A Proof-of-Concept Study of Minocycline in Autism

NCT04031755 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: CIN001 - Minocycline in ASD
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 3

Brief Summary

The purpose of the study is to determine if Minocycline shows initial evidence of efficacy, safety, and tolerability in youth with Autism Spectrum Disorder ages 12 to 22 years.

Conditions

Autism Spectrum Disorder; Autism

Keywords

Autism; Autism Spectrum Disorder; Minocycline

Outcome Measures

Primary Outcomes (3)

• Subject weight will be compared pre- and post-treatment in the drug versus placebo conditions

  Subject weight will be measured in kilograms

  Through study completion, an average of 2 years

• Aberrant Behavior will be evaluated pre- and post-treatment in the drug versus placebo conditions

  Aberrant behavior will be measured by the Aberrant Behavior Checklist total score

  Through study completion, an average of 2 years

• Incidence of liver toxicity will be evaluated in the pre- and post-treatment setting in the drug versus placebo conditions

  Liver toxicity will be defined as the development of an ALT or AST value greater than twice the upper limit of normal during a treatment period

  Through study completion, an average of 2 years

Study Arms (2)

Minocycline versus Placebo

EXPERIMENTAL

Phase 1: 4 weeks of daily minocycline 100mg BID dosing 2-week washout period Phase 2: 4 weeks of daily placebo dosing

Drug: Minocycline; Drug: Placebos

Placebo versus Minocycline

EXPERIMENTAL

Phase 1: 4 weeks of daily placebo dosing 2-week washout period Phase 2: 4 weeks of daily minocycline 100mg BID dosing

Drug: Minocycline; Drug: Placebos

Interventions

Minocycline DRUG

Minocycline 100 mg capsules or matching placebo will be prepared by the central investigational pharmacy at Cincinnati Children's Hospital Medical Center. Drug identity will be masked by over encapsulation of the study drug.

Also known as: Minocin

Minocycline versus Placebo; Placebo versus Minocycline

Placebos DRUG

Minocycline 100 mg capsules or matching placebo will be prepared by the central investigational pharmacy at Cincinnati Children's Hospital Medical Center. Drug identity will be masked by over encapsulation of the study drug.

Minocycline versus Placebo; Placebo versus Minocycline

Eligibility Criteria

• Age: 12 Years - 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• ≥ Age ≥12 years. Males and females included in study.
• Diagnostic confirmation of Autism Spectrum Disorder as confirmed by gold standard clinical interview using DSM 5 criteria and administration of the Autism Diagnostic Observation Schedule-2, Module 3 or 4.
• General good health as determined by physical exam, medical and psychiatric history and safety labs as defined by the PI or designee.
• Male study participants who are sexually active with a female partner of childbearing potential must be surgically sterilized, practicing abstinence, or agree to use highly effective methods of birth control (defined below), and not rely on barrier methods and spermicide alone, from the time of screening until 1 week after final dose of study drug.
• Female participants of childbearing potential may be included in the study provided they are practicing abstinence or are using a double barrier method from the time of screening until 1 week after the final dose of study drug. Participants using hormonal methods of birth control (oral, intravaginal, transdermal, injectable, or implantable) must be on a stable dose for at least three months prior to screening.
• Whole brain absolute cumulative gamma power (30 to 80 Hz) with median cut off at 2.5 (upward adjusted)

You may not qualify if:

• Allergy or hypersensitivity to any of the tetracyclines antibiotics.
• Inability to swallow study drug.
• Concomitant use of scheduled anti-inflammatory drugs with the exception of as needed ibuprofen or acetaminophen use.
• Unstable dosing of any mood, anxiety or behavior medications in the 5 half-lives prior to Phase 1 baseline visit.
• Concomitant use of scheduled benzodiazepines, baclofen, gabapentin, pregabalin, or supplements with impact on the GABA system.
• Concomitant daily use of antacids
• Concomitant use of oral acne medications (isotretinoin), not including lotions or creams applied to the skin
• Concomitant use of any cannabinoid or related product.
• Unstable seizure disorder as defined by any seizure in the 6 months prior to baseline visit and/or a change in any anti-convulsant drug dosing in the 60 days prior to study screen.
• Abnormal baseline safety lab assessments including, but not limited to ALT or AST greater than 1.5x the upper limit of normal, elevated ANA, total bilirubin or creatinine greater than 1x the upper limit of normal, other clinically relevant lab abnormality, or abnormality in ECG, HR or BP at screening as determined by the investigator or designee.
• History of autoimmune disorder
• History of or current abuse of drugs or alcohol including prescription medication.
• Inability to attend scheduled study visits, plans for family relocation during the study, or any other criteria that the investigator may determine to be associated with inability to complete the study

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (3)

Thompson Center for Autism & Neurodevelopmental Disorders - University of Missouri

Columbia, Missouri, 65211, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Center for Autism and Developmental Disorders - University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15203, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder; cyclopia sequence

Interventions

Minocycline

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Tetracyclines; Naphthacenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds

Study Officials

• Craig Erickson

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2019
• First Posted: July 24, 2019
• Study Start: April 19, 2019
• Primary Completion: October 30, 2020
• Study Completion: October 30, 2020
• Last Updated: December 8, 2020

Record last verified: 2020-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)