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

NCT03835117 | Completed Phase 2 Results FDA Drug

• 1 other identifier: MITO
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

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.

Conditions

Autism Spectrum Disorder

Keywords

Mitochondrial Dysfunction

Outcome Measures

Primary Outcomes (4)

• Change in Mitochondrial Activity in Study Patients: Citrate Synthase

  Mitochondrial activity and redox metabolism at baseline and after the placebo and supplement arms of the study, as determined through laboratory assessment. Normal Range 4.4 to 22 with Mean and SD of 12.1 and 5.1. It is better to be within the normal range than outside of the normal range regardless of whether the value is higher or lower.

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

• Change in Mitochondrial Activity in Study Patients: Complex I

  Mitochondrial activity and redox metabolism at baseline and after the placebo and supplement arms of the study, as determined through laboratory assessment. Normal range is 3.4 to 11.9 with a mean of 7.65. Outside of the normal range is worse regardless of whether it is above or below the normal range.

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

• Change in Mitochondrial Activity in Study Patients: Complex II

  Mitochondrial activity and redox metabolism at baseline and after the placebo and supplement arms of the study, as determined through laboratory assessment. Normal Range (mean ±SD) 0.03 -- 0.35 (0.194 ±0.08). It is better to be within the normal range regardless of whether the value is above or below the normal range

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

• Change in Mitochondrial Activity in Study Patients: Complex IV

  Mitochondrial activity and redox metabolism at baseline and after the placebo and supplement arms of the study, as determined through laboratory assessment. Normal Range (mean ±SD) 0.15 -- 0.6 (0.31 ±0.1). Better scores are inside the normal range regardless of if they are higher or lower.

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

Secondary Outcomes (9)

• Change in the Childhood Autism Rating Scale (CARS) Score

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

• Change in the Clinical Global Impression Scale (CGI) Score

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

• Change in the Children's Yale-Brown Obsessive Compulsive Scale Modified for Autism Spectrum Disorder (CYBOCS-ASD) Score

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

• Change in the Aberrant Behavior Checklist (ABC) Scores

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

• Change in the Parent-rated Anxiety Scale for ASD (PRAS-ASD) Score

  Before (Baseline), After Part I (Week 12), After Part 2 (Week 24)

Study Arms (2)

Wide-spectrum nutritional supplement

EXPERIMENTAL

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.

Drug: Wide-spectrum nutritional supplement

Placebo control

PLACEBO COMPARATOR

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.

Other: Placebo

Interventions

Wide-spectrum nutritional supplement DRUG

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

Also known as: NeuroNeeds: Spectrum Needs, NeuroNeeds:Q Needs

Wide-spectrum nutritional supplement

Placebo OTHER

Inactive placebo comparator

Placebo control

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Rossignol Medical Center: lead
• Southwest Autism Research & Resource Center: collaborator

Study Sites (1)

Southwestern Research & Resource Center

Phoenix, Arizona, 85016, United States

Location

Related Publications (3)

• 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.

  PMID: 28208802 BACKGROUND

• 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.

  PMID: 30039193 BACKGROUND

• 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.

  PMID: 25019065 BACKGROUND

MeSH Terms

Conditions

Autism Spectrum Disorder; Mitochondrial Diseases

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Richard E Frye
• Organization: Autism Discovery & Treatment Foundation

DrFrye@AutismDiscovery.org

844-ADTF-Research

Study Officials

• Richard E Frye, MD, PhD

  Rossignol Medical Center, Phoenix AZ

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Research

Model Details: Prospective Randomized 12-week Double-Blind Placebo-Controlled Cross-over

Study Record Dates

• First Submitted: February 5, 2019
• First Posted: February 8, 2019
• Study Start: February 1, 2020
• Primary Completion: July 31, 2022
• Study Completion: July 31, 2022
• Last Updated: May 9, 2025
• Results First Posted: May 9, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)