Purpura, Thrombotic ThrombocytopenicPhase 4NCT01754545
Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
A Phase 4 study of Purpura, Thrombotic Thrombocytopenic, run by St. Olavs Hospital.
Trial summary
Congenital thrombotic thrombocytopenic purpura (TTP), also called Upshaw-Schulman Syndrome or hereditary or familial TTP is a rare, but severe disease. The purpose of this study is to determine how infusions of plasma to patients with congenital TTP correlate with symptoms and signs of activity of the disease, and to… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Purpura, Thrombotic Thrombocytopenic
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Sep 2012to Nov 2012
- Phase
- Phase 4
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed diagnosis of congenital TTP
- On regular prophylactic plasma treatment: > Once weekly (group 1) or >1 <3 times monthly (group 2)
- Between 18 and 65 years
- Capable of understanding and complying with the protocol
Who cannot join
- Pregnancy
- Acute bout of TTP requiring daily plasma infusions or -exchange for more than 3 days, within the last 2 weeks before intervention period
About this study
Congenital thrombotic thrombocytopenic purpura (TTP), also called Upshaw-Schulman Syndrome or hereditary or familial TTP is a rare, but severe disease. The purpose of this study is to determine how infusions of plasma to patients with congenital TTP correlate with symptoms and signs of activity of the disease, and to determine why some patients need more frequent infusions of plasma than others to prevent acute attacks of the disease.
Read the detailed description
Patients with congenital TTP have an inherited lack of function or amount of a protein in plasma called ADAMTS13, that otherwise is responsible for cleaving large von Willebrand-molecules into smaller parts. The patients suffer recurrent attacks of clotting of small blood vessels, that can cause damage to major organs, including the central nervous system. Acute attacks can be treated successfully with infusions of human plasma, and some patients also receive regular plasma therapy for prevention of acute attacks. A small group of patients receive preventive plasma infusions twice or more weekly, indicating a much higher need for plasma than what is otherwise recommended for preventive therapy. Do these patients have an ongoing activity of their disease despite a stimulus? Or a higher turn-over of transfused ADAMTS13? Have these patients developed antibodies against transfused ADAMTS13? Are any symptoms correlated with signs of disease activity?
- Conditions
- Purpura, Thrombotic Thrombocytopenic
- Being tested
- Octaplas infusion and placebo (group 1), Octaplas infusion and placebo (group 2)
- Keywords
- Infusions, Intravenous, Plasma, ADAMTS-13 protein, human
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Octaplas infusion and placebo (group 1)Drug
Also called ATC B05A A02, Solvent/detergent plasma, Human plasma
Intervention period is Monday-Friday in two following weeks. Active treatment with Octaplas is given 2-3 times each week and placebo is given the other 2-3 days of intervention each week. Route of administration is intravenously.
Octaplas infusion and placebo (group 2)Drug
Also called ATC B05A A02, Solvent/detergent plasma, Human plasma
Intervention period is Monday-Wednesday in two separate weeks (minimum of three weeks between intervention weeks). Active treatment with Octaplas is given once and placebo is given twice each week. Route of administration is intravenously.
| Study group | Role | What they receive |
|---|---|---|
| Octaplas infusion and placebo (group 1) | Experimental | Active treatment with randomly assigned 400 ml octaplas intravenously 2-3 times a week and 400 ml placebo (for octaplas)intravenously 2-3 times a week over two weeks. Receives: Drug: Octaplas infusion and placebo (group 1) |
| Octaplas infusion and placebo (group 2) | Experimental | Active treatment with randomly assigned 400 ml octaplas intravenously once and 400 ml placebo (for octaplas)intravenously twice in two separate intervention weeks Receives: Drug: Octaplas infusion and placebo (group 2) |
What is being measured
The primary outcome is the main question the study is designed to answer.
A composite score of clinical and biochemical signs of disease activity
Biochemical signs of disease activity are scored by measurements of lactate dehydrogenase, hemoglobin, platelet counts and ADAMTS13 activity.
Clinical signs of disease activity are measured by an evaluator's score and subjects symptom screening
Measured: 4 hours and 24 hours after intervention
Secondary outcomes (2)
ADAMTS13 activity half-life
Calculation of half-life of ADAMTS13 activity based on serial measurements of ADAMTS13 activity before and after plasma infusions
Measured: 4 hours and 24 hours after intervention
ADAMTS13 allo-antibodies
Presence of allo-antibodies towards ADAMTS13
Measured: At baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2012 (estimated)
- Study startSep 2012
- Primary completionNov 2012
- Study completionNov 2012
Why it stopped: altered situation in study population; not enough patients meeting enrollment criteria
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Purpura, Thrombotic ThrombocytopenicStudies of the same condition, open and under way.
