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Tests treatment safety and results for Muscular Atrophy, Spinal
Official title A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
ClinicalTrials.gov ID: NCT05861999
What this study is testing
What is Risdiplam?
Risdiplam is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for muscular atrophy, spinal.
Also referred to as RO7034067.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
- Phase 4: studies an already-approved treatment
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 24
You may be able to join if
- \<2 years of age at the time of informed consent
- Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of...
- Confirmed presence of two SMN2 gene copies as documented through laboratory testing
- Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
- Has received onasemnogene abeparvovec for SMA no less than 13 weeks prior to enrollment
You likely can't join if
- Previous or current enrolment in investigational study prior to initiation of study treatment
- Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information
- Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide
- Concomitant or previous use of an anti-myostatin agent
- Participants requiring invasive ventilation or tracheostomy
- Presence of feeding tube and an OrSAT score of 0
See the full eligibility criteria
- \<2 years of age at the time of informed consent
- Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the Survival of Motor Neuron 1 (SMN1) gene
- Confirmed presence of two SMN2 gene copies as documented through laboratory testing
- Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
- Has received onasemnogene abeparvovec for SMA no less than 13 weeks prior to enrollment
- If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration.
- In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows...
- Previous or current enrolment in investigational study prior to initiation of study treatment
- Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information
- Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide
- Concomitant or previous use of an anti-myostatin agent
- Participants requiring invasive ventilation or tracheostomy
- Presence of feeding tube and an OrSAT score of 0
- Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening
- Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration.
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Madera, California, United States
- Palo Alto, California, United States
- Aurora, Colorado, United States
- Gainesville, Florida, United States
- Atlanta, Georgia, United States
- Grand Rapids, Michigan, United States
- Philadelphia, Pennsylvania, United States
- Dallas, Texas, United States
- Norfolk, Virginia, United States
- Berlin, Germany
- Giessen, Germany
- Beersheba, Israel
- Petah Tikva, Israel
- Tel Aviv, Israel
- Gda?sk, Poland
- Warsaw, Poland
- Al Rayyan, Qatar
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 months to 24 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Madera, California, United States; Palo Alto, California, United States; Aurora, Colorado, United States; Gainesville, Florida, United States; Atlanta, Georgia, United States and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.