A Randomized, Controlled, Open- label Study Evaluating the Efficacy and Tolerability of AMG 531 versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects with Immune (Idiopathic) Thrombocytopenic Purpura

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2006-003700-18-AT
• Date of registration: 21/12/2006
• Prospective Registration: Yes
• Primary sponsor: Amgen Inc.
• Public title: A Randomized, Controlled, Open- label Study Evaluating the Efficacy and Tolerability of AMG 531 versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects with Immune (Idiopathic) Thrombocytopenic Purpura
• Scientific title: A Randomized, Controlled, Open- label Study Evaluating the Efficacy and Tolerability of AMG 531 versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects with Immune (Idiopathic) Thrombocytopenic Purpura
• Date of first enrolment: 05/02/2007
• Target sample size: 210
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2006-003700-18
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Standard of Care as defined by international treatment guidelines and local medical practice

Countries of recruitment

Austria, Belgium, Czech Republic, France, Germany, Italy, Netherlands, Portugal, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
- Subject is = 18 years of age.
- Subject has a diagnosis of ITP according to ASH guidelines.
- If subject is > 60 years of age, subject has a written bone marrow biopsy report confirming the diagnosis of ITP.
- Subject has received at least 1 prior therapy for ITP.
- Subject has a platelet count < 50 x 109/L or their platelet count falls to < 50 x 109/L during or after a clinically- indicated taper or discontinuation of current ITP therapy.
- Before any study- specific procedure, the appropriate written informed consent must be obtained (see Section 12.1).
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Subject has had a splenectomy for any reason.
- Subject has an active malignancy.
- Subject has a history of cancer, other than basal cell carcinoma or cervical carcinoma in situ, with treatment or active disease within 5 years.
- Subject has a known history of bone marrow stem cell disorder,
• Abnormal bone marrow findings, other than those typical of ITP, must be approved by Amgen before a subject may be enrolled in the study.
- Subject has participated in any study evaluating PEG- rHuMGDF, recombinant human thrombopoietin (rHuTPO), AMG 531, or a thrombopoietic protein.
- Subject is receiving other investigational agents or procedures.
- Subject is currently enrolled in, or has completed within the last 30 days, another investigational device or drug study.
- Subject is pregnant or breast feeding.
- Subject is not using adequate contraceptive precautions.
- Subject has known sensitivity to any recombinant E coli- derived product.
- Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative.
- Subject has any kind of disorder that compromises the ability of the subject to comply with study procedures.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Immune (idiopathic) thrombocytopenic purpura (ITP)
MedDRA version: 8.1 Level: PT Classification code 10021245 Term: Idiopathic thrombocytopenic purpura

Intervention(s)

Product Name: AMG 531
Product Code: AMG 531
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: AMG 531
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 600-

Primary Outcome(s)

Secondary Objective: The secondary objectives of the study are to observe the impact of AMG 531 on various ITP symptoms and platelet parameters compared to medical SOC for ITP. These include the time to splenectomy, platelet response, and the change in ITP Patient Assessment Questionnaire (PAQ) Physical Health domains of Symptoms, Bother, Activity, and Fatigue.

Primary end point(s): This study has 2 primary endpoints, 1 related to splenectomy and the other related to treatment failure. The first primary endpoint will be the number of subjects undergoing a splenectomy by treatment group during the 52-week treatment period. The second primary endpoint will be the number of subjects with a treatment failure during the 52-week treatment period. Treatment failure is defined as:

• a lack of efficacy defined as platelet count < 20 x 109/L for 4 consecutive weeks at the highest recommended dose and schedule (AMG 531 – 10 ug/kg weekly; medical SOC for ITP – apply standard institutional practices or therapeutic guidelines); or,

• a major bleeding event; or,

• a change in therapy due to an intolerable side effect or bleeding symptoms (including a minor bleeding event).
A minor bleeding event is defined as “ dry” or “ wet” purpura (eg, petechiae, mucosal bleeding from the gums or nose). A major bleeding event is defined as a hemorrhage (eg, upper or lower gastrointestinal tract, genitourinary tract or central nervous system). The primary statistical hypothesis will be that the corresponding rates in the AMG 531 arm will be lower compared to the medical SOC for ITP arm in both the proportion of subjects having a splenectomy and the proportion of subjects with a treatment failure within the 52-week treatment period. The study will declare a success with AMG 531 treatment if the tests for both primary efficacy endpoints are statistically significant in favor of AMG 531 arm over the medical SOC for ITP arm.

Main Objective: The objective of the study is to compare the ability of AMG 531 versus medical standard of care (SOC) to prevent a splenectomy and to provide a durable treatment option for immune (idiopathic) thrombocytopenic purpura (ITP) non-splenectomized adult subjects during the 52- week treatment period.

Secondary ID(s)

20060131