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TerminatedNCT03442985MO-PedUpdated Aug 1, 2022Results posted

An Efficacy and Safety Study of Palovarotene for the Treatment of MO

A Phase 2 interventional study of Palovarotene 2.5 mg and Palovarotene 5.0 mg in Exostoses, Multiple Hereditary, sponsored by Clementia Pharmaceuticals Inc.. Terminated at 31 sites in 12 countries. Open to participants aged 2 Years to 14 Years. Per ClinicalTrials.gov, last updated 2022-08-01.

Sponsored by Clementia Pharmaceuticals Inc. · Phase 2, Interventional, and Treatment

Why this study was terminated
Trial was terminated early to analyze the accumulated data and evaluate the efficacy, safety and future of palovarotene in MO.
Phase
Phase 2
Study type
Interventional
Enrollment
193
Allocation
Randomized
Ages
2 Years to 14 Years
Sex
All
01

Study 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).

Read the detailed description

Multiple osteochondromas is a rare condition where children develop multiple benign cartilage-capped bony tumors called osteochondromas on bones throughout the body, resulting in pain, deformity, limb length discrepancy, disability, and eventually arthritis and possible malignancy. The primary objective is to compare the efficacy of two dosage regimens of palovarotene with placebo to prevent the formation of new osteochondromas in pediatric MO subjects with exostosin 1 or exostosin 2 gene mutations. Osteochondroma formation was assessed by whole body magnetic resonance imaging (MRI). Secondary efficacy objectives were to compare the effects of palovarotene with placebo on the volume of osteochondromas as assessed by MRI; the proportion of subjects with no new osteochondromas as assessed by whole-body MRI; the annualized rate of new or worsening deformities; the annualized rate of MO-related surgeries; and palatability. The overall safety and pharmacokinetics of palovarotene and the effects of palovarotene on linear growth, bone growth plates, bone mineral density, quality of life, and pain due to osteochondromas was also studied.

02

Conditions studied

  • Exostoses, Multiple Hereditary

Keywords

  • Multiple osteochondromas
  • Osteochondroma
  • Palovarotene
  • Hereditary multiple exostoses
  • HME
  • MO
  • Retinoic acid receptor gamma agonist
  • Retinoic acid receptor agonist
03

Who can participate

Ages eligible
2 Years to 14 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • 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.

Key Exclusion Criteria:

  • Weight under 10 kg.
  • Other syndromic conditions such as Langer-Giedion or Potocki-Shaffer.
  • Any subject with neurologic signs suggestive of spinal cord impingement.
  • Concomitant medications that are strong inhibitors or inducers of cytochrome P450 3A4 activity.
  • Amylase or lipase >2 times the above the upper limit of normal (>2×ULN) or with a history of chronic pancreatitis.
  • Elevated aspartate aminotransferase or alanine aminotransferase above 2.5×ULN.
  • Any surgical implant that is contraindicated for MRI.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
193 participants (actual)

Study arms

  • Experimental
    Palovarotene 2.5 mg daily regimen

    Drug: Palovarotene 2.5 mg

  • Experimental
    Palovarotene 5.0 mg daily regimen

    Drug: Palovarotene 5.0 mg

  • Placebo comparator
    Placebo regimen

    Other: Placebo

Interventions

  • DrugPalovarotene 2.5 mg

    Subjects received a weight-adjusted dose equivalent of 2.5 mg palovarotene, once daily, for up to 24 months.

  • DrugPalovarotene 5.0 mg

    Subjects received a weight-adjusted dose equivalent of 5.0 mg palovarotene, once daily, for up to 24 months.

  • OtherPlacebo

    Subjects received placebo, once daily, for up to 24 months.

05

What researchers measure

Primary outcomes

  1. Annualized Rate of New Osteochondromas (OCs)

    The annualized rate of new OCs was assessed by whole-body magnetic resonance imaging (MRI) (that is, the total number of new OCs divided by the time in years between the baseline and latest post-baseline MRI).

