Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2017-002751-28 | EudraCT Number |
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Trial was terminated early to analyze the accumulated data and evaluate the efficacy, safety and future of palovarotene in MO.
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Palovarotene 2.5 mg daily regimen | Experimental |
| |
| Palovarotene 5.0 mg daily regimen | Experimental |
| |
| Placebo regimen | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Palovarotene 2.5 mg | Drug | Subjects received a weight-adjusted dose equivalent of 2.5 mg palovarotene, once daily, for up to 24 months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline (Day 1) and Month 12 |
| Percentage of Participants With No New OCs |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Orthopaedic Center | Los Angeles | California | 90027 | United States | ||
| Shriners Hospital for Children - Sacramento |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29120519 | Background | 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. | |
| 41188357 | Derived | Sangiorgi L, Conrad EU, Shih F, Strahs A, Feldman DS. Palovarotene for patients with multiple hereditary exostosis: results of MO-Ped, a terminated, randomized, placebo-controlled, double-blind phase 2 trial. Sci Rep. 2025 Nov 4;15(1):38563. doi: 10.1038/s41598-025-22554-6. |
| Label | URL |
|---|---|
| MHE Coalition | View source |
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Study consisted of a screening period (up to 35 days), followed by a double-blind treatment period (24 months) and safety follow-up period (6 months). Participants were randomized in a 1:1:1 ratio to palovarotene 2.5 milligram (mg) or 5.0 mg or placebo. A total of 193 participants received at least 1 dose of study drug and were included in the study analysis.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants were to receive placebo matching with palovarotene capsules orally once daily, for up to 24 months. |
| FG001 | Palovarotene 2.5 mg | Participants were to receive weight-adjusted dose equivalent of palovarotene 2.5 mg capsules orally once daily, for up to 24 months. |
| Title | Milestones | Reasons Not Completed | ||||
|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 23, 2019 | Jul 8, 2021 |
Not provided
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Multicenter, randomized, double-blind, placebo-controlled
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| Palovarotene 5.0 mg | Drug | Subjects received a weight-adjusted dose equivalent of 5.0 mg palovarotene, once daily, for up to 24 months. |
|
| Placebo | Other | Subjects received placebo, once daily, for up to 24 months. |
|
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. |
| Month 12 |
| 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. | Month 12 |
| 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. | Month 12 |
| 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. | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
| 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. | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
| 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. | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
| 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. | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
| 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. | Day 1 and Month 1 |
| Sacramento |
| California |
| 95817 |
| United States |
| University of California-San Francisco | San Francisco | California | 94158 | United States |
| Children's National Medical Center | Washington D.C. | 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 | Montreal | Quebec | H3T 1C5 | Canada |
| Shriners Hospital for Children - Canada | Montreal | 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 | North 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 | İzmir | Turkey (Türkiye) |
| Bezmialem Vakif University Medical Faculty Hospital | Istanbul | 34093 | Turkey (Türkiye) |
| 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 |
| MHE Research Foundation | View source |
| FG002 | Palovarotene 5.0 mg | Participants were to receive weight-adjusted dose equivalent of palovarotene 5.0 mg capsules orally once daily, for up to 24 months. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
The Safety set included randomized participants who received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants were to receive placebo matching with palovarotene capsules orally once daily, for up to 24 months. |
| BG001 | Palovarotene 2.5 mg | Participants were to receive weight-adjusted dose equivalent of palovarotene 2.5 mg capsules orally once daily, for up to 24 months. |
| BG002 | Palovarotene 5.0 mg | Participants were to receive weight-adjusted dose equivalent of palovarotene 5.0 mg capsules orally once daily, for up to 24 months. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants | No |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants | No |
| |||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| |||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | The Full Analysis Set (FAS) included randomized participants who received at least 1 dose of study drug. Only data from the 56 participants for whom Month 12 efficacy imaging data were available were included in the analysis. | Posted | Least Squares Mean | 95% Confidence Interval | number of new OCs per year | Month 12 |
|
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| Secondary | 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. | The FAS included randomized participants who received at least 1 dose of study drug. Only data from the 56 participants for whom Month 12 efficacy imaging data were available were included in the analysis. | Posted | Mean | Standard Deviation | cubic millimeter | Baseline (Day 1) and Month 12 |
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| Secondary | 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. | The FAS included randomized participants who received at least 1 dose of study drug. Only data from the 56 participants for whom Month 12 efficacy imaging data were available were included in the analysis. | Posted | Number | percentage of participants | Month 12 |
