4-phenylbutyric acid has been researched along with Adult Spinal Muscular Atrophy in 3 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 |
|---|---|---|
| "Proximal spinal muscular atrophy (SMA) is a leading genetic cause for infant death in the world and results from the selective loss of motor neurons in the spinal cord." | 1.91 | Evaluation of the orally bioavailable 4-phenylbutyrate-tethered trichostatin A analogue AR42 in models of spinal muscular atrophy. ( Burghes, AHM; Butchbach, MER; Connell, AJ; Harris, AW; Kirk, RW; Lumpkin, CJ; Pellizzoni, L; Saieva, L; Whiting, JA, 2023) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 2 (66.67) | 29.6817 |
| 2010's | 0 (0.00) | 24.3611 |
| 2020's | 1 (33.33) | 2.80 |
| Authors | Studies |
|---|---|
| Lumpkin, CJ | 1 |
| Harris, AW | 1 |
| Connell, AJ | 1 |
| Kirk, RW | 1 |
| Whiting, JA | 1 |
| Saieva, L | 1 |
| Pellizzoni, L | 1 |
| Burghes, AHM | 1 |
| Butchbach, MER | 1 |
| Andreassi, C | 1 |
| Angelozzi, C | 2 |
| Tiziano, FD | 2 |
| Vitali, T | 1 |
| De Vincenzi, E | 1 |
| Boninsegna, A | 1 |
| Villanova, M | 2 |
| Bertini, E | 2 |
| Pini, A | 2 |
| Neri, G | 2 |
| Brahe, C | 2 |
| Mercuri, E | 1 |
| Messina, S | 1 |
| Solari, A | 1 |
| D'Amico, A | 1 |
| Battini, R | 1 |
| Berardinelli, A | 1 |
| Boffi, P | 1 |
| Bruno, C | 1 |
| Cini, C | 1 |
| Colitto, F | 1 |
| Kinali, M | 1 |
| Minetti, C | 1 |
| Mongini, T | 1 |
| Morandi, L | 1 |
| Orcesi, S | 1 |
| Pane, M | 1 |
| Pelliccioni, M | 1 |
| Vita, G | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy[NCT00374075] | Phase 1 | 42 participants | Interventional | 2003-09-30 | Completed | ||
| Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I)[NCT00661453] | Phase 1/Phase 2 | 40 participants (Actual) | Interventional | 2008-04-30 | Completed | ||
| Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)[NCT00227266] | Phase 2 | 94 participants (Actual) | Interventional | 2005-09-30 | 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] | |||||||
(NCT00661453)
Timeframe: -2 weeks, time 0, 3 months, 6 months
| Intervention | g (Mean) | |||||
|---|---|---|---|---|---|---|
| Lean Mass Baseline | Lean Mass 3 months | Lean Mass 6 months | Fat Mass Baseline | Fat Mass 3 months | Fat Mass 6 months | |
| SMA Type 1 | 4317.15 | 4993.92 | 5133.83 | 3011.37 | 3618.25 | 4316.08 |
The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mV (Mean) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 2.28 | 2.32 |
| Cohort 1b Sitters Treatment | 2.93 | 2.37 |
| Cohort 2 Standers and Walkers - Treatment | 5.52 | 6.56 |
The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mV (Median) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 1.91 | 1.44 |
| Cohort 1b Sitters Treatment | 2.2 | 1.8 |
| Cohort 2 Standers and Walkers - Treatment | 5.3 | 5.85 |
The maximum Compound Motor Action Potential (CMAP) area is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This procedure is repeated multiple times. The maximum area is the response that results in the largest area under the response curve. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mVms (Mean) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 5.46 | 5.28 |
| Cohort 1b Sitters Treatment | 5.45 | 5.26 |
| Cohort 2 Standers and Walkers - Treatment | 14.85 | 16.26 |
The maximum Compound Motor Action Potential (CMAP) area is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This procedure is repeated multiple times. The maximum area is the response that results in the largest area under the response curve. (NCT00227266)
Timeframe: 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available)
| Intervention | mVms (Median) | |
|---|---|---|
| Baseline | 6 months | |
| Cohort 1a Sitters Placebo Then Treatment | 3.6 | 3.74 |
| Cohort 1b Sitters Treatment | 4.6 | 3.4 |
| Cohort 2 Standers and Walkers - Treatment | 13.65 | 16.85 |
Comparison of Modified Hammersmith Change from baseline to 6 months. Scores range from 0 to 40. A higher score indicates a better outcome. This scale is used to assess gross motor abilities of non-ambulant children with SMA in multiple research trials as well as in clinical settings. (NCT00227266)
Timeframe: 0 months, 6 months
| Intervention | Score (Mean) | ||
|---|---|---|---|
| Baseline visit (0 weeks) | 6 Month visit (V2) | Change from Baseline | |
| Cohort 1a Sitters Placebo Then Treatment | 20.0 | 20.6 | 0.6 |
| Cohort 1b Sitters Treatment | 16.6 | 16.8 | 0.2 |
"Baseline Modified Hammersmith Extend testing. The baseline test is the score they receive during their screening visits. This scale ranges from 0 to 56. A higher score indicates a better outcome.~This scale is used to assess gross motor abilities of children with SMA in multiple research trials as well as in clinical settings." (NCT00227266)
Timeframe: 1 month prior to enrollment, at enrollment (0 months)
| Intervention | Score (Mean) | |
|---|---|---|
| Modified Hammersmith Extend at S1 (-4 weeks) | Modified Hammersmith Extend at S2 (0 weeks) | |
| Cohort 2 Experimental | 47.0 | 48.3 |
1 trial available for 4-phenylbutyric acid and Adult Spinal Muscular Atrophy
| Article | Year |
|---|---|
Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy.
Topics: Child; Child, Preschool; Double-Blind Method; Female; Humans; Male; Muscular Atrophy, Spinal; Phenyl | 2007 |
2 other studies available for 4-phenylbutyric acid and Adult Spinal Muscular Atrophy
| Article | Year |
|---|---|
Evaluation of the orally bioavailable 4-phenylbutyrate-tethered trichostatin A analogue AR42 in models of spinal muscular atrophy.
Topics: Animals; Disease Models, Animal; Histone Deacetylase Inhibitors; Mice; Motor Neurons; Muscular Atrop | 2023 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy.
Topics: Base Sequence; Blotting, Western; Cell Culture Techniques; Cyclic AMP Response Element-Binding Prote | 2004 |