A Phase 1/2, Open-Label, Dose Escalation Study of PRTX-100 in Adult Patients With Persistent/Chronic Immune Thrombocytopenia
PRTX-100-202 Open-Label, Dose Escalation Study in Adult Patients With ITP
Sponsors
Source
Protalex, Inc.
Oversight Info
Has Dmc
Yes
Brief 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. 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)
Overall Status
Terminated
Start Date
2015-09-01
Completion Date
2019-06-04
Primary Completion Date
2019-03-01
Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Measure |
Time Frame |
Platelet Response, Change from Baseline |
Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 |
Secondary Outcome
Measure |
Time Frame |
Complete platelet response, Change from Baseline |
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 |
Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 |
Durability of platelet response |
Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 |
Concomitant ITP medication use (frequency and amount) |
Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 |
Adverse Events |
Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 |
Enrollment
13
Condition
Intervention
Intervention Type
Drug
Intervention Name
Description
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.
Arm Group Label
PRTX-100
Other Name
SpA
Staphylococcal Protein A
Eligibility
Criteria
Inclusion Criteria:
1. Willing and able to provide written informed consent prior to initiation of any
study-related procedures
2. Male or female ≥ 18 years of age
3. 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.
4. Received ≥ 1 typical regime for the treatment of ITP. Splenectomy is considered one
standard ITP treatment
5. 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.)
6. 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.
7. If receiving eltrombopag or romiplostim, the dose must have been stable for ≥ 21 days
prior to the first dose of PRTX-100
8. 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.
9. Any prior treatment with rituximab or any other anti-CD20 agent must have been > 6
months prior to the first dose of PRTX-100
10. 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.
Exclusion Criteria:
1. Splenectomy ≤ 90 days prior to the first dose of PRTX-100
2. 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
3. Evidence of active infection requiring antibiotic therapy ≤ 14 days prior to the first
dose of PRTX- 100
4. 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.
5. Medical history of vasculitis or lupus erythematosus
6. Propensity to allergic reactions defined as a history of allergic reaction to more
than one medication
7. 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
8. Seropositive for human immunodeficiency virus (HIV)
9. 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)
10. History suggestive of substance abuse
11. 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
12. Treatment with IVIG ≤ 14 days prior to the first dose of PRTX-100
13. Treatment with an anti-Rh D antigen agent (e.g. WinPho) ≤ 14 days prior to the first
dose of PRTX-100
14. 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
15. Not willing to stay at the study site for 4 hours after each PRTX-100 infusion
Gender
All
Minimum Age
18 Years
Maximum Age
N/A
Healthy Volunteers
No
Overall Official
Last Name |
Role |
Affiliation |
William E Gannon Jr., MD |
Study Director |
Protalex, Inc. |
Location
Facility |
USC Norris Comprehensive Cancer Center Los Angeles California 90033 United States |
Massachusetts General Hospital Boston Massachusetts 02114 United States |
Michigan Center of Medical Research Farmington Hills Michigan 48334 United States |
Weil-Cornell Medical College New York New York 10065 United States |
Gabrail Cancer Center Canton Ohio 44718 United States |
Cleveland Clinic Foundation Cleveland Ohio 44195 United States |
Aberdeen Royal Infirmary Aberdeen AB AB25 27N United Kingdom |
Leicester Royal Infirmary Leicester Leichester LE1 5WW United Kingdom |
Queen Elizabeth Hospital Birmingham B15 2TH United Kingdom |
Glasgow Royal Infirmary Glasgow G4 0SF United Kingdom |
Norfolk and Norwich Hospital Norwich NR4 7UY United Kingdom |
Location Countries
Country
United Kingdom
United States
Verification Date
2019-07-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Keywords
Has Expanded Access
No
Condition Browse
Number Of Arms
1
Arm Group
Arm Group Label
PRTX-100
Arm Group Type
Experimental
Description
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.
Firstreceived Results Date
N/A
Why Stopped
Enough data has been collected to allow analysis of safety profile and risk-benefit.
Firstreceived Results Disposition Date
N/A
Study Design Info
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)
Study First Submitted
March 18, 2015
Study First Submitted Qc
March 23, 2015
Study First Posted
March 27, 2015
Last Update Submitted
July 2, 2019
Last Update Submitted Qc
July 2, 2019
Last Update Posted
July 4, 2019
ClinicalTrials.gov processed this data on December 13, 2019
Conditions
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conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.