NL-OMON48469
Completed
Phase 2
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects with Multiple Osteochondromas - MO-Ped
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- Clementia Pharmaceuticals Inc
- Enrollment
- 10
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
Trial ended prematurely
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Written, signed, and dated informed subject/parent consent and age
- •appropriate assent (performed according to local regulations)., 2\. A clinical
- •diagnosis of MO with a disease\-causing Ext1 or Ext2 mutations confirmed by a
- •central laboratory., 3\. Male and female subjects with a chronological age of
- •2\-14 years, inclusive., 4\. Female subjects must be premenarchal at screening.,
- •5\. Bone age at screening of \<\=14 years, 0 months per the Greulich\-Pyle method as
- •assesed by a central reader., 6\. Symptomatic MO, defined as the occurrence of
- •any one of the following at screening:, • Five or more clinically\-evident OCs
- •and the presence of a new or enlarging OC in the preceding 12 months.
- •Five or more clinically\-evident OCs and the presence of a painful OC.
Exclusion Criteria
- •1\. A weight \<10 kg., 2\. Other known syndromic conditions such as Langer\-Giedion
- •or Potocki Shaffer., 3\. Any subject with neurologic signs suggestive of spinal
- •cord impingement., 4\. If subject is currently using vitamin A or beta carotene,
- •multivitamins containing vitamin A or beta carotene, or herbal preparations,
- •fish oil, and unable or unwilling to discontinue use of these products during
- •palovarotene treatment. For eligibility, no washout is required prior to the
- •first dose of study drug., 5\. Exposure to synthetic oral retinoids within 4
- •weeks prior to enrollment., 6\. Concurrent treatment with tetracycline or any
- •tetracycline derivatives, due to the potential increased risk of pseudotumor
- •cerebri., 7\. History of allergy or hypersensitivity to retinoids, gelatin or
Outcomes
Primary Outcomes
Not specified
Similar Trials
Completed
Phase 2
A Phase 2, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Safety and Efficacy of Risankizumab in Adult Subjects with Moderate to Severe Hidradenitis Supperativa.NL-OMON50087AbbVie B.V.15
Completed
Not Applicable
A Phase 1, Randomized, Placebo-Controlled, Double-Blind, First in Man, Single Escalating Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of GAL-475 in Healthy Male Volunteers and Male Subjects with Sleep Apneasleep disorderSleep apneaNL-OMON55229eurim Pharmaceuticals Ltd.36
Completed
Not Applicable
A phase I, randomised, double-blind, placebo-controlled study in patients with amyotrophic lateral sclerosis to further assess the safety and tolerability of intracerebroventricular administration of sNN0029 infusion solution.ALSLou Gehrig's disease1002930510009720NL-OMON40611ewron Sweden AB9
Completed
Not Applicable
A phase I randomized, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, tolerability and pharmacokinetics of NPT189 in healthy subjectsNL-OMON48151Proclara Biosciences, Inc.24
Completed
Phase 3
A randomized, double-blind, placebo-controlled intervention study to assess the therapeutic effect of an extensively hydrolyzed infant formula with an added synbiotic mixture in infants with atopic dermatitis.atopic dermatitiseczema1003871610040785NL-OMON39465Danone Research - Centre for Specialised Nutrition144