Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers

Completed

• Conditions: Friedreich Ataxia; Healthy Volunteers
• Interventions: Diagnostic Test: Buccal Swabs, Blood Draws and Skin Punch Biopsy

• Registration Number: NCT05028764
• Lead Sponsor: Larimar Therapeutics, Inc.

Brief Summary

To examine range of tissue frataxin (FXN) concentrations, specific ribonucleic acids, other proteins and specialized lipids in the buccal cells, blood, and skin cells of normal healthy volunteers without Friedreich's ataxia (FRDA).

Detailed Description

Primary Objective: To examine the range of tissue frataxin (FXN) concentrations in the buccal cells, blood, and skin cells of normal healthy volunteers without FRDA.

Secondary Objective: To examine the range of specific ribonucleic acids (RNAs), other proteins, and specialized lipids in the buccal cells, blood, and skin cells of normal healthy volunteers without FRDA.

Study Timeline

• Study Start: 2021-08-04
• Study First Submitted: 2021-08-25
• Study First Submitted QC: 2021-08-25
• Study First Posted: 2021-08-31
• Status Verified: 2022-02
• Primary Completion: 2022-02-04
• Study Completion: 2022-02-04
• Last Update Submitted: 2022-02-22
• Last Update Posted: 2022-02-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1. Subject is a healthy male or female,18-50 years of age
2. Subject has a body mass index (BMI) ≥18.0 and ≤32.0 as of the screening visit.
3. Subject is racially Caucasian, Asian, Native Hawaiian, or Native American.

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Exclusion Criteria

1. Subject used erythropoietin, etravirine, or gamma interferon within 3 months prior to the Day 1 (assessment) visit.
2. Subject has a chronic condition that requires ongoing prescription drug treatment.
3. Subject use of any statin medications within 3 months before the Day 1 (assessment) visit.
4. Subject use of any lipid-lowering agents, other than statins, within 6 weeks before the Day 1 (assessments) visit.
5. Subject is racially black or African American.
6. Pregnant or breast-feeding female subjects.
7. Subject has a known history of drug or alcohol abuse or current suspected drug or alcohol abuse.
8. Subject is positive for human immunodeficiency virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg), or hepatitis A virus (HAV).
9. Subject has any condition, disease, or situation, that in the opinion of the principal investigator (PI), could confound the results of the study or put the subject at undue risk, making participation inadvisable.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
18 to 30 years of age group	Buccal Swabs, Blood Draws and Skin Punch Biopsy	Consisting of at least 13 males and 13 females
31 to 50 years of age group	Buccal Swabs, Blood Draws and Skin Punch Biopsy	Consisting of at least 13 males and 13 females

Primary Outcome Measures

Name	Time	Method
Examine tissue frataxin concentrations in buccal cells, blood and skin cells	1 day	Range of tissue frataxin levels in buccal cells, blood and skin cells

Secondary Outcome Measures

Name	Time	Method
Examine specific ribonucleic acids (RNAs), other proteins, and specialized lipids in buccal cells and blood	1 day	Range of specific ribonucleic acids (RNAs), other proteins, and specialized lipids in buccal cells and blood

Trial Locations

Locations (1)

Clinilabs Drug Development Corporation; 🇺🇸 Eatontown, New Jersey, United States