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ADAMTS13 Replacement Therapy

BAX 930 for TTP

Phase 3
Recruiting
Research Sponsored by Baxalta now part of Shire
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Participant has a documented diagnosis of severe hereditary ADAMTS13 deficiency, confirmed by molecular genetic testing and ADAMTS13 activity < 10 % as measured by the fluorescent resonance energy transfer- von Willebrand factor73 (FRETS-VWF73) assay
Participant is 0 to 70 years of age, inclusive, at the time of screening
Timeline
Screening 3 weeks
Treatment Varies
Follow Up start of treatment periods 1, 2 and 3: within 60 min pre-infusion and at study completion/early termination visit (up to 19 months)
Awards & highlights

Study Summary

This trial is testing a new treatment, BAX 930, for people with thrombotic thrombocytopenic purpura (TTP), a condition where blood clots form in small blood vessels throughout the body. The study will compare the number of TTP events in people treated with BAX 930 versus those treated with the standard treatment.

Who is the study for?
This trial is for children, teenagers, and adults born with severe hereditary Thrombotic Thrombocytopenic Purpura (TTP). Participants must have a confirmed diagnosis of TTP due to ADAMTS13 deficiency, be willing to follow the study protocol, and use effective contraception if applicable. They should not have acute illnesses or a history of drug/alcohol abuse recently.Check my eligibility
What is being tested?
The trial tests BAX 930 against standard care in preventing or controlling TTP flare-ups. It involves three periods: starting with either BAX 930 or standard treatment for about six months each before switching; then everyone receives BAX 930 again. Treatments are given through vein infusion.See study design
What are the potential side effects?
Potential side effects from BAX 930 and standard treatments will be monitored but are not specified here. Side effects could include reactions related to infusions such as discomfort at the injection site, allergic reactions, or other unspecified systemic responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a severe hereditary ADAMTS13 deficiency confirmed by genetic testing.
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I am between 0 and 70 years old.
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I don't have severe blood clotting issues or very low platelets.
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I, or my legal guardian, have signed the consent form and I am 18 years or older.
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I am on, or can tolerate, standard preventive treatment for blood clots.
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I am not pregnant, can take a pregnancy test, and will use birth control during the study.
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I either don't have hepatitis C, or if I do, it's stable and chronic.
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I am over 16 and can care for myself, or if under 16, I can do most activities.
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I am using effective birth control and will continue for at least 16 days after my last treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~start of treatment periods 1, 2 and 3: within 60 min pre-infusion and at study completion/early termination visit (up to 19 months)
This trial's timeline: 3 weeks for screening, Varies for treatment, and start of treatment periods 1, 2 and 3: within 60 min pre-infusion and at study completion/early termination visit (up to 19 months) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Acute Thrombotic Thrombocytopenic Purpura (TTP) Events
Secondary outcome measures
Area Under the Plasma curve [AUC] of ADMATS13 Activity and ADMATS13 Antigen for SoC Agent and BAX 930 in Plasma
Assessment of ADAMTS13 Activity (Pre-Infusion ADAMTS13 Levels) and Select VWF Parameters
Assessment of Anti-Chinese Hamster Ovary (Anti-CHO) Protein Antibodies
+33 more

