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Sirolimus for Tuberous Sclerosis (TSC-STEPS Trial)

Phase 2
Recruiting
Led By Darcy A Krueger, MD, PhD
Research Sponsored by Darcy Krueger
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
Subject must be 0-6 months of age at the time of enrollment with a corrected age of at least 39 weeks
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 and 24 months of age
Awards & highlights

TSC-STEPS Trial Summary

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

Who is the study for?
This trial is for infants diagnosed with Tuberous Sclerosis Complex (TSC), aged between 0-6 months. They must not have started any seizure medications, special diets, or other treatments related to TSC and should be generally healthy without significant prematurity (born after at least 30 weeks of gestation).Check my eligibility
What is being tested?
The study tests the safety and effectiveness of Sirolimus in preventing or delaying seizures in infants with TSC. It's a Phase II trial where participants are randomly given either Sirolimus or a placebo without knowing which one they receive.See study design
What are the potential side effects?
While specific side effects for this trial aren't listed, Sirolimus can typically cause immune system suppression leading to increased infection risk, mouth sores, acne-like skin conditions, diarrhea, nausea, and potentially abnormal liver or kidney function.

TSC-STEPS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with TSC through clinical tests or genetic analysis.
Select...
My baby is between 0-6 months old and corrected age is at least 39 weeks.

TSC-STEPS Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 and 24 months of age
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 and 24 months of age for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Seizures
Safety -- adverse events
Secondary outcome measures
EEG Biomarkers
MRI Biomarkers
Neurodevelopmental Outcomes
+2 more

Side effects data

From 2008 Phase 4 trial • 293 Patients • NCT00118742
29%
Diarrhoea
18%
Abdominal Pain
16%
Nausea
16%
Headache
16%
Fatigue
16%
Hepatitis C
14%
Vomiting
14%
Pyrexia
14%
Leukopenia
12%
Oedema Peripheral
11%
Insomnia
10%
Hyperkalaemia
10%
Tremor
10%
Back Pain
10%
Hypertension
10%
Anaemia
9%
Cough
9%
Pruritis
9%
Arthralgia
8%
Neutropenia
8%
Abdominal Pain Upper
8%
Dizziness
8%
Pain in Extremity
8%
Hepatic Enzyme Increased
7%
Dyspnoea
7%
Constipation
7%
Sinusitis
7%
Weight Decreased
6%
Blood Creatinine Increased
6%
Liver Function Test Abnormal
6%
White Blood Cell Count Decreased
5%
Jaundice
5%
Decreased Appetite
5%
Muscle Spasms
5%
Renal Failure
5%
Weight Increased
5%
Upper Respiratory Tract Infection
5%
Nasopharyngitis
5%
Asthenia
5%
Incision Site Pain
5%
Depression
4%
Anorexia
4%
Night Sweats
4%
Oropharyngeal Pain
4%
Rhinorrhoea
3%
Hyperlipidaemia
3%
Pleural Effusion
3%
Thrombocytopenia
3%
Myalgia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Hypokalaemia
2%
Pneumonia
2%
Sepsis
1%
Urinary Retention
1%
Spinal Osteoarthritis
1%
Hypercholesterolaemia
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Hypoglycaemia
1%
Blood Glucose Increased
1%
Portal Vein Thrombosis
1%
Cardiac Failure Congestive
1%
Clostridium Difficile Colitis
1%
Abdominal Hernia
1%
Blood Alkaline Phosphatase Increased
1%
Ventricular Tachycardia
1%
Gastrointestinal Tract Adenoma
1%
Encephalopathy
1%
Febrile Neutropenia
1%
Gastritis
1%
Renal Failure Acute
1%
Multi-Organ Failure
1%
Crohn's Disease
1%
Hepatic Neoplasm Malignant
1%
Chest Pain
1%
Non-Small Cell Lung Cancer Metastatic
1%
Transplant Rejection
1%
Benign Prostatic Hyperplasia
1%
Cerebral Haemorrhage
1%
Confusional State
1%
Convulsion
1%
Peritonitis
1%
Haemorrhage Intracranial
1%
Deep Vein Thrombosis
1%
Inguinal Hernia
1%
Viral Infection
1%
Acarodermatitis
1%
Gastrointestinal Haemorrhage
1%
Atrial Fibrillation
1%
Malaise
1%
Hepatic Cancer Metastatic
1%
Adenocarcinoma
1%
B-Cell Lymphoma
1%
Desmoid Tumour
1%
Pulmonary Embolism
1%
Stomatitis
1%
Influenza
1%
Staphylococcal Infection
1%
Umbilical Hernia
1%
Hepatic Function Abnormal
1%
Atrial Flutter
1%
Hyponatraemia
1%
Bacteraemia
1%
Cellulitis
1%
Clostridial Infection
1%
Diverticulitis
1%
Escherichia Urinary Tract Infection
1%
Lactobacillus Infection
1%
Lobar Pneumonia
1%
Pseudomonal Sepsis
1%
Post Procedural Haemorrhage
1%
Procedural Pain
1%
Biliary Anastomosis Complication
1%
Complications of Transplanted Kidney
1%
Bile Duct Obstruction
1%
Bile Duct Stenosis
1%
Biliary Tract Disorder
1%
Autoimmune Hepatitis
1%
Cholestasis
1%
Hepatic Artery Stenosis
1%
Hepatic Failure
1%
Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%
Lung Disorder
1%
Pulmonary Oedema
1%
Sinus Congestion
1%
Embolism Venous
1%
Orthostatic Hypotension
1%
Vasculitis
1%
Hyperglycaemia
1%
Graft Versus Host Disease
100%
80%
60%
40%
20%
0%
Study treatment Arm
CellCept + CNI (Tacrolimus or Cyclosporine)
CellCept + Sirolimus

TSC-STEPS Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: SirolimusExperimental Treatment1 Intervention
Sirolimus
Group II: PlaceboPlacebo Group1 Intervention
Placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
2013
Completed Phase 4
~2750

Find a Location

Who is running the clinical trial?

Darcy KruegerLead Sponsor
Darcy A Krueger, MD, PhDPrincipal InvestigatorChildren's Hospital Medical Center, Cincinnati
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

Media Library

Placebo Clinical Trial Eligibility Overview. Trial Name: NCT05104983 — Phase 2
Tuberous Sclerosis Research Study Groups: Sirolimus, Placebo
Tuberous Sclerosis Clinical Trial 2023: Placebo Highlights & Side Effects. Trial Name: NCT05104983 — Phase 2
Placebo 2023 Treatment Timeline for Medical Study. Trial Name: NCT05104983 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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)."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

Who else is applying?

What state do they live in?
Arizona
What portion of applicants met pre-screening criteria?
Met criteria
~22 spots leftby Jun 2025