TOLER-ENT Study: A Study to Investigate the Tolerance of the High Energy, High Protein Formula Sondalis® HP 2 kcal
A multicentre, prospective, open-label, single arm study for 2 months, involving adult patients under poorly tolerated Home Enteral Nutrition (HEN).
- Trial name
- SONDALIS (NCT05429853)
- Testing
- Sondalis® HP 2 kcal (with or without fibre)
- Where
- 2 sites in France
- Volunteers
- 17 people
- Sponsor
- Société des Produits Nestlé (SPN)
Chronic Kidney Disease; Iron Deficiency Anemia
A Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA Secondary Objective: To…
- Trial name
- NCT03619850
- Testing
- Ferumoxytol, Iron sucrose
- Where
- 21 sites in United States, Hungary and 3 more
- Volunteers
- 129 people
- Sponsor
- AMAG Pharmaceuticals, Inc.
Parkinson Disease, Amyotrophic Lateral Sclerosis
Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy
The purpose of this research study is to assess safety and tolerability of a single intravenous (given through a vein) dose of the investigational retinal tracer AMDX-2011P in patients with neurodegenerative diseases (Parkinson's…
- Trial name
- PROBE (NCT05542576)
- Testing
- AMDX2011P
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Amydis Inc.
Amyotrophic Lateral Sclerosis
Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis
A Phase I, single center, prospective, non-randomized, open label, safety/efficacy study of the infusion of autologous bone marrow-derived stem cells, in 6 patients with Amyotrophic Lateral Sclerosis according to established…
- Trial name
- ALS (NCT01082653)
- Testing
- autologous bone marrow-derived stem cells
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- TCA Cellular Therapy
Amyloidosis, AL Amyloidosis
Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis
This is a single arm, Open-Label, Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis.
- Trial name
- NCT05199337
- Testing
- ZN-d5
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 18 people
- Sponsor
- K-Group Alpha, Inc., a wholly owned subsidiary of Zentalis Pharmaceuticals, Inc.
Amyotrophic Lateral Sclerosis
Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Amisodin in Healthy Adult Subjects With Amyotrophic Lateral Sclerosis
Researchers will evaluate the safety, tolerability, and pharmacokinetics (PK) of orally administered Amisodin in healthy adult subjects through a randomized, double-blind, placebo-controlled Phase 1 study consisting of two parts…
- Trial name
- ALS (NCT07221240)
- Testing
- Amisodin
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- PRG Science & Technology Co., Ltd.
Renal Insufficiency, Chronic, Iron-Deficiency Anemia
Study With Oral Ferric Maltol for the Treatment of Iron Deficiency Anemia in Subjects With Chronic Kidney Disease
To evaluate the efficacy of oral ferric maltol compared with placebo in the treatment of IDA in subjects with CKD
- Trial name
- AEGIS-CKD (NCT02968368)
- Testing
- Ferric maltol
- Where
- 27 sites in United States
- Volunteers
- 167 people
- Sponsor
- Shield Therapeutics
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
Iron Deficiency Anaemia
Time to Relapse of Iron Deficiency Anaemia After Standard Treatment With The Intravenous Iron (Monofer®)
The objective is to monitor and quality assure the efficacy, including effects on quality of life, and safety of Monofer® in Chronic Kidney Disease and Inflammatory Bowel Disease patient populations when Monofer® is used…
- Trial name
- NCT02546154
- Testing
- 10% Iron Isomaltoside 1000
- Where
- United Kingdom
- Volunteers
- 359 people
- Sponsor
- Pharmacosmos UK Ltd
Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial
To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).
