Evaluation of Intralesional Triamcinolone Acetonide Injections for Prevention of Refractory Strictures in Pediatric Esophageal Atresia Repair

The primary objective of this study is to determine whether **intralesional steroid injections** (ISI) in children with **esophageal atresia** can prevent the development of refractory strictures and subsequently reduce the total number of dilatations required within a 28-day interval. This is clinically relevant as it aims to improve patient outcomes by minimizing the need for repeated interventions, which can be burdensome and carry potential risks.

Secondary objectives include:

• Comparing the level of **dysphagia** between groups.
• Assessing the efficacy of ISI on the luminal esophageal diameter and stricture length.
• Evaluating the influence of co-medication on stricture formation.
• Analyzing potential systemic effects of ISI.
• Assessing the cost-effectiveness of the intervention between the two groups.

The clinical trial involves **children** diagnosed with **esophageal atresia** who have undergone primary anastomotic surgery within the first days of life. The study population includes both male and female participants, aged 3 months and older, who are in need of a third or fourth dilatation. The trial does not specifically target a vulnerable population. Participants were selected based on their medical history and the requirement for additional dilatations. The sponsor has not provided information regarding the total number of participants. Lifestyle considerations such as diet, physical activity, or habits are not specified in the available data. Key inclusion criteria include obtaining written informed consent from both parents or legal representatives, if applicable.

The clinical trial is designed to evaluate the efficacy of **intralesional steroid injections** in preventing refractory strictures in children with esophageal atresia. This is a **randomized, double-blind, controlled** trial, with a primary objective to assess whether the intervention can reduce the number of dilatations required within a 28-day interval. The trial is set to run until December 31, 2028, with recruitment having commenced on October 28, 2019. Participants will be involved in the study for a period of six months from the day of the study dilatation.

The trial involves several key study visits. Initially, an inclusion (screening) visit will be conducted to confirm eligibility based on criteria such as age (≥3 months), previous surgical intervention for esophageal atresia, and the need for a third or fourth dilatation. Following the screening, participants will undergo the study dilatation, which marks the start of the trial period. Subsequent follow-up visits will be scheduled to monitor the number of dilatations required and assess the development of any refractory strictures. The end-of-study visit will occur six months post-study dilatation, concluding the participant's involvement in the trial.

Participants may be withdrawn from the study prematurely if they experience adverse reactions to the treatment, fail to comply with the study protocol, or if the investigator deems it in the participant's best interest. The trial employs **triamcinolone acetonide** as the active substance, administered via local injection in the form of a suspension for injection. The maximum daily and total dose is set at 10 mg, with a treatment period not exceeding one day. The trial is conducted under the authorization of relevant regulatory bodies, ensuring adherence to ethical and safety standards throughout its duration.

The clinical trial involves the administration of two experimental medications, both containing the active substance **triamcinolone acetonide**. The first medication, Kenacort-A 10, is a suspension for injection with a concentration of 10 mg/ml. It is manufactured by Bristol-Myers Squibb B.V. and is authorized in the Netherlands. The pharmaceutical form is a suspension for injection, and the medication is administered via local injection. The maximum daily dose is 10 mg, with a total dose not exceeding 10 mg over the treatment period. The maximum treatment period is one day. Participant compliance with the dosing schedule will be monitored throughout the trial.

The second experimental medication is Kenacort-T 40 mg/ml, a solution for suspension for injection, also containing **triamcinolone acetonide**. This medication is produced by Bristol-Myers Squibb AB and is authorized in Sweden. Similar to Kenacort-A 10, it is administered via local injection. The maximum daily and total dose is 10 mg, with a treatment period limited to one day. The pharmaceutical form is a solution for suspension for injection, ensuring consistency in administration and absorption. Compliance with the dosing regimen will be closely monitored to ensure adherence to the study protocol.

Both medications are chemically derived and are not formulated for pediatric use. The trial does not include any non-experimental treatments such as standard-of-care therapy, placebo, or comparator treatments. The primary objective of the trial is to evaluate the efficacy of intralesional steroid injections in preventing refractory strictures in patients with esophageal atresia, thereby potentially reducing the number of dilatations required within a 28-day interval. The trial is conducted under authorized conditions, ensuring adherence to regulatory standards and participant safety.

Efficacy in this clinical trial will be assessed by evaluating the primary endpoint, which is the total number of dilatations required within a 28-day interval per patient during the study period. This period extends from the day of the study dilatation until six months later. The trial aims to determine whether intralesional steroid injections (ISI) in children with **esophageal atresia** can prevent the development of refractory strictures and consequently reduce the need for dilatations. The efficacy assessment will involve collecting data on the number of dilatations each patient undergoes within the specified timeframe. This data will be analyzed to determine the effectiveness of the treatment in achieving the primary objective of the trial.