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Antisense Oligonucleotide for Intellectual Disability

Age: Any Age
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: n-Lorem Foundation
Disqualifiers: Other trial participation, others
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug intended for a single participant with Schuurs-Hoeijmakers syndrome (SHMS) due to a pathogenic, de novo, heterozygous missense gain-of-function mutation in PACS1

Are You a Good Fit for This Trial?

Inclusion Criteria

I have SHMS caused by a PACS1 gene mutation (c.607C>T, p.Arg203Trp).
* Informed consent/assent provided by the participant's parent(s) or legally authorized representative(s)
I can travel to the study site and attend all follow-up visits.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Administration of personalized antisense oligonucleotide (ASO) treatment

24 months
Regular visits for administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months
Periodic assessments every 6 months

What Are the Treatments Tested in This Trial?

Interventions

  • nL-PACS1-001

How Is the Trial Designed?

1

Treatment groups

Experimental Treatment

Group I: Open LabelExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

n-Lorem Foundation

Lead Sponsor

Trials
5
Recruited
5+

The Hospital for Sick Children

Collaborator

Trials
724
Recruited
6,969,000+

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