PRTX-100-203 Open-Label, Dose Escalation Study in Adult Patients With ITP

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02566603
• Date of registration: 30/09/2015
• Prospective Registration: Yes
• Primary sponsor: Protalex, Inc.
• Public title: PRTX-100-203 Open-Label, Dose Escalation Study in Adult Patients With ITP
• Scientific title: A Phase 1b Open-Label, Dose Escalation Study of PRTX-100 in Adult Patients With Persistent/Chronic Immune Thrombocytopenia
• Date of first enrolment: November 2015
• Target sample size: 15
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02566603
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

France, United Kingdom

Contacts

• Name: William E Gannon, MD
• Affiliation: Protalex, Inc.

Key inclusion & exclusion 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 regimen for the treatment of ITP. Splenectomy is considered one
regimen.

5. A mean platelet count of < 30,000/µL with no individual platelet count > 55,000/µL.
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 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.

8. Any prior treatment with rituximab or any other anti-CD20 agent must have been > 6
months prior to the first dose of PRTX-100

9. If female, must not be pregnant (as indicated by screening negative pregnancy test),
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. Exposure to TPO-RA within 2 weeks before inclusion

3. Previous treatment with rituximab within <6 months prior to the first dose of PRTX-100

4. Bleeding score = 8 (Khellaf M et al. Haematologica 2005)

5. 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

6. Evidence of active infection requiring antibiotic therapy = 14 days prior to the first
dose of PRTX-100

7. 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.

8. Medical history systemic lupus erythematosus or any cause of secondary ITP

9. 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

10. Seropositive for human immunodeficiency virus (HIV)

11. 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)

12. History suggestive of substance abuse

13. Clinically significant abnormalities in screening laboratory tests, including:

- Absolute neutrophil count < 1.0 x109/L

- Hemoglobin < 10 g/dL

- Absolute lymphocyte count < 0.8 x109/L

- Alanine transaminase (ALT) or aspartate transaminase (AST) > 2 x upper limit of
normal (ULN)

- Lactate dehydrogenase > 3 x ULN

- Total bilirubin level >1.5 x ULN

- Serum creatinine level > 0.14 mmol/L (1.6 mg/dL) in males or 0.12 mmol/L (1.4
mg/dL) in females

14. Treatment with IVIG = 14 days prior to the first dose of PRTX-100

15. Treatment with an anti-Rh D antigen agent (e.g. WinRho®) = 14 days prior to the first
dose of PRTX-100

16. Use of any investigational drug = 30 days or 5 half-lives of the investigational drug
(whichever is longer) prior to the first dose of PRTX-100



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Immune Thrombocytopenia

Intervention(s)

Drug: PRTX-100

Primary Outcome(s)

Number of participants with treatment-related adverse events as assessed by Toxicity Grading Criteria based on RCTC v 2.0 and CTCAE v 4.03 [Time Frame: 337 Days]

Secondary Outcome(s)

Overall platelet response, change from baseline (Day 1) [Time Frame: Days 1, 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337]

Concomitant ITP medication use (number of subjects) [Time Frame: 337 Days]

Time to platelet response (number of days) [Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337]

Complete platelet response (number of patients) [Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337]

Durability of platelet response (number of days) [Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337]

Secondary ID(s)

2014-005626-35

PRTX-100-203

Source(s) of Monetary Support

Please refer to primary and secondary sponsors