Tuberous SclerosisPhase 1/2NCT00792766
Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)
A Phase 1/2 study testing everolimus (RAD001) in people with Tuberous Sclerosis and Angiolipoma, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
This is an open label long term follow up study, open to those subjects who were previously enrolled in"RAD001 Therapy of Angiomyolipomata in Patients with Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis", CCHMC IRB #2008-0812 and who meet the criteria for this long-term follow-up study. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Tuberous Sclerosis, Angiolipoma
- What is being tested?
- everolimus (RAD001)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2008to Sep 2013
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects must have met all eligibility criteria for the initial RAD001 protocol (CCHMC IRB 2008-0812)
- Subjects with documented angiomyolipoma volume decrease from baseline measures at the end of 12 months on study drug of thirty percent or more during the initial RAD001 protocol OR subjects with less than thirty percent decrease in angiomyolipomas at the end of 12 months on study drug but with documented improvement, or stabilization, of baseline clinical status per physical, pulmonary function and/or laboratory examination at the end of 12 months on study drug that was not maintained during a period of 12 or more months off study drug.
- If female and of child-bearing potential, documentation of negative pregnancy test prior to start of study drug
- Creatinine <3 mg/dl, within 30 days prior to start of drug
Who cannot join
- Inability to complete the initial RAD001 protocol (CCHMC IRB # 2008-0812) due to toxicities requiring discontinuation of treatment.
- Demonstrated an increase in the size of the angiomyolipoma from baseline at the end of 12 months on study drug on the initial RAD001 study.
- Significant hematologic or hepatic abnormality (i.e. ALT and AST >2.5x ULN), serum albumin <3 g/dl, HCT <30%, platelets <75,000/cumm, adjusted absolute neutrophil count <1,000/cumm, total WBC <3,000/cumm).
- Continuous requirement for supplemental oxygen.
- Intercurrent infection at initiation of RAD001.
- Embolization of angiomyolipoma within one month; any other recent surgery (within 2 months of initiation of RAD001).
Show all 9Show fewer
- Pregnant or lactating women or women who plan on becoming pregnant during the course of this study due to unknown effects of RAD001 on the fetus.
- Inadequate contraception (participants who are fertile must maintain adequate contraception throughout the trial and for three months after stopping the drug). Acceptable contraceptive measures include non estrogen-containing birth control contraceptive regimen (progestin based contraceptives), prior hysterectomy, tubal ligation, complete abstinence, barrier methods which include a cervical diaphragm and spermicidal jelly, IUD, or vasectomy in partner.
- Use of an investigational drug, including rapamycin, within the last 30 days.
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
This is an open label long term follow up study, open to those subjects who were previously enrolled in"RAD001 Therapy of Angiomyolipomata in Patients with Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis", CCHMC IRB #2008-0812 and who meet the criteria for this long-term follow-up study.
The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly even cause regression.
Read the detailed description
Tuberous sclerosis complex is a genetic disorder with a birth incidence of approximately one in six thousand. Angiomyolipomas occur in eighty percent of persons with TS, and in up to 60% of persons with LAM. They are fatty tumors that often occur in kidney or liver as well as other parts of the body. They can grow and cause damage to surrounding kidney tissue and even renal failure. They may also leak blood causing potentially life-threatening hemorrhage.
Laboratory studies have shown that RAD001 suppresses the chemical that cause tumors to grow in tuberous sclerosis and sporadic LAM. The use of RAD001 to treat angiomyolipomas in tuberous sclerosis or sporadic LAM is considered experimental.
- Conditions
- Tuberous Sclerosis, Angiolipoma
- Being tested
- everolimus (RAD001)
- Keywords
- Tuberous Sclerosis Complex (TSC), Angiolipoma, mTOR, Everolimus
- Medical subject headings
- Tuberous Sclerosis, Angiolipoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
everolimus (RAD001)Drug
Also called RAD001
Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study.
| Study group | Role | What they receive |
|---|---|---|
| RAD001 | Experimental | Not described Receives: Drug: everolimus (RAD001) |
What is being measured
The primary outcome is the main question the study is designed to answer.
