Tuberous SclerosisPhase 2NCT00457808
Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
A Phase 2 study testing Rapamycin, sirolimus in people with Tuberous Sclerosis and Lymphangioleiomyomatosis, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Tuberous Sclerosis, Lymphangioleiomyomatosis
- What is being tested?
- Rapamycin, sirolimus
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2002to Mar 2006
- Phase
- Phase 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
- Diagnosed with angiomyolipomas and have either Tuberous Sclerosis complex or Lymphangioleiomyomatosis
- Between the gaes of 18 and 65 years
- Competency to voluntarily consent
- Clinically definite diagnosis of tuberous sclerosis or S-LAM
- Adequate contraception
- At least one angiomyolipoma of 1 cm or greater in largest diameter
Who cannot join
- Use of continuous supplemental oxygen
- Concurrent infection
- Recent surgery
- Ongoing or planned pregnancy
- Lactation
- Use of an investigational drug within the last 30 days of study entrance
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229-3039
About this study
Official title: Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
Read the detailed description
The study design was an open label, phase I/II trial of sirolimus for one year followed by one year off therapy.Patients were seen at baseline,at two weeks to four weeks, and at 2,4,6,9,12,18 and 24 months. Angiomyolipomata imaging was performed at all but the two to four week visit. Complete pulmonary function tests and six-minute walk were obtained at baseline,6 or 9 month, 12 and 24 month visits, while simple spirometry only was performed at all other visits.
- Conditions
- Tuberous Sclerosis, Lymphangioleiomyomatosis
- Being tested
- Rapamycin, sirolimus
- Keywords
- TSC and LAM, Tuberous Sclerosis Complex and sporadic LAM
- Medical subject headings
- Tuberous Sclerosis, Lymphangioleiomyomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rapamycin, sirolimusDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Volume of Angiomyolipomata
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2007 (estimated)
- Study startDec 2002
- Study completionMar 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- The LAM Foundation, Tuberous Sclerosis Alliance
- Investigators
- John Bissler, MD, Principal investigator, Cincinnati Childrens Hospital Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00457808
- Sponsor's protocol ID
- CCHMC IRB # 02-10-16
- Other IDs
- R21CA103486
- Status last verified
- Apr 2007
- First submitted
- 6 Apr 2007
- Last updated
- 9 Apr 2007
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564.
PubMed 18184959
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
- ClinicalTrials.gov ID
- NCT00457808
- Acronym / sponsor ID
- CCHMC IRB # 02-10-16
- Conditions
- Tuberous Sclerosis, Lymphangioleiomyomatosis
- Keywords
- TSC and LAM, Tuberous Sclerosis Complex and sporadic LAM
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- The LAM Foundation, Tuberous Sclerosis Alliance
- Study start
- Dec 2002
- Study completion
- Mar 2006 (actual)
- First posted
- 9 Apr 2007 (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.
The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
Detailed description
The study design was an open label, phase I/II trial of sirolimus for one year followed by one year off therapy.Patients were seen at baseline,at two weeks to four weeks, and at 2,4,6,9,12,18 and 24 months. Angiomyolipomata imaging was performed at all but the two to four week visit. Complete pulmonary function tests and six-minute walk were obtained at baseline,6 or 9 month, 12 and 24 month visits, while simple spirometry only was performed at all other visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosed with angiomyolipomas and have either Tuberous Sclerosis complex or Lymphangioleiomyomatosis
- Between the gaes of 18 and 65 years
- Competency to voluntarily consent
- Clinically definite diagnosis of tuberous sclerosis or S-LAM
- Adequate contraception
Key exclusion criteria (6 listed)
- Use of continuous supplemental oxygen
- Concurrent infection
- Recent surgery
- Ongoing or planned pregnancy
- Lactation
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 |
|---|---|---|
| Rapamycin, sirolimus | Drug | – |
Primary outcome
Volume of Angiomyolipomata
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 9 Apr 2007.
- Derived from this study: PubMed 18184959Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564.
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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
Cincinnati Childrens Hospital Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Rapamycin, sirolimus in people with Tuberous Sclerosis and Lymphangioleiomyomatosis, 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 6 inclusion and 6 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 9 Apr 2007. 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 phase mean?
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?
Volume of Angiomyolipomata. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00457808, which also lists 1 linked publication.
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 9 Apr 2007.