Thrombotic Microangiopathy
Conditions
Keywords
TMA, aHUS, TTP, stem cell transplant-associated TMA
Brief summary
The purpose of this compassionate use study, for two patients with thrombotic microangiopathy, is to provide expanded access to patients who have participated in the clinical trial OMS721-TMA-001 and in whom improvement in their disease markers was observed while on treatment or to patients who could otherwise benefit from the treatment. This is a treatment protocol; not a research protocol.Therefore, only patients in study OMS721-TMA-001 deemed eligible by the investigator may participate.
Detailed description
The study is open-label, expanded access study to provide continued OMS721 treatment for compassionate use to patients who have participated in clinical trial OMS721-TMA-001. Patients will be eligible if markers of disease activity, such as platelet count, LDH, and haptoglobin, were observed to improve during the study or if the patients disease activity remained stable, but they could be treated with a higher dose that may be beneficial. Safety measures of adverse events and laboratory measures will be monitored. Markers of disease activity, pharmacokinetics and ex vivo pharmacodynamics will also be monitored.
Interventions
BIOLOGICALOMS721
Human IgG4 MASP-2 Monoclonal Antibody
Sponsors
Michal Nowicki
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Inclusion criteria
* Have a diagnosis of thrombotic microangiopathy related to aHUS, TTP or stem cell transplant. * Have completed treatment in clinical trial OMS721-TMA-001. * Investigator determined that continued treatment with OMS721 could be beneficial. * Aged 18 years or older.
Exclusion criteria
* Hypersensitivity to OMS721 or any excipients. * Have a serious medical condition that increases the risk of OMS721 treatment to the patient.
Contacts
Primary ContactMichal Nowicki, MD, PhD
Outcome results
None listed