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Trial record 1 of 2 for:    REN001
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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.
ClinicalTrials.gov Identifier: NCT03833128
Recruitment Status : Not yet recruiting
First Posted : February 6, 2019
Last Update Posted : February 6, 2019
Sponsor:
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)
Estimated Study Start Date : March 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : July 2020

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

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

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 ClinicalTrials.gov identifier (NCT number): NCT03833128


Contacts
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Contact: Lynn Purkins, PhD +44 (0) 1304 809360 lpurkins@reneopharma.com

Sponsors and Collaborators
Reneo Pharma Ltd
Investigators
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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
ClinicalTrials.gov Identifier: NCT03833128     History of Changes
Other Study ID Numbers: REN001-102
First Posted: February 6, 2019    Key Record Dates
Last Update Posted: February 6, 2019
Last Verified: February 2019

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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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Disease
Pathologic Processes