Clinical trial NCT00440037: Open Label Extension Study of AMG 531 in Japanese Subjects With ITP
Official title: An Open Label Extension Study Evaluating the Safety and Efficacy of Long Term Dosing of AMG 531 in Thrombocytopenic Japanese Subjects With Immune (Idiopathic) Thrombocytopenic Purpura
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Keywords: AMG 531
ITP
Long term treatment
Japanese
Romiplostim
Lead sponsor: Kyowa Kirin Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2007-02-26
Enrollment: 44
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the safety and efficacy of long term dosing of AMG 531 in thrombocytopenic Japanese subjects with ITP.

It is anticipated that AMG 531 will be a safe and well tolerated in long term treatment and that AMG 531 will effectively raise and maintain platelet counts to a desired therapeutic range, when individual dose adjustments based on platelet counts are permitted.

This study is available to subjects who have completed any previous AMG 531 ITP study in Japan and meet the eligibility criteria of this study.

Detailed description: Romiplostim was administered by subcutaneous (SC) injection once per week. If subjects entered the extension study within 12 weeks from the last investigational product administration in the previous study and had shown an increase in platelet counts ≥ 20 x 109/L from baseline at least once during the 13-week treatment period (excluding 4 weeks after receiving rescue medication), they were treated with romiplostim at the same weekly dose (last dose on study) received in the previous study. Otherwise, subjects were treated with romiplostim at a starting dose of 3 μg/kg. Dose adjustment based on platelet counts was allowed throughout the treatment period to allow subjects to maintain platelet counts in the target range of ≥ 50 to ≤ 200 x 109/L, up to a maximum permitted dose of 10 μg/kg.

Eligibility criteria: Inclusion Criteria

* Subjects must have previously completed an AMG 531 ITP study in Japan.
* Platelet count taken at the screening visit must be \< 50 x 109/L.
* Before any study-specific procedure, the appropriate written informed consent must be obtained.

Exclusion Criteria

* Any significant change in medical history since completion of the previous AMG 531 ITP study including bone marrow stem cell disorders or new active malignancies
* known positive result from a test for neutralizing antibodies to AMG 531 in the previous AMG 531 ITP study
* Currently receiving any treatment for ITP except oral corticosteroids, azathioprine and/or danazol administered at a constant dose and schedule from at least 4 weeks prior to the screening visit
* received intravenous immunoglobulin, anti-D immunoglobulin, or any drug administered to increase platelet counts (eg, immunosuppressants etc) within 1 week before the screening visit
* received anti-malignancy agents (eg, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, Interferon-alfa etc) within 4 weeks before the screening visit
* received any monoclonal antibody drugs (eg, rituximab etc) within 8 weeks before the screening visit
* Less than 4 weeks since receipt of any therapeutic drug or device that is not Ministry of Health, Labor and Welfare (MHLW) approved for any indication before the screening visit (excluding AMG 531)
* Pregnant or breast feeding
* Subjects of reproductive potential who are not using adequate contraceptive precautions, in the judgment of the investigator
* known severe drug hypersensitivity
* Concerns for subject's compliance with the protocol

Primary outcomes:
measure: Percentage of Participants With Adverse Events Including Clinically Significant Changes in Laboratory Values.
timeFrame: Through study completion, the median treatment duration (time between first dose and last dose) was 146.0 weeks (range: 12 to 243 weeks)
Secondary outcomes:
measure: Incidence of Anti AMG 531 Antibody Formation
timeFrame: Through study completion, the median treatment duration (time between first dose and last dose) was 146.0 weeks (range: 12 to 243 weeks)
measure: Incidence of Platelet Response (Platelet Response is Defined as a Doubling of Baseline Platelet Counts and More Than 50 x 10^9/L; Baseline Platelet Counts is That Obtained in the Previous Study)
timeFrame: Through study completion, the median treatment duration (time between first dose and last dose) was 146.0 weeks (range: 12 to 243 weeks)
measure: Percentage of Subjects Able to Reduce or Discontinue Their Concurrent ITP Therapies (for Subjects That Are Receiving Oral Corticosteroids at a Constant Dose and Schedule at the Screening Visit)
timeFrame: Through study completion, the median treatment duration (time between first dose and last dose) was 146.0 weeks (range: 12 to 243 weeks)
measure: Change From Baseline in Patient-reported Outcome (PRO) Endpoints at Each Time Point (Baseline PRO is Obtained at Day 1 Predose)
timeFrame: By Week 48
Other outcomes:
Locations:
city: Sapporo
country: Japan
facility: Research Site
state: Hokkaido
city: Tsukuba
country: Japan
facility: Research Site
state: Ibaraki
city: Isehara-shi
country: Japan
facility: Research Site
state: Kanagawa
city: Sagamihara
country: Japan
facility: Research Site
state: Kanagawa
city: Suita
country: Japan
facility: Research Site
state: Osaka
city: Chuo
country: Japan
facility: Research Site
city: Hirakata
country: Japan
facility: Research Site
city: Hiroshima
country: Japan
facility: Research Site
city: Kumamoto
country: Japan
facility: Research Site
city: Moriguchi
country: Japan
facility: Research Site
city: Tokyo
country: Japan
facility: Research Site
city: Tokyo
country: Japan
facility: Research Site
city: Tokyo
country: Japan
facility: Research Site
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: Arranz R, Garcia-Noblejas A, Grande C, Cannata-Ortiz J, Sanchez JJ, Garcia-Marco JA, Alaez C, Perez-Calvo J, Martinez-Sanchez P, Sanchez-Gonzalez B, Canales MA, Conde E, Martin A, Arranz E, Terol MJ, Salar A, Caballero D. First-line treatment with rituximab-hyperCVAD alternating with rituximab-methotrexate-cytarabine and followed by consolidation with 90Y-ibritumomab-tiuxetan in patients with mantle cell lymphoma. Results of a multicenter, phase 2 pilot trial from the GELTAMO group. Haematologica. 2013 Oct;98(10):1563-70. doi: 10.3324/haematol.2013.088377. Epub 2013 Jun 10.