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| Secondary | 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. | The FAS included randomized participants who received at least 1 dose of study drug. Only data from the 56 participants for whom Month 12 efficacy imaging data were available were included in the analysis. | Posted | Least Squares Mean | 95% Confidence Interval | number of deformities per year | Month 12 |
|
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| Secondary | 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. | The FAS included randomized participants who received at least 1 dose of study drug. Participants with planned surgeries within 6 months of enrollment to remove symptomatic OCs or to correct deformities present at baseline, and/or had surgical procedures that were a continuation of a previous procedure were excluded in the analysis. | Posted | Least Squares Mean | 95% Confidence Interval | number of MO-related surgeries per year | Month 12 |
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| Secondary | 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. | The Pharmacokinetic Set (PKS) included participants receiving treatment with palovarotene and with evaluable PK data. | Posted | Geometric Mean | Geometric Coefficient of Variation | nanogram per milliliter (ng/mL) | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
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| Secondary | 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. | The PKS included participants receiving treatment with palovarotene and with evaluable PK data. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng/mL | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | The PKS included participants receiving treatment with palovarotene and with evaluable PK data. | Posted | Median | Full Range | hour | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | The PKS included participants receiving treatment with palovarotene and with evaluable PK data. | Posted | Geometric Mean | Geometric Coefficient of Variation | hour*ng/mL | Month 1: pre-dose and 3, 6, 10 and 24 hours post-dose |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | The Safety set included randomized participants who received at least 1 dose of study drug. Only data from the participants analyzed at Day 1 and Month 1 were included in the analysis. | Posted | Count of Participants | Participants | No | Day 1 and Month 1 |
|
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).
The Safety set included randomized participants who received at least 1 dose of study drug.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Participants were to receive placebo matching with palovarotene capsules orally once daily, for up to 24 months. | 0 | 62 | 0 | 62 | 41 | 62 |
| EG001 | Palovarotene 2.5 mg | Participants were to receive weight-adjusted dose equivalent of palovarotene 2.5 mg capsules orally once daily, for up to 24 months. | 0 | 66 | 2 | 66 | 56 | 66 |
| EG002 | Palovarotene 5.0 mg | Participants were to receive weight-adjusted dose equivalent of palovarotene 5.0 mg capsules orally once daily, for up to 24 months. | 0 | 65 | 2 | 65 | 56 | 65 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Blood Loss Anaemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Radius Fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Ulna Fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Status Epilepticus | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dry Skin | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash Generalised | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Lip Dry | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dry Mouth | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
|
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.
Not provided
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Ipsen | see email | clinical.trials@ipsen.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 14, 2020 | Jul 22, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D005097 | Exostoses, Multiple Hereditary |
| D015831 | Osteochondroma |
| ID | Term |
|---|---|
| D018216 | Osteochondromatosis |
| D018213 | Neoplasms, Bone Tissue |
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009386 | Neoplastic Syndromes, Hereditary |
| D010009 | Osteochondrodysplasias |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D005096 | Exostoses |
| D015576 | Hyperostosis |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| ID | Term |
|---|---|
| C546535 | Palovarotene |
Not provided
Not provided
Not provided
| 6 to 10 years |
|
| 11 to 14 years |
|
| Male |
|
| Black Or African American |
|
| Asian |
|
| American Indian Or Alaska Native |
|
| Multiple |
|
| Other |
|
| Missing |
|
| Not Hispanic or Latino |
|
| Missing |
|
|
Palovarotene 2.5 mg vs Placebo: The annualized rate for number of new OCs was estimated using an unadjusted negative binomial regression model, offsetted by log-transformed follow-up time (years) to obtain annualized rate. |
| Negative binomial regression model |
| 0.1788 |
The p-values were not adjusted for multiple testing due to small sample size. |
| Risk Ratio (RR) |
| 3.040 |
| 2-Sided |
| 95 |
| 0.601 |
| 15.373 |
| Other |
| Palovarotene 5.0 mg vs Placebo: The annualized rate for number of new OCs was estimated using an unadjusted negative binomial regression model, offsetted by log-transformed follow-up time (years) to obtain annualized rate. | Negative binomial regression model | 0.6570 | The p-values were not adjusted for multiple testing due to small sample size. | Risk Ratio (RR) | 1.441 | 2-Sided | 95 | 0.287 | 7.234 | Other |
| Units | Counts |
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| Participants |
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| Units | Counts |
|---|---|
| Participants |
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| Not sure |
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| Like a little |
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| Like very much |
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