Immune ThrombocytopeniaPhase 1/2NCT02401061
PRTX-100-202 Open-Label, Dose Escalation Study in Adult Patients With ITP
A Phase 1/2 study testing PRTX-100 in people with Immune Thrombocytopenia, run by Protalex, Inc. at 11 sites in 2 countries.
Trial summary
Pre-clinical and clinical evaluations show that PRTX- 100 has biological activity that may lead to improved platelet levels where these are decreased due to immunological pathologies and that PRTX-100 has an acceptable safety profile. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- PRTX-100
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 11 sitesin 2 countries
- Study dates
- Sep 2015to 4 Jun 2019
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing and able to provide written informed consent prior to initiation of any study-related procedures
- Male or female ≥ 18 years of age
- ITP that has persisted for ≥ 3 months. ITP must be diagnosed in accordance The American Society of Hematology 2011 Evidence-based Practice Guideline for Immune Thrombocytopenia (Neunert et al. 2011) or the International Consensus Report on The Investigation and Management of Primary Immune Thrombocytopenia (Provan et al. 2010), as locally applicable.
- Received ≥ 1 typical regime for the treatment of ITP. Splenectomy is considered one standard ITP treatment
- A mean platelet count of < 30,000/μL, with no individual platelet count > 35,000/μL; or for those subjects receiving a constant dose of permitted treatments for ITP: a mean platelet count < 50,000/μL, with no count greater than 55,000/μL. (Note: The mean platelet count must be determined based on 2 platelet counts including one obtained within ≤ 7 days of first PRTX-100 dose and the other within ≤ 30 days of the first dose of PRTX-100.)
- If on corticosteroids, a dose of < 1 mg/kg prednisone per day or equivalent that has been stable for ≥ 21 days prior to the first dose of PRTX-100. High-dose pulse steroid therapy is NOT allowed within 14 days prior to the first dose of PRTX-100.
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- If receiving eltrombopag or romiplostim, the dose must have been stable for ≥ 21 days prior to the first dose of PRTX-100
- If on steroid-sparing adjunctive immunosuppression with cyclosporine, azathioprine, mycophenolate, or 6-mercaptopurine, the dose must have been stable for ≥ 30 days prior to the first dose of PRTX-100 and must be expected to remain stable through study Day 29, unless dose reduction is required due to toxicities. Treatment with other cytotoxic agents (e.g. cyclophosphamide, vincristine) are not allowed within three months prior to the first dose of PRTX- 100.
- Any prior treatment with rituximab or any other anti-CD20 agent must have been > 6 months prior to the first dose of PRTX-100
- If female, must not be pregnant (pregnancy testing will be performed locally in all female patients of childbearing potential), must not be nursing and must be one of the following:
- Surgically sterile (bilateral tubal ligation, hysterectomy)
- Postmenopausal with last natural menses > 24 months prior
- Premenopausal and using an acceptable form of birth control. Acceptable forms of birth control include: hormonal contraceptives (implantable, oral, patch) used for ≥ 2 months prior to screening or double barrier methods (any combination of two of the following: intrauterine device [IUD], male or female condom with spermicidal gel, diaphragm, sponge, cervical cap). All premenopausal females must have a negative urine or serum pregnancy test at screening and on Day 1 prior to first PRTX-100 treatment.
Who cannot join
- Splenectomy ≤ 90 days prior to the first dose of PRTX-100
- Unstable coronary artery disease or other medical condition (such as type 1 diabetes) that, in the Investigator's opinion, might increase the risk to the patient
- Evidence of active infection requiring antibiotic therapy ≤ 14 days prior to the first dose of PRTX- 100
- Myelodysplastic syndrome. If clinically significant anemia or pancytopenia exists, documentation of a bone marrow aspirate within 24 months prior to the first dose of PRTX-100 showing no evidence of myelodysplasia is required.
