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Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

ConditionPelizaeus-Merzbacher Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexMale
Age2-17
SponsorIonis Pharmaceuticals, Inc.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study requirements.

Diagnosis of PMD with genetic confirmation of PLP1 gene duplication.

Clinical phenotype and brain imaging consistent with a diagnosis of PMD.

Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.

Disqualifiers

Clinically significant abnormalities in medical history, laboratory tests or physical examination.

Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator.

Any contraindication or unwillingness to undergo magnetic resonance imaging (MRI).

Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of the investigational agent, whichever is longer.

Trial design

Design model

Sequential

Treatments tested in this trial

  • ION356

    Drug

    Administered as intrathecal (IT) injection.

Treatment groups

24 Participants
are divided into 2 treatment groups
Group A: Cohort A: ION356 Dose AExperimental treatment 1 intervention
Group B: Cohort B: ION356 Dose BExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs

Time frame
Up to Week 145
2

Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments

Time frame
Baseline up to Week 145
3

Number of Participants With Clinically Significant Change From Baseline in Neurological Examination Findings

Time frame
Baseline up to Week 145
4

Number of Participants With Clinically Significant Change From Baseline in Vital Signs

Time frame
Baseline up to Week 145

Secondary outcomes

1

Maximum Plasma Concentration (Cmax) of ION356

Time frame
Pre-dose and at multiple points post-dose up to Week 145
2

Area Under the Concentration-time Curve (AUC) of ION356

Time frame
Pre-dose and at multiple points post-dose up to Week 145
3

Plasma Terminal Elimination Half-life (t½) of ION356

Time frame
Pre-dose and at multiple points post-dose up to Week 145
4

Plasma Concentration of ION356

Time frame
Pre-dose and at multiple points post-dose up to Week 145

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Ionis Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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