    Time frame: Month 12

Secondary outcomes

  1. Mean Change From Baseline in the Total Volume of New OCs at Month 12

    The change from baseline in the total volume of OCs was assessed by whole-body MRI. Baseline was defined as the last available value prior to first administration of study drug.

    Time frame: Baseline (Day 1) and Month 12

  2. Percentage of Participants With No New OCs

    The percentage of participants with no new OCs as assessed by whole-body MRI. Participants with new OCs not identified by MRI due to surgical resection during the treatment period were categorized as having new OCs for this analysis.

    Time frame: Month 12

  3. Annualized Rate of New or Worsening Deformities

    The annualized rate of new or worsening deformities as assessed by radiographic imaging of both upper and lower limbs.

    Time frame: Month 12

  4. Annualized Rate of MO-Related Surgeries

    The MO-related surgeries included any procedure indicated for the treatment of MO, such as an excision of a symptomatic OC or correction of a limb deformity.

    Time frame: Month 12

  5. Maximum Observed Plasma Drug Concentrations at Steady State (Cmax,ss) of Palovarotene

    The Cmax,ss of palovarotene was evaluated. The pharmacokinetic (PK) sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

    Time frame: Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose

  6. Minimum Observed Plasma Drug Concentrations at Steady State (Cmin,ss) of Palovarotene

    The Cmin,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

    Time frame: Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose

  7. Time to Maximum Observed Drug Concentration at Steady State (Tmax,ss) of Palovarotene

    The Tmax,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

    Time frame: Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose

  8. Area Under the Plasma Concentration-Time Curve at Steady State From Time 0 to 24 Hours After Dosing (AUC0-24,ss) of Palovarotene

    The AUC0-24,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

    Time frame: Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose

  9. Number of Participants With Palatability of Sprinkled Palovarotene and Placebo

    Palatability of palovarotene and placebo when sprinkled on specific foods as assessed with a 5-point hedonic face scale at the first dose (Day 1) and at Month 1 in all participants (including \<4 years old) who sprinkled the palovarotene or placebo onto a spoonful of specific foods. The hedonic face scale ranges from 1 to 5 where, 1= dislike very much, 2= dislike slightly, 3= neither like nor dislike, 4= like slightly, 5= like very much. Higher scores indicate positive outcome.

    Time frame: Day 1 and Month 1

06

Results

Posted Aug 1, 2022
Limitations and caveats
The Sponsor terminated the study early due to a partial clinical hold instituted by the Food and Drug Administration. Recruitment was stopped before full enrollment was reached, and study drug administration was discontinued.

Participant flow

This Phase 2 placebo-controlled study was conducted in pediatric participants with multiple osteochondromas (MO) at 29 study sites in 11 countries between 22 March 2018 and 30 October 2020. For sites in the European Union, participants from 7 to \<15 years of age were enrolled first and participants from 2 to \<7 years of age were enrolled after the 6-month bone safety data from at least 20 skeletally immature participants.

Participant flow — Overall Study
MilestonePlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Started626665
Completed000
Not completed626665
Withdrew: Sponsor request556054
Withdrew: Lost to follow-up527
Withdrew: Withdrawal by subject234
Withdrew: Adverse event010

Outcome measures

PrimaryAnnualized Rate of New Osteochondromas (OCs)

The annualized rate of new OCs was assessed by whole-body magnetic resonance imaging (MRI) (that is, the total number of new OCs divided by the time in years between the baseline and latest post-baseline MRI).

Time frame:
Month 12
Reported as:
Least squares mean · number of new OCs per year
Annualized Rate of New Osteochondromas (OCs)
number of new OCs per yearPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Annualized Rate of New Osteochondromas (OCs)0.119 (0.031 to 0.461)0.363 (0.148 to 0.888)0.172 (0.073 to 0.404)
Statistical analysis
  • Palovarotene 2.5 mg vs Palovarotene 5.0 mg · Negative binomial regression model · p = 0.2556 (The p-values were not adjusted for multiple testing due to small sample size.) · Risk ratio (rr): 2.109 · 95% CI 0.583 to 7.638
  • Placebo vs Palovarotene 2.5 mg · Negative binomial regression model · p = 0.1788 (The p-values were not adjusted for multiple testing due to small sample size.) · Risk ratio (rr): 3.040 · 95% CI 0.601 to 15.373
  • Placebo vs Palovarotene 5.0 mg · Negative binomial regression model · p = 0.6570 (The p-values were not adjusted for multiple testing due to small sample size.) · Risk ratio (rr): 1.441 · 95% CI 0.287 to 7.234
SecondaryMean Change From Baseline in the Total Volume of New OCs at Month 12

