A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects With Multiple Osteochondromas

Status: Terminated
Location: See all (31) locations...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

This is a randomized, double-blind, placebo-controlled study comparing the safety and efficacy of 2 dosage regimens of palovarotene versus placebo in preventing disease progression in pediatric subjects with multiple osteochondromas (MO).

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 14
Healthy Volunteers: No
View:

• Written, signed, and dated informed subject/parent consent and age-appropriate assent (performed according to local regulations).

• A clinical diagnosis of MO with disease-causing exostosin 1 or 2 gene mutations.

• Male or female from 2 to 14 years of age.

• Female subjects must be premenarchal at screening.

• A bone age at screening of 14 years or less.

• Symptomatic MO, defined as five or more clinically evident osteochondromas and a new or enlarged osteochondroma that occurred in the preceding 12 months, five or more clinically evident osteochondromas and the presence of a painful osteochondroma, a skeletal deformity, a joint limitation, or prior surgery for a MO-related complication.

• The ability to undergo whole body MRI with or without sedation/general anesthesia.

• Use of two effective methods of birth control during treatment, and for 1 month after treatment discontinuation, unless committed to true abstinence from heterosexual sex. Sexually active females of child-bearing potential must also agree to start effective methods of birth control at screening.

Locations
United States
California
Children's Orthopaedic Center
Los Angeles
Shriners Hospital for Children - Sacramento
Sacramento
University of California-San Francisco
San Francisco
Washington, D.c.
Children's National Medical Center
Washington
Florida
The Paley Institute
West Palm Beach
Illinois
Shriners Hospital for Children - Chicago
Chicago
Massachusetts
Boston Children's Hospital
Boston
Maryland
Johns Hopkins University
Baltimore
Minnesota
Mayo Clinic - PPDS
Rochester
Oregon
Shriners Hospitals for Children - Portland
Portland
Pennsylvania
Shriners Hospital for Children - Philadelphia
Philadelphia
The Children's Hospital of Philadelphia (CHOP)
Philadelphia
Texas
Memorial Hermann Hospital
Houston
Other Locations
Australia
Westmead Children's Hospital
Westmead
Belgium
UZ Antwerpen
Edegem
Canada
Centre Hospitalier Universitaire Sainte-Justine
Montréal
Shriners Hospital for Children - Canada
Montréal
Hospital for Sick Children
Toronto
France
Hôpital universitaire Necker - Enfants Malades
Paris
Hôpital des Enfants, CHU de Toulouse
Toulouse
Italy
Istituti Ortopedici Rizzoli
Bologna
Japan
Nagoya University Hospital
Nagoya
Osaka University Hospital
Suita
Netherlands
OLVG locatie Oost
Amsterdam
Portugal
Hospital Pediátrico de Coimbra
Coimbra
Spain
Hospital Universitario La Paz
Madrid
Turkey
Ege University Medical Faculty Hospital
Bornova
Bezmialem Vakif University Medical Faculty Hospital
Istanbul
United Kingdom
Evelina London Children's Hospital
London
Royal Manchester Childrens Hospital
Manchester
Royal National Orthopaedic Hospital
Stanmore
Time Frame
Start Date: March 22, 2018
Completion Date: October 30, 2020
Participants
Target number of participants: 193
Treatments
Experimental: Palovarotene 2.5 mg daily regimen
Experimental: Palovarotene 5.0 mg daily regimen
Placebo Comparator: Placebo regimen
Authors
Assoc. Prof. Dr. Levent Kucuk, Pam Scott, Drod Paley, Brooke Nelson, Pr Jean Pierre Salles, Assoc. Prof. Gozde Yesil, Mathilde Debeurme, Luisa Diogo Matos, Dr. Hidetatsu Otani, Dilek Bayraktar, Sofia Fernandes, Paula Garcia, Dr. Satoshi Takenaka, Gaspar González Morán, Esra Işık, Dr. Kenichiro Hamada, Hiroto Takahashi, Jessica Tenney, Richard Goldberg, Ingrid Holm, Purnendu Gupta, Michael A Levine, Craig Robbins, Samantha Spencer, Robert Pignolo, Daniel J. Bouton, Bethany Lipa, Holly Leshikar, Jeffrey Ackman, Peter Smith, Dan Zlotolow, Edna Mancilla, Christina Jacobsen, Stephen Broski, Krister Freese, Vernon T Tolo, Julie Hoover-Fong, Shiraz Younas, Robert Henshaw, Haluk Altiok, Edward Hsiao, Scott Kozin, Alexander Meves, David Feldman, Amy Mori, Ann Reina, Ernest Conrad, Dr. Kiyoshi Yoshida, John Ham, Zilla Huma, Franck Accadbled, Valerie Porquet-Bordes, Valerie Cormier-Daire, Thomas Edouard, Janet Lee, Hiroyuki Saitou, Rachel Y Goldstein, Hüseyin Günay, Arnard van der Zwan, Michelle James, Özgür Mert Bakan, Fernando Santos Simarro, Andreas Zankl, Chantal Janelle, Cathleen E. Buckon, Jennifer Stimec, Irene Lara-Corrales, Noelle Larson, AeRang Kim, Susan Sienko, Craig Munns, Taichi Kitaoka, Philippe Campeau, Andac Celasun Alsina, Masaki Matsushita, Kenichi Mishima, Hiroshi Kitoh, Keiichi Ozono, Andrew Howard, Tadashi Nagata, Vermon T Tolo, Matthew Drake, Lucie Dupuis, Geert Mortier, Gokcer Uzer, Cristina Alves, Peter Kannu, Karen Heath, Luca Sangiorgi, Reggie Hamdy, Roberto Mendoza-Londono, Inês Balacó, Melita Irving, Sergio Sousa, Raja Padidela, Nara Sobreira, Tahir Atik, Ayca Dilruba Aslanger, Mars Skae, Genevieve Baujat
Sponsors
Leads: Clementia Pharmaceuticals Inc.

This content was sourced from clinicaltrials.gov

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