Spinal muscular atrophy Nervous System Diseases
Conditions
Interventions
Adult male participants with SMA, who are currently on treatment with risdiplam or were previously treated with risdiplam, will be enrolled in this study. Participants actively trying to conceive will
Sponsors
Genentech
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Diagnosis of SMA 2. Currently receiving or previously ever received risdiplam treatment 3. Actively trying to conceive or conceived in the past (during or after exposure to risdiplam) 4. Consent to a baseline and annual questionnaire for the duration of the study 5. Able to complete a questionnaire, in English, with or without assistance 6. Able to access a smartphone with internet connection
Exclusion criteria
Exclusion criteria: 1. Participants who are using/used donor sperm for conception 2. Participants who are using/used their own cryopreserved sperm that was not exposed to risdiplam for conception
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Whether risdiplam-exposed adult males with SMA conceive, measured using questionnaires completed by the participants at study initiation and annually for up to 3 years | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Presence of confounding factors that may impact fertility in enrolled participants, measured using questionnaires completed by the participants at study initiation and annually for up to 3 years 2. Fertility-related healthcare resource utilization, management, and treatment decisions in enrolled participants, measured using questionnaires completed by the participants at study initiation and annually for up to 3 years 3. Outcome of pregnancies, measured using questionnaires completed by the participants at study initiation and annually for up to 3 years | — |
Countries
United States of America
Contacts
Public ContactClinical Trials
Outcome results
None listed