The change from baseline in the total volume of OCs was assessed by whole-body MRI. Baseline was defined as the last available value prior to first administration of study drug.

Time frame:
Baseline (Day 1) and Month 12
Reported as:
Mean · cubic millimeter
Mean Change From Baseline in the Total Volume of New OCs at Month 12
cubic millimeterPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Mean Change From Baseline in the Total Volume of New OCs at Month 1210476.7 ± 23294.95250.5 ± 11754.710911.0 ± 35869.6
Statistical analysis
  • Palovarotene 2.5 mg vs Palovarotene 5.0 mg · Unadjusted estimation equation model · p = 0.4252 (The p-values were not adjusted for multiple testing due to small sample size.) · Risk ratio (rr): -4412.6 · 95% CI -15257.3 to 6432.1
  • Placebo vs Palovarotene 2.5 mg · Unadjusted estimation equation model · p = 0.4053 (The p-values were not adjusted for multiple testing due to small sample size.) · Risk ratio (rr): -4640.9 · 95% CI -15570.8 to 6289.0
  • Placebo vs Palovarotene 5.0 mg · Unadjusted estimation equation model · p = 0.9677 (The p-values were not adjusted for multiple testings due to small sample size.) · Risk ratio (rr): -228.3 · 95% CI -11265.0 to 10808.4
SecondaryPercentage of Participants With No New OCs

The percentage of participants with no new OCs as assessed by whole-body MRI. Participants with new OCs not identified by MRI due to surgical resection during the treatment period were categorized as having new OCs for this analysis.

Time frame:
Month 12
Reported as:
Number · percentage of participants
Percentage of Participants With No New OCs
percentage of participantsPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Percentage of Participants With No New OCs62.547.156.5
Statistical analysis
  • Palovarotene 2.5 mg vs Palovarotene 5.0 mg · Regression, Logistic · p = 0.5025 · Odds ratio (or): 0.643 · 95% CI 0.177 to 2.335Logistic regression was adjusted for the following covariates: baseline age, sex, and Ext1/2 mutation status.
  • Placebo vs Palovarotene 2.5 mg · Regression, Logistic · p = 0.3763 · Odds ratio (or): 0.528 · 95% CI 0.128 to 2.175Logistic regression was adjusted for the following covariates: baseline age, sex, and Ext1/2 mutation status.
  • Placebo vs Palovarotene 5.0 mg · Regression, Logistic · p = 0.7714 · Odds ratio (or): 0.820 · 95% CI 0.215 to 3.123Logistic regression was adjusted for the following covariates: baseline age, sex, and Ext1/2 mutation status.
SecondaryAnnualized Rate of New or Worsening Deformities

The annualized rate of new or worsening deformities as assessed by radiographic imaging of both upper and lower limbs.

Time frame:
Month 12
Reported as:
Least squares mean · number of deformities per year
Annualized Rate of New or Worsening Deformities
number of deformities per yearPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Annualized Rate of New or Worsening Deformities1.797 (1.272 to 2.537)1.802 (1.209 to 2.684)1.895 (1.584 to 2.266)
Statistical analysis
  • Palovarotene 2.5 mg vs Palovarotene 5.0 mg · Negative binomial regression model · p = 0.8155 (The p-values were not adjusted for multiple testing due to small sample size. The correlation matrix type = compound symmetry was used.) · Risk ratio (rr): 0.951 · 95% CI 0.623 to 1.451
  • Placebo vs Palovarotene 2.5 mg · Negative binomial regression model · p = 0.9918 (The p-values were not adjusted for multiple testing due to small sample size. The correlation matrix type = compound symmetry was used.) · Risk ratio (rr): 1.003 · 95% CI 0.592 to 1.699
  • Placebo vs Palovarotene 5.0 mg · Negative binomial regression model · p = 0.7997 (The p-values were not adjusted for multiple testing due to small sample size. The correlation matrix type = compound symmetry was used.) · Risk ratio (rr): 1.055 · 95% CI 0.700 to 1.589
SecondaryAnnualized Rate of MO-Related Surgeries