- Medical history of vasculitis or lupus erythematosus
- Propensity to allergic reactions defined as a history of allergic reaction to more than one medication
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- History of any treatment for cancer within the past two years other than basal cell or squamous cell carcinoma of the skin that has been treated with curative intent
- Seropositive for human immunodeficiency virus (HIV)
- History of acute/chronic hepatitis B or C and/or carriers of hepatitis B or C (positive for hepatitis B surface antigen or positive anti-hepatitis C antibody test) and evidence of current or active infection (e.g. HCV RNA test)
- History suggestive of substance abuse
- History or evidence on physical examination or screening laboratory tests of any systemic disease or any acute or chronic illness that, in the opinion of the investigator, may interfere with the evaluation of the safety or immunogenicity of the study drug
- Treatment with IVIG ≤ 14 days prior to the first dose of PRTX-100
- Treatment with an anti-Rh D antigen agent (e.g. WinPho) ≤ 14 days prior to the first dose of PRTX-100
- Use of any investigational drug, other than eltrombopag or romiplostim, ≤ 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to the first dose of RTX-100
- Not willing to stay at the study site for 4 hours after each PRTX-100 infusion
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States6 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Gabrail Cancer Center
Canton, Ohio, 44718
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Michigan Center of Medical Research
Farmington Hills, Michigan, 48334
USC Norris Comprehensive Cancer Center
Los Angeles, California, 90033
Weil-Cornell Medical College
New York, New York, 10065
United Kingdom5 sites
Aberdeen Royal Infirmary
Aberdeen, AB, AB25 27N
Queen Elizabeth Hospital
Birmingham, B15 2TH
Glasgow Royal Infirmary
Glasgow, G4 0SF
Leicester Royal Infirmary
Leicester, Leichester, LE1 5WW
Norfolk and Norwich Hospital
Norwich, NR4 7UY
About this study
Official title: A Phase 1/2, Open-Label, Dose Escalation Study of PRTX-100 in Adult Patients With Persistent/Chronic Immune Thrombocytopenia
Pre-clinical and clinical evaluations show that PRTX- 100 has biological activity that may lead to improved platelet levels where these are decreased due to immunological pathologies and that PRTX-100 has an acceptable safety profile. In vivo treatment with PRTX-100 has been shown to raise platelet counts in a mouse model of immune thrombocytopenia (ITP). The primary objective of the study is to assess the efficacy of PRTX-100 in terms of platelet response in patients with chronic/persistent ITP.
Funding Source - FDA OOPD (1R01FD005750-01A1)
- Conditions
- Immune Thrombocytopenia
- Being tested
- PRTX-100
- Keywords
- ITP, Thrombocytopenia
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRTX-100Drug
Also called SpA, Staphylococcal Protein A
Four weekly infusions of PRTX-100 at a level of 1 (3, 6,12, 18, or 24) microgram of PRTX-100 per kilogram of patient weight, infused over 30 minutes, followed by four hours of observation.
| Study group | Role | What they receive |
|---|---|---|
| PRTX-100 | Experimental | Patients will be assigned to consecutive PRTX-100 interventions as they are enrolled into the study. Between two and six patients will be enrolled per intervention level. Intervention levels range from one (1) to twenty four (24) micrograms of PRTX-100 per kilogram of patient weight. Patients may receive up to four weekly infusions of PRTX-100 over the study treatment period. PRTX-100 doses ≤ 500 μg will be infused intravenously over 30 minutes. PRTX-100 doses > 500 μg will be infused over 60 minutes. Patients will remain under observation for 4 hours after completion of PRTX-100 dosing for safety management. Receives: Drug: PRTX-100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet Response, Change from Baseline
The primary efficacy endpoint is platelet response defined as a platelet count ≥ 30,000/μL and at least a doubling of baseline platelet count (determined on Day 1 prior to PRTX-100 administration) in patients with a baseline platelet count < 30,000/μL. In patients receiving permitted treatments for ITP with a baseline platelet count ≥ 30,000/μL and < 50,000/μL, an increase in platelet count to ≥ 50,000/μL and at least a doubling of baseline platelet count or an increase to > 100,000/μL be considered a platelet response.
Measured: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Secondary outcomes (5)
Complete platelet response, Change from Baseline
Defined as a platelet count ≥ 100,000/μL
Measured: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Time to platelet response defined as the mean number of days from first PRTX-100 dose until platelet response
The mean number of days from first PRTX-100 dose until platelet response
Measured: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Durability of platelet response
The number of days from first documented platelet response to first platelet count below platelet response criteria
Measured: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Concomitant ITP medication use (frequency and amount)
ITP medications include eltrombopag, romiplostim, steroid-sparing adjunctive treatment (e.g. cyclosporine, azathioprine, mycophenolate, danazol, dapsone, or 6-mercaptopurine), and any ITP rescue medications (e.g. IVIG) received during the study Screening and Treatment Periods
Measured: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Adverse Events
Safety will be described by AEs, SAEs, infusion reactions, clinical laboratory tests (hematology, blood chemistry and urinalysis), vital signs, physical findings and ECGs. AE severity will be graded according to Toxicity Grading Criteria derived from published standards
Measured: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2015 (estimated)
- Study startSep 2015
- Primary completionMar 2019
- Study completion4 Jun 2019
Why it stopped: Enough data has been collected to allow analysis of safety profile and risk-benefit.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Immune ThrombocytopeniaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protalex, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- William E Gannon Jr., MD, Study director, Protalex, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02401061
- Sponsor's protocol ID
- PRTX-100-202
- Status last verified
- Jul 2019
- First submitted
- 18 Mar 2015
- Last updated
- 5 Jul 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-Label, Dose Escalation Study of PRTX-100 in Adult Patients With Persistent/Chronic Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT02401061
- Acronym / sponsor ID
- PRTX-100-202
- Conditions
- Immune Thrombocytopenia
- Keywords
- ITP, Thrombocytopenia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Enough data has been collected to allow analysis of safety profile and risk-benefit.
