Haemophagocytic lymphohistiocytosis, macrophage activation syndrome, relapsed/refractory solid tumours Cancer Malignant neoplasms of ill-defined, secondary and unspecified sites
Conditions
Interventions
Current interventions as of 16/03/2023:
GOTHAM is a Phase II multicentre clinical trial of gemtuzumab ozogamicin. The trial aims to determine whether gemtuzumab ozogamicin elicits a biological respons
Sponsors
University of Birmingham
Eligibility
Sex/Gender
All
Age
1 Years to 100 Years
Inclusion criteria
Inclusion criteria: 1. Aged >1 year old at the time of trial entry 2. Diagnosis of primary or secondary HLH or MAS disease that is relapsing/refractory to treatment at time of enrolment (Group 1) OR 3. 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) (Group 2) Note: patients who meet the inclusion criteria for both groups 1 and 2 should be entered into group 1 3.1.Group 2 only – must have adequate liver function: 3.1.1. Total bilirubin =x2 upper limit of normal (ULN) 3.1.2. Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) =2.5 x ULN 4. Documented negative pregnancy test for female patients of childbearing potential within 7 days prior to trial entry 5. 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 6. Written informed consent given by patient and/or parents/legal guardian
Exclusion criteria
Exclusion criteria: 1. Evidence of sinusoidal obstruction syndrome (SOS)/veno-occlusive disease (VOD) 2. Previous treatment with another CD33 targeting antibody or immunotoxin 3. Hypersensitivity to gemtuzumab ozogamicin or to any of the excipients 4. Pregnant or lactating female
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Current primary outcome measure as of 16/03/2023: CD33+ cell count in the blood samples of patients collected on day 1, day 8, day 15, day 22, 43, 50 and 57 Previous primary outcome measure: CD33+ cell count in the blood samples of patients collected on day 1, day 8, day 15, day 22 and day 29 | — |
Secondary
| Measure | Time frame |
|---|---|
| Measured using patient records up to 1 year after the end of treatment: 1. Overall Survival time - defined as the time from the date of entry into the trial to the date of death 2. Progression-free survival (PFS) time (Group 2 only) - defined as the time from the date of entry into the trial to the date of disease progression 3. Grade 3 and 4 adverse events | — |
Countries
England, United Kingdom
Outcome results
None listed