4-phenylbutyric acid and Amyotrophic Lateral Sclerosis

4-phenylbutyric acid has been researched along with Amyotrophic Lateral Sclerosis in 12 studies

4-phenylbutyric acid: RN refers to the parent cpd
4-phenylbutyric acid : A monocarboxylic acid the structure of which is that of butyric acid substituted with a phenyl group at C-4. It is a histone deacetylase inhibitor that displays anticancer activity. It inhibits cell proliferation, invasion and migration and induces apoptosis in glioma cells. It also inhibits protein isoprenylation, depletes plasma glutamine, increases production of foetal haemoglobin through transcriptional activation of the gamma-globin gene and affects hPPARgamma activation.

Amyotrophic Lateral Sclerosis: A degenerative disorder affecting upper MOTOR NEURONS in the brain and lower motor neurons in the brain stem and SPINAL CORD. Disease onset is usually after the age of 50 and the process is usually fatal within 3 to 6 years. Clinical manifestations include progressive weakness, atrophy, FASCICULATION, hyperreflexia, DYSARTHRIA, dysphagia, and eventual paralysis of respiratory function. Pathologic features include the replacement of motor neurons with fibrous ASTROCYTES and atrophy of anterior SPINAL NERVE ROOTS and corticospinal tracts. (From Adams et al., Principles of Neurology, 6th ed, pp1089-94)

Research

Studies (12)

Authors

Clinical Trials (1)

Trial Overview

Trial Outcomes

Accurate Testing of Limb Isometric Strength (ATLIS) Total Score Change

The ATLIS device assess the isometric muscle strength of six upper-limb and six lower-limb muscle groups. At least two trials are performed for each muscle group to assess change in rate of decline of isometric muscle strength over treatment duration. Values are standardized to the percentage of predicted normal strength based on sex, age, weight, and height. Results are presented as percent of predicted normal. (NCT03127514)
Timeframe: 24 Weeks

Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) Slope Change

Change in slope of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over treatment duration. The ALSFRS-R consists of 12 items across 4 subdomains of function (bulbar, fine motor, gross motor, and breathing) with each item scored on a scale from 0 (total loss of function) to 4 (no loss of function). Total scores range from 0 to 48, with higher scores indicating better function. (NCT03127514)
Timeframe: 24 Weeks

Change in Plasma Levels of Phosphorylated Axonal Neurofilament H Subunit (pNF-H)

Neuronal degeneration releases phosphorylated axonal neurofilament H subunit (pNF-H) into the cerebrospinal fluid and subsequently the blood and is thought to be a potential biomarker of motor neuron degeneration; elevated plasma levels of pNF-H are presumed to correlate with neuronal injury. Change in levels of plasma pNF-H were measured from baseline to week 24 (NCT03127514)
Timeframe: 24 Weeks

Death, Tracheostomy, and Hospitalization

The composite outcome was defined as death, a death-equivalent event (which consisted of only tracheostomy in one participant in this trial), or hospitalization, whichever occurred first; there were no instances of permanent ventilation delivered by noninvasive means in the study. (NCT03127514)
Timeframe: 24 Weeks

Number of Participants in Each Group Able to Remain on Study Drug Until Planned Discontinuation

A comparison of the number of participants in each group able to remain on study drug until planned discontinuation between groups (NCT03127514)
Timeframe: 24 weeks

Number of Participants With Adverse Events

Comparison Between Groups of Number of Participants With Adverse Events Until Planned Completion (NCT03127514)
Timeframe: 24 Weeks

Rate of Decline in Slow Vital Capacity (SVC)

Respiratory muscle function was assessed according to slow vital capacity (SVC). SVC was measured in an upright position for at least three trials per assessment. SVC volumes were standardized to the percentage of predicted normal value based on age, sex, and height. (NCT03127514)
Timeframe: 24 Weeks

Reviews

3 reviews available for 4-phenylbutyric acid and Amyotrophic Lateral Sclerosis

Trials

2 trials available for 4-phenylbutyric acid and Amyotrophic Lateral Sclerosis

Other Studies

7 other studies available for 4-phenylbutyric acid and Amyotrophic Lateral Sclerosis