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mTOR inhibitor
Sirolimus for Castleman Disease
Phase 2
Waitlist Available
Led By David C Fajgenbaum, MD, MBA, MS
Research Sponsored by University of Pennsylvania
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Ability to consume oral medication in the form of a tablet
Evidence of active disease, defined as at least two abnormalities in the criteria comprising the CBR criteria, including at least one objective measurement (hemoglobin, weight loss, or lymph node size)
Must not have
Subjects cannot have any of the following: ECOG >3 (or Karnofsky/Lansky score ≤ 60 in children); Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2 or creatinine > 3.0 mg/dL; Absolute neutrophil count (ANC) < 1000 x 109/L ((< 500 x 109/L in children); Hemoglobin ≤ 6.5 g/dL (transfusion independent, defined as not receiving a red blood cell transfusion for ≥ 7 days prior); Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) laboratory values greater than three times the upper limit of normal; Albumin < 2 g/dL (transfusion independent, defined as not receiving intravenous albumin for ≥ 7 days prior); Platelet count ≤ 40 x 109/L (transfusion independent, defined as not receiving platelet transfusion for ≥ 7 days prior); Pulmonary involvement or interstitial pneumonitis with dyspnea (adequate pulmonary function is defined as pulse oximetry > 94% on room air if there is clinical indication for determination (e.g. dyspnea at rest, history of interstitial pneumonitis, etc.)); Fasting cholesterol > 300 mg/dL or fasting triglyceride > 400 mg/dL
Subjects cannot have a documented history of human immunodeficiency virus (HIV) or HHV-8 infection, or severe combined immunodeficiency syndrome
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3, 6, 9, and 12 months ± 2 weeks
Awards & highlights
Summary
This trial tests sirolimus, a medication that controls the immune system, on patients with iMCD who haven't responded to other treatments. Sirolimus blocks a pathway to reduce immune response and abnormal cell growth. Sirolimus has shown potential in treating various conditions.
Who is the study for?
This trial is for people aged 2-80 with idiopathic multicentric Castleman disease who haven't responded well to anti-IL-6 therapy or can't tolerate it. Participants must have active disease, be able to take oral medication, and not be pregnant or nursing. They shouldn't have had sirolimus before, any recent other systemic therapies except corticosteroids, severe infections mimicking iMCD, certain cancers within the last year, serious psychiatric disorders affecting consent ability, or very poor health as defined by specific medical criteria.
What is being tested?
The study tests the effect of Sirolimus on patients with idiopathic multicentric Castleman disease who haven’t seen improvement with standard treatments. It aims to understand how this drug impacts their condition.
What are the potential side effects?
Sirolimus may cause side effects like mouth sores, diarrhea, nausea; increased risk of infection; abnormal blood test results indicating liver or kidney issues; and potentially lung problems.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can take pills by mouth.
Select...
My condition shows active disease with at least two symptoms, including one measurable change.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I do not have HIV, HHV-8, or severe combined immunodeficiency syndrome.
Select...
I am not currently in, nor planning to join, another clinical trial for my condition.
Select...
I do not have any uncontrolled infections that could be confused with iMCD.
Select...
I have never been treated with sirolimus for my Castleman disease.
Select...
