Purpose of this Study
This study is testing a new medicine called TGD001 for people who have a serious condition called thrombotic microangiopathy, or TMA. TMA can cause very small blood clots to form in blood vessels and block blood flow to important organs. TGD001 is designed to help dissolve these clots and is given through a vein.
Participants will be placed into one of three groups based on the type of TMA they have. The study will begin with lower doses of TGD001. If the medicine is shown to be safe, later participants may receive higher doses so researchers can find the best dose to use. Once the safest and most effective dose is found, more people will receive that dose.
During the study, participants will have blood tests and physical exams so doctors can monitor their health and how well the treatment is working. Participation in the study lasts about 90 days.
Who Can Participate?
Eligibility
People can join this study if they are having an active episode of a serious blood vessel condition called thrombotic microangiopathy, or TMA. They must have a low platelet count, which can increase the risk of bleeding and other health problems.
People cannot join if they are currently receiving medicines that strongly suppress the immune system or if they have had a major bleeding event within the past 30 days. Researchers are studying a new treatment called TGD001 to see if it is safe and if it can help people during an active TMA episode.
Age Range
0-110
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study is testing a new medicine called TGD001 for people who have a serious condition called thrombotic microangiopathy, or TMA. In TMA, very small blood clots can form in blood vessels and block blood flow to important organs. TGD001 is designed to help break up these clots and improve blood flow.
Researchers want to learn if TGD001 is safe and if it can help treat people with TMA. The study will test different dose levels of the medicine to find the best dose and learn how well it works. The goal is to develop a new treatment that may help people with this condition in the future.
Locations
Duke University Hospital
Visit Timing
Weekdays
Weekends
Evenings
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
An Adaptive Dose Escalation and Expansion Basket Trial to Explore the Safety, Pharmacology, and Clinical Activity of TGD001 in Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) and Other Thrombotic Microangiopathies
Principal Investigator
Ara
Metjian
Protocol Number
PRO00120526
NCT ID
NCT07459114
Phase
I/II
Enrollment Status
Pending Open to Enrollment