PIXI for Neurogenetic Disorders

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: RTI International

Disqualifiers: Blindness, Severe hearing impairment

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

Compensation: Varies

Number of Visits: 20

120 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether participants need to stop taking their current medications. It is best to consult with the trial coordinators for specific guidance.

What data supports the effectiveness of the treatment Parent-Infant Inter(X)Action Intervention (PIXI) for neurogenetic disorders?

The PIXI treatment is designed to help parents support their infants with neurogenetic conditions, and while direct effectiveness data is not available, similar parent-mediated therapies have shown improvements in socio-communicative skills in children with genetic conditions like fragile X syndrome. This suggests that parent-focused interventions can be beneficial in supporting child development. ^{1 2 3 4 5}

Is the PIXI intervention safe for humans?

The available research on the PIXI intervention focuses on its feasibility and acceptability, but does not provide specific safety data. However, since it is a remotely delivered program involving psychoeducation and parent support, it is generally considered low-risk. ^{1 6 7 8 9}

How is the PIXI treatment different from other treatments for neurogenetic disorders?

The PIXI treatment is unique because it is a remotely delivered, two-phase program specifically designed for infants with neurogenetic conditions and their caregivers. It focuses on psychoeducation and parent support to establish routines and teach targeted skills to support infant development, which is different from traditional medical or drug-based treatments. ^{1 3 4 9 10}

What is the purpose of this trial?

This trial tests a program called PIXI, which helps parents of infants with genetic disorders. The program educates parents about their baby's condition, coaches them on how to interact with their baby, and provides family support. The goal is to improve the baby's development and family well-being.

Research Team

Anne Wheeler, PhD

Principal Investigator

RTI International

Eligibility Criteria

This trial is for infants under 12 months old diagnosed with certain neurogenetic disorders, such as Down Syndrome or Duchenne Muscular Dystrophy. The family must primarily speak English at home. Infants who are blind or have severe hearing impairments cannot participate.

Inclusion Criteria

My infant was diagnosed through screening, not just because of my concerns.

English is the main language spoken in my home.

Exclusion Criteria

My child is not blind and does not have severe hearing loss.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 1 Intervention

Development and testing of the PIXI intervention with a pilot sample of families, focusing on parent education and direct parent coaching

Approximately 6 months

20 sessions

Phase 2 Intervention

Continuation of the PIXI intervention, focusing on family and child functioning assessments and examining feasibility and acceptability

Approximately 6 months

Follow-up

Participants are monitored for early developmental outcomes and autism symptoms

Approximately 30 months

Treatment Details

Interventions

• Parent-Infant Inter(X)Action Intervention (PIXI)

Trial Overview The PIXI program is being tested to support infant development and help parents of children with neurogenetic disorders. It includes parent education, coaching on interaction with their child, and well-being support, along with assessments of the child's and family's progress.

Participant Groups

Group I: Infants with a rare neurogenetic condition and their parent/primary caregiver(s) Experimental Treatment 1 Intervention

PIXI

Who Is Running the Clinical Trial?

RTI International

Lead Sponsor

Trials

201

Recruited

942,000+

University of North Carolina, Chapel Hill

Collaborator

Trials

1,588

Recruited

4,364,000+

References

1. United States pubmed.ncbi.nlm.nih.gov

Pilot protocol for the Parent and Infant Inter(X)action Intervention (PIXI) feasibility study. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Brief Report: Assessment Experiences of Children with Neurogenetic Syndromes: Caregivers' Perceptions and Suggestions for Improvement. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Cooperative Parent-Mediated Therapy in Children with Fragile X Syndrome and Williams Beuren Syndrome: A Pilot RCT Study of a Transdiagnostic Intervention-Preliminary Data. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Group-based parent training programme for children with neurological conditions: a feasibility study. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Extending the Parent-Delivered Early Start Denver Model to Young Children with Fragile X Syndrome. [2020]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Bringing the Laboratory Home: PANDABox Telehealth-Based Assessment of Neurodevelopmental Risk in Children. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

The PRETEND Program: Evaluating the Feasibility of a Remote Parent-Training Intervention for Children With Prader-Willi Syndrome. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

Prepulse inhibition in fragile X syndrome: feasibility, reliability, and implications for treatment. [2021]

9. United States pubmed.ncbi.nlm.nih.gov

Family and Caregiver Characteristics Contribute to Caregiver Change in Use of Strategies and Growth in Child Spoken Language in a Parent-Implemented Language Intervention in Fragile X Syndrome. [2023]

10. United States pubmed.ncbi.nlm.nih.gov

Stress in Parents of Children With Genetically Determined Leukoencephalopathies: A Pilot Study. [2021]