HemophiliaPhase 1NCT01191372
First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients
A Phase 1 study testing ARC19499 in people with Hemophilia, run by Baxalta now part of Shire at 1 site in United Kingdom.
Trial summary
The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia. Read more about this study
- Who is it for?
- Ages 18 to 75 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- ARC19499
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 15 Sep 2010to 28 Dec 2011
- Phase
- Phase 1
- 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
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult male patients ≥18 to ≤75 years of age.
- Hemophilia of any type or severity.
- Patients who are negative for hepatitis B surface antigen (HBsAg), and human immunodeficiency virus (HIV) I and II antibody tests at screening.
- Male patients who, with their partners, are willing to use 2 effective, methods of contraception (i.e., for both self and partner) throughout the study and for at least 3 months after discontinuation of study drug treatment.
- All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.
Who cannot join
- Female patients;
- If on a prophylactic coagulation factor concentrate regimen, inability or unwillingness to discontinue prophylaxis during participation in this study.
- Existence of other co-existing bleeding disorder (e.g., von Willebrand Disease).
- Medical history of venous or arterial thromboembolism.
- Scheduled for elective surgical procedure during the conduct of this study.
- Use of an investigational drug within 30 days of study entry.
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- Transaminase values > 3 x upper limit of normal (ULN) at time of screening.
- Haemoglobin <12.0 g/dL.
- Participants who, in the opinion of the Investigator, have a significant infection or known inflammatory process on screening.
- Participants who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.
- Any medical condition the investigator believes would place the patient at increased risk as a result of participation in the study e.g. history of thromboembolic disease or stroke.
- Any medication the investigator considers may increase the risk of adverse effects during the study.
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.
- Conditions
- Hemophilia
- Being tested
- placebo control, ARC19499
- Keywords
- Hemophilia A, Hemophilia B, pharmacokinetics, pharmacodynamics, Tissue Factor Pathway Inhibitor
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
placebo controlDrug
sterile saline for injection
ARC19499Drug
Anti-tissue factor pathway inhibitor (TFPI) aptamer
| Study group | Role | What they receive |
|---|---|---|
| saline for injection | Placebo comparator | Not described Receives: Drug: placebo control |
| ARC19499 Low Dose | Experimental | Not described Receives: Drug: ARC19499 |
| ARC19499 Mid Dose | Experimental | Not described Receives: Drug: ARC19499 |
| ARC19499 High Dose | Experimental | Not described Receives: Drug: ARC19499 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics (PK) of ARC19499
The PK profile of ARC19499 administered by single and multiple subcutaneous injections will be characterized. The bioavailability of subcutaneously injected ARC19499 relative to that of intravenously infused ARC19499 will be determined.
Measured: 2 weeks
Secondary outcomes (1)
Coagulation system pharmacodynamic (PD) effects of ARC19499.
The PD profile of ARC19499 with respect to the kinetics of thrombin generation and clot formation will be characterized.
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2010 (estimated)
- Study start15 Sep 2010
- Primary completion28 Dec 2011
- Study completion28 Dec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01191372
- Sponsor's protocol ID
- ARC19499-001
- Other IDs
- 2010-020373-17, 271101
- Status last verified
- May 2021
- First submitted
- 26 Aug 2010
- Last updated
- 5 May 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients
- ClinicalTrials.gov ID
- NCT01191372
- Acronym / sponsor ID
- ARC19499-001
- Conditions
- Hemophilia
- Keywords
- Hemophilia A, Hemophilia B, pharmacokinetics, pharmacodynamics, Tissue Factor Pathway Inhibitor
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 15 Sep 2010 (actual)
- Primary completion
- 28 Dec 2011 (actual)
- Study completion
- 28 Dec 2011 (actual)
- First posted
- 30 Aug 2010 (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 study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.
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 |
|---|---|---|
| saline for injection | Placebo comparator | Drug: placebo control |
| ARC19499 Low Dose | Experimental | Drug: ARC19499 |
| ARC19499 Mid Dose | Experimental | Drug: ARC19499 |
| ARC19499 High Dose | Experimental | Drug: ARC19499 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Adult male patients ≥18 to ≤75 years of age.
- Hemophilia of any type or severity.
- Patients who are negative for hepatitis B surface antigen (HBsAg), and human immunodeficiency virus (HIV) I and II antibody tests at screening.
- Male patients who, with their partners, are willing to use 2 effective, methods of contraception (i.e., for both self and partner) throughout the study and for at least 3 months after discontinuation of study drug treatment.
- All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.
Key exclusion criteria (12 listed)
- Female patients;
- If on a prophylactic coagulation factor concentrate regimen, inability or unwillingness to discontinue prophylaxis during participation in this study.
- Existence of other co-existing bleeding disorder (e.g., von Willebrand Disease).
- Medical history of venous or arterial thromboembolism.
- Scheduled for elective surgical procedure during the conduct of this study.
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 |
|---|---|---|
| placebo controlsterile saline for injection | Drug | saline for injection |
| ARC19499Anti-tissue factor pathway inhibitor (TFPI) aptamer | Drug | ARC19499 High Dose, ARC19499 Low Dose, ARC19499 Mid Dose |
Primary outcome
Pharmacokinetics (PK) of ARC19499
Time frame: 2 weeks
The PK profile of ARC19499 administered by single and multiple subcutaneous injections will be characterized. The bioavailability of subcutaneously injected ARC19499 relative to that of intravenously infused ARC19499 will be determined.
1 secondary outcome
Coagulation system pharmacodynamic (PD) effects of ARC19499.
Time frame: 2 weeks
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2021.
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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.
Study Director study director
Takeda
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ARC19499 in people with Hemophilia, run by Baxalta now part of Shire at 1 site in United Kingdom.
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 5 inclusion and 12 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 5 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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. 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?
Pharmacokinetics (PK) of ARC19499 (time frame: 2 weeks). 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 5 May 2021.