Effects of Metformin on Androgens and Other Steroid Hormones in Affected Subjects With Autism (Metformin)

January 6, 2025 updated by: Insel Gruppe AG, University Hospital Bern

A Cohort Study to Elucidate the Effects of Metformin on Androgens and Other Steroid Hormones in Affected Subjects With Autism - a First Pilot Study

Autism is diagnosed with increasing frequency in recent years despite remaining uncertainty concerning the cause. The disorder is characterized by deficits in social behavior, a lack of communication skills, and repetitive and stereotypical interests. As part of the research, It attempted to pursue the hypothesis that the disorder is signed by an endocrine involvement. Therefore, the original description of Hans Asperger was analyzed first. This was followed by comprehensive steroid hormone analyses in girls and boys with autism. Based on the assumption that steroid hormones are involved, dysregulation of the adrenal gland for all metabolite classes - mineralocorticoid, glucocorticoid, androgen - were identified. The subsequently followed animal experiments yielded to the conclusion that a dysregulation of the hypothalamic-pituitary system might be responsible for the autistic behavior. This is also probably associated with overactivity of 17/20 lyase, an orchestrating enzyme of oxidative stress, which is driven by p38. It is suspected that the increased oxidative stress is of mitochondrial origin and thus other metabolic cascades are involved. Due to the developed understanding of the suspected dysregulation, new therapeutic options for treatment are opening up, with metformin in particular - known for its antiandrogenic effect used in poly cystic ovarian syndrome - appearing to have the best effect on social withdrawal in the developed mouse model. Initial urine analyses allow the assumption that metformin directly influences steroidogenesis, and thus opens up the possibility of a clinical trial for affected subjects with autism.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

To date, the etiopathogenesis or the biochemical basis of social withdrawal behavior have not been conclusively clarified and accordingly the treatment options are not exhausted. Investigators know from analyzes of those affected by the most severe form of social impairment respectively from subjects with autism that there appears to be a change in the cholesterol-dependent steroid metabolism. Furthermore, it can be assumed that treatment with different substances could be promising. Metformin seems to cause a direct inhibition of the androgen synthesis pathways while parallel influencing social behavior. In principle, Metformin is mainly used to treat Diabetes mellitus type 2. It is a biguanide drug that has been used for more than 60 years. Studies have shown that metformin improves mortality rates in diabetes patients, and younger studies show additional effects in treating cancer, obesity, nonalcoholic fatty liver disease, polycystic ovary syndrome and metabolic syndrome.Treatment with metformin mimics some of the benefits of caloric restriction, such as improved physical performance while yielding to increased insulin sensitivity and reduced low-density lipoprotein and cholesterol levels. Furthermore, as chronic inflammation is also influenced by exercise training it is allowed to assume similar mechanism of Metformin and physical activity. At a molecular level, metformin increases activated protein kinase activity and increases antioxidant protection, resulting in reductions in both oxidative damage accumulation and chronic inflammation thereby mimicking positive effects of endurance training.In consequence, it was indicated that these actions may contribute to the beneficial effects of metformin on health- and lifespan. Focusing on neurologic diseases affected by Metformin positive effects on Alzheimer's disease were shown. Metformin attenuated spatial memory deficit, neuron loss in the hippocampus and enhanced neurogenesis in mice. In another mouse model the metformin treatment counteracted the development of depression-like behaviors in mice suffering social defeat stress when administered. This directly yields to the aim of the study: to assess effects of Metformin on steroid hormones in subjects affected with autism.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
20

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Subjects with autism

Description

The inclusion criteria:

  • Diagnosis with autism according to DSM-V.
  • Written informed consent

The exclusion criteria:

  • Clinically significant concomitant disease states (e.g. advanced renal failure, hepatic Dysfunction - risk factor of lactate acidosis)
  • Significant musculoskeletal disease
  • Active infection
  • Immunosuppressive medical therapy
  • Known or suspected non-compliance, drug or alcohol abuse
  • Pregnant or breastfeeding mothers are excluded
  • Inability to follow the procedures of the study, e.g. due to insufficient language skills, psychological disorders, very severe dementia inability to report adequately

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Metformin Treatment
Drug: Metformin treatment
Interested subjects with autism are treated with metformin with a standard dosage
Other Names:
  • Metformin treatment in subjects with autism

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Steroid hormone concentrations of total androgens
Time Frame: After 6-9 month of treatment with Metformin
Steroid hormones
After 6-9 month of treatment with Metformin

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Insel Gruppe AG, University Hospital Bern

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Markus Mohaupt, Prof. Dr., University of Berne

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 30, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 30, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 30, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 21, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 6, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 25, 2025

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 6, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • Steroidlabs
  • Lindenhofhospital Berne (Other Identifier: Lindenhofhospital Berne)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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