RECRUITING

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.

Official Title

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Quick Facts

Study Start:2024-04-26

Study Completion:2025-08-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05808764

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified to 19 Days

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
* Male or female newborn infant aged \<20 days at first dose * Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing. * Gestational age equal to or greater than 37 weeks * Receiving adequate nutrition and hydration at the time of screening * Adequately recovered from any acute illness at baseline and considered well enough to participate in the study * Parent/caregiver is willing to consider nasogastric, nasojejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator.	* Presence of clinical symptoms or signs consistent with SMA Type 0 * In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures * Systolic blood pressure or diastolic blood pressure or heart rate abnormalities * Presence of clinically relevant electrocardiogram (ECG) abnormalities * The infant (or the person breastfeeding the infant) taking any of the following: any inhibitor of CYP3A4 taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing, any inducer of CYP3A4 taken within 4 weeks (or within 5 times the elimination half-life, whichever is longer prior to dosing, and/or use of any multidrug and toxin extrusion (MATE) substrates taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing * Concurrent or previous administration of nusinersen or onasemnogene abeparvovec * Clinically significant abnormalities in laboratory test

Inclusion Criteria

Exclusion Criteria

* Male or female newborn infant aged \<20 days at first dose
* Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing.
* Gestational age equal to or greater than 37 weeks
* Receiving adequate nutrition and hydration at the time of screening
* Adequately recovered from any acute illness at baseline and considered well enough to participate in the study
* Parent/caregiver is willing to consider nasogastric, nasojejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator.

* Presence of clinical symptoms or signs consistent with SMA Type 0
* In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures
* Systolic blood pressure or diastolic blood pressure or heart rate abnormalities
* Presence of clinically relevant electrocardiogram (ECG) abnormalities
* The infant (or the person breastfeeding the infant) taking any of the following: any inhibitor of CYP3A4 taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing, any inducer of CYP3A4 taken within 4 weeks (or within 5 times the elimination half-life, whichever is longer prior to dosing, and/or use of any multidrug and toxin extrusion (MATE) substrates taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing
* Concurrent or previous administration of nusinersen or onasemnogene abeparvovec
* Clinically significant abnormalities in laboratory test

Contacts and Locations

Study Contact

Reference Study ID Number: BN44619 https://forpatients.roche.com/

CONTACT

888-662-6728 (U.S. Only)

global-roche-genentech-trials@gene.com

Principal Investigator

Clinical Trials

STUDY_DIRECTOR

Hoffmann-La Roche

Study Locations (Sites)

Ann and Robert H. Lurie Children Hospital of Chicago

Chicago, Illinois, 60611

United States

University Of Michigan; Pediatric Nephrology

Ann Arbor, Michigan, 48109

United States

Clinic for Special Children.

Gordonville, Pennsylvania, 17529

United States

Collaborators and Investigators

Sponsor: Hoffmann-La Roche

Clinical Trials, STUDY_DIRECTOR, Hoffmann-La Roche

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-04-26

Study Completion Date2025-08-31

Study Record Updates

Study Start Date2024-04-26

Study Completion Date2025-08-31

Terms related to this study

Additional Relevant MeSH Terms

Muscular Atrophy, Spinal