Haemophagocytic lymphohistiocytosis (HLH) or Macrophage activation syndrome (MAS) and relapsed/refractory solid tumours MedDRA version: 21.1 Level: PT Classification code 10071583 Term: Haemophagocytic lymphohistiocytosis System Organ Class: 10021428 - Immune system disorders MedDRA version: 21.1 Level: LLT Classification code 10053867 Term: Macrophage activation syndrome System Organ Class: 10021428 - Immune system disorders MedDRA version: 20.0 Level: LLT Classification code 10007050 Term:
Conditions
Interventions
Trade Name: gemtuzumab ozogamicin
Product Name: gemtuzumab ozogamicin
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: hP67.6 antibody-NAc-gamma calicheamicin
Sponsors
University of Birmingham
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Aged >1 year old at the time of trial entry •Diagnosis of HLH or MAS disease primary, secondary or refractory to treatment at time of enrolment OR Histologically confirmed diagnosis of solid cancer with radiological or clinical evidence of disease progression (during or after completion of at least one previous treatment) or any subsequent recurrence (biopsy at relapse is not mandated) •Must have adequate liver function (solid cancer patients only): o Total bilirubin = x2 upper limit of normal (ULN) o Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) = 2.5 x ULN •Documented negative pregnancy test for female patients of childbearing potential within 7 days prior to trial entry •Sexually active patients must agree to use 2 methods of adequate and appropriate contraception while on trial drug and for 4 months (male) and 7 months (female) following treatment discontinuation •Written informed consent given by patient and/or parents/legal guardian Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 9 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: •Evidence of sinusoidal obstruction syndrome (SOS) / veno-occlusive disease (VOD) •Previous treatment with another CD33 targeting antibody or immunotoxin •Hypersensitivity to Gemtuzumab Ozogamicin or to any of the excipients •Pregnant or lactating female
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To see if gemtuzumab ozogamicin can reduce the number of CD33 myeloid cells in two groups of people 1) who have Haemophagocytic lymphohistiocytosis (HLH) or Macrophage activation syndrome (MAS), or 2)solid tumours which have not responded to previous treatment (refractory) or come back after previous treatment (relapsed). ;Secondary Objective: To assess the effect of gemtuzumab ozogamicin on overall survival time (OS) and progression free survival time (PFS) (PFS is applicable only to those patients with relapsed/refractory solid tumours). To assess the feasibility of delivering gemtuzumab ozogamicin in these patient groups. ;Primary end point(s): CD33+ cell count in the blood samples of patients collected at the pre-specified time points (day 1, day 8, day 15, day 22 and 29);Timepoint(s) of evaluation of this end point: CD33+ cell count will be assessed on days 1, 8, 15, 22 and 29. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Overall survival (OS) time is defined as the time from the date of entry into the trial to the date of death. Patients lost to follow-up will be censored accordingly at date last seen. Progression free survival (PFS) time (Patients with solid tumours only) is defined as the time from the date of entry into the trial to the date of disease progression. Patients lost to follow-up will be censored accordingly at date last seen. Incidence of grade 3 and 4 adverse events is the frequency of adverse events (as defined in the protocol). ;Secondary end point(s): Overall Survival (OS) time Progression Free Survival (PFS) time (Patients with solid tumours only) Incidence of grade 3 and 4 adverse events | — |
Countries
United Kingdom
Contacts
Public ContactSenior Trial Coordinator
University of Birmingham
Outcome results
None listed