Clinical Studies And Their Impact On Autism Spectrum Disorder


Autism spectrum disorder [ASD] is a neurological and developmental disorder which affects how people interact with others, communicate, learn and behave. Although Autism can be diagnosed at any age, it is described as a developmental disorder because the symptoms and traits generally appear in the first 2 years of life. Asperger’s syndrome or simply Asperger’s was retired as an official diagnosis in 2013. Asperger’s syndrome is now diagnosed as level 1 Autism Spectrum Disorder [ASD] or Autism with low support needs.

ASD is now the name used for a wide range of autism-like disorders. However, certain personnel may still use the term Asperger’s or ASD without intellectual or language impairment.

ASD is a developmental disability caused by differences in the brain. People with ASD often have problems with social communication and interaction and have restricted or repetitive behaviors or interests. People with Autism may also have different ways of learning, moving or paying attention.

It is pertinent to note that ASD as the name implies is a spectrum disorder. The spectrum in question may be imagined as a circular wheel of the symptoms rather than a lineal line with high functioning at the end of one line and low functioning at the other end of the line. This means that an autistic person may possess different traits and symptoms from said wheel and ASD may manifest itself differently as it is not a one-size-fits-all disorder but there are certain common traits in which majority of autistic people can relate to such as monotropism, lack of spatial awareness, inability to maintain eye contact, prolonged eye contact, inability to understand social cues, stimming, being non-verbal, experiencing alexithymia, burn out, overstimulation, emotional dysregulation, echolalia, savant syndrome, poor proprioception skills, etc.

Clinical researches have been conducted in order to further improve the quality of life of people with Autism Spectrum Disorder. Some of the researches by NHIC include;

1. Double Blind Trial in Children With ASD
The trial, funded by PaxMedica, was aimed at investigating the effectiveness of suramin as a safe treatment for the core symptoms of autism spectrum disorder (ASD). The study involved two dose levels of suramin vs. placebo in a multicenter, prospective, randomized, double- blind, and placebo-controlled multiple dose experiment. 52 male participants were randomized to undergo treatment interventions for ASD; around 17 participants were assigned to each treatment arm.

Male children between the ages of 4 and 17 who had received a diagnosis of ASD from the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V) and had been receiving sustained treatments for a minimum of two months were included in the study. Participants with acute medical problems, Rett syndrome, Microcephaly, Tuberous Sclerosis, Neurofibromatosis, Epilepsy, and clinically significant liver, kidney, or adrenal disease were not allowed to enroll in the study. Neither were those who planned to begin a new medication, diet, or behavioral intervention during the study.

An unblinded chemist created a 10% (100 mg/mL) solution of suramin by reconstituting a 1g vial of suramin in 10 mL of sterile water for infusion. When prepared, suramin and placebo were both obvious solutions. It was a double-blind study. The main goals were to compare the safety and effectiveness of two low-dose suramin dosage levels to a placebo in children with ASD receiving conventional treatment. The Clinical Global Impression (CGI) ratings and the Aberrant Behavior Checklist (ABC) were utilized to assess efficacy. The study is finished, and the findings are accessible. The following NCT06058962 is the study’s identification number.

The Double Blind Trial in Children With Autism Spectrum Disorder was a Phase II clinical trial. Here is how it broke down into phases:

Phase I (Not part of this trial): This phase involves a small number of healthy participants to test the safety and dosage of the medication.

Phase II (This trial):
Randomization: 52 male participants with Autism Spectrum Disorder (ASD) were randomly assigned to one of three treatment arms: two different doses of suramin or a placebo.

Double-Blind: Neither the participants nor the investigators knew which treatment arm the participants were in.

Treatment: Participants received the assigned treatment for a set period.

Efficacy Evaluation: The Aberrant Behavior Checklist (ABC) and Clinical Global Impression (CGI) scales were used to evaluate the effectiveness of the treatment.

Safety Monitoring: Participants were monitored for side effects and safety issues.

Phase III (Not part of this trial): If the results of Phase II were promising, the trial would have moved on to Phase III, which would involve a larger number of participants and a comparison to existing treatments.

Phase IV (Not part of this trial): After FDA approval, the medication would be monitored in a real-world setting to gather more information about its safety and effectiveness. This trial was a Phase II trial, which aimed to evaluate the safety and efficacy of suramin in children with ASD. Though encouraging, the trial’s findings have not yet been generally embraced.

The following are salient features of the findings and their possible implications:

Positive Outcomes: With a good safety and tolerability profile, the trial’s primary and multiple secondary endpoints revealed consistent improvements above placebo in PAX-101 (IV suramin).

Prospective Therapy: PAX-101 may be the first medication authorized to address the primary symptoms of autism spectrum disorder (ASD).

Next Actions: In order to get ready to perform more clinical trials, the firm is actively preparing to submit a Clinical Trial Application (CTA) in Europe and an Investigational New Drug (IND) in the United States.

Impact: Should PAX-101 be approved, it may alter the way that ASD is treated as well as other neurodevelopmental disorders.

2. Early Naturalistic Hands-on Autism Caregiver training (EHNANCE)
For young children with autism spectrum disorder (ASD), ENHANCE (Early Naturalistic Hands-on Autism Caregiver Training) is a caregiver-mediated intervention programme. It seeks to provide parents and other carers with the knowledge and techniques needed to promote their child’s growth and lessen ASD symptoms.