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Protalex, Inc.Industry (pharma, biotech, devices)
- Study start
- Sep 2015
- Primary completion
- Mar 2019 (actual)
- Study completion
- 4 Jun 2019 (actual)
- First posted
- 27 Mar 2015 (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.
Pre-clinical and clinical evaluations show that PRTX- 100 has biological activity that may lead to improved platelet levels where these are decreased due to immunological pathologies and that PRTX-100 has an acceptable safety profile. In vivo treatment with PRTX-100 has been shown to raise platelet counts in a mouse model of immune thrombocytopenia (ITP). The primary objective of the study is to assess the efficacy of PRTX-100 in terms of platelet response in patients with chronic/persistent ITP.
Funding Source - FDA OOPD (1R01FD005750-01A1)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PRTX-100 | Experimental | Drug: PRTX-100Patients will be assigned to consecutive PRTX-100 interventions as they are enrolled into the study. Between two and six patients will be enrolled per intervention level. Intervention levels range from one (1) to twenty four (24) micrograms of PRTX-100 per kilogram of patient weight. Patients may receive up to four weekly infusions of PRTX-100 over the study treatment period. PRTX-100 doses ≤ 500 μg will be infused intravenously over 30 minutes. PRTX-100 doses > 500 μg will be infused over 60 minutes. Patients will remain under observation for 4 hours after completion of PRTX-100 dosing for safety management. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Willing and able to provide written informed consent prior to initiation of any study-related procedures
- Male or female ≥ 18 years of age
- ITP that has persisted for ≥ 3 months. ITP must be diagnosed in accordance The American Society of Hematology 2011 Evidence-based Practice Guideline for Immune Thrombocytopenia (Neunert et al. 2011) or the International Consensus Report on The Investigation and Management of Primary Immune Thrombocytopenia (Provan et al. 2010), as locally applicable.
- Received ≥ 1 typical regime for the treatment of ITP. Splenectomy is considered one standard ITP treatment
- A mean platelet count of < 30,000/μL, with no individual platelet count > 35,000/μL; or for those subjects receiving a constant dose of permitted treatments for ITP: a mean platelet count < 50,000/μL, with no count greater than 55,000/μL. (Note: The mean platelet count must be determined based on 2 platelet counts including one obtained within ≤ 7 days of first PRTX-100 dose and the other within ≤ 30 days of the first dose of PRTX-100.)
Key exclusion criteria (15 listed)
- Splenectomy ≤ 90 days prior to the first dose of PRTX-100
- Unstable coronary artery disease or other medical condition (such as type 1 diabetes) that, in the Investigator's opinion, might increase the risk to the patient
- Evidence of active infection requiring antibiotic therapy ≤ 14 days prior to the first dose of PRTX- 100
- Myelodysplastic syndrome. If clinically significant anemia or pancytopenia exists, documentation of a bone marrow aspirate within 24 months prior to the first dose of PRTX-100 showing no evidence of myelodysplasia is required.
- Medical history of vasculitis or lupus erythematosus
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 |
|---|---|---|
| PRTX-100Also: SpA, Staphylococcal Protein AFour weekly infusions of PRTX-100 at a level of 1 (3, 6,12, 18, or 24) microgram of PRTX-100 per kilogram of patient weight, infused over 30 minutes, followed by four hours of observation. | Drug | PRTX-100 |
Primary outcome
Platelet Response, Change from Baseline
Time frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
The primary efficacy endpoint is platelet response defined as a platelet count ≥ 30,000/μL and at least a doubling of baseline platelet count (determined on Day 1 prior to PRTX-100 administration) in patients with a baseline platelet count < 30,000/μL. In patients receiving permitted treatments for ITP with a baseline platelet count ≥ 30,000/μL and < 50,000/μL, an increase in platelet count to ≥ 50,000/μL and at least a doubling of baseline platelet count or an increase to > 100,000/μL be considered a platelet response.
5 secondary outcomes
Complete platelet response, Change from Baseline
Time frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Time to platelet response defined as the mean number of days from first PRTX-100 dose until platelet response
Time frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Durability of platelet response
Time frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Concomitant ITP medication use (frequency and amount)
Time frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Adverse Events
Time frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337
Results and safety
The study was stopped early (reason given: Enough data has been collected to allow analysis of safety profile and risk-benefit.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jul 2019.
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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.
William E Gannon Jr., MD study director
Protalex, Inc.
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PRTX-100 in people with Immune Thrombocytopenia, run by Protalex, Inc. at 11 sites in 2 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 13 inclusion and 15 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 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
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: Enough data has been collected to allow analysis of safety profile and risk-benefit.). 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?
Platelet Response, Change from Baseline (time frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337). 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 Jul 2019.