4-phenylbutyric acid has been researched along with Disease Exacerbation in 7 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.
| Excerpt | Relevance | Reference |
|---|---|---|
| "Proteinuria is one of the primary risk factors for the progression of chronic kidney disease (CKD) and has been implicated in the induction of endoplasmic reticulum (ER) stress." | 1.46 | Endoplasmic reticulum stress inhibition limits the progression of chronic kidney disease in the Dahl salt-sensitive rat. ( Ask, K; Brimble, E; Carlisle, RE; Chahal, J; Dickhout, JG; Lu, C; Upagupta, C; Yum, V, 2017) |
| "The therapeutic potency of 4PB in these patients was tested by oral administration of this drug with gradually increasing dosage (200, 350, and 500 mg/kg/day) for 6 months." | 1.40 | Intractable itch relieved by 4-phenylbutyrate therapy in patients with progressive familial intrahepatic cholestasis type 1. ( Bessho, K; Hanada, K; Hasegawa, Y; Hayashi, H; Igarashi, K; Kimura, T; Kondou, H; Konishi, A; Kusuhara, H; Miyoshi, Y; Nagasaka, H; Nakao, K; Naoi, S; Ozono, K, 2014) |
| "Experimental allergic encephalomyelitis (EAE) is the animal model for multiple sclerosis." | 1.32 | Sodium phenylacetate inhibits adoptive transfer of experimental allergic encephalomyelitis in SJL/J mice at multiple steps. ( Banik, NL; Dasgupta, S; Jana, M; Pahan, K; Zhou, Y, 2003) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 2 (28.57) | 29.6817 |
| 2010's | 3 (42.86) | 24.3611 |
| 2020's | 2 (28.57) | 2.80 |
| Authors | Studies |
|---|---|
| Alfahel, L | 1 |
| Argueti-Ostrovsky, S | 1 |
| Barel, S | 1 |
| Ali Saleh, M | 1 |
| Kahn, J | 1 |
| Azoulay-Ginsburg, S | 1 |
| Rothstein, A | 1 |
| Ebbinghaus, S | 1 |
| Gruzman, A | 1 |
| Israelson, A | 1 |
| Paganoni, S | 1 |
| Macklin, EA | 1 |
| Hendrix, S | 1 |
| Berry, JD | 1 |
| Elliott, MA | 1 |
| Maiser, S | 1 |
| Karam, C | 1 |
| Caress, JB | 1 |
| Owegi, MA | 1 |
| Quick, A | 1 |
| Wymer, J | 1 |
| Goutman, SA | 1 |
| Heitzman, D | 1 |
| Heiman-Patterson, T | 1 |
| Jackson, CE | 1 |
| Quinn, C | 1 |
| Rothstein, JD | 1 |
| Kasarskis, EJ | 1 |
| Katz, J | 1 |
| Jenkins, L | 1 |
| Ladha, S | 1 |
| Miller, TM | 1 |
| Scelsa, SN | 1 |
| Vu, TH | 1 |
| Fournier, CN | 1 |
| Glass, JD | 1 |
| Johnson, KM | 1 |
| Swenson, A | 1 |
| Goyal, NA | 1 |
| Pattee, GL | 1 |
| Andres, PL | 1 |
| Babu, S | 1 |
| Chase, M | 1 |
| Dagostino, D | 1 |
| Dickson, SP | 1 |
| Ellison, N | 1 |
| Hall, M | 1 |
| Hendrix, K | 1 |
| Kittle, G | 1 |
| McGovern, M | 1 |
| Ostrow, J | 1 |
| Pothier, L | 1 |
| Randall, R | 1 |
| Shefner, JM | 1 |
| Sherman, AV | 1 |
| Tustison, E | 1 |
| Vigneswaran, P | 1 |
| Walker, J | 1 |
| Yu, H | 1 |
| Chan, J | 1 |
| Wittes, J | 1 |
| Cohen, J | 1 |
| Klee, J | 1 |
| Leslie, K | 1 |
| Tanzi, RE | 1 |
| Gilbert, W | 1 |
| Yeramian, PD | 1 |
| Schoenfeld, D | 1 |
| Cudkowicz, ME | 1 |
| Hasegawa, Y | 1 |
| Hayashi, H | 1 |
| Naoi, S | 1 |
| Kondou, H | 1 |
| Bessho, K | 1 |
| Igarashi, K | 1 |
| Hanada, K | 1 |
| Nakao, K | 1 |
| Kimura, T | 1 |
| Konishi, A | 1 |
| Nagasaka, H | 1 |
| Miyoshi, Y | 1 |
| Ozono, K | 1 |
| Kusuhara, H | 1 |
| Yum, V | 1 |
| Carlisle, RE | 1 |
| Lu, C | 1 |
| Brimble, E | 1 |
| Chahal, J | 1 |
| Upagupta, C | 1 |
| Ask, K | 1 |
| Dickhout, JG | 1 |
| Schultz, J | 1 |
| Ibrahim, SM | 1 |
| Vera, J | 1 |
| Kunz, M | 1 |
| Chan, JY | 1 |
| Luzuriaga, J | 1 |
| Bensellam, M | 1 |
| Biden, TJ | 1 |
| Laybutt, DR | 1 |
| Dasgupta, S | 1 |
| Zhou, Y | 1 |
| Jana, M | 1 |
| Banik, NL | 1 |
