A study to see if a drug called gemtuzumab ozogamicin is a useful treatment for patients with immune disorders called haemophagocytic lymphohistiocytosis (HLH) or macrophage activation syndrome (MAS), or for patients with cancer

Phase 2

• Conditions: Haemophagocytic lymphohistiocytosis, macrophage activation syndrome, relapsed/refractory solid tumours; Cancer; Malignant neoplasms of ill-defined, secondary and unspecified sites

• Registration Number: ISRCTN89158144
• Lead Sponsor: niversity of Birmingham

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-12-15
• Last Update Posted: 17 April 2023
• Study Completion: 2025-03-01

Recruitment & Eligibility

• Status: Ongoing
• Sex: All
• Target Recruitment: 20

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified