ongitudinal study of clinical outcome measures in adult patients with Spinal Muscular Atrophy (SMA) treated with Risdiplam. - version 4.0

Z Leuven0 sites20 target enrollmentSeptember 10, 2022

ConditionsSpinal Muscular Atrophy (SMA)MedDRA version: 20.1Level: PTClassification code: 10041582Term: Spinal muscular atrophy Class: 100000004850 Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Spinal Muscular Atrophy (SMA)
Sponsor: Z Leuven
Enrollment: 20
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

September 10, 2022

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Z Leuven

Eligibility Criteria

Inclusion Criteria

•Participants eligible for inclusion in this Trial must meet all of the following criteria: 1\)Voluntary written informed consent of the participant or their legally authorized representative has been obtained prior to any screening procedures; 2\) Use of highly effective methods of birth control; Highly effective contraceptives are methods that can achieve a failure rate of less than 1% per year when used consistently and correctly. Such methods include: • combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal); • progestogen\-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable); • intrauterine device (IUD); • intrauterine hormone\-releasing system ( IUS); • bilateral tubal occlusion; • vasectomised partner; • sexual abstinence. A male participant who is sexually active with a woman of childbearing potential and who has not had a vasectomy must agree to use a barrier method of birth control (eg, either a condom \[with spermicidal foam/gel/film/cream/suppository or a partner with an occlusive cap \[diaphragm or cervical/vault caps] plus spermicidal foam/gel/film/cream/suppository), during the study and for at least 4 months after receiving the last administration of Risdiplam. The time period during which highly effective contraception is required after the last dose of Risdiplam is 1 month for female patients of childbearing potential and 4 months for male patients. Male participants will be informed that they may consider sperm preservation prior to treatment initiation; 3\)Male or female patients of an adult age (i.e., 18 years or older); 4\)Confirmed genetic diagnosis of 5q\-autosomal recessive Spinal Muscular Atrophy (SMA) (i.e., deletions of both SMN1\-genes or mutations in both SMN1\- genes have been genetically proven); 5\)Patients eligible for treatment with Risdiplam (Evrysdi®) (i.e., SMA type 2 or 3 or patients with up to 4 SMN2\-gene copies and no permanent invasive ventilation, corresponding to the RIZIV reimbursement criteria for Evrysdi® (Risdiplam) in Belgium) ), that are either treatment\-naive or are already being treated with Evrysdi® (Risdiplam) in Standard\-Of\-Care.

Exclusion Criteria

•Participants eligible for this Trial must not meet any of the following criteria: 1\)Any disorder, which in the Investigator’s opinion might jeopardise the participant’s safety or compliance with the protocol; 2\) Female who is pregnant, breast\-feeding or intends to become pregnant or is of child\-bearing potential and not using an adequate, highly effective contraceptive. A woman is considered of childbearing potential (WOCBP), i.e. fertile, following menarche and until becoming post\-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. Exclusion criterion is a male participant who is sexually active with a woman of childbearing potential and who has not had a vasectomy and does not agree to use a barrier method of birth control (e.g. a condom) during the study until at least 4 months after the last dose of Risdiplam; 3\)Participation in an interventional Trial with an investigational medicinal product (IMP) or device; 4\) Patients younger than 18 years of age at the time of the study; 5\) Patients without genetically confirmed diagnosis of 5q\-SMA at the time of the study; 6\) SMA patients not eligible for treatment with Risdiplam according to the RIZIV reimbursement criteria for Risdiplam (Evrysdi®) in Belgium; 7\) Patients treated with Risdiplam (Evrysdi®) in a Compassionate Use Programme (CUP) at the time of inclusion; 8\) Patients treated with nusinersen (Spinraza®) before or at the time of inclusion.