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A Phase 1b Study of the Safety of REN001 in Patients With Fatty Acid Oxidation Disorders

NCT ID: NCT03833128

Last Updated: 2022-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-04-04

Study Completion Date

2022-03-21

Brief Summary

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The purpose of this Phase 1b study is to assess REN001 safety in subjects with fatty acid oxidation disorders.

Detailed Description

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This is a Phase 1b, open-label, multiple-dose study of the safety and tolerability of 2 dose levels of REN001 in subjects with fatty acid oxidation disorders (FAODs) with confirmed mutations in the Carnitine palmitoyltransferase II deficiency (CPT2), Very long-chain Acyl-CoA dehydrogenase deficiency (VLCAD), Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency (LCHAD) or Trifunctional Protein Deficiency (TFP). All subjects will provide written consent prior to commencing any study related activities or assessments. Potential subjects will be screened for study participation up to 8 weeks prior to the start of dosing.The study is divided into two parts, Part A and Part B. Part A has finished enrollment and further eligible patients will participate in Part B only.

Conditions

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Fatty Acid Oxidation Disorders

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Group 1 - Part A

REN001 Low Dose oral once daily x 12 weeks

Group Type EXPERIMENTAL

Low Dose REN001

Intervention Type DRUG

Oral

Group 2 - Part A

REN001 High Dose oral once daily x 12 weeks

Group Type EXPERIMENTAL

High Dose REN001

Intervention Type DRUG

Oral

Group 3 - Part B

REN001 High Dose oral once daily x 12 weeks

Group Type EXPERIMENTAL

High Dose REN001

Intervention Type DRUG

Oral

Interventions

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Low Dose REN001

Oral

Intervention Type DRUG

High Dose REN001

Oral

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Subjects must give written, signed and dated informed consent

Confirmed diagnosis of FAOD

A diagnostic acylcarnitine profile, in blood or cultured fibroblasts

A stable treatment regimen for at least 30 days prior to enrollment

Exclusion Criteria

Unstable or poorly controlled disease

Treatment with an investigational drug within 1 month or within 5 half-lives, whichever is longer

Have been hospitalized within 3 months prior to screening for any major medical event

Pregnant or nursing females
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Reneo Pharma Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jerry Vockley, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

University of Pittsburgh Medical Center

Locations

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Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Division of Medical Genetics, University Utah

Salt Lake City, Utah, United States

Site Status

Neurology department, Raymond-Poincaré Teaching Hospital, Nord/Est/Ile de France Neuromuscular Reference Center

Garches, , France

Site Status

Servicio de Neurología - Unidad de Neuromuscular Centro de Referencia Nacional de Enfermedades Neuromusculares raras Instituto de Investigación i+12

Madrid, , Spain

Site Status

Countries

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United States France Spain

Other Identifiers

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REN001-102

Identifier Type: -

Identifier Source: org_study_id