A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor Rilzabrutinib (PRN1008) in Moderate to Severe Pemphigus

TerminadoFase 3ClinicalTrials.gov

ID: NCT03762265Tipo: INTERVENTIONALInicio: 8 de ene de 2019Fin estimado: 17 de dic de 2021

Traducción no disponible, mostrando original

Resumen

This was a Phase 3 randomized, parallel-group, double-blind, placebo-controlled trial (blinded treatment \[BT\] period) followed by an open-label extension \[OLE\] period intended to evaluate the efficacy and safety of oral PRN1008 in moderate to severe pemphigus. After completing the open-label extension period, eligible participants might continue in a long term extension (LTE) Period of 48 weeks.

Descripción detallada

A total of 131 male or female participants with newly diagnosed or relapsing moderate to severe pemphigus (pemphigus vulgaris \[PV\] or pemphigus foliaceus \[PF\]) were enrolled in the trial worldwide. The trial would last 68 weeks (approximately 17 months) for each participant. For participants eligible to enroll in the LTE, the trial might last up to 116 weeks. Participants were randomized at Day 1, using a 1:1 ratio to receive PRN1008 or placebo twice per day, by relapsing/newly diagnosed disease history (newly diagnosed defined as within 6 months of screening).

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 80 YearsSexo: ALL

Criterios de inclusión

Male or female participants, aged 18 to 80 years old with moderate to severe, newly diagnosed or relapsing PV or PF, with a clinical presentation and histopathology consistent with PV or PF.
Positive circulating anti-desmoglein 1 (anti-dsg1) or 3 autoantibody titer.
At screening, pemphigus disease area index score of at least 9 points for relapsing participants or at least 15 points for newly diagnosed participants.
Adequate hematologic, hepatic, and renal function.
Effective means of contraception.

Criterios de exclusión

Suspected paraneoplastic pemphigus and other forms of pemphigus that were not PV or PF.
Previous use of a Bruton tyrosine kinase inhibitor.
Pregnant or lactating women.
Electrocardiogram clinically significant abnormalities.
A history of malignancy of any type within 5 years before Day 1, other than surgically excised non-melanoma skin cancers or in situ cervical cancer.
Use of immunologic response modifiers as concomitant medication and with the washout period.
Use of proton pump inhibitor drugs such as omeprazole and esomeprazole within 3 days of Day 1.
Concomitant use of known strong-to-moderate inducers or inhibitors of cytochrome P450 3A (CYP3A) within 3 days or 5 half-lives (whichever is longer) of Day 1
Use of CYP3A-sensitive substrate drugs.
Had received any investigational drug within the 30 days before Day 1.
History of drug abuse within the previous 12 months.
Alcoholism or excessive alcohol use.
Any other clinically significant disease, condition or medical history that, in the opinion of the Investigator, would interfere with participant safety, trial evaluations, and/or trial procedures.

Intervenciones

drug

Rilzabrutinib

Pharmaceutical form: Tablet Route of administration: Oral

drug

Placebo

Pharmaceutical form: Tablet Route of administration: Oral

Ubicaciones

Central Recruiting (Principia Biopharma)

Buenos Aires, Argentina

Central Recruiting (Principia Biopharma)

Buenos Aires, Argentina

Central Recruiting (Principia Biopharma)

San Nicolás, Argentina

Patrocinadores

PrincipalPrincipia Biopharma, a Sanofi Company

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