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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02338609
Other study ID # CRAD001M2305
Secondary ID 2013-003795-13
Status Completed
Phase Phase 4
First received
Last updated
Start date December 17, 2014
Est. completion date December 18, 2023

Study information

Verified date February 2024
Source Novartis
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The primary objective of CRAD001M2305 is to report the long-term effects of everolimus treatment on height, weight and sexual development (using Tanner Stages) in children and adolescents with Tuberous Sclerosis Complex (TSC)-associated with Subependymal Giant Cell Astrocytoma (SEGA). The study will monitor the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in CRAD001M2301 until they reach Tanner Stage V, or until age 16 for females or 17 for males whichever occurs first.


Description:

CRAD001M2305 is a prospective, multi-center phase IIIb/IV study. This study will investigate if the physical and sexual development of pediatric patients is affected by previous or ongoing treatment with everolimus. The study will monitor the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in CRAD001M2301 until they reach Tanner Stage V, or until age 16 for females or 17 for males whichever occurs first. Continued treatment with everolimus is at investigator discretion and is not required for participation in CRAD001M2305. Growth (height, weight), and sexual development (Tanner Stages, sex hormone levels, age at menarche, thelarche (females) and adrenarche (males)), and brain development (assessed by TAND Checklist, dates of brain MRI) of patients participating in this long-term follow-up study will be followed at annual visits to the site until patients achieve Tanner Stage V or age 16 (females), age 17 (males) whichever occurs first. Adverse events, concomitant medication, appearance of menarche, will be monitored and data collected every 3 months ("3-monthly"). Unless clinically indicated these 3-monthly visits can be performed per telephone.


Recruitment information / eligibility

Status Completed
Enrollment 15
Est. completion date December 18, 2023
Est. primary completion date December 18, 2023
Accepts healthy volunteers No
Gender All
Age group 0 Years to 17 Years
Eligibility Inclusion Criteria: - Pediatric female patients who were on study treatment in study [CRAD001M2301] within the past 63 months and have not reached Tanner Stage V or age 16 at the time of completion of [CRAD001M2301] or - Pediatric male patients who were on study treatment in study [CRAD001M2301] within the past 6 months and have not reached Tanner Stage V or age 17 at the time of completion of [CRAD001M2301] - Written informed consent according to local guidelines Exclusion Criteria: - Pediatric female patients who were on study treatment in CRAD001M2301 and have not reached Tanner Stage V but are within 3 month of turning age 16 or - Pediatric male patients who were on study treatment in CRAD001M2301 and have not reached Tanner Stage V but are within 3 months of turning age 17 - Any patient who was pregnant prior to start of CRAD001M2305 Other protocol-defined Inclusion/Exclusion criteria

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Everolimus
Blood sample and Tanner Stage assessment
Envirolimus drug class as prescribed by Physician
At the discretion of the investigator, patients may be treated with commercially available everolimus, as per local product information/ standard of care.

Locations

Country Name City State
Belgium Novartis Investigative Site Brussel
Russian Federation Novartis Investigative Site Moscow
United States Cinn Children Hosp Medical Center SC Cincinnati Ohio
United States Texas Scottish Rite Hospital for Children SC Dallas Texas
United States University Of California Los Angeles SC Los Angeles California
United States Minnesota Epilepsy Group Saint Paul Minnesota

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Belgium,  Russian Federation, 

Outcome

Type Measure Description Time frame Safety issue
Primary Percentage of patients who achieved Tanner Stage V at or before age 16 (females) or 17 (males) Patients will be assessed annually until Tanner Stage 5 is achieved Annually up to 12 years
Primary Height/Body Mass Index (BMI) standard deviation score since baseline Height and weight (for BMI) will be assessed to monitor growth. Annually up to 12 years
Primary Mean Endocrine laboratory values Blood sample will be drawn to assess FSH, LH, testosterone (male) or estrogen (female) by age Annually up to 12 years
Secondary Long-term safety as assessed by National Cancer Institute (NCI) Common Toxicity Criteria or Adverse Events (CTCAE) Safety as assessed by National Cancer Institute (NCI) Common Toxicity Criteria or Adverse Events (CTCAE). Adverse Events (AEs)/Serious Adverse Events (SAEs), concomitant medications (conmeds) will be collected as needed. Annually up to 12 years
Secondary Age at menarche/thelarche (females) or adrenarche (males) Age when patient achieve each benchmark will be recorded and compared with standards. Menarche = start of menses Thelarche = onset of breast development Adrenarche = pubic haor development Annually up to 12 years
Secondary Age at Tanner Stage II, III, IV, V Age at when each Tanner Stage is achieved will be collected and compared against known standards. Annually up to 12 years
Secondary Neuropsychological development TSC-Associated-Neuropsychiatric-Disorders (TAND) Checklist will be used to follow-up patient neuropsychological development Annually up to 12 years
Secondary Height/BMI standard deviation score by year since baseline Height, weight, Tanner staging will be compared to data from TSC patients who have not been treated with everolimus Annually up to 12 years
Secondary Percentage of patients who achieved Tanner Stage V at or before age 16 (females) or 17 (males) Patients will be assessed annually until Tanner Stage 5 is achieved Annually up to 12 years
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