Autism Spectrum DisorderPhase 1NCT04031755
A Proof-of-Concept Study of Minocycline in Autism
A Phase 1 study testing Minocycline in people with Autism Spectrum Disorder and Autism, run by Children's Hospital Medical Center, Cincinnati at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 22 years
- Conditions the study is for
- Autism Spectrum Disorder, Autism
- What is being tested?
- Minocycline
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 53 participants
- Where
- 3 sitesUnited States
- Study dates
- 19 Apr 2019to 30 Oct 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 22 ≥ 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)
Who cannot join
- 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
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- 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.
- Women who are pregnant (i.e. have a positive pregnancy test), intending to become pregnant, breast feeding, or women of child-bearing potential who are unwilling to use contraception as required in the study inclusion criteria or maintain abstinence during the course of the study
- 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
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Thompson Center for Autism & Neurodevelopmental Disorders - University of Missouri
Columbia, Missouri, 65211
Center for Autism and Developmental Disorders - University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15203
About this study
Official title: Comparison of the Pharmacodynamic and Tolerability Profiles of Minocycline Versus Placebo in Autism Spectrum Disorder: a Double-blind, Placebo-controlled, Crossover, Proof-of-concept Study
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.
Read the 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
- Being tested
- Minocycline, Placebos
- Keywords
- Autism, Autism Spectrum Disorder, Minocycline
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder, cyclopia sequence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MinocyclineDrug
Also called Minocin
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.
PlacebosDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Drug: Minocycline; Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Subject weight will be compared pre- and post-treatment in the drug versus placebo conditions
Subject weight will be measured in kilograms
Measured: 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
Measured: 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
Measured: Through study completion, an average of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2019
- Study start19 Apr 2019
- Primary completion30 Oct 2020
- Study completion30 Oct 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Craig Erickson, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04031755
- Sponsor's protocol ID
- CIN001 - Minocycline in ASD
- Status last verified
- Dec 2020
- First submitted
- 10 May 2019
- Last updated
- 8 Dec 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of the Pharmacodynamic and Tolerability Profiles of Minocycline Versus Placebo in Autism Spectrum Disorder: a Double-blind, Placebo-controlled, Crossover, Proof-of-concept Study
- ClinicalTrials.gov ID
- NCT04031755
- Acronym / sponsor ID
- CIN001 - Minocycline in ASD
- Conditions
- Autism Spectrum Disorder, Autism
- Keywords
- Autism, Autism Spectrum Disorder, Minocycline
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Study start
- 19 Apr 2019 (actual)
- Primary completion
- 30 Oct 2020 (actual)
- Study completion
- 30 Oct 2020 (actual)
- First posted
- 24 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Minocycline versus Placebo | Experimental | Drug: Minocycline, Drug: PlacebosPhase 1: 4 weeks of daily minocycline 100mg BID dosing 2-week washout period Phase 2: 4 weeks of daily placebo dosing |
| Placebo versus Minocycline | Experimental | Drug: Minocycline, Drug: PlacebosPhase 1: 4 weeks of daily placebo dosing 2-week washout period Phase 2: 4 weeks of daily minocycline 100mg BID dosing |
Who may qualify
- Ages
- 12 to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 22 ≥ 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MinocyclineAlso: MinocinMinocycline 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. | Drug | Minocycline versus Placebo, Placebo versus Minocycline |
| PlacebosMinocycline 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. | Drug | Minocycline versus Placebo, Placebo versus Minocycline |
Primary outcome
Subject weight will be compared pre- and post-treatment in the drug versus placebo conditions
Time frame: Through study completion, an average of 2 years
Subject weight will be measured in kilograms
Aberrant Behavior will be evaluated pre- and post-treatment in the drug versus placebo conditions
Time frame: Through study completion, an average of 2 years
Aberrant behavior will be measured by the Aberrant Behavior Checklist total score
Incidence of liver toxicity will be evaluated in the pre- and post-treatment setting in the drug versus placebo conditions
Time frame: Through study completion, an average of 2 years
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Craig Erickson principal investigator
Children's Hospital Medical Center, Cincinnati
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Minocycline in people with Autism Spectrum Disorder and Autism, run by Children's Hospital Medical Center, Cincinnati at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Subject weight will be compared pre- and post-treatment in the drug versus placebo conditions (time frame: Through study completion, an average of 2 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2020.