| Pahan, K | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS[NCT03127514] | Phase 2 | 137 participants (Actual) | Interventional | 2017-06-22 | Completed | ||
| Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy[NCT04937062] | Early Phase 1 | 50 participants (Anticipated) | Interventional | 2021-03-01 | Enrolling by invitation | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
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
| Intervention | % of Predicted Normal Change Per Month (Least Squares Mean) |
|---|---|
| Placebo | -3.54 |
| AMX0035 | -3.03 |
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
| Intervention | Change in ALSFRS-R Total Score Per Month (Least Squares Mean) |
|---|---|
| Placebo | -1.66 |
| AMX0035 | -1.24 |
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
| Intervention | pg/ml Per Month (Least Squares Mean) |
|---|---|
| Placebo | -2.34 |
| AMX0035 | 3.58 |
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
| Intervention | events (Number) |
|---|---|
| Placebo | 17 |
| AMX0035 | 18 |
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
| Intervention | Participants (Count of Participants) |
|---|---|
| Placebo | 38 |
| AMX0035 | 61 |
Comparison Between Groups of Number of Participants With Adverse Events Until Planned Completion (NCT03127514)
Timeframe: 24 Weeks
| Intervention | Participants (Count of Participants) |
|---|---|
| Placebo | 46 |
| AMX0035 | 86 |
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
| Intervention | % of Predicted Normal Change Per Month (Least Squares Mean) |
|---|---|
| Placebo | -4.03 |
| AMX0035 | -3.10 |
1 trial available for 4-phenylbutyric acid and Disease Exacerbation
| Article | Year |
|---|---|
Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis.
Topics: Aged; Amyotrophic Lateral Sclerosis; Disease Progression; Double-Blind Method; Drug Combinations; Fe | 2020 |
6 other studies available for 4-phenylbutyric acid and Disease Exacerbation
| Article | Year |
|---|---|
4-Phenylbutyric Acid (4-PBA) Derivatives Prevent SOD1 Amyloid Aggregation In Vitro with No Effect on Disease Progression in SOD1-ALS Mice.
Topics: Amyloid; Amyloidogenic Proteins; Amyotrophic Lateral Sclerosis; Animals; Butylamines; Disease Models | 2022 |
Intractable itch relieved by 4-phenylbutyrate therapy in patients with progressive familial intrahepatic cholestasis type 1.
Topics: Administration, Oral; Adolescent; Child; Child, Preschool; Cholestasis, Intrahepatic; Disease Progre | 2014 |
Endoplasmic reticulum stress inhibition limits the progression of chronic kidney disease in the Dahl salt-sensitive rat.
Topics: Animals; Blood Pressure; Disease Models, Animal; Disease Progression; Endoplasmic Reticulum Stress; | 2017 |
14-3-3sigma gene silencing during melanoma progression and its role in cell cycle control and cellular senescence.
Topics: 14-3-3 Proteins; Antineoplastic Agents; Azacitidine; Biomarkers, Tumor; Cell Cycle; Cell Line, Tumor | 2009 |
Failure of the adaptive unfolded protein response in islets of obese mice is linked with abnormalities in β-cell gene expression and progression to diabetes.
Topics: Animals; Antioxidants; Apoptosis Regulatory Proteins; Cytokines; Diabetes Mellitus, Type 2; Disease | 2013 |
Sodium phenylacetate inhibits adoptive transfer of experimental allergic encephalomyelitis in SJL/J mice at multiple steps.
Topics: Acute Disease; Administration, Oral; Adoptive Transfer; Animals; Anti-Inflammatory Agents, Non-Stero | 2003 |