ION356 for Pelizaeus-Merzbacher Disease

Recruiting at 6 trial locations

Overseen By Ionis Pharmaceuticals, Inc.

Age: < 18

Sex: Male

Trial Phase: Phase 1

Sponsor: Ionis Pharmaceuticals, Inc.

Must not be taking: Investigational drugs

Disqualifiers: Obstructive hydrocephalus, Brain disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

24 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you are taking another investigational drug, you must stop at least 1 month before screening, or longer depending on the drug's characteristics.

What is the purpose of this trial?

The primary purpose of this study is to evaluate the safety and tolerability of ION356.

Eligibility Criteria

This trial is for boys aged 2-17 with Pelizaeus-Merzbacher Disease (PMD), confirmed by PLP1 gene duplication. They must be able to travel to the study center and comply with all requirements. Parents or guardians need to consent and participate in reporting symptoms.

Inclusion Criteria

I am a male aged between 2 and 17 years.

You are able and willing to complete all the study tasks and visits, including traveling to the study center.

My brain scans and symptoms match those of Pelizaeus-Merzbacher Disease.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Multiple-Ascending Dose (MAD)

Participants receive multiple ascending doses of ION356 for safety and pharmacokinetics evaluation

48 weeks

Open-label Long-term Extension (LTE)

Participants continue to receive ION356 in an open-label extension to evaluate long-term safety and tolerability

109 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Treatment Details

Interventions

• ION356

Trial Overview The Orbit Study is testing ION356, administered into the spinal fluid, to check its safety and how it affects the body in young males with PMD. The main goal is to see if it's safe and tolerable for these patients.

Participant Groups

Group I: Cohort B: ION356 Dose B Experimental Treatment 1 Intervention

Participants will receive ION356 intrathecally at Dose B in the MAD Period, followed by ION356 Dose B in the LTE Period.

Group II: Cohort A: ION356 Dose A Experimental Treatment 1 Intervention

Participants will receive ION356 intrathecally at Dose A in the MAD Period, followed by ION356 Dose A in the LTE Period.

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD