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Sodium Phenylbutyrate for MCADD

Phase 2

Recruiting

Led By Gerard L Vockley, MD, PhD

Research Sponsored by Jerry Vockley, MD, PhD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

A diagnosis of MCADD and molecular confirmation of at least one copy of the common c.985A>G mutation.

Able to perform and comply with study activities including overnight admission to the PCTRC, placement of an IV catheter, and all blood draws.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 7 weeks

Awards & highlights

Study Summary

This trial will study if a medication (sodium phenylbutyrate) can safely and effectively treat a disease (MCADD) caused by a common mutation in 10+ year olds.

Who is the study for?

This trial is for people aged 10 and older with MCADD, a genetic disorder affecting fat metabolism. Participants must have the ACADM c.985 A>G mutation, be able to undergo study procedures like IV placement, and use contraception if of childbearing age. Those under certain medications or with conditions that increase risk are excluded.Check my eligibility

What is being tested?

The study tests Sodium Phenylbutyrate (ACER-001), an FDA-approved drug for Urea Cycle Disorders, now being investigated for treating MCADD in patients with a specific genetic mutation. The focus is on its safety and effectiveness in this new application.See study design

What are the potential side effects?

While not explicitly listed here, potential side effects may include those known from current uses of Sodium Phenylbutyrate such as digestive issues (nausea, vomiting), headaches, fatigue, skin rash or changes in blood chemistry.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have MCADD with at least one common mutation confirmed.

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I can stay overnight for the study, have an IV placed, and get blood drawn.

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I am not pregnant or have confirmed it with a test.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 7 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~7 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 7 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of participants with treatment related adverse events as assessed by CTCAE v5.0

Secondary outcome measures

Length of fast before hypoglycemia develops

Side effects data

From 2017 Phase 4 trial • 88 Patients • NCT01257737

22%

Hyperammonaemia

16%

Upper respiratory tract infection

13%

Vomiting

13%

Headache

Ammonia increased

Diarrhoea

Ear infection

Amino acid level decreased

Cough

Amino acid level increased

Pyrexia

Dehydration

Nausea

Oropharyngeal pain

Influenza

Gastroenteritis

Hypokalaemia

Bronchitis

Sinusitis

Pain

Dizziness

100%

80%

60%

40%

20%

Study treatment Arm

HPN-100- Pediatric

HPN-100 - Adult

Trial Design

3Treatment groups

Experimental Treatment

Group I: 4.0 g/m2/day BID sodium phenylbutyrateExperimental Treatment1 Intervention

Up to 8 subjects (4 - ages 10-15 years old and 4 - 16 years of age and older) will be randomized to take 4.0 g/m2/day divided into two daily doses taken 12 hours apart

Group II: 3.0 g/m2/day QD sodium phenylbutyrateExperimental Treatment1 Intervention

Up to 8 subjects (4 - ages 10-15 years old and 4 - 16 years of age and older) will be randomized to take 3.0 g/m2/day in one daily dose

Group III: 3.0 g/m2/day BID sodium phenylbutyrateExperimental Treatment1 Intervention

Up to 8 subjects (4 - ages 10-15 years old and 4 - 16 years of age and older) will be randomized to take 3.0 g/m2/day divided into two daily doses taken 12 hours apart

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Jerry Vockley, MD, PhDLead Sponsor

4 Previous Clinical Trials

408 Total Patients Enrolled

Acer Therapeutics Inc.Industry Sponsor

2 Previous Clinical Trials

199 Total Patients Enrolled

Gerard L Vockley, MD, PhDPrincipal InvestigatorUPMC Children's Hospital of Pittsburgh

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are individuals currently able to apply for participation in this trial?

"Evidenced by the clinicaltrials.gov data, this research is presently trying to find participants. It was first posted on November 15th 2023 and recently updated on October 4th of the same year."

Answered by AI

Has the FDA endorsed a 3.0 g/m2/day dosage of sodium phenylbutyrate administered once daily?

"Our assessment of 3.0 g/m2/day QD sodium phenylbutyrate's safety is a 2, as this Phase 2 trial has only offered preliminary indications that the drug does not pose an undue risk to patients."

Answered by AI

What is the total enrollment number for this clinical trial?

"Affirmative. The information hosted on clinicaltrials.gov suggests that this medical research is actively seeking enrolment, with 24 participants required from a single centre. This experiment was originally posted on November 15th 2023 and has since been updated October 4th 2023."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Jerry Vockley, MD, PhD 2024. "Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06069375.