The MO-related surgeries included any procedure indicated for the treatment of MO, such as an excision of a symptomatic OC or correction of a limb deformity.

Time frame:
Month 12
Reported as:
Least squares mean · number of MO-related surgeries per year
Annualized Rate of MO-Related Surgeries
number of MO-related surgeries per yearPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Annualized Rate of MO-Related Surgeries2.087 (1.099 to 3.964)3.454 (1.850 to 6.451)2.231 (1.638 to 3.038)
Statistical analysis
  • Palovarotene 2.5 mg vs Palovarotene 5.0 mg · Poisson regression model · p = 0.2186 (The p-values were not adjusted for multiple testing due to small sample size. The correlation matrix type = independent was used.) · Risk ratio (rr): 1.548 · 95% CI 0.772 to 3.108
  • Placebo vs Palovarotene 2.5 mg · Poisson regression model · p = 0.2700 (The p-values were not adjusted for multiple testing due to small sample size. The correlation matrix type = independent was used.) · Risk ratio (rr): 1.655 · 95% CI 0.676 to 4.052
  • Placebo vs Palovarotene 5.0 mg · Poisson regression model · p = 0.8546 (The p-values were not adjusted for multiple testing due to small sample size. The correlation matrix type = independent was used.) · Risk ratio (rr): 1.069 · 95% CI 0.524 to 2.178
SecondaryMaximum Observed Plasma Drug Concentrations at Steady State (Cmax,ss) of Palovarotene

The Cmax,ss of palovarotene was evaluated. The pharmacokinetic (PK) sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

Time frame:
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Reported as:
Geometric mean · nanogram per milliliter (ng/mL)
Maximum Observed Plasma Drug Concentrations at Steady State (Cmax,ss) of Palovarotene
nanogram per milliliter (ng/mL)Palovarotene 2.5 mgPalovarotene 5.0 mg
Maximum Observed Plasma Drug Concentrations at Steady State (Cmax,ss) of Palovarotene18.0 ± 50.234.9 ± 63.3
SecondaryMinimum Observed Plasma Drug Concentrations at Steady State (Cmin,ss) of Palovarotene

The Cmin,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

Time frame:
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Reported as:
Geometric mean · ng/mL
Minimum Observed Plasma Drug Concentrations at Steady State (Cmin,ss) of Palovarotene
ng/mLPalovarotene 2.5 mgPalovarotene 5.0 mg
Minimum Observed Plasma Drug Concentrations at Steady State (Cmin,ss) of Palovarotene0.314 ± 86.10.674 ± 83.3
SecondaryTime to Maximum Observed Drug Concentration at Steady State (Tmax,ss) of Palovarotene

The Tmax,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

Time frame:
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Reported as:
Median · hour
Time to Maximum Observed Drug Concentration at Steady State (Tmax,ss) of Palovarotene
hourPalovarotene 2.5 mgPalovarotene 5.0 mg
Time to Maximum Observed Drug Concentration at Steady State (Tmax,ss) of Palovarotene3.00 (2.47 to 10.00)3.01 (2.42 to 24.25)
SecondaryArea Under the Plasma Concentration-Time Curve at Steady State From Time 0 to 24 Hours After Dosing (AUC0-24,ss) of Palovarotene

The AUC0-24,ss of palovarotene was evaluated. The PK sampling was performed at Month 1. If samples could not be obtained at Month 1, then one additional attempt was made at a subsequent visit.

