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RAD001 for Hamartoma Syndrome

Phase 1 & 2
Waitlist Available
Led By Mustafa Sahin, MD, PhD
Research Sponsored by Boston Children's Hospital
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 6 months
Awards & highlights

Study Summary

This study is evaluating whether a drug called RAD001 can improve brain function in people with a genetic mutation that causes intellectual disability.

Eligible Conditions
  • PTEN Syndrome
  • Hamartoma Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~6 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Secondary outcome measures
Change in Attention at 6 Months
Change in Communication Ability at 6 Months
Change in Fine Motor Skills at 6 Months
+5 more

Side effects data

From 2018 Phase 3 trial • 106 Patients • NCT01544491
30%
Diarrhoea
28%
Urinary tract infection
20%
Pyrexia
20%
Headache
20%
Anaemia
19%
Neutropenia
19%
Cough
15%
Vomiting
15%
BK virus infection
15%
Tonsillitis
11%
Hypertension
11%
Nasopharyngitis
11%
Leukopenia
11%
Abdominal pain
11%
Upper respiratory tract infection
11%
Blood creatinine increased
9%
Ear infection
9%
Gastroenteritis
9%
Ear pain
9%
Abdominal pain upper
9%
Rhinitis
9%
Weight decreased
7%
Hypomagnesaemia
7%
Pruritus
7%
Nausea
7%
Bronchitis
7%
Vitamin D deficiency
7%
Tremor
6%
Cytomegalovirus viraemia
6%
Cytomegalovirus infection
6%
Pain
6%
Oral herpes
6%
Acidosis
6%
Obesity
6%
Skin papilloma
6%
Sinusitis
6%
Haematuria
6%
Oropharyngeal pain
4%
Epstein-Barr virus infection
4%
Rash
4%
Epstein-Barr viraemia
4%
Hypertriglyceridaemia
4%
Constipation
4%
Mouth ulceration
4%
Fatigue
4%
Weight increased
4%
Proteinuria
2%
Hypokalaemia
2%
Aphthous ulcer
2%
Cystitis
2%
Influenza
2%
Otitis media
2%
Pharyngitis
2%
Hepatic enzyme increased
2%
Pain in extremity
100%
80%
60%
40%
20%
0%
Study treatment Arm
MMF+sTAC
EVR+rTAC

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: RAD001Experimental Treatment1 Intervention
RAD001 is formulated as tablets of 5.0 mg or 2.5mg strength, blister-packed under aluminum foil in units of 10 tablets and dosed on a regular basis.
Group II: PlaceboPlacebo Group1 Intervention
Matching placebo will be provided as a matching tablet and will also be blister packed under aluminum foil in units of 10.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
RAD001
2012
Completed Phase 3
~1570

Find a Location

Who is running the clinical trial?

Office of Rare Diseases (ORD)NIH
43 Previous Clinical Trials
12,283 Total Patients Enrolled
National Center for Advancing Translational Sciences (NCATS)NIH
329 Previous Clinical Trials
402,108 Total Patients Enrolled
PTEN ResearchUNKNOWN
1 Previous Clinical Trials
10 Total Patients Enrolled
~6 spots leftby Jul 2025
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