Integrated healthcare of new therapies through telemedicine, empowerment, gene therapeutics, registry establishment, drug safety, therapy pathways and reimbursement

Recruiting

• Conditions: spinal muscular atrophy, severe combined immunodeficiency due to adenosindesamniase deficiency, metachromatic leukodystrophy, folicular lymphoma, mantle cell lymphoma, diffuse large B-cell lymphoma, other and unspecified types of non-Hodgkin's lymphoma

• Registration Number: DRKS00030630
• Lead Sponsor: niversitätsklinikum Heidelberg, Klinik für Hämatologie, Onkologie und Rheumatologie Innere Medizin V

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-02-03
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 650

Inclusion Criteria

Participating patients must be insured with a statutory health insurance
- Signed treatment contract between practitioner and patient/parents according to §630a BGB
- Treatment in one of the participating treatment centers
- Age of CAR-T-cell therapy patients: = 18 years
- Age of patients for SMA, ADA-SCID and MLD: ? 7 years
- Ability to understand the patient's information and sign the consent form
- Ability to consent by patients themselves or by parents
- Consent to the exchange of data between practitioners, the telemedicine platform and the INTEGRATE register
- Indication for ATMP treatment within the existing marketing authorisation for one of the following indications prior to inclusion:
o CAR-T cell therapy: patients with diseases of the lymphatic system and planned commercial CAR-T cell therapy in an approved indication
o Spinal muscular atrophy (SMA): patients with SMA and planned commercial gene therapy with onasemnogen abeparvovec in the approved indication
o Severe combined immunodeficiency in adenosine desaminase deficiency (ADA-SCID): patients with ADA-SCID and planned gene therapy with autologous CD34+ cells transduced to express adenosine desaminase (ADA) in the approved indication.
o Metachromatic leukodystrophy (MLD): patients with MLD and planned gene therapy in the approved indication (Atidarsagen Autotemcel – autologous lentiviral transduced CD34+ cells to replace the arylsulfatase A gene).

Exclusion Criteria

- ATMP patients without a statutory health insurance
- Treatment with ATMP as part of a clinical trial or off-label use
- patients that are not able to give informed consent
- Project content is not comprehensible to patients

Study & Design

• Study Type: observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Health-related quality of life of patients

Secondary Outcome Measures

Name	Time	Method
1) Patient and healthcare provider satisfaction with care.<br>2) Overall survival<br>3) Event-free survival including adverse drug reaction (ADR) recording & disease-specific endpoints: <br>Lymphoma: progression, recurrence of the underlying disease, death<br>SMA: free sitting >30 seconds <br>MLD: gross motor function <br>ADA-SCID: Immune reconstitution