OAV101 for Atrophy

Phase-Based Progress Estimates
Novartis Investigative Site, Randwick, Australia
Atrophy+3 More
OAV101 - Genetic
< 18
All Sexes
Eligible conditions

Study Summary

This study is evaluating whether a drug called AVXS-101 can improve the health of people with spinal muscular atrophy.

See full description

Eligible Conditions

  • Atrophy
  • Spinal Muscular Atrophy (SMA)

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Other trials for Atrophy

Study Objectives

This trial is evaluating whether OAV101 will improve 7 primary outcomes and 3 secondary outcomes in patients with Atrophy. Measurement will happen over the course of 12 months.

12 months
Achievement of development motor milestones according to the modified and combined WHO-MGRS and Bayley scale of Infant and Toddler Development.
Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE), as appropriate according to participant age
Change from baseline in Revised Upper Limb Module (RULM), as appropriate according to participant age.
Change from baseline in vital signs measurements - oxygen saturation level
Change from baseline in vital signs measurements - pulse
Change from baseline in vital signs measurements - respiratory rate
Change from baseline in vital signs measurements - systolic and diastolic blood pressure
Change from baseline in vital signs measurements - temperature
Number of participants with important identified and important potential risks (Adverse Events of Special Interest (AESI))
Number of participants with treatment emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Other trials for Atrophy

Trial Design

1 Treatment Group

1 of 1
Experimental Treatment

This trial requires 24 total participants across 1 different treatment group

This trial involves a single treatment. OAV101 is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

Participants will receive a single IV dose administration of OAV101

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 12 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 12 months for reporting.

Closest Location

Novartis Investigative Site - Boston, MA

Eligibility Criteria

This trial is for patients born any sex aged 18 and younger. There are 9 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
The test is used to diagnose SMA in someone who has symptoms of the disease, and it involves looking for gene mutations in the SMN1 and SMN2 genes. show original
for an illness without medical consultation People who discontinue an approved drug treatment without first consulting a doctor may be naive to the treatment or have a medical condition that requires a different treatment. show original
products Previous use of any AAV9 gene therapy products is not a contraindication to treatment with AAV101. show original
The child has a BMI that is lower than the 3rd percentile when compared to the World Health Organization's Child Growth Standard. show original
People who have a history of aspiration pneumonia or any signs of aspiration within 4 weeks prior to screening are not eligible to participate in the study. show original
The subject has an anti-AAV9 antibody titer of greater than 1:50, as determined by a ligand binding immunoassay at the time of screening. show original
The history of gene therapy, hematopoietic transplantation, or solid organ transplantation is full of both successes and failures show original
The weight of the baby must be between 8.5 kg and 21 kg at the time of screening visit number 2. show original

Patient Q&A Section

Does muscular atrophy run in families?

"Muscular atrophy is an inherited condition. All families with muscular atrophy are related to a common ancestor, and the affected gene is localised to chromosome 12q21.1." - Anonymous Online Contributor

Unverified Answer

What causes muscular atrophy?

"Muscular atrophy is a nonspecific finding that may be observed when the biopsy shows muscle involvement with myxomatous degenerated muscle fibers containing sarcoplasmic reticulum accumulations but not fibrosis. Muscle-associated conditions do not cause muscular atrophy, nor do muscular abnormalities cause muscle wasting. This latter finding is the common artifact of a muscle biopsy showing myopathic change (i.e. muscle cells contain more than two fiber types)." - Anonymous Online Contributor

Unverified Answer

What are the signs of muscular atrophy?

"Symptoms of muscular atrophy include difficulty moving or lifting items, weakness in muscles, and lack of stamina. Patients with muscle atrophy have trouble moving things like the table they sat on, or picking up the plates on the plate above the microwave. These patients have a weakness in their arms, hands, and legs. They also have poor endurance. This can also present as poor stamina during sporting activity. These symptoms are visible only by physical inspection by an expert.\n" - Anonymous Online Contributor

Unverified Answer

What are common treatments for muscular atrophy?

"Very few treatments have been found to be effective for the treatment of muscular atrophy. No medications are available for the treatment of muscular atrophy. There is no proven medical or cosmetic value to performing surgery. Exercise is an alternative treatments for many cases of muscular atrophy, even if it cannot be proved to work to cure the condition of muscular atrophy.\n" - Anonymous Online Contributor

Unverified Answer

How many people get muscular atrophy a year in the United States?

"Around 11 million Americans have a diagnosis of muscular atrophy every year. The incidence of muscular atrophy increases with age and is highest in men 75 years and older." - Anonymous Online Contributor

Unverified Answer

Can muscular atrophy be cured?

"Even in the postoperative period, MAF patients who are overweight might have a significant risk of relapse after their operations. On the other hand, patients who are overweight or obese have no or only a slight risk of relapse postoperatively." - Anonymous Online Contributor

Unverified Answer

What is muscular atrophy?

"Muscular atrophy is a disease of old age in which muscle mass is diminished, muscle fibers are smaller, and the number of muscle fibers is diminished. It is the principal cause of muscle weakness of old age. It can lead to progressive muscle weakening that becomes progressively more severe as old age continues. It is the leading medical diagnosis in old age as the primary disease causing progressive muscular weakness. Muscular atrophy is present in many types of weakness as well as in chronic fatigue syndrome. There are several other diseases with muscular weakness as a feature." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in oav101 for therapeutic use?

"Currently there were about half of the oav101 clinical trials for therapeutic use. There are many new therapy options. Oav101 continues to be a reliable therapy for ovoidal benign tumours and benign paralyses. Ovine regenerative medicine continues to be an important adjunct to limb salvage surgery. Further clinical trials using oav101 in traumatic limb loss are planned. oav101 has been shown to have a regenerative effect on injured muscle and tendon. Oav101 remains a promising adjunct to surgical therapy in ovoidal benign tumours. Oav101 has been shown to be an effective in situ therapy for acute paralysis when surgically applied. Oav103 is also currently under development." - Anonymous Online Contributor

Unverified Answer

Has oav101 proven to be more effective than a placebo?

"An orally administered form of oav101 had proven to increase muscle volume and strength in an older population by a higher percentage than a placebo at one month post-treatment. The oav101-mediated effects were greater than those from the placebo. This is because of the increased size of both muscle fibers and muscle masses." - Anonymous Online Contributor

Unverified Answer

Does oav101 improve quality of life for those with muscular atrophy?

"O-O-Ave101 is effective in the treatment of muscular atrophy, resulting in significant improvements in both quality of life and fatigue. Further studies are warranted to determine the long-term impact of O-O-Ave101 treatment." - Anonymous Online Contributor

Unverified Answer

What is the latest research for muscular atrophy?

"Muscle atrophy is the term to describe those changes that result in skeletal muscle loss, particularly those changes in the skeletal muscle associated with a reduction in its growth potential. This occurs even in skeletal muscle with less satellite cell content (Type II fibers) due to the lack of satellite cell stimulation or re-growth after muscle injury or disuse. In this scenario, it is impossible to rehabilitate or to recover muscular strength. The use of growth factors or other methods of stimulating satellite cell proliferation and proliferation-stimulating factor may hopefully offer a new therapeutic option for the management of skeletal muscle atrophic disorders." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets muscular atrophy?

"On average, the time of diagnosis is 9.1 years later for men than women. In a cohort of all cases diagnosed from 2002 to 2006, the onset of muscular atrophy is relatively early and mostly affects the anterior compartment of the lower extremities and legs." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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