RAD001 tolerance
Measured: Every three months while on study drug
Secondary outcomes (1)
Angiomyolipoma volume reduction
Measured: Every year while on study drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2008 (estimated)
- Study startDec 2008
- Primary completionSep 2013
- Study completionSep 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous SclerosisStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Sponsor
- Investigators
- John Bissler, MD, Principal investigator, LeBonheur Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00792766
- Sponsor's protocol ID
- CCHMC IRB# 2008-0333
- Status last verified
- Sep 2013
- First submitted
- 17 Nov 2008
- Last updated
- 18 Sep 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
- ClinicalTrials.gov ID
- NCT00792766
- Acronym / sponsor ID
- CCHMC IRB# 2008-0333
- Conditions
- Tuberous Sclerosis, Angiolipoma
- Keywords
- Tuberous Sclerosis Complex (TSC), Angiolipoma, mTOR, Everolimus
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Dec 2008
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 18 Nov 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open label long term follow up study, open to those subjects who were previously enrolled in"RAD001 Therapy of Angiomyolipomata in Patients with Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis", CCHMC IRB #2008-0812 and who meet the criteria for this long-term follow-up study.
The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly even cause regression.
Detailed description
Tuberous sclerosis complex is a genetic disorder with a birth incidence of approximately one in six thousand. Angiomyolipomas occur in eighty percent of persons with TS, and in up to 60% of persons with LAM. They are fatty tumors that often occur in kidney or liver as well as other parts of the body. They can grow and cause damage to surrounding kidney tissue and even renal failure. They may also leak blood causing potentially life-threatening hemorrhage.
Laboratory studies have shown that RAD001 suppresses the chemical that cause tumors to grow in tuberous sclerosis and sporadic LAM. The use of RAD001 to treat angiomyolipomas in tuberous sclerosis or sporadic LAM is considered experimental.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RAD001 | Experimental | Drug: everolimus (RAD001) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Subjects must have met all eligibility criteria for the initial RAD001 protocol (CCHMC IRB 2008-0812)
- Subjects with documented angiomyolipoma volume decrease from baseline measures at the end of 12 months on study drug of thirty percent or more during the initial RAD001 protocol OR subjects with less than thirty percent decrease in angiomyolipomas at the end of 12 months on study drug but with documented improvement, or stabilization, of baseline clinical status per physical, pulmonary function and/or laboratory examination at the end of 12 months on study drug that was not maintained during a period of 12 or more months off study drug.
- If female and of child-bearing potential, documentation of negative pregnancy test prior to start of study drug
- Creatinine <3 mg/dl, within 30 days prior to start of drug
Key exclusion criteria (9 listed)
- Inability to complete the initial RAD001 protocol (CCHMC IRB # 2008-0812) due to toxicities requiring discontinuation of treatment.
- Demonstrated an increase in the size of the angiomyolipoma from baseline at the end of 12 months on study drug on the initial RAD001 study.
- Significant hematologic or hepatic abnormality (i.e. ALT and AST >2.5x ULN), serum albumin <3 g/dl, HCT <30%, platelets <75,000/cumm, adjusted absolute neutrophil count <1,000/cumm, total WBC <3,000/cumm).
- Continuous requirement for supplemental oxygen.
- Intercurrent infection at initiation of RAD001.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| everolimus (RAD001)Also: RAD001Subjects will resume the dosing regimen that they were receiving at the completion of the initial RAD001 study. | Drug | RAD001 |
Primary outcome
RAD001 tolerance
Time frame: Every three months while on study drug
1 secondary outcome
Angiomyolipoma volume reduction
Time frame: Every year while on study drug
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2013.
Related trials for Tuberous Sclerosis
RecruitingObservational study
Study of the Disease Process of Lymphangioleiomyomatosis
Lung Disease, Pneumothorax
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Active, not recruitingPhase 2
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Tuberous Sclerosis Complex, Epilepsy
RecruitingObservational study
Study of Skin Tumors in Tuberous Sclerosis
Tuberous Sclerosis
RecruitingPhase 2/3
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
Tuberous Sclerosis Complex
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Bissler, MD principal investigator
LeBonheur Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing everolimus (RAD001) in people with Tuberous Sclerosis and Angiolipoma, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Sep 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
RAD001 tolerance (time frame: Every three months while on study drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2013.