I have never had a liver or lung transplant.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3, 6, 9, and 12 months ± 2 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3, 6, 9, and 12 months ± 2 weeks
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Disease activity, as measured by the CHAP scale
Disease activity, as measured by the MCD-related Overall Symptom Score
Side effects data
From 2008 Phase 4 trial • 293 Patients • NCT0011874229%
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%
Anaemia
10%
Hyperkalaemia
10%
Tremor
10%
Back Pain
10%
Hypertension
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%
Renal Failure
5%
Muscle Spasms
5%
Decreased Appetite
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%
Pleural Effusion
3%
Myalgia
3%
Hyperlipidaemia
3%
Thrombocytopenia
3%
Rash
3%
Acne
3%
Incisional Hernia
2%
Sepsis
2%
Pneumonia
2%
Hypokalaemia
1%
Renal Failure Acute
1%
Urinary Retention
1%
Clostridium Difficile Colitis
1%
Benign Prostatic Hyperplasia
1%
Hypoglycaemia
1%
Blood Alkaline Phosphatase Increased
1%
Gastritis
1%
Cerebral Haemorrhage
1%
Crohn's Disease
1%
Abdominal Hernia
1%
Chest Pain
1%
Hepatic Failure
1%
Multi-Organ Failure
1%
Hepatic Neoplasm Malignant
1%
Non-Small Cell Lung Cancer Metastatic
1%
Ventricular Tachycardia
1%
Inappropriate Antidiuretic Hormone Secretion
1%
Gastrointestinal Haemorrhage
1%
Cardiac Failure Congestive
1%
Hepatic Artery Stenosis
1%
Portal Vein Thrombosis
1%
Epstein-Barr Virus Associated Lymphoproliferative Disorder
1%
Gastrointestinal Tract Adenoma
1%
Febrile Neutropenia
1%
Encephalopathy
1%
Atrial Flutter
1%
Blood Glucose Increased
1%
Transplant Rejection
1%
Confusional State
1%
Spinal Osteoarthritis
1%
Hypercholesterolaemia
1%
Convulsion
1%
Peritonitis
1%
Haemorrhage Intracranial
1%
Deep Vein Thrombosis
1%
Inguinal Hernia
1%
Viral Infection
1%
Acarodermatitis
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%
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%
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
Trial Design
1Treatment groups
Experimental Treatment
Group I: SirolimusExperimental Treatment1 Intervention
Oral sirolimus: For adults, loading dose of 5 mg/m\^2, rounded to the nearest mg, on day 1. For adults, starting on day 2, oral sirolimus daily at 2.5 mg/m\^2/day (rounded to the nearest mg), target trough level 10-15 ng/mL by HPLC, for 12 months. For children, 2 mg/m\^2/day, target trough level 5-15 ng/mL by HPLC.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
2013
Completed Phase 4
~2750
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Castleman Disease treatments often target the dysregulated immune system and abnormal cell growth. Sirolimus, an mTOR inhibitor, works by blocking the mTOR pathway, which is crucial for cell proliferation and survival.
This inhibition can reduce the overactive immune response and abnormal lymph node growth seen in Castleman Disease. Other treatments may include monoclonal antibodies like siltuximab, which target interleukin-6 (IL-6), a cytokine involved in inflammation and immune response.
By understanding and targeting these pathways, treatments can help manage symptoms and improve outcomes for Castleman Disease patients.
Find a Location
Who is running the clinical trial?
University of PennsylvaniaLead Sponsor
2,060 Previous Clinical Trials
42,647,582 Total Patients Enrolled
2 Trials studying Castleman Disease
1,130 Patients Enrolled for Castleman Disease
David C Fajgenbaum, MD, MBA, MSPrincipal InvestigatorUniversity of Pennsylvania
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I do not have HIV, HHV-8, or severe combined immunodeficiency syndrome.My condition did not improve with anti-IL-6 therapy, or I couldn't tolerate it.You have a medical history that matches the criteria for a specific disease called iMCD (idiopathic multicentric Castleman disease).I am not currently in, nor planning to join, another clinical trial for my condition.I can take pills by mouth.I am between the ages of 2 and 80.I do not have any uncontrolled infections that could be confused with iMCD.My condition shows active disease with at least two symptoms, including one measurable change.You have had an allergic reaction or sensitivity to sirolimus or similar medications in the past.I haven't had any treatment for iMCD, except possibly anti-IL6 therapy over 14 days ago.I have never been treated with sirolimus for my Castleman disease.I have never had a liver or lung transplant.I do not have any rheumatic diseases that could be confused with iMCD.I have no cancer history, except for certain skin cancers, cervical cancer in situ, or any cancer I haven't been treated for in the last year.
Research Study Groups:
This trial has the following groups:- Group 1: Sirolimus
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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