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Sirolimus for Tuberous Sclerosis

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
University of North Carolina at Chapel Hill, Chapel Hill, NCTuberous Sclerosis+1 MoreSirolimus - Drug
Eligibility
1 - 6
All Sexes
What conditions do you have?
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Study Summary

This trial is studying whether sirolimus can prevent or delay seizure onset in infants with Tuberous Sclerosis Complex.

Eligible Conditions
  • Tuberous Sclerosis
  • Epilepsy

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

1
mFOLFIRINOX
1
Reparixin
1
Sirolimus

Study Objectives

2 Primary · 5 Secondary · Reporting Duration: 12 and 24 months of age

Month 24
EEG Biomarkers
MRI Biomarkers
Neurodevelopmental Outcomes
Quality of Life Outcomes
12 months of age
Seizures
Safety -- adverse events
Sirolimus Precision Dosing

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Similar Trials

1
mFOLFIRINOX
2
Reparixin
2
Sirolimus

Side Effects for

CellCept + CNI (Tacrolimus or Cyclosporine)
29%Diarrhoea
18%Abdominal Pain
16%Hepatitis C
16%Nausea
16%Fatigue
16%Headache
14%Vomiting
14%Pyrexia
14%Leukopenia
12%Oedema Peripheral
11%Insomnia
10%Back Pain
10%Hyperkalaemia
10%Tremor
10%Anaemia
10%Hypertension
9%Arthralgia
9%Pruritis
9%Cough
8%Pain in Extremity
8%Abdominal Pain Upper
8%Hepatic Enzyme Increased
8%Dizziness
8%Neutropenia
7%Sinusitis
7%Constipation
7%Weight Decreased
7%Dyspnoea
6%White Blood Cell Count Decreased
6%Blood Creatinine Increased
6%Liver Function Test Abnormal
5%Nasopharyngitis
5%Depression
5%Jaundice
5%Asthenia
5%Upper Respiratory Tract Infection
5%Decreased Appetite
5%Weight Increased
5%Muscle Spasms
5%Incision Site Pain
5%Renal Failure
4%Night Sweats
4%Oropharyngeal Pain
4%Anorexia
4%Rhinorrhoea
3%Rash
3%Hyperlipidaemia
3%Thrombocytopenia
3%Incisional Hernia
3%Pleural Effusion
3%Acne
3%Myalgia
2%Sepsis
2%Pneumonia
2%Hypokalaemia
1%Benign Prostatic Hyperplasia
1%Convulsion
1%Bile Duct Stenosis
1%Gastrointestinal Haemorrhage
1%Autoimmune Hepatitis
1%Lung Disorder
1%Blood Glucose Increased
1%Biliary Tract Disorder
1%Acarodermatitis
1%Lobar Pneumonia
1%Inguinal Hernia
1%Bile Duct Obstruction
1%Hepatic Failure
1%Ventricular Tachycardia
1%Procedural Pain
1%Hyperglycaemia
1%Clostridium Difficile Colitis
1%Viral Infection
1%Bacteraemia
1%Escherichia Urinary Tract Infection
1%Cellulitis
1%Clostridial Infection
1%Diverticulitis
1%Influenza
1%Lactobacillus Infection
1%Pseudomonal Sepsis
1%Staphylococcal Infection
1%Umbilical Hernia
1%Crohn's Disease
1%Abdominal Hernia
1%Peritonitis
1%Gastritis
1%Biliary Anastomosis Complication
1%Complications of Transplanted Kidney
1%Post Procedural Haemorrhage
1%Cholestasis
1%Hepatic Artery Stenosis
1%Portal Vein Thrombosis
1%Hepatic Function Abnormal
1%Chest Pain
1%Multi-Organ Failure
1%Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%Desmoid Tumour
1%Gastrointestinal Tract Adenoma
1%Adenocarcinoma
1%Malaise
1%Hepatic Cancer Metastatic
1%B-Cell Lymphoma
1%Hepatic Neoplasm Malignant
1%Pulmonary Embolism
1%Febrile Neutropenia
1%Non-Small Cell Lung Cancer Metastatic
1%Sinus Congestion
1%Embolism Venous
1%Pulmonary Oedema
1%Atrial Fibrillation
1%Cardiac Failure Congestive
1%Encephalopathy
1%Orthostatic Hypotension
1%Cerebral Haemorrhage
1%Deep Vein Thrombosis
1%Vasculitis
1%Hypoglycaemia
1%Haemorrhage Intracranial
1%Blood Alkaline Phosphatase Increased
1%Atrial Flutter
1%Hyponatraemia
1%Transplant Rejection
1%Confusional State
1%Urinary Retention
1%Graft Versus Host Disease
1%Renal Failure Acute
1%Spinal Osteoarthritis
1%Stomatitis
1%Inappropriate Antidiuretic Hormone Secretion
1%Hypercholesterolaemia
This histogram enumerates side effects from a completed 2008 Phase 4 trial (NCT00118742) in the CellCept + CNI (Tacrolimus or Cyclosporine) ARM group. Side effects include: Diarrhoea with 29%, Abdominal Pain with 18%, Hepatitis C with 16%, Nausea with 16%, Fatigue with 16%.

