Study of the self-administration of AOP2014 using a pre-filled pen, developed for the treatment of Polycythemia Vera patients

Phase 1

Conditions: Polycythemia Vera
MedDRA version: 18.0Level: LLTClassification code 10036061Term: Polycythemia veraSystem Organ Class: 100000004864
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2014-001356-31-CZ

Lead Sponsor: AOP Orphan Pharmaceuticals AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 30

Inclusion Criteria

1. Patients who either completed the 12 months AOP2014 treatment arm of the PROUD-PV study, or are currently participating in the CONTINUATION-PV, and at the end-of-treatment visit (EoT) of the PROUD-PV study or two weeks after the last visit of the CONTINUATION-PV study, fulfill at least one of the following criteria:
a. Normalization of at least two out of three main blood parameters (Hct, PLTs and WBCs) if these parameters were moderately increased (Hct<50%, WBC<20 x 10 9/L, PLTs<600 x 10 9/L) at baseline visit of the PROUD-PV study, OR
b. >35% decrease of at least two out of three main blood parameters (Hct, PLTs and WBCs) if these parameters were massively increased (Hct>50%, WBCs>20 x 10 9/L, PLTs>600 x 10 9/L) at baseline visit of the PROUD-PV study, OR
c. Normalization of spleen size, if spleen was enlarged at baseline visit of the PROUD-PV study, OR
d. Otherwise a clear, medically verified benefit from treatment with AOP2014 (e.g. normalization of disease-related micro-vasculatory symptoms, substantial decrease of JAK2 allelic burden).
2. Signed written ICF.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 8

Exclusion Criteria

Withdrawal criteria, as specified in the PROUD-PV and CONTINUATION-PV studies, which mandate treatment discontinuation:
1. Non-recovery from the AOP2014 related toxicities to the grade (usually, Grade I) which allows continuation of the treatment.
2. Hospital Anxiety Depression Scale (HADS) score of 11 or higher on either or both of the subscales, and /or development or worsening of clinically significant depression or suicidal thoughts.
3. Progressive and clinically significant increase of liver enzyme levels despite dose reduction, or if such increase is accompanied by increased bilirubin level, any signs or symptoms of a clinically significant autoimmune disease.
4. Clinically significant development of a new ophthalmologic disorder, or worsening of a pre-existing one, during the study.
5. Loss of efficacy of AOP2014 or any comparable situation where no further benefits of treatment continuation are expected by the investigator.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the ease of AOP2014 self-administration using dedicated questionnaires;Secondary Objective: - To assess safety and tolerability: adverse events (AEs), laboratory parameters, electrocardiogram (ECG) throughout study.<br>- To assess maintenance of the blood efficacy parameters Hct, WBC and PLTs and spleen size (comparing values at Visit P7 vs. values at Visit P1).<br>- To assess the feasibility of AOP2014 self-administration: defined as the ability of the patients to use the pen as a self-administration tool (ease of handling, safety, tolerability and efficacy).;Primary end point(s): To evaluate ease of self-administration of AOP2014, as assessed by staff and patients using dedicated questionnaires, using rates of full success and failure rates.;Timepoint(s) of evaluation of this end point: every two weeks

Secondary Outcome Measures

Name	Time	Method
Timepoint(s) of evaluation of this end point: every two weeks;Secondary end point(s): Efficacy Evaluation<br>1. Disease response rate.<br>2. Number of phlebotomies performed during the study.<br>3. Hct, erythrocyte (RBC), WBC and PLT changes comparing values at the assessment visit (P7) vs. values at baseline visit (P1) and changes at every visit.<br>4. Spleen size at the end of the study vs. baseline visit (P1).<br><br>Safety evaluation<br>1. Disease-related symptoms (microvascular disturbances, pruritus, headache), assessed at every visit.<br>2. AEs, incl. adverse events of special interest (AESI).<br>3. Laboratory data.<br>4. Standard 12-lead ECG.<br>5. Physical examination.<br>6. Vital signs: heart rate, systolic and diastolic blood pressure.<br>7. Immunogenicity.<br>8. Urine.

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