Time frame:
Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose
Reported as:
Geometric mean · hour*ng/mL
Area Under the Plasma Concentration-Time Curve at Steady State From Time 0 to 24 Hours After Dosing (AUC0-24,ss) of Palovarotene
hour*ng/mLPalovarotene 2.5 mgPalovarotene 5.0 mg
Area Under the Plasma Concentration-Time Curve at Steady State From Time 0 to 24 Hours After Dosing (AUC0-24,ss) of Palovarotene112 ± 29.1241 ± 42.7
SecondaryNumber of Participants With Palatability of Sprinkled Palovarotene and Placebo

Palatability of palovarotene and placebo when sprinkled on specific foods as assessed with a 5-point hedonic face scale at the first dose (Day 1) and at Month 1 in all participants (including \<4 years old) who sprinkled the palovarotene or placebo onto a spoonful of specific foods. The hedonic face scale ranges from 1 to 5 where, 1= dislike very much, 2= dislike slightly, 3= neither like nor dislike, 4= like slightly, 5= like very much. Higher scores indicate positive outcome.

Time frame:
Day 1 and Month 1
Reported as:
Count of participants · Participants
Number of Participants With Palatability of Sprinkled Palovarotene and Placebo
ParticipantsPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Day 1 — Dislike very much010
Day 1 — Dislike a little110
Day 1 — Not sure633
Day 1 — Like a little353
Day 1 — Like very much1155
Month 1 — Dislike very much100
Month 1 — Dislike a little111
Month 1 — Not sure513
Month 1 — Like a little851
Month 1 — Like very much686

Adverse events

Collected over Treatment-emergent adverse events were to be collected from the start of the first study drug (Day 1) up to 7 days after last study drug intake, assessed until data cut-off for study termination (maximum of 595 days).. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Placebo0/62 (0%)0/62 (0%)41/62 (66.1%)
Palovarotene 2.5 mg0/66 (0%)2/66 (3%)56/66 (84.8%)
Palovarotene 5.0 mg0/65 (0%)2/65 (3.1%)56/65 (86.2%)
Most frequent serious events
Most frequent serious events
EventPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
Blood Loss AnaemiaBlood and lymphatic system disorders0/620/661/65
Status EpilepticusNervous system disorders0/620/661/65
PneumoniaInfections and infestations0/621/660/65
Radius FractureInjury, poisoning and procedural complications0/621/660/65
Ulna FractureInjury, poisoning and procedural complications0/621/660/65
Most frequent other events
Showing 10 of 11
Most frequent other events
EventPlaceboPalovarotene 2.5 mgPalovarotene 5.0 mg
RashSkin and subcutaneous tissue disorders7/6217/6625/65
Dry SkinSkin and subcutaneous tissue disorders7/6216/6619/65
Lip DryGastrointestinal disorders3/626/6611/65
PyrexiaGeneral disorders0/623/669/65
PruritusSkin and subcutaneous tissue disorders6/628/667/65
ArthralgiaMusculoskeletal and connective tissue disorders7/623/661/65
VomitingGastrointestinal disorders2/623/667/65
HeadacheNervous system disorders6/624/663/65
Rash GeneralisedSkin and subcutaneous tissue disorders2/623/665/65
NasopharyngitisInfections and infestations3/623/665/65

Baseline characteristics

The Safety set included randomized participants who received at least 1 dose of study drug.

Age, Continuous
Age, Continuous(years)PlaceboPalovarotene 2.5 mgPalovarotene 5.0 mgTotal
Mean7.9 ± 2.57.8 ± 3.17.4 ± 3.17.7 ± 2.9
Age, Customized
Age, Customized(Participants)PlaceboPalovarotene 2.5 mgPalovarotene 5.0 mgTotal
2 to 5 years13171949
6 to 10 years383335106
11 to 14 years11161138
Sex: Female, Male
Sex: Female, Male(Participants)PlaceboPalovarotene 2.5 mgPalovarotene 5.0 mgTotal
Female23262776
Male394038117
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)PlaceboPalovarotene 2.5 mgPalovarotene 5.0 mgTotal
White485052150
Black Or African American0224
Asian53311
American Indian Or Alaska Native0101
Multiple67518
Other0011
Missing3328
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)PlaceboPalovarotene 2.5 mgPalovarotene 5.0 mgTotal
Hispanic or Latino67720
Not Hispanic or Latino565857171
Missing0112
07

