48 Participants Needed

ION440 for MECP2 Syndrome

Recruiting at 4 trial locations
IP
Overseen ByIonis Pharmaceuticals
Age: Any Age
Sex: Male
Trial Phase: Phase 1 & 2
Sponsor: Ionis Pharmaceuticals, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify that you must stop taking your current medications. However, you need to be on stable doses of your current medications for at least 3 months before starting the trial, unless the investigator decides otherwise.

What is the purpose of this trial?

The primary purpose of this study is to evaluate the safety and tolerability of ION440.

Eligibility Criteria

This trial is for children and adults with MECP2 Duplication Syndrome (MDS). Children must be aged 2-7, have a caregiver to consent and attend visits, while adults are eligible from ages 8-65. Participants need genetic confirmation of MDS and stable medication doses for at least 3 months.

Inclusion Criteria

I am a male between 2 and 65 years old.
I am between 8 and 65 years old.
I am between 2 and 7 years old.
See 3 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Multiple Ascending Dose (MAD)

Participants are randomized to receive ION440 or sham in a double-blind manner

36 weeks
Regular visits for dosing and monitoring

Open-label Long-term Extension (LTE)

Participants receive ION440 for long-term evaluation

Up to 156 weeks
Regular visits for dosing and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • ION440
Trial Overview The ATTUNE study tests the safety of ION440 when administered into the spinal fluid compared to a sham procedure. It aims to understand how the body handles ION440 and its effects on participants with MDS.
Participant Groups
4Treatment groups
Experimental Treatment
Placebo Group
Group I: Cohort 3: ION440 Dose CExperimental Treatment1 Intervention
Participants will receive ION440 intrathecally at Dose C during Part 1/MAD, followed by ION440 Dose C during Part 2/LTE.
Group II: Cohort 2: ION440 Dose BExperimental Treatment1 Intervention
Participants will receive ION440 intrathecally at Dose B during Part 1/MAD, followed by ION440 Dose B during Part 2/LTE.
Group III: Cohort 1: ION440 Dose AExperimental Treatment1 Intervention
Participants will receive ION440 intrathecally at Dose A during Part 1/MAD, followed by ION440 Dose A during Part 2/LTE.
Group IV: Sham ProcedurePlacebo Group1 Intervention
During the Part 1/MAD period, a lumbar procedure (LP) will be performed at the same frequency as ION440 administration. Participants will not receive ITB injections during this period. It will be followed by the open-label Part 2/LTE period, where participants will receive ION440 at the same dose as their enrolled cohort (e.g. Dose A, Dose B or Dose C).

Find a Clinic Near You

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials
151
Recruited
27,800+
Dr. Brett P. Monia profile image

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 profile image

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD

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