Phase 4
N=15
Long-term Monitoring of Growth and Development of Pediatric Patients Previously Treated With Everolimus
Growth and Development
Bottom Line
View on ClinicalTrials.gov: NCT02338609 ↗Enrolled (actual)
15
Serious AEs
42.9%
Results posted
Aug 2024
Primary outcome: Primary: Number of Participants Who Achieved Tanner Stage V at or Before Age 16 (Females) or 17 (Males) — 4; 6 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Everolimus (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Dec 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Achieved Tanner Stage V at or Before Age 16 (Females) or 17 (Males) |
4; 6 | — |
| PRIMARY Number of Participants With Notably Low and Notably High Height and Body Mass Index (BMI) Standard Deviation Score (SDS) |
2; 1; 2; 2; 2; 2 | — |
| PRIMARY Endocrine Laboratory Values LH and FSH in Male Participants |
1.0; 1.5; 1.0; 1.7; 1.8; 2.1 | — |
| PRIMARY Endocrine Laboratory Values LH and FSH in Female Participants |
5.1; 3.4; 3.2; 3.5; 7.8; 5.6 | — |
| PRIMARY Endocrine Laboratory Values of Testosterone in Male Participants |
0.7; 2.5; 3.8; 4.3; 7.2; 12.9 | — |
| PRIMARY Endocrine Laboratory Values of Estrogen in Female Participants |
139.8; 80.5; 43.7; 50.0; 139.0; 47.7 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
12; 6; 0; 0 | — |
| SECONDARY Participants Age at Menarche/Thelarche (Females) or Adrenarche (Males) |
12.5; 10.2; 11.0 | — |
| SECONDARY Participants Age at Tanner Stage II, III, IV, V |
11.8; 11.9; 13.3; 12.6; 14.2; 14.2 | — |
| SECONDARY TAND Checklist: Number of Participants Achieved Basic Developmental Milestones and the Age at Which Participants Achieved the Basic Developmental Milestones |
2.9; 2.9; 7.5; 7.5; 16.5; 16.5 | — |
| SECONDARY TAND Checklist: Number of Participants With Behavioral Disorders |
12; 13; 6; 8; 5; 7 | — |
| SECONDARY TAND Checklist - Number of Participants With Psychiatric Disorders |
4; 7; 5; 9; 4; 7 | — |
| SECONDARY TAND Checklist - Number of Participants With Scholastic Issues |
12; 14; 12; 14; 12; 14 | — |
| SECONDARY TAND Checklist: Number of Participants With Difficulty in Specific Brain Skills |
7; 12; 12; 14; 12; 14 | — |
| SECONDARY TAND Checklist - Number of Participants With Psychological Issues |
2; 6; 6; 8; 5; 7 | — |
| SECONDARY TAND Checklist - Number of Participants With Varied Levels of Language Skills |
2; 6; 4; 3 | — |
| SECONDARY TAND Checklist - Number of Participants With Different Levels of Physical Dependency |
4; 7; 1; 3 | — |
| SECONDARY TAND Checklist: Number of Participants With Different Levels of Mobility |
1; 1; 0; 10; 3 | — |
| SECONDARY TAND Checklist: Number of Participants With Different Levels of Intelligence Quotient |
2; 2; 1; 5; 0; 1 | — |
| SECONDARY TAND Checklist: Number of Participants With Different Levels of Intellectual Ability |
1; 8; 3; 3 | — |
Summary
The primary objective of CRAD001M2305 was 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 monitored the growth and development of pediatric patients with TSC-associated SEGA, previously enrolled in CRAD001M2301 (NCT00789828) until they reach Tanner Stage V, or until age 16 for females or 17 for males whichever occurred first.
Eligibility Criteria
Inclusion Criteria
- Pediatric female patients who were on study treatment in study [CRAD001M2301] within the past 6 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
Data sourced from ClinicalTrials.gov (NCT02338609). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.