Study to Evaluate the Efficacy and Safety of MAS825 in NLRC4-GOF Patients

A Three-period Multicenter Study, With a Randomized-withdrawal, Double-blinded, Placebo-controlled Design in Period 2 to Evaluate the Clinical Efficacy, Safety, and Tolerability of MAS825 in NLRC4-GOF Patients

ClinicalTrials.gov Identifier: NCT04641442

Novartis Reference Number: CMAS825D12201

Last Update: May 17, 2022

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF

Condition 
NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis)
Phase 
Phase 2
Overall status 
Recruiting
Start date 
Dec 18, 2020
Completion date 
Apr 25, 2027
Gender 
All
Age(s)
(Child, Adult, Older Adult)

Interventions

Biological
MAS825
Experimental drug
Biological
Placebo
matching placebo

Eligibility Criteria

Inclusion Criteria:

Male and female patients weighing at least 3 kg
Written informed consent by parent(s)/legal guardian(s) for the pediatric patients and assent by the pediatric patient (depending on local requirements) must be obtained before any study-specific assessment is performed. For adult patients, written informed consent by patients capable of giving consent, or when the patient is not capable of giving consent, by his/her legal/authorized representative (if allowed according to local requirements).
Patients with genetic diagnosis of NLRC4-GOF
Clinical history and investigations consistent with autoinflammation with infantile enterocolitis (AIFEC/NLRC4-GOF)
At first treatment, evidence of active disease

Exclusion Criteria:

History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients.

Signs and symptoms, in the judgment of the investigator, of clinically significant systemic recurrent and/or evidence of active bacterial, fungal, parasitic or viral infections.

- COVID-19 specific: If in line with health and governmental authority guidance, it is highly recommended that testing to exclude COVID-19 using PCR or comparable approved methodology be completed within 1 week prior to first dosing.

Any conditions or significant medical problems, which in the opinion of the investigator places the patient at unacceptable risk for MAS825 therapy
Previous treatment with anti-rejection and/or immunomodulatory drugs within the past 28 days or 5 half-lives (whichever is the longer) for immunomodulatory therapeutic antibodies (or as listed in the prohibited medications section) prior to MAS825 treatment with the exceptions of glucocorticoids, cyclosporin and targeted binding or blocking therapies.
A positive HIV test result at Screening. Evidence of prior testing within 3 months is sufficient.A positive HIV test result at Screening. Evidence of prior testing within 3 months is sufficient.
A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C test result. Evidence of prior testing within 3 months is sufficient.
Presence of tuberculosis infection as defined by a positive TB test at Screening. Evidence of prior testing within 3 months is sufficient.
Live vaccinations within 1 month prior to MAS825 treatment, during the trial, and up to 3 months following the last dose.
Female patients of child-bearing potential (or Tanner stage 2 or above) who are or might become sexually active, Female patients of child-bearing potential (or Tanner stage 2 or above) who are or might become sexually active, agree to use highly effective contraceptive methods to prevent pregnancy while on MAS825 therapy

Study Locations

United States
Cincinnati Children's Hospital Medical Center
Recruiting
Cincinnati, 45229 - Ohio
Contact: Alexei Grom (513-636-4676) - [email protected] -
United States
Czech Republic
Novartis Investigative Site
Recruiting
Praha, 121 00
CZ
Czech Republic
France
Novartis Investigative Site
Recruiting
Bordeaux Cedex, 33076
-
France
Novartis Investigative Site
Recruiting
Bron Cedex, 69677
-
France
Novartis Investigative Site
Recruiting
Paris cedex 15, 75015
-
France
Novartis Investigative Site
Recruiting
Paris, 75020
-
France
Italy
Novartis Investigative Site
Recruiting
Roma, 00165 - RM
Contact: Fabrizio De Benedetti (+39 06-68592659-4393) - [email protected] - Fabrizio De Benedetti
Italy
Japan
Novartis Investigative Site
Recruiting
Obu, 474 8710
Aichi
Japan

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
Name: 
Novartis Pharmaceuticals
Phone: 

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Call 1-888-669-6682 or email [email protected]