Thrombotic MicroangiopathyPhase 2NCT00726544
Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
A Phase 2 study testing ARC1779 Injection in people with Thrombotic Microangiopathy and Thrombotic Thrombocytopenic Purpura, run by Archemix Corp. at 17 sites in 5 countries.
Trial summary
The purpose of this ascending-dose research study is to determine whether the administration of ARC1779 Injection improves subject's health profile by protecting the brain, heart, and kidney from damage due to formation of small blood clots in blood vessels. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Thrombotic Microangiopathy, Thrombotic Thrombocytopenic Purpura
- What is being tested?
- ARC1779 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 17 sitesin 5 countries
- Study dates
- Dec 2008to Mar 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female;
- ≥18 to ≤75 years of age;
- Diagnosis of TMA based on presence of:
- Thrombocytopenia, defined as a platelet count <100 x 109 per liter;
- Microangiopathic hemolytic anemia, defined by negative findings on direct antiglobulin test, and evidence of accelerated red blood cell (RBC) production and destruction); AND
- Absence of a clinically apparent alternative explanation for thrombocytopenia and anemia, e.g., disseminated intravascular coagulation (DIC), eclampsia, HELLP syndrome, Evans syndrome;
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- Females: non-pregnant and commit to use of effective, redundant methods of contraception (i.e., for both self and male partner) throughout the study and for at least 30 days after discontinuation of study drug treatment;
- Males: commit to use of a medically acceptable contraceptive (abstinence or use of a condom with spermicide) throughout the study and for at least 30 days after discontinuation of study drug treatment;
- Not received an unlicensed investigational agent (drug, device, or blood-derived product) within 30 days prior to randomization, and may not receive such an investigational agent in the 30 days post-randomization (note: investigational use for treatment of TMA of a licensed immunomodulator, e.g., rituximab, is permitted at any time relative to randomization);
- Capable of understanding and complying with the protocol, and he/she (or a legal representative) must have signed the informed consent document prior to performance of any study-related procedures.
- Patients who have again become acutely ill following recent treatment and achievement of a brief remission of acute TMA may be enrolled in the study if ALL of the following conditions are met:
- Disease activity in the patient in unabated (e.g. persistent thrombocytopenia and microangiopathic hemolytic anemia with ongoing neurological symptoms and/or troponin elevation);
- The last plasma exchange of the patient's preceding course of treatment occurred at least 7 days prior;
- The patient did not undergo splenectomy during the preceding course of treatment;
- The new course of plasma exchange has not been ongoing for more than 3 days.
Who cannot join
- Females: pregnant or <24 hours post-partum, or breastfeeding;
- History of bleeding diathesis or evidence of active abnormal bleeding within the previous 30 days;
- Disseminated malignancy or other co-morbid illness limiting life expectancy to ≤3 months independent of the TMA disorder.
- Diagnosis other than TMA which can account for the findings of thrombocytopenia and hemolytic anemia (e.g., DIC, HELLP syndrome, Evans syndrome);
- Diagnosis of DIC verified by laboratory values for D-dimer, fibrinogen, prothrombin time (PT), and activated partial thromboplastin time (aPTT).
- Patients who have again become acutely ill following recent treatment and achievement of a brief remission of acute TMA may not be enrolled in the study if ANY of the following conditions are met:
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- The last plasma exchange of the patient's preceding course of treatment occurred less than 7 days prior;
- The patient underwent splenectomy during the preceding course of treatment;
- The new course of plasma exchange has been ongoing for more than 3 days.
Trial locations and contacts
17 sites in 5 countries.
