NCT06645769

Brief Summary

This study will investigate if the drug sodium phenylbutyrate (NaPB) impacts blood pressure and vascular function in healthy volunteers and in patients with diabetes.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P50-P75 for early_phase_1 diabetes

Timeline
37mo left

Started Jun 2026

Longer than P75 for early_phase_1 diabetes

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 4, 2024

Completed
13 days until next milestone

First Posted

Study publicly available on registry

October 17, 2024

Completed
1.6 years until next milestone

Study Start

First participant enrolled

June 1, 2026

Expected
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2028

1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2029

Last Updated

March 12, 2026

Status Verified

March 1, 2026

Enrollment Period

1.6 years

First QC Date

October 4, 2024

Last Update Submit

March 10, 2026

Conditions

Diabetes

Outcome Measures

Primary Outcomes (1)

  • Systemic vascular resistance

    Arterial hemodynamics and echocardiography to assess systemic vascular resistance, in dynes Ă— sec/cm5.

    from enrollment to the end of participation at 8 weeks

Secondary Outcomes (3)

  • Plasma BCAAs

    from enrollment to the end of participation at 8 weeks

  • Flow-mediated dilation (FMD)

    from enrollment to the end of participation at 8 weeks

  • Carotid femoral pulse wave velocity (PWV)

    from enrollment to the end of participation at 8 weeks

Other Outcomes (2)

  • Home ambulatory mean pressure and pulse pressure

    from enrollment to the end of participation at 8 weeks

  • Cardiac Ejection Fraction

    from enrollment to the end of participation at 8 weeks

Study Arms (2)

NaPB administration

EXPERIMENTAL
Drug: Sodium Phenylbutyrate

Placebo control

PLACEBO COMPARATOR
Other: Placebo

Interventions

Sodium PhenylbutyrateDRUG

will be compared to placebo

NaPB administration
PlaceboOTHER

will be compared to NaPB

Placebo control

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Over 18 years of age and less than 60.
  • Healthy subjects will have no history of diabetes or prediabetes.
  • Diabetes subjects can be either insulin dependent or not.
  • Able to provide written consent and to comply with the procedures of the study protocol.

You may not qualify if:

  • Age \<18 or \>60
  • Pregnant or lactating women.
  • Subjects with hypertension or taking any vasodilatory medications, any steroidal drugs, haloperidol, or valproic acid.
  • Patients with congestive heart failure, severe renal insufficiency, hepatic failure, or known sodium retention with oedema.
  • Active alcohol or substance abuse.
  • Use of tobacco within the previous six months.
  • Unble to provide written consent and to comply with the procedures of the study protocol.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (2)

  • Murashige D, Jung JW, Neinast MD, Levin MG, Chu Q, Lambert JP, Garbincius JF, Kim B, Hoshino A, Marti-Pamies I, McDaid KS, Shewale SV, Flam E, Yang S, Roberts E, Li L, Morley MP, Bedi KC Jr, Hyman MC, Frankel DS, Margulies KB, Assoian RK, Elrod JW, Jang C, Rabinowitz JD, Arany Z. Extra-cardiac BCAA catabolism lowers blood pressure and protects from heart failure. Cell Metab. 2022 Nov 1;34(11):1749-1764.e7. doi: 10.1016/j.cmet.2022.09.008. Epub 2022 Oct 11.

    PMID: 36223763BACKGROUND

  • Vanweert F, Neinast M, Tapia EE, van de Weijer T, Hoeks J, Schrauwen-Hinderling VB, Blair MC, Bornstein MR, Hesselink MKC, Schrauwen P, Arany Z, Phielix E. A randomized placebo-controlled clinical trial for pharmacological activation of BCAA catabolism in patients with type 2 diabetes. Nat Commun. 2022 Jun 18;13(1):3508. doi: 10.1038/s41467-022-31249-9.

    PMID: 35717342BACKGROUND

MeSH Terms

Conditions

Diabetes Mellitus

Interventions

4-phenylbutyric acid

Condition Hierarchy (Ancestors)

Glucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System Diseases

Study Officials

  • Zoltan Arany, MDPHD

    University of Pennsylvania

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Claire Brady

CONTACT

8573344274Claire.Brady@Pennmedicine.upenn.edu

Study Design

Study Type
interventional
Phase
early phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
BASIC SCIENCE
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 4, 2024

First Posted

October 17, 2024

Study Start (Estimated)

June 1, 2026

Primary Completion (Estimated)

January 1, 2028

Study Completion (Estimated)

June 1, 2029

Last Updated

March 12, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share