Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis With an arg15leu Pathogenic Variant in CHCHD10.

Overview

About this study

The purpose of this study is to evaluate the clinical efficacy of nL-CHCHD-001 in motor function and survival in a single ALS participant., and to evaluate further the effects of nL-CHCHD-001 in slowing, halting, or reversing the rate of decline of clinical functioning and improvement in biomarkers of disease severity in a single ALS participant.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).
* Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.
* Genetically confirmed neurological disorder.

Exclusion Criteria:

* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.
* Use of an investigational medication within less than 5 half-lives of the drug at enrollment

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 5/03/2024. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location	Status	Contact
Jacksonville, Fla. Mayo Clinic principal investigator Bjorn Oskarsson, M.D.	Closed for enrollment	Contact information: ALS Research Team (904) 953-6912 mayofloridaALSresearch@mayo.edu

Mayo Clinic Location

Status

Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Bjorn Oskarsson, M.D.

Closed for enrollment

Contact information:

ALS Research Team

(904) 953-6912

mayofloridaALSresearch@mayo.edu

More information

Publications

Publications are currently not available

Clinical Trials