ENHANCE is play-based and naturalistic, emphasizing relationship-building and caregiver-
child contact. The curriculum offers techniques for handling difficult behaviors and places a strong emphasis on play, social engagement, and communication skills. Furthermore, ENHANCE can be provided in a clinic, home, or community setting and is tailored to the specific requirements and circumstances of each family. Carers also receive coaching and ongoing assistance. ENHANCE aims to improve the quality of interactions between carers and children, improve social and communication skills in children, increase play and cognitive development in children, decrease problem behaviors in children, support carer competence and confidence, and enhance family well-being and quality of life. ENHANCE seeks to improve the lives of children with ASD and their families by arming carers with practical techniques and resources.

Unlike the Double Blind trial previously mentioned, this is regarded as a study as it doesn’t test any products, only the interactions between people who care for those on the autism spectrum in order to lessen their symptoms and help them grow. The viability and efficacy of Early Naturalistic Hands-on Autism Carer Training (ENHANCE) are being researched and piloted. The goal of the hands-on, naturalistic intervention is to enhance children’s social communication and repetitive behavior. The study is still in progress, and the findings are not yet accessible. Here are a few salient features of the research:

Objective: The stud’s objectives are to assess the ENHANCE intervention’s viability, methodology, and initial carer satisfaction levels through a pilot test.

Methodology: A single-arm pre-post design will be used in this study, and parent feedback, participant completion and attrition rates, the interventionist study log, and the interventionist/ caregiver fidelity will all be measured.

Results: The interventionist study log, parent feedback, participant completion and attrition rates, and interventionist/caregiver/fidelity will be the main measures of success. Repetitive behavior and social communication in children will be the secondary outcomes.

Sample: The study will recruit parents and their children with autism spectrum disorder (ASD).

Location: The study will be conducted at the Mayo Clinic in Rochester, MN.

It is important to note that the study is still under progress and that the findings are not yet accessible. The study’s conclusions, however, will add to the body of knowledge already available on autism medical procedures and could influence further study and use. To further assist people with autism and their carers, additional research and interventions are being created and put into practice. Enhancing access to early intervention, meeting the needs of marginalized communities, and advancing evidence-based methods are the goals of these initiatives and research.
3. Multisite Controlled Secretin Trial In Autism
The University of Washington and the University of Colorado, Denver, two prestigious academic institutions, collaborated on the Multisite Controlled Secretin Trial in Autism. The National Institute on Deafness and Other Communication Disorders (NIDCD) provided extra assistance for this rigorous clinical investigation, which was supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

This multi-site trial, which ran from June 1999 to May 2000, involved 85 participants and lasted for almost a year. The results of the study had a significant impact on how we saw Secretin’s effectiveness as a possible cure for autism spectrum disorder (ASD). The Multisite Controlled Secretin experiment in Autism is one extensive clinical experiment aimed at evaluating the safety and effectiveness of Secretin as a possible treatment for Autism Spectrum Disorder (ASD). An extensive scientific study was required because anecdotal evidence suggested that Secretin, a hormone generated in the intestine, could be used as a treatment for ASD.

To guarantee impartial and objective outcomes, the study used a randomized, double-blind, placebo-controlled design, which is regarded as the gold standard in clinical research. A total of eighty-five participants, aged three to twelve, with ASD, were randomized at random to receive a placebo, biologically derived porcine secretin (bPS), or synthetic porcine secretin (sPS). The results of this multi-site study are more broadly applicable because it was carried out at multiple places across the United States.

The intervention consisted of injecting 0.1 ml of the prescribed medication intravenously at first, and if an allergic reaction did not occur, administering the entire dose over the course of a minute. The researchers tracked negative occurrences and assessed the impact of Secretin on the main characteristics of ASD, such as social interaction, communication, and behavioral functioning. There was no significant difference in the primary symptoms of ASD between the secretin groups and the placebo group, according to the study’s finding.

More specifically, there was no evidence that Secretin improved social interaction, improved communication, or lessened behavioral issues related to ASD. Furthermore, no appreciable variations in adverse events were observed between the Secretin and placebo groups in the trial.

The study’s conclusions are unambiguous: Secretin is not a suggested or appropriate treatment for ASD and is not a successful treatment. Strong evidence opposing the use of Secretin as a treatment for ASD is provided by these results, which are in line with other randomised controlled trials that have examined the substance’s effectiveness in treating the disorder. The trial was carried out at several locations in an effort to improve the study’s external validity and raise the result’s generalizability. The collaboration between two prestigious institutions and the presence of experienced investigators significantly increased the study’s legitimacy and scientific rigor.

The trial’s findings have greatly advanced the area of autism research and given physicians, researchers, and families of ASD patients insightful information. The study’s findings have influenced future research projects and treatment choices, ultimately advancing the use of evidence-based techniques in the field of autism. Families and medical professionals should proceed with caution when contemplating novel and potentially hazardous treatments for ASD in light of these findings. Rather, they ought to concentrate on evidence-based treatments that have undergone extensive testing and been shown to be successful in enhancing the lives of people with ASD. Helpful treatments for ASD must be found through further study, and projects like the Multisite Controlled Secretin Trial in Autism greatly advance our knowledge of the strategies that are helpful and ineffective in treating this difficult condition.

We highlighted 3 clinical trials and research currently going on to improve the standard of living of people living with ASD. Thousands more clinical trials and research are currently looking into innovative and patient-centric methods, approaches and drugs to further improve the quality of life. The role that quality data management of these clinical researches plays cannot be overemphasized.

At Zilla Clinicals, we give utmost care to ensure ethical conducts are adhered to during rigorous data management processes. We aim to change the world one clinical trial at a time and ASD patients deserve a good life in that world.

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