Effect of a Wide Spectrum Nutritional Supplement on Mitochondrial Function in Children With Autism Spectrum Disorder

NCT ID: NCT03835117

Last Updated: 2025-05-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-02-01
• Study Completion Date: 2022-07-31

Brief Summary

The objective of this study is to evaluate the metabolic effects of a comprehensive wide-spectrum supplement for children with ASD to determine whether it physiologically targets mitochondrial pathways known to be abnormal in children with ASD.The intervention is a commonly used wide-spectrum nutritional supplement, which is theoretically designed to normalize mitochondrial function. The investigators aim to determine if the supplement does have the hypothesized effect on physiology in individuals with ASD. The investigator will enroll up to 50 children, aged 4 to 14 years of age with confirmed ASD and mitochondrial dysfunction, and participation will last 26 weeks.

Detailed Description

Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder often with life-long consequences that affects young children during critical developmental periods. The Centers for Disease Control estimates that ASD affects as many as 17 per 1000 children (1 in 59) in the United States suggesting that the prevalence is higher than previous estimates.Despite the dramatic rise in the detected prevalence of ASD over the past two decades, there is no effective medical treatment for core ASD symptoms (social communication and repetitive behavior), the closely associated problem of language impairment, or the underlying pathophysiology of ASD. Currently, the only accepted treatment for core ASD symptoms is behavior therapy, which may entail intensive one-on-one treatment over several years.

The primary aims of this study are to evaluate the effect of a wide-spectrum nutritional supplement on mitochondrial function in individuals with ASD. Participants entered into the trial will have abnormalities in mitochondrial function that are known to be associated with ASD (approximately 50+% of children with ASD) but are not diagnostic of mitochondrial disease. The investigators hypothesize that nutritional supplements designed for children with ASD have a physiological action of normalizing mitochondrial function and cellular physiology throughout the body.

To test whether the targeted nutritional supplement is superior to placebo, the investigators will study 50 children, between the ages of 4 years to 14 years, with confirmed ASD and known abnormal variations in mitochondrial at baseline. Participants will be randomly assigned to receive active treatment or placebo for 12-weeks under double-blind conditions and at the end of the 12 weeks switch to the opposite condition after a 2-week wash out period. Mitochondrial function will be measured at baseline and after each treatment arm in order to determine if the supplement positively influences cellular biochemistry. The investigator will also evaluate the effectiveness of the supplement on core and associated ASD symptoms using several behaviors assessments.

Conditions

Autism Spectrum Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Wide-spectrum nutritional supplement

The Wide-spectrum nutritional supplement used will be a combination of NeuroNeeds: SpectrumNeeds and QNeeds. Weight based dosing will be used. The daily serving size will be divided into two oral daily doses in the form of a powder which can be mixed into liquid or food. Together, there are 34 different dietary supplements in the products. Except for ubiquinol, all of these nutrients are provided in a powder form in SpectrumNeeds. Ubiquinol is provided separately in QNeeds gel capsules. These capsules can be swallowed whole, or cut with scissors and the contents squeezed out and added to SpectrumNeeds just before ingestion.

Group Type: EXPERIMENTAL

Wide-spectrum nutritional supplement

Intervention Type: DRUG

Comprehensive powder with 33 dietary supplements and 1 dietary supplement via gel capsule.

Placebo control

Participants randomized to receive placebo will take placebo in an oral form divided into powder and a gel capsule in the same manner as treatment. For the second phase of the cross over, participants will be part of the opposite group they were assigned to in Phase I (Placebo or Treatment). Quantities for placebo or treatment will match across phases for each subject, utilizing the same weight based dosing.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Inactive placebo comparator

Interventions

Wide-spectrum nutritional supplement

Comprehensive powder with 33 dietary supplements and 1 dietary supplement via gel capsule.

Intervention Type: DRUG

Placebo

Inactive placebo comparator

Intervention Type: OTHER

Other Intervention Names

NeuroNeeds: Spectrum Needs, NeuroNeeds:Q Needs

Eligibility Criteria

Inclusion Criteria

• Weight ≥ 15 kg and ≤ 100kg;
• DSM-5 diagnosis of Autism Spectrum Disorder as established by formal clinical assessment which includes a gold-standard tool such as the Autism Diagnostic Observational Schedule.
• Current Clinical Global Impression Severity score ≥ 4
• Stable educational and therapy plan (one month) with no planned changes in the intensity of treatment for 12 weeks.
• English is spoken in the home and at least one parent is able to read, write and speak English.
• Stable medication (no changes in past 6 weeks and no planned changes for the study duration.
• Electron Transport Chain Complex (I, II, III, IV) or Citrate Synthase Activity which is >= 2.0 Standard Deviation Above or Below Average (outside the normal range)

Exclusion Criteria

• Presence of serious behavioral problems (tantrums, aggression, self-injury) for which another treatment is warranted.
• Current Clinical Global Impression Severity score < 7 (Extremely Ill)
• Significant medical condition by history or by physical examination or lab tests that would be incompatible with the treatment.
• Children taking anticonvulsant medication for seizures or active epilepsy.
• Diagnosis of Mitochondrial Disease

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Southwest Autism Research & Resource Center

OTHER

Sponsor Role: collaborator

Rossignol Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Richard Frye

Director of Research

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Richard E Frye, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Rossignol Medical Center, Phoenix AZ

Locations

Southwestern Research & Resource Center

Phoenix, Arizona, United States

Countries

United States

References

Delhey LM, Nur Kilinc E, Yin L, Slattery JC, Tippett ML, Rose S, Bennuri SC, Kahler SG, Damle S, Legido A, Goldenthal MJ, Frye RE. The Effect of Mitochondrial Supplements on Mitochondrial Activity in Children with Autism Spectrum Disorder. J Clin Med. 2017 Feb 13;6(2):18. doi: 10.3390/jcm6020018.

Reference Type: BACKGROUND

PMID: 28208802 (View on PubMed)

Rose S, Niyazov DM, Rossignol DA, Goldenthal M, Kahler SG, Frye RE. Clinical and Molecular Characteristics of Mitochondrial Dysfunction in Autism Spectrum Disorder. Mol Diagn Ther. 2018 Oct;22(5):571-593. doi: 10.1007/s40291-018-0352-x.

Reference Type: BACKGROUND

PMID: 30039193 (View on PubMed)

Frye RE, Rossignol DA. Treatments for biomedical abnormalities associated with autism spectrum disorder. Front Pediatr. 2014 Jun 27;2:66. doi: 10.3389/fped.2014.00066. eCollection 2014.

Reference Type: BACKGROUND

PMID: 25019065 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

MITO

Identifier Type: -

Identifier Source: org_study_id