Trial Design

4Treatment groups
Experimental Treatment
Group I: Prophylaxis Cohort IIExperimental Treatment2 Interventions
Participants randomized to BAX-930 arm in prophylactic cohort will receive a PK dose of their current SoC product followed by a single dose IV infusions of 40 IU/kg BAX-930 ORT at 14 days later in PK I with a washout period of 14 days (+ or - 2 days). In period 1 participants will receive a single dose IV infusions of 40 IU/kg BAX-930 ORT once Q2W for the next six months followed by SOC for 6 months in period 2. Thereafter participants will receive a single dose IV infusions of 40 IU/kg BAX-930 SIN followed by IV infusions of 40 IU/kg BAX-930 ORT in PK II with a washout period of 14 days (+ or - 2 days). All the participants in period 3 will receive BAX-930 SIN prophylactic dose IV infusions of 40 IU/kg for another 6 months.
Group II: Prophylaxis Cohort IExperimental Treatment2 Interventions
Participants randomized to SOC arm in prophylactic treatment cohort will receive a single dose intravenous (IV) infusions of 40 international units per kilogram (IU/kg) BAX-930 ORT product followed by a PK dose of their current SoC at 14 days later in PK I with a washout period of 14 days (+ or - 2 days). In period 1 participants will receive SOC for 6 months followed by IV infusions of 40 IU/kg dose of BAX-930 ORT once every 2 weeks (Q2W) in period 2 for the next six months. After period 2, participants will receive a single dose IV infusions of 40 IU/kg BAX-930 ORT followed by IV infusions of 40 IU/kg BAX-930 SIN in PK II with a washout period of 14 days (+ or - 2 days). All the participants in period 3 will receive BAX-930 SIN prophylactic dose of IV infusions of 40 IU/kg for another 6 months.
Group III: On Demand Cohort IIExperimental Treatment2 Interventions
Participants randomized to BAX-930 arm in On-demand cohort will receive initial dose of IV infusions 40 IU/kg [+/- 4 IU/kg] BAX-930 ORT or BAX-930 SIN infusion then a subsequent dose IV infusions of 20 IU/kg [+/- 2 IU/kg] BAX-930 ORT or BAX-930 SIN infusion on Day 2 and an additional daily dose IV infusions of 15 IU/kg [+/- 1.5 IU/kg] BAX 930 until 2 days after the acute event is resolved. In period 1 participants will receive SOC for the next six months followed by IV infusions of 40 IU/kg dose of BAX-930 ORT once Q2W for 6 months in period 2. Thereafter participants will receive a single dose IV infusions of 40 IU/kg BAX-930 SIN followed by IV infusions of 40 IU/kg BAX-930 ORT in PK II with a washout period of 14 days (+ or - 2 days). All the participants in period 3 will receive BAX-930 SIN prophylactic dose IV infusions of 40 IU/kg for another 6 months.
Group IV: On Demand Cohort IExperimental Treatment2 Interventions
Participants randomized to SOC arm in On-demand cohort will receive the investigator-recommended SOC and dosing regimen during the acute event. In period 1 participants will receive IV infusions of 40 IU/kg dose of BAX-930 ORT once every 2 weeks (Q2W) for 6 months followed by SOC in period 2 for the next six months. Thereafter participants will receive a single dose IV infusions of 40 IU/kg BAX-930 ORT followed by IV infusions of 40 IU/kg BAX-930 SIN in PK II with a washout period of 14 days (+ or - 2 days). All the participants in period 3 will receive BAX-930 SIN prophylactic dose IV infusions of 40 IU/kg for another 6 months.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Standard of care
2019
Completed Phase 4
~16000

Find a Location

Who is running the clinical trial?

Baxalta now part of ShireLead Sponsor
110 Previous Clinical Trials
9,047 Total Patients Enrolled
Takeda Development Center Americas, Inc.Industry Sponsor
56 Previous Clinical Trials
11,973 Total Patients Enrolled
Study DirectorStudy DirectorShire
1,203 Previous Clinical Trials
489,522 Total Patients Enrolled

Media Library

BAX 930 (ADAMTS13 Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03393975 — Phase 3
Thrombotic Thrombocytopenic Purpura Research Study Groups: Prophylaxis Cohort II, On Demand Cohort II, On Demand Cohort I, Prophylaxis Cohort I
Thrombotic Thrombocytopenic Purpura Clinical Trial 2023: BAX 930 Highlights & Side Effects. Trial Name: NCT03393975 — Phase 3
BAX 930 (ADAMTS13 Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03393975 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are we still looking for patients who want to participate in this experiment?

"This clinical trial, which was first posted on October 13, 2017 and last updated on May 4, 2022, is currently searching for participants."

Answered by AI

Might I be eligible for this experiment?

"This trial is looking for 57 people with thrombotic thrombocytopenic purpura (ttp) between the ages of 0 and 70. Most notable, candidates should meet the following criteria: Participant is currently on a prophylactic dosing regimen or has a documented history of at least 1 TTP event and an ability to tolerate SoC prophylactic dosing (prophylactic cohort only)., If female of childbearing potential, participant presents with a negative blood or urine pregnancy test, confirmed no more than 7 days before the first administration, and agrees to employ adequate birth control measures for the duration"

Answered by AI

Are there different versions of this study taking place in different states?

"This trial is recruiting at various locations including Durham's Duke University Medical Center, Oklahoma City's University of Oklahoma, and Atlanta's Winship Cancer Institute. There are a total of 6 recruiting sites."

Answered by AI

Are geriatric patients within the scope of this research?

"This clinical trial is recruiting people who are between 0 and 70 years old."

Answered by AI

Could you please give some clarity on the potential risks of BAX930?

"BAX930's safety is based on data from previous Phase 3 trials, and it was given a score of 3."

Answered by AI

Are there any other similar studies ongoing at this time?

"BAX930 has a long clinical history, with the first study taking place in 2017. The trial was sponsored by Takeda Development Center Americas, Inc.. After this initial Phase 1 study involving 57 people, BAX930 received approval for Phase 3 trials. Now, there are 2 active studies being conducted in 20 cities and 10 countries around the world."

Answered by AI

How many patients have been recruited for this clinical trial?

"In order to move forward, this trial requires 57 individuals that match the pre-determined inclusion criteria. These participants can come from different hospitals, such as Duke University Medical Center or University of Oklahoma."

Answered by AI

Could you please elaborate on other research that has included BAX930?

"BAX930 is being trialled in 2 Phase 3 studies currently underway. 51 different research sites are conducting investigations into BAX930, with many of them located in Fukuoka-shi, Fukuoka-Ken."

Answered by AI
~1 spots leftby Jun 2024