Suramin has been found to correct the symptoms, metabolism, and brain synaptic abnormalities in two classical genetic and environmental mouse models of autism. A preliminary clinical trial (SAT-1) examined the safety and activity of a single low-dose of suramin in children with ASD and concluded suramin showed promise as a novel approach to treatment of ASD. The current study, STAT-2A, will be a randomized, double-blind, crossover, 30-week study to evaluate the preliminary proof of concept, safety, and PK of suramin sodium (KZ101) with repeat dosing by IV infusion in males 5-14 years of age who have been diagnosed with ASD. The study will be conducted at approximately 3 sites contributing approximately 15 subjects per site. Total enrollment of approximately 45 subjects is planned to achieve approximately 36 participants completing the study.
After up to a 4-week screening period, participants will undergo 8 weeks of active or placebo treatment (Period 1), followed by an 8-week washout period, and then cross over to 8 weeks of placebo or active treatment (Period 2). Patients will be followed for 2 weeks after completion of Period 2. Two dosing groups are designated as Group A, who are randomly assigned to active treatment with KZ101 in Period 1 and saline in Period 2, and Group B, who are randomly assigned to saline infusion in Period 1 and active treatment with KZ101 in Period 2. Dosing in both periods will consist of 2 IV infusions of either saline (placebo) or KZ101 (active treatment), given 4 weeks apart.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
45
For active treatment with KZ101, a loading dose of 454 mg/m2 (salt-free) will be followed by a treatment dose of 363 mg/m2 (salt-free).
Dosing in the placebo group will consist of a volume of normal saline equivalent to that given during the active treatment period for each participant.
Southwest Autism Research and Resource Center
Phoenix, Arizona, United States
NOT_YET_RECRUITINGChildren's Hospital Orange County, Thompson Autism and Neurodevelopmental Center
Orange, California, United States
RECRUITINGKennedy Krieger Institute
Baltimore, Maryland, United States
RECRUITINGPrimary Efficacy Endpoint - Vineland-3 Socialization Domain
The primary efficacy endpoint will be the change in standard score on the Socialization Domain of the Comprehensive Interview Form of the Vineland Adaptive Behavior Scale Third Edition (Vineland-3), which includes subdomains for coping skills, play skills, and interpersonal relation skills.
Time frame: Change from Screening to Week 8 (before washout) will be compared to change from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Vineland-3, Additional Scores
Vineland-3 Composite Score, Communication Domain Score, and Daily Living Skills Domain Score will also be assessed.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Global Impresssion, Severity/Change
Clinician Global Impression of Severity and Change, and Caregiver/Parent Global Impression of Severity and Change, will be collected and assessed weighted for socialization.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Social Responsiveness Scale-2
The Social Responsiveness Scale-2 (SRS-2) is a well-established tool to evaluate socialization that was also used in a prior pilot study.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Aberrant Behavior Checklist, Second Edition (ABC-2)
The ABC-2 provides measures of repetitive behaviors and includes domains of irritability, social withdrawal, stereotypy, hyperactivity, and inappropriate speech.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Childhood Sleep Habits Questionnaire (CSHQ)
As a large proportion of the adolescents with ASD population is highly impacted by sleep concerns, the CSHQ will be administered to investigate relationships between KZ101 and sleep.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Parenting Stress Impact (PSI)
Data from the PSI will be used to evaluate whether changes in symptoms in the ASD participant is associated with changes in parental stress throughout the study.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Child Behavior Check List (CBCL)
The CBCL is a parental measure of internalizing and externalizing behaviors as well as common DSM-5 conditions co-occurring with ASD.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
Secondary Efficacy Endpoint - Ohio State University Autism Rating Scale, 5th Edition
The Ohio State University Autism Rating Scale 5 (OARS-5) will provide three summary scores to be evaluated with KZ101 administration and placebo administration: autism symptom count, weighted mean severity score of autism symptoms, and impairment index based on level of support needed.
Time frame: Secondary outcome measures will be used to assess change from Week 0 to Week 8 (before washout) and from Week 16 to Week 24 (after washout).
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