Study locations

31 sites
  • Children's Orthopaedic Center
    Los Angeles, California 90027, United States
  • Shriners Hospital for Children - Sacramento
    Sacramento, California 95817, United States
  • University of California-San Francisco
    San Francisco, California 94158, United States
  • Children's National Medical Center
    Washington, District of Columbia 20010, United States
  • The Paley Institute
    West Palm Beach, Florida 330407, United States
  • Shriners Hospital for Children - Chicago
    Chicago, Illinois 60707, United States
  • Johns Hopkins University
    Baltimore, Maryland 21205, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • Mayo Clinic - PPDS
    Rochester, Minnesota 55905, United States
  • Shriners Hospitals for Children - Portland
    Portland, Oregon 97239, United States
  • The Children's Hospital of Philadelphia (CHOP)
    Philadelphia, Pennsylvania 19104, United States
  • Shriners Hospital for Children - Philadelphia
    Philadelphia, Pennsylvania 19410-4160, United States
  • Memorial Hermann Hospital
    Houston, Texas 77030, United States
  • Westmead Children's Hospital
    Westmead, New South Wales 2145, Australia
  • UZ Antwerpen
    Edegem, Antwerp 2650, Belgium
  • Hospital for Sick Children
    Toronto, Ontario M5G 1X8, Canada
  • Centre Hospitalier Universitaire Sainte-Justine
    Montréal, Quebec H3T 1C5, Canada
  • Shriners Hospital for Children - Canada
    Montréal, Quebec H4A 0A9, Canada
  • Hôpital universitaire Necker - Enfants Malades
    Paris, 75015, France
  • Hôpital des Enfants, CHU de Toulouse
    Toulouse, 31059, France
  • Istituti Ortopedici Rizzoli
    Bologna, Emilia-Romagna 40136, Italy
  • Nagoya University Hospital
    Nagoya, Aiti 4668560, Japan
  • Osaka University Hospital
    Suita, Osaka 565-0871, Japan
  • OLVG locatie Oost
    Amsterdam, Noord-Holland 1091 AC, Netherlands
  • Hospital Pediátrico de Coimbra
    Coimbra, 3000-602, Portugal
  • Hospital Universitario La Paz
    Madrid, 28046, Spain
  • Ege University Medical Faculty Hospital
    Bornova, Izmir, Turkey
  • Bezmialem Vakif University Medical Faculty Hospital
    Istanbul, 34093, Turkey
  • Evelina London Children's Hospital
    London, SE1 7EH, United Kingdom
  • Royal Manchester Childrens Hospital
    Manchester, M13 9WL, United Kingdom
  • Royal National Orthopaedic Hospital
    Stanmore, HA7 4LP, United Kingdom
08

References and documents

Publications

  • Inubushi T, Lemire I, Irie F, Yamaguchi Y. Palovarotene Inhibits Osteochondroma Formation in a Mouse Model of Multiple Hereditary Exostoses. J Bone Miner Res. 2018 Apr;33(4):658-666. doi: 10.1002/jbmr.3341. Epub 2017 Nov 30. PubMed 29120519 ↗

Study documents

  • Study protocol · Apr 23, 2019
  • Statistical analysis plan · May 14, 2020

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

09

Registry details

Key details

Study ID
NCT03442985
Lead sponsor
Clementia Pharmaceuticals Inc.
Responsible party
Sponsor
First posted
Feb 22, 2018
Start date
Mar 22, 2018
Primary completion
Mar 24, 2020
Completion
Oct 30, 2020
Results posted
Aug 1, 2022
Last update
Aug 1, 2022

Study contacts

Ipsen Medical Director
study director · Ipsen

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
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