Prednisolone for Infantile Spasms

Overseen By Angela L. Martinez

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: University of California, Los Angeles

Must be taking: Prednisolone, ACTH, Vigabatrin

Must not be taking: Artisanal cannabinoids

Disqualifiers: Hypertension, Infection, Ketogenic diet, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 4 months

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether low-dose prednisolone, a corticosteroid medication, can reduce the risk of relapse in children with infantile spasms, a condition that can lead to serious issues like autism or other types of epilepsy. Researchers aim to determine if this treatment is safe, manageable, and effective. The trial includes two groups: one receiving the actual treatment and another receiving a placebo, a non-active substance. Children diagnosed with infantile spasms who have already responded well to standard treatments are suitable candidates. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot be on a ketogenic diet or have recently used artisanal cannabinoid products. It's best to discuss your current medications with the study team.

Is there any evidence suggesting that low-dose prednisolone is likely to be safe for infants with spasms?

Research has shown that prednisolone is generally well-tolerated for treating infantile spasms. In past studies, prednisolone effectively stopped spasms in many children with this condition. Specifically, high doses of prednisolone worked well, with many infants becoming free from spasms within two weeks.

Regarding safety, prednisolone is considered a reliable option. Its established use in treating infantile spasms means its safety profile is well-known. While high doses are more common in research, this trial tests low-dose prednisolone, which might be even easier for infants to handle. Although specific information on side effects for low doses is not available in the sources, its use in other situations suggests it is safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Prednisolone is unique because it offers a potential alternative to the standard treatments for infantile spasms, which typically include medications like ACTH (adrenocorticotropic hormone) or vigabatrin. One of the key features of prednisolone is its corticosteroid action, which might provide a different mechanism of addressing the seizures associated with this condition. Researchers are excited about prednisolone because it could offer a more accessible and potentially cost-effective treatment option, with a different side effect profile compared to current therapies.

What evidence suggests that low-dose prednisolone might be an effective treatment for infantile spasms?

Research has shown that prednisolone can help treat infantile spasms. In one study, 63% of children had a complete response to prednisolone. Another study found that about 43.5% of cases responded fully to the treatment. Additionally, 41% of children taking oral steroids, such as prednisolone, experienced a lasting stop to spasms within two weeks. These studies suggest that prednisolone may lower the chance of spasms returning. Participants in this trial will receive either low-dose prednisolone or a placebo to further evaluate its effectiveness. ^{2 6 7 8 9}

Who Is on the Research Team?

Shaun A. Hussain, MD, MS

Principal Investigator

University of California, Los Angeles

Are You a Good Fit for This Trial?

This trial is for infants aged 2 to 18 months who have been diagnosed with infantile spasms syndrome and showed a complete response to standard treatment. It's not suitable for those outside this age range or without EEG-confirmed recovery.

Inclusion Criteria

My child is between 2 to 18 months old.

My infantile spasms responded completely to standard treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either low-dose prednisolone or placebo for 4 months

4 months

Monthly visits with clinic and EEG evaluations

Follow-up

Participants are monitored for safety and effectiveness after treatment, with a final evaluation at age 2 years

Up to age 2 years

Final visit at age 2 years

What Are the Treatments Tested in This Trial?

Interventions

• Prednisolone

Trial Overview The study tests if low-dose prednisolone can safely prevent relapse in children who've responded well to initial treatments for infantile spasms, compared with a placebo.

How Is the Trial Designed?

Group I: Low-dose prednisolone Active Control 2 Interventions

Prednisolone and famotidine.

Group II: Placebo Placebo Group 1 Intervention

Placebo (prednisolone) and placebo (famotidine)

Who Is Running the Clinical Trial?

University of California, Los Angeles

Lead Sponsor

Trials

1,594

Recruited

10,430,000+

Pediatric Epilepsy Research Foundation

Collaborator

Trials

2

Recruited

430+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3904676/

Treatment of infantile spasms with very high dose ... Sixty-three percent (17/27) of patients responded completely to prednisolone. Subsequently, 40% (4/10) of prednisolone non-responders exhibited a complete ...

2. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0887899421000096

Epilepsy Outcome at Four Years in a Randomized Clinical ... There was no difference between ACTH or prednisolone in four-year seizure outcome after infantile spasms. These data may help the clinicians decide between the ...

3. cureus.com cureus.com/articles/88299-comparison-of-efficacy-and-safety-of-low--versus-high-dose-oral-prednisolone-in-infantile-spasm-is-an-open-label-randomized-controlled-trial-at-the-childrens-hospital--institute-of-child-health-multan-pakistan

Versus High-Dose Oral Prednisolone in Infantile Spasm (IS ... In the present study, overall efficacy in terms of complete response of prednisolone was observed in 43.5% of the total cases. Complete spasm ...

4. neurology.org neurology.org/doi/10.1212/WNL.0000000000201232

Association of Time to Clinical Remission With Sustained ... Sustained resolution of infantile spasms to standard treatment by day 14 was observed in 48% (99/205) on ACTH, 41% (41/99) on oral steroids, and 34% (31/91) on ...

5. frontiersin.org frontiersin.org/journals/neurology/articles/10.3389/fneur.2021.756746/full

Safety and Effectiveness of Oral Methylprednisolone ... The short and intermediary-term efficacy and recurrence rates of oMP are not inferior to those of imACTH and oP for the treatment of IS.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9009990/

Versus High-Dose Oral Prednisolone in Infantile Spasm (IS) Hancock and Osborne found 71.4% infants having complete spasms freedom by the end of 14 days were on the high-dose prednisolone regimen [23].

7. sciencedirect.com sciencedirect.com/science/article/pii/S1090379822001726

Prednisolone or tetracosactide depot for infantile epileptic ... For Day 14–42 cessation of spasms, on tetracosactide depot, 41 of 61 (67%) were free of spasms compared to 35 of 62 (56%) on prednisolone (difference 11%, 95% ...

8. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/10.1111/dmcn.15153

Response to treatment and outcomes of infantile spasms in ... Prednisolone is an effective and well-tolerated medication for treating infantile spasms in Down syndrome. Despite the high percentage of spasm ...

9. aesnet.org aesnet.org/abstractslisting/outcome-and-challenges-in-treating-infantile-spasms-with-high-dose-oral-prednisolone

Abstracts - American Epilepsy Society OUTCOME AND CHALLENGES IN TREATING INFANTILE SPASMS WITH HIGH DOSE ORAL PREDNISOLONE. Abstract number : 1.151. Submission category : 4 ...