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Triheptanoin for Pyruvate Dehydrogenase Deficiency

Phase 1

Recruiting

Led By Jirair Bedoyan, MD, PhD

Research Sponsored by Jirair Krikor Bedoyan

Eligibility Criteria Checklist

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

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

Study Summary

This trial will test a medication called triheptanoin in patients with a disease called Pyruvate Dehydrogenase Complex (PDC) Deficiency. The medication is already approved by

Who is the study for?

This trial is for children with a condition called Pyruvate Dehydrogenase Complex (PDC) Deficiency. Specific eligibility criteria are not provided, but typically participants must meet certain health standards and may be required to have a confirmed diagnosis of PDC Deficiency.Check my eligibility

What is being tested?

The study is testing the safety and effectiveness of triheptanoin, a medication approved for Long-Chain Fatty Acid Oxidation Disorders, in treating PDC Deficiency in children.See study design

What are the potential side effects?

While specific side effects for triheptanoin in this trial aren't listed, common ones from similar treatments could include stomach pain, diarrhea, nausea, or potential liver issues. The study aims to assess these as part of its safety evaluation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

More efficacious disease control

More efficacious metabolic control

More efficacious seizure control

+7 more

Secondary outcome measures

Improved long-term maintenance and tolerance of diet

Improved quality of life

Side effects data

From 2019 Phase 2 trial • 20 Patients • NCT02036853

83%

Diarrhea

33%

Fever

33%

Headache

33%

Stomach Virus

17%

Convulsive Seizures

17%

Skin Scale Hands

17%

Rash-Right Leg

17%

Decreased Appetite

17%

Lethargy

17%

Hand Pain

17%

Tongue Pain

17%

Gastric Reflux

17%

Urinary Incontinence

17%

Amenorrhea

17%

Otalgia

17%

Hirsutism

17%

Abdominal Discomfort

17%

Constipation

17%

Emesis

17%

Nausea

17%

Nasal Fracture

17%

Right Arm Fracture

17%

Decreased Hematocrit

17%

Weight Gain

17%

Seizure

17%

Insomnia

17%

Moodiness

17%

Upper Respiratory Infection

17%

Impetigo

17%

Onychomycosis

17%

Rhinovirus

100%

80%

60%

40%

20%

Study treatment Arm

Schedule A

Schedule B

Trial Design

1Treatment groups

Experimental Treatment

Group I: TriheptanoinExperimental Treatment1 Intervention

Open label study

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Triheptanoin

2020

Completed Phase 2

~200

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncIndustry Sponsor

89 Previous Clinical Trials

179,069 Total Patients Enrolled

Jirair Krikor BedoyanLead Sponsor

Jirair Bedoyan, 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.

Is the current clinical trial actively enrolling participants?

"The current status on clinicaltrials.gov reveals that this research study is actively seeking eligible individuals. Originally posted on March 29, 2024, the trial's most recent update was also made on the same day."

Answered by AI

Do I meet the necessary criteria to participate in this clinical investigation?

"Eligible candidates for this study must be aged between 1 year and 17 years, presenting with pyruvate dehydrogenase deficiency. The trial aims to recruit approximately six participants."

Answered by AI

What is the current number of participants being enrolled in this research project?

"Indeed, the details on clinicaltrials.gov indicate that this research endeavor is actively seeking eligible participants. The trial was originally posted on March 29th, 2024, and underwent its last revision on the same date. A total of 6 individuals are sought to take part in this study at a single designated site."

Answered by AI

Are individuals who are older than 50 years eligible to participate in this clinical trial?

"This research is seeking participants older than 12 months but under the age of 17 years."

Answered by AI

What is the level of risk associated with Triheptanoin when administered to patients?

"Our team at Power has evaluated the safety of Triheptanoin as a 1 on our scale, given that this trial is in its initial Phase 1 stage with minimal available data supporting both safety and efficacy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency

Jirair Krikor Bedoyan 2024. "Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06340685.

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