- Conditions
- Amyloidosis in Transthyretin (TTR), Amyloidosis, Familial, Amyloid Cardiomyopathy, Amyloid Cardiomyopathy, Transthyretin-Related
- Trial name
- NCT07116473
- Testing
- Acoramidis (AG10)
- Volunteers
- 250 people
- Sponsor
- Eidos Therapeutics, a BridgeBio company
ALS
Riluzole Oral Soluble Film Safety and Tolerability in Amyotrophic Lateral Sclerosis
The primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for…
- Trial name
- NCT03457753
- Testing
- Riluzole Oral Soluble Film
- Where
- United States
- Sponsor
- Aquestive Therapeutics
Primary Progressive Nonfluent Aphasia
A 24-month Phase 1 Pilot Study of AADvac1 in Patients With Non Fluent Primary Progressive Aphasia
This study is a pilot trial evaluating the safety and immunogenicity of AADvac1 in patients with the non-fluent variant of Primary Progressive Aphasia.
- Trial name
- AIDA (NCT03174886)
- Testing
- AADvac1 40 µg, AADvac1 160 µg
- Where
- 3 sites in Germany
- Volunteers
- 33 people
- Sponsor
- Axon Neuroscience SE
Granulin Mutation
Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.
- Trial name
- NCT02149160
- Testing
- FRM-0334
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 30 people
- Sponsor
- FORUM Pharmaceuticals Inc
FGF23-related Hypophosphataemic Rickets and Osteomalacia
Post Marketing Surveillance Study to Observe Safety and Effectiveness of CRYSVITA® in S. Korean Patients
The objective of this post-marketing surveillance (PMS) study is to assess the safety and effectiveness of CRYSVITA injection 10, 20, and 30mg, equivalent to in routine clinical settings
- Trial name
- NCT06202027
- Testing
- FGF23-related hypophosphataemic rickets and osteomalacia
- Where
- 13 sites in South Korea
- Volunteers
- 100 people
- Sponsor
- Kyowa Kirin Korea Co., Ltd.
Amyotrophic Lateral Sclerosis
A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.
- Trial name
- NCT01281631
- Testing
- NP001
- Where
- 17 sites in United States
- Volunteers
- 136 people
- Sponsor
- Neuraltus Pharmaceuticals, Inc.
Malnutrition, Complication of Hemodialysis
Intradialytic Parenteral Nutrition in Hemodialysis Patients
Malnutrition is a major cause of death in chronic hemodialysis patients. Primary treatment of malnutrition in these patients is dietetic counseling, additional enteral nutrition and occasionally drug therapy.
- Trial name
- IDPNHD (NCT00501956)
- Testing
- Intradialytic Parenteral Nutrition (IDPN)
- Where
- Germany
- Volunteers
- 140 people
- Sponsor
- Fresenius Kabi
Amyotrophic Lateral Sclerosis
CARE Canadian ALS Riluzole Evaluation
Evaluate the efficacy of riluzole 50-mg bid defined by comparing the percentage of riluzole-treated subjects who experienced death, permanently assisted ventilation (PAV) or tracheostomy, to a group of recent historical controls…
- Trial name
- NCT00542412
- Testing
- Riluzole
- Where
- Canada
- Volunteers
- 414 people
- Sponsor
- Sanofi
AL Amyloidosis
Study of Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients With AL Amyloidosis
The purpose of this study is to examine the tolerance, safety, pharmacokinetics, and possible clinical benefit of the good manufacturing practice (GMP)-grade amyloid fibril-reactive chimeric (Ch) IgG1 mAb 11-1F4 in patients with…
- Trial name
- NCT02245867
- Testing
- Chimeric Fibril-Reactive Monoclonal Anti-body 11-1F4
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Tetraplegia/Tetraparesis, Quadriplegia
GB-PRIME: An Early Feasibility Study of a Precise Robotically Implanted Brain-Computer Interface for the Control of External Devices
The GB-PRIME Study is an early feasibility study designed to assess the clinical safety and functionality of the Neuralink N1 Implant and R1 Robot.
- Conditions
- Tetraplegia/Tetraparesis, Quadriplegia, Quadriplegia/Tetraplegia, Cervical Spinal Cord Injury
- Trial name
- GB-PRIME (NCT07127172)
- Testing
- N1 Implant, R1 Robot
- Where
- 2 sites in United Kingdom
- Volunteers
- 7 people
- Sponsor
- Neuralink Corp