17 sites in 5 countries
United States7 sites
Northwestern University
Chicago, Illinois, 60611
The Ohio State University Research Foundation
Columbus, Ohio, 43235
The Methodist Hospital
Houston, Texas, 77030
Indiana University Simon Cancer Center
Indianapolis, Indiana, 46202
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Washington University
St Louis, Missouri, 63110
New York Medical College
Valhalla, New York, 10595
Italy5 sites
Ospedale Ferrarotto
Catania, 95100
Policlinico Mangiagalli Regina Elena-Fondazione L.Villa
Milan
Fondazione Ospedale Maggiore Policlinico
Milan, 20122
Azienda Ospedaliera S.Maria Nuova
Reggio Emilia, 42100
Università Cattolica del Sacro Cuore
Rome
Canada3 sites
CICM/Hospital Charles LeMoyne
Greenfield Park, Quebec, J4V 2H1
QEII CDHA Centre
Halifax, Nova Scotia, B3H 2Y9
CHA-Hospital de L'Enfant-Jesus
Québec, Quebec, G1J 1Z4
United Kingdom1 site
University College London Hospital
London, W1T 4EU
About this study
Official title: A Randomized, Double-blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
The purpose of this ascending-dose research study is to determine whether the administration of ARC1779 Injection improves subject's health profile by protecting the brain, heart, and kidney from damage due to formation of small blood clots in blood vessels. It will also determine the safety of ARC1779 Injection, how ARC1779 Injection enters and leaves the blood and tissue over time, and its effect on laboratory tests related to blood clotting, heart and brain function, and other body systems.
- Being tested
- ARC 1779 Placebo, ARC1779 Injection
- Keywords
- thrombocytopenia, microangiopathic hemolytic anemia, von Willebrand Factor, ADAMTS13
- Medical subject headings
- Thrombotic Microangiopathies, Purpura, Thrombotic Thrombocytopenic, Thrombocytopenia, Anemia, Hemolytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARC 1779 PlaceboDrug
ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper.
ARC1779 InjectionDrug
ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 3μg/mL.
ARC1779 InjectionDrug
ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 6μg/mL.
ARC1779 InjectionDrug
ARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 12μg/mL.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: ARC 1779 Placebo |
| Low Dose | Active comparator | Not described Receives: Drug: ARC1779 Injection |
| Medium Dose | Active comparator | Not described Receives: Drug: ARC1779 Injection |
| High Dose | Active comparator | Not described Receives: Drug: ARC1779 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of the clinical composite of death (all-cause mortality), stroke, coma, seizures, renal failure, or acute myocardial infarction (AMI)
Measured: 6 weeks post randomization
Secondary outcomes (4)
Neurocognitive function is to be assessed with the CogState® test system.
Measured: Once during the hospitalization period and again at the 6 week clinic visit.
The incidence of death, stroke, or acute renal failure/injury requiring dialysis is to be assessed.
Measured: During the extended clinical follow-up for each patient from the time of the 6 week clinic visit until the study is closed.
Safety- and efficacy-related clinical laboratory parameters and biomarkers will be analyzed in relation to ARC1779 exposure in terms of the dose administered and the observed plasma concentration.
Measured: During initial hospitalization and at 6 week clinic visit.
The incidence of the composite of complications associated with plasma exchange therapy (i.e., catheter-related infection, thrombosis, internal hemorrhage, or pneumothorax) is to be assessed.
Measured: During initial hospitalization and at the 6 week clinic visit.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2008 (estimated)
- Study startDec 2008
- Primary completionDec 2010 (estimated)
- Study completionMar 2011 (estimated)
Why it stopped: Enrollment into study was slower than expected.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic MicroangiopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Archemix Corp.
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00726544
- Sponsor's protocol ID
- ARC1779-006
- Status last verified
- Nov 2009
- First submitted
- 30 Jul 2008
- Last updated
- 26 Nov 2009
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo Controlled, Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy
- ClinicalTrials.gov ID
- NCT00726544
- Acronym / sponsor ID
- ARC1779-006
- Keywords
- thrombocytopenia, microangiopathic hemolytic anemia, von Willebrand Factor, ADAMTS13
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enrollment into study was slower than expected.
