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mTOR inhibitor

Long-Term Safety of Everolimus for Tuberous Sclerosis

Phase 3
Waitlist Available
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1 up to approximately 10 years, assessed every 12 weeks,
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is for patients with TSC and refractory seizures who are currently receiving everolimus treatment and are determined to be benefiting from it, in order to evaluate the long-term safety of the treatment.

Who is the study for?
This trial is for patients with Tuberous Sclerosis Complex (TSC) and hard-to-treat seizures who are already part of the EXIST-3 study, taking everolimus, and seeing benefits. They must have followed the previous study's rules well and agree to keep doing so. People can't join if they've stopped everolimus in EXIST-3 or if it's now approved and paid for in their country.
What is being tested?
The trial is looking at the long-term safety of a drug called everolimus in patients with TSC who experience seizures that don’t respond well to other treatments. These patients have seen improvements from this drug during a prior study (EXIST-3) and will continue treatment under observation.
What are the potential side effects?
Everolimus may cause mouth ulcers, skin problems, infections due to a weakened immune system, high blood sugar levels, kidney issues, lung or breathing problems, delayed wound healing among others.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1 up to approximately 10 years, assessed every 12 weeks,
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 up to approximately 10 years, assessed every 12 weeks, for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Occurances of adverse events and serious adverse events
Secondary study objectives
Percentage of patients with clinical benefit

Side effects data

From 2019 Phase 3 trial • 235 Patients • NCT03176238
63%
Stomatitis
27%
Decreased appetite
26%
Hyperglycaemia
25%
Aspartate aminotransferase increased
24%
Rash
23%
Alanine aminotransferase increased
22%
Cough
19%
Anaemia
16%
Fatigue
16%
Blood cholesterol increased
16%
Weight decreased
15%
Diarrhoea
15%
Gamma-glutamyltransferase increased
13%
Pneumonitis
12%
Hypertriglyceridaemia
12%
Upper respiratory tract infection
12%
Headache
12%
Nausea
11%
Back pain
11%
Pruritus
9%
Myalgia
9%
Blood lactate dehydrogenase increased
9%
Insomnia
8%
Productive cough
8%
Dyspnoea
8%
Dyspepsia
8%
Oedema peripheral
8%
Arthralgia
7%
Dysgeusia
7%
Pain in extremity
6%
Constipation
6%
Pyrexia
6%
Hypertension
5%
Dyslipidaemia
5%
Aphthous ulcer
5%
Lymphoedema
5%
Hypercholesterolaemia
5%
Urinary tract infection
5%
Epistaxis
5%
Asthenia
5%
Pneumonia
5%
Blood alkaline phosphatase increased
5%
Neutrophil count decreased
5%
Dry skin
5%
Thrombocytopenia
4%
Rhinorrhoea
4%
Acne
4%
Abdominal pain
4%
Peripheral swelling
4%
Blood creatinine increased
4%
Hypokalaemia
4%
Hypophosphataemia
4%
Urticaria
4%
Mouth ulceration
4%
Toothache
3%
Abdominal discomfort
3%
Influenza
3%
Hyperlipidaemia
3%
Nail disorder
3%
Cellulitis
3%
Paraesthesia
3%
Alopecia
3%
Abdominal pain upper
3%
Diabetes mellitus
3%
Oropharyngeal pain
3%
Depression
3%
Hyperkalaemia
3%
Face oedema
3%
Non-cardiac chest pain
3%
Nasopharyngitis
3%
Pharyngitis
3%
Haemoglobin decreased
3%
White blood cell count decreased
3%
Dermatitis acneiform
3%
Vomiting
2%
Musculoskeletal pain
2%
Pleural effusion
2%
Onychomadesis
2%
Dizziness
2%
Hypoaesthesia
2%
Influenza like illness
2%
Mucosal inflammation
2%
Pain
2%
Blood triglycerides increased
2%
Platelet count decreased
2%
Vitamin D deficiency
2%
Flank pain
2%
Neck pain
2%
Eczema
2%
Rash maculo-papular
2%
Haemorrhoids
1%
Soft tissue infection
1%
Left ventricular failure
1%
Hyponatraemia
1%
Hydrocephalus
1%
Pulmonary oedema
1%
Respiratory failure
1%
Eye pain
1%
Gastric haemorrhage
1%
Tumour pain
1%
Central nervous system haemorrhage
1%
Dysphagia
1%
Herpes virus infection
1%
Gastroenteritis
1%
Haemoptysis
1%
Abdominal hernia
1%
Ascites
1%
Furuncle
1%
Lower respiratory tract infection
1%
Interstitial lung disease
1%
Atypical pneumonia
1%
Skin ulcer
1%
Cardiopulmonary failure
1%
Lethargy
1%
Ventricular tachycardia
1%
Acute kidney injury
1%
Joint effusion
1%
Dyspnoea exertional
1%
Wheezing
1%
Leukopenia
1%
Metastases to lung
1%
Gait inability
1%
Renal failure
1%
Abdominal distension
1%
Musculoskeletal chest pain
1%
Pulmonary embolism
1%
Asthma
1%
Palmar-plantar erythrodysaesthesia syndrome
1%
Vision blurred
1%
Folliculitis
1%
Cardiac failure
1%
Contrast media allergy
1%
Dry eye
1%
Gingivitis
1%
Muscular weakness
1%
Dental caries
1%
Intracranial aneurysm
1%
Onycholysis
1%
Oral herpes
1%
Sinusitis
1%
Hypocalcaemia
1%
Hypomagnesaemia
1%
Bone pain
1%
Joint stiffness
1%
Pain in jaw
1%
Spinal pain
1%
Breast pain
1%
Ingrowing nail
1%
Rash papular
1%
Rash pruritic
1%
Hot flush
1%
Atrial fibrillation
1%
Cardiac arrest
1%
Gastric ulcer
1%
Haematochezia
1%
Large intestinal haemorrhage
1%
Urethritis
1%
Urosepsis
1%
Viral infection
1%
Cartilage injury
1%
Femur fracture
1%
Ligament sprain
1%
Overdose
1%
Eastern cooperative oncology group performance status worsened
1%
Liver function test increased
1%
Dehydration
1%
Diabetes mellitus inadequate control
1%
Tumour necrosis
1%
Neutropenia
1%
Tachycardia
1%
Gastrooesophageal reflux disease
1%
Oral pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Asian
Non-Asian

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: everolimusExperimental Treatment1 Intervention
everolimus, 2mg dispersible tablets
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
everolimus
2005
Completed Phase 4
~1490

Find a Location

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,911 Previous Clinical Trials
4,250,471 Total Patients Enrolled
8 Trials studying Tuberous Sclerosis
769 Patients Enrolled for Tuberous Sclerosis

Media Library

Everolimus (mTOR inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT02962414 — Phase 3
Tuberous Sclerosis Research Study Groups: everolimus
Tuberous Sclerosis Clinical Trial 2023: Everolimus Highlights & Side Effects. Trial Name: NCT02962414 — Phase 3
Everolimus (mTOR inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02962414 — Phase 3
~54 spots leftby Aug 2027
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