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mTOR inhibitor

Arm 1 for Tuberous Sclerosis

Phase 2
Waitlist Available
Research Sponsored by Aucta Pharmaceuticals, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 12
Awards & highlights

Study Summary

This trial is testing a possible treatment for a skin condition called angiofibromas, which is caused by a genetic disorder called tuberous sclerosis complex.

Eligible Conditions
  • Tuberous Sclerosis
  • Facial Angiofibromas

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 12
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 12 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
The proportion of subjects with a clinical response of treatment success.
Secondary outcome measures
Overall response of angiofibroma assessed by the IRC at Week 12 compared to baseline based on Modified Nobel Scoring System
Overall response of angiofibroma assessed by the investigator at Week 12 as compared to baseline based on Modified Nobel Scoring System
The proportion of subjects with an investigator assessed IGA score of clear or almost clear with at least a 2-grade improvement on the Week 12 IGA of the facial skin lesions
+8 more

Trial Design

3Treatment groups
Active Control
Placebo Group
Group I: Arm 1Active Control1 Intervention
Sirolimus 0.2% ointment applied topically hs x 12 weeks
Group II: Arm 2Active Control1 Intervention
Sirolimus 0.4% ointment applied topically hs x 12 weeks
Group III: Arm 3Placebo Group1 Intervention
Placebo ointment applied topically hs x 12 weeks

Find a Location

Who is running the clinical trial?

Aucta Pharmaceuticals, IncLead Sponsor
1 Previous Clinical Trials
40 Total Patients Enrolled
Shoufeng Li, Ph.DStudy DirectorAucta Pharmaceuticals, Inc

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who meets the eligibility criteria to participate in this research project?

"This clinical study seeks 45 participants with adenoma sebaceum aged between 2 and 21. It is essential that applicants have 3 or more facial angiofibromas of grade ≥2 colour for each lesion, agree to abstain from intercourse or use an accepted form of birth control if their partner does not get a vasectomy, be in good health based on medical history/physical exam results, and are willing/able to comply with trial requirements."

Answered by AI

What past research has been conducted on the first arm of this experiment?

"Presently, Arm 1 has 125 ongoing studies with 13 of them in the third phase. Of these trials some are occurring within Cincinnati, Ohio while 1074 sites across the globe host clinical trials for this arm."

Answered by AI

What safety precautions have been taken with respect to participants in Arm 1?

"We rate Arm 1's safety at 2, as Phase 2 trials have generated some data affirming security without the same level of evidence for efficacy."

Answered by AI

What is the intake capacity for this research initiative?

"The prerequisite number of eligible participants needed to commence the trial is 45. Aucta Pharmaceuticals, Inc will be managing recruitment and administration from multiple sites including Children's Hospital of Los Angeles' Division of Neurology in LA and Boston Children's Hospital in Massachusetts."

Answered by AI

Which ailments are typically targeted with Arm 1 treatments?

"Rejection of a transplanted liver is typically handled with Arm 1, which is also suitable for treating disease, kidney issues and renal angiomyolipomas."

Answered by AI

Is the current experiment open to individuals aged 55 and above?

"Per the rules of this medical experiment, participants must be no older than 21 years old and at least 2 years old."

Answered by AI

Are there vacancies for prospective participants in this clinical experiment?

"According to clinicaltrials.gov, this research is currently recruiting participants with the initial posting date being April 12th 2017 and modifications made on February 2nd 2022."

Answered by AI
Recent research and studies
~3 spots leftby Apr 2025