SPONSOR
Ionis Pharmaceuticals, Inc.
Total Trials
2
Recruiting
2
Phases
Phase 1, Phase 2
Conditions studied: Methyl CpG Binding Protein 2 (MECP2) Duplication SyndromePelizaeus-Merzbacher Disease
Ionis Pharmaceuticals, Inc. is a clinical research sponsor with 2 registered studies on ClinicalTrials.gov, of which 2 are actively recruiting participants. Research covers conditions including Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, Pelizaeus-Merzbacher Disease.
The trial portfolio spans Phase 1, Phase 2 studies, reflecting a pipeline that may include early-stage safety evaluations, mid-stage efficacy assessments, and large-scale confirmatory trials. All studies listed here are sourced directly from ClinicalTrials.gov and updated daily.
Frequently Asked Questions — Ionis Pharmaceuticals, Inc.
How many clinical trials has Ionis Pharmaceuticals, Inc. sponsored?
ClinicalMetric currently tracks 2 clinical trials sponsored by Ionis Pharmaceuticals, Inc., of which 2 are actively recruiting participants. These trials cover conditions including Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, Pelizaeus-Merzbacher Disease. Trial counts are updated daily from ClinicalTrials.gov and reflect currently registered studies.
What therapeutic areas does Ionis Pharmaceuticals, Inc. research focus on?
Ionis Pharmaceuticals, Inc. clinical trials span Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome, Pelizaeus-Merzbacher Disease. The research pipeline includes Phase 1, Phase 2 trials, reflecting work from early-stage safety evaluation through large-scale efficacy studies.
How do I participate in a Ionis Pharmaceuticals, Inc. clinical trial?
To participate in a Ionis Pharmaceuticals, Inc. trial, review the active studies listed on this page, check each trial's specific eligibility criteria on ClinicalTrials.gov, and contact the trial site directly—not the sponsor's headquarters. Trial sites are academic medical centers, hospitals, or specialized research clinics. Your physician can facilitate a referral. ClinicalMetric lists all Ionis Pharmaceuticals, Inc. trials with current recruitment status updated daily.
Clinical Trials by Ionis Pharmaceuticals, Inc.
NCT06430385 Phase 1, Phase 2
Recruiting
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
NCT05659901
Recruiting
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Pelizaeus-Merzbacher Disease
ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer · Last Reviewed: September 2026 · Data Methodology