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

R

Reneo Pharma

Status and phase

Completed
Phase 1

Conditions

Fatty Acid Oxidation Disorders

Treatments

Drug: Low Dose REN001
Drug: High Dose REN001

Study type

Interventional

Funder types

Industry

Identifiers

NCT03833128
REN001-102

Details and patient eligibility

About

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

Full description

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.

Enrollment

24 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

24 participants in 3 patient groups

Group 1 - Part A
Experimental group
Description:
REN001 Low Dose oral once daily x 12 weeks
Treatment:
Drug: Low Dose REN001
Group 2 - Part A
Experimental group
Description:
REN001 High Dose oral once daily x 12 weeks
Treatment:
Drug: High Dose REN001
Group 3 - Part B
Experimental group
Description:
REN001 High Dose oral once daily x 12 weeks
Treatment:
Drug: High Dose REN001

Trial contacts and locations

7

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Data sourced from clinicaltrials.gov

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