- More trials run by St. Olavs HospitalThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Olavs Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Norwegian University of Science and Technology
- Responsible party
- Sponsor
- Investigators
- Anne S von Krogh, MD, Principal investigator, St. Olavs Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01754545
- Sponsor's protocol ID
- 133995
- Status last verified
- Dec 2012
- First submitted
- 12 Dec 2012
- Last updated
- 21 Dec 2012
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prophylactic Plasma Infusion Therapy for Congenital Thrombotic Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT01754545
- Acronym / sponsor ID
- 133995
- Conditions
- Purpura, Thrombotic Thrombocytopenic
- Keywords
- Infusions, Intravenous, Plasma, ADAMTS-13 protein, human
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: altered situation in study population; not enough patients meeting enrollment criteria
- Enrolment
- 0 participants (actual)
- Lead sponsor
- St. Olavs HospitalUniversity, hospital or other
- Collaborators
- Norwegian University of Science and Technology
- Study start
- Sep 2012
- Primary completion
- Nov 2012 (actual)
- Study completion
- Nov 2012 (actual)
- First posted
- 21 Dec 2012 (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.
Congenital thrombotic thrombocytopenic purpura (TTP), also called Upshaw-Schulman Syndrome or hereditary or familial TTP is a rare, but severe disease. The purpose of this study is to determine how infusions of plasma to patients with congenital TTP correlate with symptoms and signs of activity of the disease, and to determine why some patients need more frequent infusions of plasma than others to prevent acute attacks of the disease.
Detailed description
Patients with congenital TTP have an inherited lack of function or amount of a protein in plasma called ADAMTS13, that otherwise is responsible for cleaving large von Willebrand-molecules into smaller parts. The patients suffer recurrent attacks of clotting of small blood vessels, that can cause damage to major organs, including the central nervous system. Acute attacks can be treated successfully with infusions of human plasma, and some patients also receive regular plasma therapy for prevention of acute attacks. A small group of patients receive preventive plasma infusions twice or more weekly, indicating a much higher need for plasma than what is otherwise recommended for preventive therapy. Do these patients have an ongoing activity of their disease despite a stimulus? Or a higher turn-over of transfused ADAMTS13? Have these patients developed antibodies against transfused ADAMTS13? Are any symptoms correlated with signs of disease activity?
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Octaplas infusion and placebo (group 1) | Experimental | Drug: Octaplas infusion and placebo (group 1)Active treatment with randomly assigned 400 ml octaplas intravenously 2-3 times a week and 400 ml placebo (for octaplas)intravenously 2-3 times a week over two weeks. |
| Octaplas infusion and placebo (group 2) | Experimental | Drug: Octaplas infusion and placebo (group 2)Active treatment with randomly assigned 400 ml octaplas intravenously once and 400 ml placebo (for octaplas)intravenously twice in two separate intervention weeks |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of congenital TTP
- On regular prophylactic plasma treatment: > Once weekly (group 1) or >1 <3 times monthly (group 2)
- Between 18 and 65 years
- Capable of understanding and complying with the protocol
Key exclusion criteria (2 listed)
- Pregnancy
- Acute bout of TTP requiring daily plasma infusions or -exchange for more than 3 days, within the last 2 weeks before intervention period
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 |
|---|---|---|
| Octaplas infusion and placebo (group 1)Also: ATC B05A A02, Solvent/detergent plasma, Human plasmaIntervention period is Monday-Friday in two following weeks. Active treatment with Octaplas is given 2-3 times each week and placebo is given the other 2-3 days of intervention each week. Route of administration is intravenously. | Drug | Octaplas infusion and placebo (group 1) |
| Octaplas infusion and placebo (group 2)Also: ATC B05A A02, Solvent/detergent plasma, Human plasmaIntervention period is Monday-Wednesday in two separate weeks (minimum of three weeks between intervention weeks). Active treatment with Octaplas is given once and placebo is given twice each week. Route of administration is intravenously. | Drug | Octaplas infusion and placebo (group 2) |
Primary outcome
A composite score of clinical and biochemical signs of disease activity
Time frame: 4 hours and 24 hours after intervention
Biochemical signs of disease activity are scored by measurements of lactate dehydrogenase, hemoglobin, platelet counts and ADAMTS13 activity. Clinical signs of disease activity are measured by an evaluator's score and subjects symptom screening
2 secondary outcomes
ADAMTS13 activity half-life
Time frame: 4 hours and 24 hours after intervention
ADAMTS13 allo-antibodies
Time frame: At baseline
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2012.
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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.
Anne S von Krogh, MD principal investigator
St. Olavs Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 4 study of Purpura, Thrombotic Thrombocytopenic, run by St. Olavs Hospital.
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 4 inclusion and 2 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: withdrawn, as of the sponsor's last update on 21 Dec 2012. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 withdrawn before enrolling participants, so there are no results.
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?
A composite score of clinical and biochemical signs of disease activity (time frame: 4 hours and 24 hours after intervention). 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 21 Dec 2012.