A Proof-of-Concept Study of Minocycline in Autism

NCT ID: NCT04031755

Last Updated: 2020-12-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 53 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-04-19
• Study Completion Date: 2020-10-30

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.

Detailed Description

This is a double-blind, placebo-controlled, crossover, proof-of-concept study that compares the pharmacodynamic and tolerability profiles of minocycline versus placebo in autism spectrum disorder.

Conditions

Autism Spectrum Disorder; Autism

Keywords

Autism; Autism Spectrum Disorder; Minocycline

Study Design

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

Study Groups

Minocycline versus Placebo

Phase 1: 4 weeks of daily minocycline 100mg BID dosing

2-week washout period

Phase 2: 4 weeks of daily placebo dosing

Group Type: EXPERIMENTAL

Minocycline

Intervention Type: 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.

Placebos

Intervention Type: 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.

Placebo versus Minocycline

Phase 1: 4 weeks of daily placebo dosing

2-week washout period

Phase 2: 4 weeks of daily minocycline 100mg BID dosing

Group Type: EXPERIMENTAL

Minocycline

Intervention Type: 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.

Placebos

Intervention Type: 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.

Interventions

Minocycline

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.

Intervention Type: DRUG

Placebos

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.

Intervention Type: DRUG

Other Intervention Names

Minocin

Eligibility Criteria

Inclusion Criteria

1. 22 ≥ Age ≥12 years. Males and females included in study.

2. 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.

3. General good health as determined by physical exam, medical and psychiatric history and safety labs as defined by the PI or designee.

4. 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.

5. 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.

6. Whole brain absolute cumulative gamma power (30 to 80 Hz) with median cut off at 2.5 (upward adjusted)

14. 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

Exclusion Criteria

1. Allergy or hypersensitivity to any of the tetracyclines antibiotics.

2. Inability to swallow study drug.

3. Concomitant use of scheduled anti-inflammatory drugs with the exception of as needed ibuprofen or acetaminophen use.

4. Unstable dosing of any mood, anxiety or behavior medications in the 5 half-lives prior to Phase 1 baseline visit.

5. Concomitant use of scheduled benzodiazepines, baclofen, gabapentin, pregabalin, or supplements with impact on the GABA system.

6. Concomitant daily use of antacids

7. Concomitant use of oral acne medications (isotretinoin), not including lotions or creams applied to the skin

8. Concomitant use of any cannabinoid or related product.

9. 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.

10. 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.

11. History of autoimmune disorder

12. History of or current abuse of drugs or alcohol including prescription medication.

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 22 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Craig Erickson

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Locations

Thompson Center for Autism & Neurodevelopmental Disorders - University of Missouri

Columbia, Missouri, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

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

Pittsburgh, Pennsylvania, United States

Countries

United States

Other Identifiers

CIN001 - Minocycline in ASD

Identifier Type: -

Identifier Source: org_study_id