Trial Design

2 Treatment Groups

Sirolimus
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

64 Total Participants · 2 Treatment Groups

Primary Treatment: Sirolimus · Has Placebo Group · Phase 2

Sirolimus
Drug
Experimental Group · 1 Intervention: Sirolimus · Intervention Types: Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 12 and 24 months of age

Who is running the clinical trial?

Darcy KruegerLead Sponsor
Martina Bebin, MD, MPAPrincipal InvestigatorUniversity of Alabama at Birmingham
1 Previous Clinical Trials
84 Total Patients Enrolled
1 Trials studying Tuberous Sclerosis
84 Patients Enrolled for Tuberous Sclerosis
Darcy A Krueger, MD, PhDPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati

Eligibility Criteria

Age 1 - 6 · All Participants · 2 Total Inclusion Criteria

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Who else is applying?

What state do they live in?
Arizona100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%
References

Frequently Asked Questions

How many research locations are participating in this investigative trial?

"This trial has been extended to 8 sites, including the medical centres at Stanford University (Palo Alto), Washington University -- St. Louis (Saint Louis), and Boston Children's Hospital (Boston). The other 5 locations will be listed in subsequent communications." - Anonymous Online Contributor

Unverified Answer

What potential dangers do patients face when taking Sirolimus?

"Based on the information at hand, our team provides Sirolimus a safety score of 2 due to data suggesting its efficacy has yet to be empirically proven." - Anonymous Online Contributor

Unverified Answer

Is this experiment still enlisting participants?

"Affirmative. As verified by clinicaltrials.gov, this medical trial is actively recruiting participants since its initial post date on October 13th 2021 and the recent edit it underwent on September 2nd 2022. The experiment calls for 64 individuals to be recruited from 8 separate sites." - Anonymous Online Contributor

Unverified Answer

What are the aims of this research endeavor?

"This 12-month study will primarily evaluate the safety of sirolimus in terms of adverse events, but also assess neurodevelopmental outcomes after treatment, the efficacy of precise dosing for this particular therapy, and EEG biomarkers to measure neuronal connectivity." - Anonymous Online Contributor

Unverified Answer

What is the cap on the number of individuals involved in this research?

"Affirmative. According to clinicaltrials.gov, this medical trial is currently seeking suitable participants. It was first posted on October 13 2021 and most recently amended on September 2 2022 with a requirement for 64 patients from 8 locations." - Anonymous Online Contributor

Unverified Answer

Do I meet the qualifications to participate in this trial?

"64 infants with aura are being accepted into the research trial. The necessary criteria includes a corrected age of at least 39 weeks, and they must be below 7 months old when randomized for treatment initiation. Additionally, applicants need to have been diagnosed clinically or genetically as having tuberous sclerosis complex (TSC)." - Anonymous Online Contributor

Unverified Answer

Are there any other research projects that have incorporated Sirolimus into their methods?

"Currently, 125 clinical trials examining Sirolimus are underway. Of those experiments, 13 have progressed to the third phase of testing. The majority of these studies are situated in Cincinnati, Ohio while other locations across the US host a total of 1074 sites conducting this research." - Anonymous Online Contributor

Unverified Answer

What is the usual purpose of prescribing Sirolimus?

"Sirolimus is a viable therapy for the prevention of transplant rejection, as well as diseases affecting the liver and kidneys." - Anonymous Online Contributor

Unverified Answer

Is the minimum age requirement for this clinical trial higher than twenty years old?

"This clinical trial requires infants aged 1 Day to 6 Months for enrollment. There are 194 other trials that cater specifically to minors and 600 studies designated for elderly patients." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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