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New treatment option for Spinal Muscular Atrophy
Official title A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
ClinicalTrials.gov ID: NCT07047144
What this study is testing
What is Apitegromab?
Apitegromab is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for spinal muscular atrophy.
Also referred to as SRK-015.
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 double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score \<55.
- Phase 2: a mid-size study of how well it works
- This is a blinded study.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 2
You may be able to join if
- Is \<2 years old at the time of the informed consent
- Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
- Has confirmed diagnosis of 5q autosomal recessive SMA
- Has confirmed presence of SMN2 gene copy(ies)
- Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved...
You likely can't join if
- Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are...
- Major orthopedic issues such as severe scoliosis or severe contractures or treatment procedure, including spine or hip surgery, which is considered...
- Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome...
See the full eligibility criteria
- Is \<2 years old at the time of the informed consent
- Had a gestational age of ≥35 weeks and gestational body weight ≥2.0 kg at birth
- Has confirmed diagnosis of 5q autosomal recessive SMA
- Has confirmed presence of SMN2 gene copy(ies)
- Must have been treated with an approved SMN1-targeted therapy (ie, onasemnogene abeparvovec-xioi) or are continuing to be treated with an approved SMN2-targeted therapy (ie, nusinersen or risdiplam)
- Body weight for age is no less than 1st percentile based on the WHO Child Growth Standards at the Screening Visit
- Has delayed motor milestones for age attributed to SMA at the discretion of the Investigator or a CHOP-INTEND score \<55
- Nutritional status that is not anticipated to be stable throughout the study or medical necessity for a gastric feeding tube, where most feeds are administered by this route
- Major orthopedic issues such as severe scoliosis or severe contractures or treatment procedure, including spine or hip surgery, which is considered to have the potential to substantially limit the ability of the subject...
- Any other physical limitations (eg, the subject requires cast for contractures) that would prevent the subject from undergoing motor function outcome measures throughout the study.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Orange, California, United States
- Palo Alto, California, United States
- Aurora, Colorado, United States
- Atlanta, Georgia, United States
- Iowa City, Iowa, United States
- Grand Rapids, Michigan, United States
- Winston-Salem, North Carolina, United States
- Philadelphia, Pennsylvania, United States
- Memphis, Tennessee, United States
- Dallas, Texas, United States
- Flower Mound, Texas, United States
- Fort Worth, Texas, United States
- Houston, Texas, United States
- Ghent, East-Flanders, Belgium
- Leuven, Belgium
- Liège, Belgium
- Paris, France
- Milan, Italy
- Rome, Italy
+ 4 more site(s).
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, up to 2 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Orange, California, United States; Palo Alto, California, United States; Aurora, Colorado, United States; Atlanta, Georgia, United States; Iowa City, Iowa, United States and 18 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.