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Archemix Corp.Industry (pharma, biotech, devices)
- Study start
- Dec 2008
- Primary completion
- Dec 2010 (estimated)
- Study completion
- Mar 2011 (estimated)
- First posted
- 1 Aug 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this ascending-dose research study is to determine whether the administration of ARC1779 Injection improves subject's health profile by protecting the brain, heart, and kidney from damage due to formation of small blood clots in blood vessels. It will also determine the safety of ARC1779 Injection, how ARC1779 Injection enters and leaves the blood and tissue over time, and its effect on laboratory tests related to blood clotting, heart and brain function, and other body systems.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: ARC 1779 Placebo |
| Low Dose | Active comparator | Drug: ARC1779 Injection |
| Medium Dose | Active comparator | Drug: ARC1779 Injection |
| High Dose | Active comparator | Drug: ARC1779 Injection |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Male or female;
- ≥18 to ≤75 years of age;
- Diagnosis of TMA based on presence of:
- Thrombocytopenia, defined as a platelet count <100 x 109 per liter;
- Microangiopathic hemolytic anemia, defined by negative findings on direct antiglobulin test, and evidence of accelerated red blood cell (RBC) production and destruction); AND
Key exclusion criteria (9 listed)
- Females: pregnant or <24 hours post-partum, or breastfeeding;
- History of bleeding diathesis or evidence of active abnormal bleeding within the previous 30 days;
- Disseminated malignancy or other co-morbid illness limiting life expectancy to ≤3 months independent of the TMA disorder.
- Diagnosis other than TMA which can account for the findings of thrombocytopenia and hemolytic anemia (e.g., DIC, HELLP syndrome, Evans syndrome);
- Diagnosis of DIC verified by laboratory values for D-dimer, fibrinogen, prothrombin time (PT), and activated partial thromboplastin time (aPTT).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARC 1779 PlaceboARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. | Drug | Placebo |
| ARC1779 InjectionARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 3μg/mL. | Drug | Low Dose |
| ARC1779 InjectionARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 6μg/mL. | Drug | Medium Dose |
| ARC1779 InjectionARC1779 Injection or placebo is administered intravenously to patients as an initial loading dose followed by continuous infusion of up to 14 days plus 2 day taper. Treatment with ARC1779 Injection is to be given at low dosage that is intended to produce target steady-state ARC1779 plasma concentrations during infusion of 12μg/mL. | Drug | High Dose |
Primary outcome
The incidence of the clinical composite of death (all-cause mortality), stroke, coma, seizures, renal failure, or acute myocardial infarction (AMI)
Time frame: 6 weeks post randomization
4 secondary outcomes
Neurocognitive function is to be assessed with the CogState® test system.
Time frame: Once during the hospitalization period and again at the 6 week clinic visit.
The incidence of death, stroke, or acute renal failure/injury requiring dialysis is to be assessed.
Time frame: During the extended clinical follow-up for each patient from the time of the 6 week clinic visit until the study is closed.
Safety- and efficacy-related clinical laboratory parameters and biomarkers will be analyzed in relation to ARC1779 exposure in terms of the dose administered and the observed plasma concentration.
Time frame: During initial hospitalization and at 6 week clinic visit.
The incidence of the composite of complications associated with plasma exchange therapy (i.e., catheter-related infection, thrombosis, internal hemorrhage, or pneumothorax) is to be assessed.
Time frame: During initial hospitalization and at the 6 week clinic visit.
Results and safety
The study was stopped early (reason given: Enrollment into study was slower than expected.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2009.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
17 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ARC1779 Injection in people with Thrombotic Microangiopathy and Thrombotic Thrombocytopenic Purpura, run by Archemix Corp. at 17 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 26 Nov 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Italy, Canada, Austria, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Enrollment into study was slower than expected.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of the clinical composite of death (all-cause mortality), stroke, coma, seizures, renal failure, or acute myocardial infarction (AMI) (time frame: 6 weeks post randomization). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Nov 2009; AzTrials last refreshed its copy on 7 Nov 2026.