8/27/2016
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`Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM - Full Text View - Clinica|Tria|s.gov
`
`Clin icalTrials.gov
`A service of the U.S. National Institutes of Health
`
`Trial record 1 of 1 for: NCTOO457808
`
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`Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
`
`This study has been completed.
`
`Sponsor:
`Children's Hospital Medical Center, Cincinnati
`Collaborators:
`The LAM Foundation
`Tuberous Sclerosis Alliance
`
`Information provided by:
`Children's Hospital Medical Center, Cincinnati
`
`C|inicaITrials.gov Identifier:
`NCTOO457808
`
`First received: April 6. 2007
`Last updated: NA
`Last verified: April 2007
`History: No changes posted
`
`Full Text View
`
`Tabular View
`
`No Study Results Posted
`
`Disclaimer
`
`How to Read a Study Record
`
`F Purpose
`
`The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
`
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`
`Tuberous Sclerosis
`Lymphangioleiomyomatosis
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`Drug: Rapamycin, sirolimus
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`Phase 2
`
`lnterventional
`Study Type:
`Study Design: Allocation: Non-Randomized
`Endpoint Classification: Safety/Efficacy Study
`Intervention Model: Single Group Assignment
`Masking: Open Label
`Primary Purpose: Treatment
`
`Official Title:
`
`Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic
`Lymphangioleiomyomatosis
`
`Resource links provided by NLM:
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`Further study details as provided by Children's Hospital Medical Center, Cincinnati:
`
`Primary Outcome Measures:
`- Volume of Angiomyolipomata
`
`25
`Estimated Enrollment:
`December 2002
`Study Start Date:
`Study Completion Date: March 2006
`
`https://clinicaltrials.gov/ct2Ishow/N CT00457808?term= NCT00457808&rank=1
`
`Par Pharm., Inc.
`Exhibit 1095
`
`Par Pharm., Inc. v. Novartis AG
`Case |PR2016-00084
`
`Ex. 1095-0001
`
`

`
`Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM - Full Text View - Clinica|Tria|s.gov
`
`8/27/2016
`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 baseIine,6 or 9 month, 12 and 24 month visits, while simple spirometry
`only was performed at all other visits.
`
`P Eligibility
`
`Ages Eligible for Study:
`Genders Eligible for Study:
`Accepts Healthy Volunteers:
`Criteria
`
`Inclusion Criteria:
`
`18 Years to 65 Years
`Both
`No
`
`(Adult)
`
`- 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
`Exclusion Criteria:
`
`- Use of continuous supplemental oxygen
`- Concurrent infection
`
`- Reoentsurgery
`- Ongoing or planned pregnancy
`- Lactation
`
`- Use of an investigational drug within the last 30 days of study entrance
`
`F Contacts and Locations
`
`Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a
`study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general
`information, see Learn About Clinical Studies.
`
`Please refer to this study by its CIinica|Tria|s.gov identifier: NCTOO457808
`
`Locations
`
`United States, Ohio
`
`Cincinnati Childrens Hospital Medical Center
`Cincinnati, Ohio, United States, 45229-3039
`
`Sponsors and Collaborators
`
`Children's Hospital Medical Center, Cincinnati
`The LAM Foundation
`
`Tuberous Sclerosis Alliance
`
`Investigators
`
`Principal Investigator:
`
`John Bissler, MD Cincinnati Childrens Hospital Medical Center
`
`F More Information
`
`Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
`
`Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN.
`
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`
`C|inica|Tria|s.gov Identifier:
`Other Study ID Numbers:
`
`t
`CCHMC IRB # 02-10-16
`
`https://clinicaItriaIs.gov/ct2Ishow/N CT00457808?term= NCT00457808&rank=1
`
`2/3
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`Ex. 1095-0002
`
`Ex. 1095-0002
`
`

`
`Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM - Full Text \fiew - C|inicalTria|s.gov
`8/27/2016
`Study First Received:
`April 6, 2007
`Last Updated:
`April 6, 2007
`Health Authority:
`United States: Food and Drug Administration
`
`Keywords provided by Children's Hospital Medical Center, Cincinnati:
`TSC and LAM
`Tuberous Sclerosis Complex and sporadic LAM
`
`Additional relevant MeSH terms:
`Sclerosis
`Tuberous Sclerosis
`Lymphangioleiomyomatosis
`Pathologic Processes
`Hamartoma
`Neoplasms
`Neoplasms, Multiple Primary
`Neoplastic Syndromes, Hereditary
`Malformations of Cortical Development, Group I
`Malformations of Cortical Development
`Nervous System Malformations
`Nervous System Diseases
`Neurocutaneous Syndromes
`Heredodegenerative Disorders, Nervous System
`Neurodegenerative Diseases
`
`ClinicalTria|s.gov processed this record on August 25, 2016
`
`Congenital Abnormalities
`Genetic Diseases, lnborn
`Lymphangiomyoma
`Lymphatic Vessel Tumors
`Neoplasms by Histologic Type
`Perivascular Epithelioid Cell Neoplasms
`Neoplasms, Connective and Soft Tissue
`Lymphoproliferative Disorders
`Lymphatic Diseases
`lmmunoproliferative Disorders
`Immune System Diseases
`Sirolimus
`Everolimus
`Anti-Bacterial Agents
`Anti-infective Agents
`
`https1/clinica|tria|s.gov/ct2/show/N CT00457808?term=NCT004578D8&rank=1
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`3/3
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`Ex. 1095-0003
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`Ex. 1095-0003