Clinical trial NCT03751280: Study of Efficacy of PEAR-004 in Schizophrenia
Official title: A Randomized, Sham-Controlled Study of PEAR-004 as an Adjunct to Standard-of-care Treatment for Schizophrenia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Schizophrenia
Keywords: Schizophrenia
Digital Therapeutic
CBT
smartphone app
mental disorder
psychosis
acute psychotic reaction
chronic psychosis
failure to recognize what is real
false beliefs
unclear thinking,
confused thinking
auditory hallucinations
paranoia
reduced social engagement
reduced emotional expression
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2018-11-23
Enrollment: 112
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of the study was to determine in patients currently being administered antipsychotic pharmacotherapy whether PEAR-004 could further reduce symptoms of schizophrenia as measured by the Positive and Negative Syndrome Scale (PANSS).

The overall rationale for the study was to assess the first prescription digital therapeutic (PDT) in schizophrenia using a form of proven psychosocial intervention, cognitive behavioral therapy (CBT), to supplement standard of care with antipsychotic medications.

Detailed description: This was a randomized, sham-controlled, rater-blinded, parallel group trial. Overall, 112 subjects were randomized 1:1 in to the following groups:

* Group A: Clinician-directed pharmacotherapy + PEAR-004
* Group B: Clinician-directed pharmacotherapy + sham app An up to 28-day screening period included standard screening assessments as defined in the assessment schedule. Eligible subjects were randomized on Day 1 into one of the treatment groups.

Subjects in both groups continued to receive their clinician-directed standard-of-care treatment for schizophrenia, including pharmacotherapy. Subjects in Group A used PEAR 004 and subjects in Group B used a sham for a period of 12 weeks. Subjects returned to the clinic for outpatient visits at Week 4 (day 29), Week 8 (day 57), and Week 12 (day 85). At each visit, standard assessments were performed according to the assessment schedule, including PANSS, ISST-Plus, CGI, BMQ, MAP-SR, WHOQOL-BREF, BDI-II, ISI, and adverse events (AEs). A final follow-up visit was performed at Week 16 (day 115),

Eligibility criteria: Key Inclusion Criteria:

* Signed informed consent must be obtained prior to participation in the study.
* Healthy male and female subjects 18 to 65 years of age, inclusive, and in good health as determined by medical history, physical examination, and vital signs at screening
* SCID-based DSM-5 diagnosis of schizophrenia and a total PANSS score \> 60
* Proficient in English at 5th grade reading level or higher, in the judgement of the investigator
* Capable of using a mobile device (compatible with PEAR-004) and using common applications, in the judgement of the investigator

Key Exclusion Criteria:

* Major change in primary antipsychotic medication in the prior 4 weeks before screening (e.g., switching to a new agent or a dose adjustment within two weeks of randomization)
* Planning to move out of the geographic area within 3 months
* Unable to use English to participate in the consent process, the interventions or assessments
* Inability to comply with study procedures, due to severe medical conditions or otherwise
* Meet DSM-5 diagnosis for a current episode of major depression, mania, or hypomania in the past month
* Meet DSM-5 diagnosis for a current moderate or severe alcohol or cannabis use disorder in the past 2 months
* Meet DSM-5 diagnosis for a current substance use disorder (other than alcohol or cannabis) in the past 2 months
* Considered high risk for suicidal behavior based on ISST-Plus score at screening, or in the judgement of the investigator
* Previously participated in a clinical study involving PEAR-004

Primary outcomes:
measure: Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) Score
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Percent of Dropout
timeFrame: Day 115
Secondary outcomes:
measure: Change From Baseline in the Positive PANSS Score
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Change From Baseline in the General Psychopathology PANSS Score
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Change From Baseline in the Negative PANSS Score
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Change From Baseline in the Motivation and Pleasure Self-report (MAP-SR) Score
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Change From Baseline in the World Health Organization Quality of Life (WHOQOL-BREF) Scale
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Change From Baseline in the Beck Depression Inventory, Second Ed. (BDI-II) Total Score
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Percentage Change From Baseline in Total PANSS Score (Within Assigned Treatment Group)
timeFrame: Baseline, Day 29, Day 57, Day 85
measure: Percentage of Responders as Assessed by the Brief Medication Questionnaire (BMQ)
timeFrame: Day 29, Day 57, and Day 85
measure: Percentage of Responders as Assessed by the Total PANSS Score
timeFrame: Day 85
measure: Number of Patients With Adverse Events
timeFrame: Day 115
measure: Number of Patients With Vital Sign Measurements
timeFrame: Day 85
measure: InterSePT Scale for Suicidal Thinking-Plus (ISST-Plus) Score
timeFrame: Baseline, Day 29, 57, 85, and 115
Other outcomes:
Locations:
city: Garden Grove
country: United States
facility: Novartis Investigative Site
state: California
city: Oakland
country: United States
facility: Novartis Investigative Site
state: California
city: Torrance
country: United States
facility: Novartis Investigative Site
state: California
city: Maitland
country: United States
facility: Novartis Investigative Site
state: Florida
city: Grand Rapids
country: United States
facility: Novartis Investigative Site
state: Michigan
city: Albuquerque
country: United States
facility: Novartis Investigative Site
state: New Mexico
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Ghaemi SN, Sverdlov O, van Dam J, Campellone T, Gerwien R. A Smartphone-Based Intervention as an Adjunct to Standard-of-Care Treatment for Schizophrenia: Randomized Controlled Trial. JMIR Form Res. 2022 Mar 28;6(3):e29154. doi: 10.2196/29154.