EUCTR2006-006304-11-ES进行中（未招募）1 期
A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa - TKT034
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 41
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient glucocerebrosidase (GCB) activity in leukocytes or by genotype analysis.
- •The patient has received imiglucerase at a dose = 60 U/kg and = 15 U/kg every other
- •week for a minimum of 30 consecutive months. Patients must have received the
- •same dose and dose regimen during the 6 months prior to study enrollment. (Note:
- •Patients who are anti-imiglucerase antibody positive will be allowed to enter this
- •The patient is at least 2 years of age.
- •Female patients of child bearing potential must agree to use a medically acceptable
- •method of contraception at all times during the study and must have negative results
- •to a pregnancy test performed at Screening and as required throughout their
- •participation in the study.
- •The patient, the patient’s parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
- •The patient must be sufficiently cooperative to participate in this clinical study as
- •judged by the Investigator.
- •Are the trial subjects under 18? yes
- •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
排除标准
- •The patient has a hemoglobin concentration = 10 g/dL and a platelet count = 80,000 platelets/cu mm.
- •The difference between the patient’s highest and lowest hemoglobin concentration
- •collected over 3 consecutive evaluations is > 1 g/dL, AND/OR, the difference
- •between the patient’s highest and lowest platelet count collected over 3 onsecutive evaluations is > 20%. (Note: At least 1 of these values must have been collected at
- •least 6 months prior to the Screening evaluation and 1 value must be collected during Screening).
- •The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease.
- •The patient has received treatment with any investigational drug or device within the
- •30 days prior to study entry; such use during the study is not permitted.
- •The patient is known to be positive for human immunodeficiency virus (HIV), i.e.,
- •has a documented positive result. Patients who do not have a documented positive
- •result will be tested for HIV during Screening.
- •The patient is known to be positive for hepatitis B and/or C, i.e., has a documented
- •positive result. Patients who do not have a documented positive result will be tested
- •for hepatitis during Screening.
- •The patient presents with sustained iron, folic acid and/or vitamin B12 deficiencyrelated anemia during Screening (i.e., non-Gaucher disease-related anemia).
- •The patient, patient’s parent(s), or patient’s legal guardian(s) is/are unable to
- •understand the nature, scope, and possible consequences of the study.
- •The patient has a significant comorbidity that might affect study data or confound the
- •study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease,
- •The patient is unable to comply with the protocol, e.g., has a clinically relevant
- •medical condition making implementation of the protocol difficult, has an
- •uncooperative attitude, is unable to return for safety evaluations, or is otherwise
- •unlikely to complete the study, as determined by the Investigator.
- •The patient has had inconsistent treatment with imiglucerase in the last 6 months such as a drug holiday.
- •The patient has experienced an anaphylactic reaction during treatment with
- •imiglucerase.
- •The patient has received miglustat during the 6 months prior to study enrollment.
- •The patient is currently receiving erythropoietin or chronic systemic corticosteroids in
- •the last 6 months. (NOTE: Use of intermittent corticosteroids as premedication to
- •prevent infusion reactions is allowed.)
- •The patient has experienced spleen infarction.
- •The patient has bone necrosis.
研究者
相似试验
进行中（未招募）
1 期
A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase. - TKT034Type I Gaucher DiseaseMedDRA version: 9.1Level: LLTClassification code 10018048Term: Gaucher's diseaseEUCTR2006-006304-11-DEShire Human Genetic Therapies Inc40
进行中（未招募）
1 期
A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase - TKT034Type I Gaucher DiseaseMedDRA version: 9.1 Level: LLT Classification code 10018048 Term: Gaucher's diseaseEUCTR2006-006304-11-GBShire Human Genetic Therapies Inc41
进行中（未招募）
不适用
A Multicenter Open-Label Study of Gene-Activated Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease Previously Treated with Imiglucerase - NDEUCTR2006-006304-11-ITSHIRE HUMAN GENETIC THERAPIES, INC.40
进行中（未招募）
1 期
An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher DiseaseType I Gaucher diseaseMedDRA version: 9.1 Level: LLT Classification code 10018048 Term: Gaucher's diseaseEUCTR2008-001965-27-GBShire Human Genetic Therapies, Inc.95
进行中（未招募）
1 期
A phase IA/II, multi-center, open-label study of HCD122 administered intravenously once weekly for four weeks in adult patients with advanced non-Hodgkin's or Hodgkin's lymphoma who have progressed after at leastr two prior therapies - CHCD122A2103EUCTR2007-004888-22-BEovartis Pharma AG230