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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03833128
Recruitment Status : Recruiting
First Posted : February 6, 2019
Last Update Posted : August 26, 2020
Information provided by (Responsible Party):
Reneo Pharma Ltd

Brief Summary:
The purpose of this study is to assess REN001 safety in subjects with fatty acid oxidation disorders.

Condition or disease Intervention/treatment Phase
Fatty Acid Oxidation Disorders Drug: Low Dose REN001 Drug: High Dose REN001 Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Study to Determine the Safety and Tolerability of 12 Weeks Treatment With Oral REN001 in Subjects With Fatty Acid Oxidation Disorders (FAOD)
Actual Study Start Date : April 4, 2019
Estimated Primary Completion Date : October 2021
Estimated Study Completion Date : November 2021

Arm Intervention/treatment
Experimental: Group 1
REN001 Low Dose
Drug: Low Dose REN001
Once daily for 12 weeks

Experimental: Group 2
REN001 High Dose
Drug: High Dose REN001
Once daily for 12 weeks

Primary Outcome Measures :
  1. Adverse Events [ Time Frame: Continous to Week 12 ]
    Number of participants with Adverse Events (AEs) as a measure of safety and tolerability

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03833128

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Contact: Lynn Purkins, PhD +44 (0) 1304 809360

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United States, Illinois
Ann & Robert H. Lurie Children's Hospital Of Chicago Withdrawn
Chicago, Illinois, United States, 60611-2605
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Ashley Gregor   
Principal Investigator: Melanie Gillingham         
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Elizabeth McCracken   
Principal Investigator: Jerry Vockley         
Sponsors and Collaborators
Reneo Pharma Ltd
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Principal Investigator: Jerry Vockley, M.D., Ph.D. University of Pittsburgh Medical Center
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Responsible Party: Reneo Pharma Ltd Identifier: NCT03833128    
Other Study ID Numbers: REN001-102
First Posted: February 6, 2019    Key Record Dates
Last Update Posted: August 26, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pathologic Processes