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Trial record 10 of 23 for:    "Adenosine deaminase deficiency"

Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

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ClinicalTrials.gov Identifier: NCT03232203
Recruitment Status : Recruiting
First Posted : July 27, 2017
Last Update Posted : January 8, 2019
Sponsor:
Information provided by (Responsible Party):
Orchard Therapeutics Limited

Brief Summary:
STRIMVELIS is a medicinal product that restores adenosine deaminase (ADA) function in hematopoietic cell lineages, thereby preventing impaired immune function. STRIMVELIS is indicated for the treatment of patients with ADA- severe combined immunodeficiency (SCID), for whom suitable human leukocyte antigen (HLA)-matched related stem cell donor is not available. The objective of this study is to evaluate the effectiveness of routine and additional risk minimization measures by assessing the understanding of referring health care providers (HCPs) and parents/carers (hereby referred as participants) with regard to the specific risks associated with STRIMVELIS. In this cross-sectional study, surveys will be provided to referring HCPs and parents/carers of children approximately six months after treatment with STRIMVELIS. The study will recruit for approximately two years until 10 referring HCPs and 10 parents/carers have completed their respective surveys.

Condition or disease Intervention/treatment
Severe Combined Immunodeficiency Due to ADA Deficiency Drug: STRIMVELIS

Study Type : Observational [Patient Registry]
Estimated Enrollment : 10 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Target Follow-Up Duration: 2 Years
Official Title: Evaluation of Referring HCPs' and Parents'/Carers' Understanding of Specific Risks Associated With Strimvelis™ Treatment
Actual Study Start Date : April 12, 2018
Estimated Primary Completion Date : September 30, 2020
Estimated Study Completion Date : September 30, 2020


Group/Cohort Intervention/treatment
Health care providers
A HCP survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS summary of product characteristics and educational materials
Drug: STRIMVELIS
It is the Autologous cluster of differentiation (CD) 34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA complementary Deoxyribonucleic acid (cDNA) sequence. HCP who have previously referred a patient for STRIMVELIS treatment or a parent's/carer's child who previously received treatment with STRIMVELIS will be recruited to the study

Parent/carer
A parent/carer survey instrument of approximately 20 questions will be provided. Survey questions will be based on the STRIMVELIS Patient Information Leaflet and educational materials
Drug: STRIMVELIS
It is the Autologous cluster of differentiation (CD) 34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA complementary Deoxyribonucleic acid (cDNA) sequence. HCP who have previously referred a patient for STRIMVELIS treatment or a parent's/carer's child who previously received treatment with STRIMVELIS will be recruited to the study




Primary Outcome Measures :
  1. Proportion of HCPs providing the correct response [ Time Frame: Up to 2 years ]
    A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.

  2. Proportion of Parents/Carers providing the correct response [ Time Frame: Up to 2 years ]
    A series of questions concerning the specific risks associated with STRIMVELIS will be asked during survey. Data from all survey respondents will be analyzed and reported as descriptive statistics.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
Study Population
Parents/carers and referring HCPs of patients enrolled within this registry or patients treated with STRIMVELIS outside of the registry will be contacted separately and asked to participate in their respective surveys evaluating the effectiveness of risk minimization measures.
Criteria

Inclusion Criteria:

  • HCPs or HCPs' close family members may not have been employees of GSK, Pharmaceutical Product Development, LLC (PPD), the Food and Drug Administration (FDA), or the European Medicines Agency (EMA).
  • HCPs must be licensed
  • An HCP must not have previously completed a survey regarding STRIMVELIS educational materials.
  • An HCP must have previously referred a patient for STRIMVELIS treatment.
  • Parents/carers or parents'/carers' close family members may not have been employees of GSK, PPD, FDA, or EMA.
  • A parent/carer must not have previously completed a survey regarding STRIMVELIS educational materials.
  • A parent's or carer's child must have previously received treatment with STRIMVELIS

Exclusion Criteria:

  • No exclusion criteria

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03232203


Contacts
Contact: Orchard Clinical Trials +44 (0) 203 384 6700 medinfo@orchard-tx.com

Locations
Italy
Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Recruiting
Milan, Italy, 20132
Contact: Orchard Clinical Trials         
Sponsors and Collaborators
Orchard Therapeutics Limited
Investigators
Study Director: Clinical Trials Orchard Therapeutics Limited

Responsible Party: Orchard Therapeutics Limited
ClinicalTrials.gov Identifier: NCT03232203     History of Changes
Other Study ID Numbers: STRIM-001
First Posted: July 27, 2017    Key Record Dates
Last Update Posted: January 8, 2019
Last Verified: January 2019

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Orchard Therapeutics Limited:
Human leukocyte antigen
Key Risk Message
ADA-SCID
Health care providers

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Severe Combined Immunodeficiency
Immune System Diseases
Infant, Newborn, Diseases
DNA Repair-Deficiency Disorders
Metabolic Diseases