276. 軟骨無形成症 [臨床試験数:11,薬物数:8(DrugBank:2),標的遺伝子数:1,標的パスウェイ数:5]
Searched query = "Achondroplasia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT04085523 | November 2019 | 30 September 2019 | A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia | ACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 12 Months in Prepubertal Children With Achondroplasia | Achondroplasia | Drug: TransCon CNP;Drug: Placebo for TransCon CNP | Ascendis Pharma A/S | Not recruiting | 2 Years | 10 Years | All | 60 | Phase 2 | United States;Australia | |
2 | NCT03989947 | June 12, 2019 | 26 August 2019 | An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111 | BioMarin Pharmaceutical | Recruiting | 15 Months | N/A | All | 70 | Phase 2 | United States | |
3 | NCT03583697 | May 23, 2018 | 3 June 2019 | A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months | Achondroplasia | Drug: BMN 111;Drug: Placebo | BioMarin Pharmaceutical | Recruiting | N/A | 59 Months | All | 70 | Phase 2 | United States;Australia;United Kingdom | |
4 | NCT03424018 | December 12, 2017 | 4 November 2019 | An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111 | BioMarin Pharmaceutical | Recruiting | 6 Years | N/A | All | 110 | Phase 3 | United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom | |
5 | EUCTR2015-003836-11-GB | 20/01/2017 | 30 April 2019 | A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. | achondroplasia MedDRA version: 20.0 Level: LLT Classification code 10000452 Term: Achondroplasia System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11] | Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 Pharmaceutical Form: Lyophilisate for solution for injection INN or Proposed INN: Vosoritide CAS Number: 1480724-61-5 Current Sponsor code: BMN 111 Other descriptive name: MODIFIED RHCNP Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 0.8- Pharmaceutical form of the placebo: Lyophilisate and solvent for solution for injection Route of administration of the placebo: Subcutaneous use Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 Pharmaceutical Form: Lyophilisate for solution for injection INN or Proposed INN: Vosoritide CAS Number: 1480724-61-5 Current Sponsor code: BMN 111 Other descriptive name: MODIFIED RHCNP Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 2- Pharmaceutical form of the placebo: Lyophilisate and solvent for solution for injection Route of administration of the placebo: Subcutaneous use Product Name: modified recombinant human C-type natriuretic peptide Product Code: BMN 111 Pharmaceutical Form: Lyophilisate for solution for injection INN or Proposed INN: Vosoritide CAS Number: 1480724-61-5 Current Sponsor code: BMN 111 Other descriptive name: MODIFIED RHCNP Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | BioMarin Pharmaceutical Inc. | Authorised | Female: yes Male: yes | 110 | Phase 3 | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | |||
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
6 | NCT03197766 | December 12, 2016 | 11 June 2019 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111;Drug: Placebo | BioMarin Pharmaceutical | Not recruiting | 5 Years | 18 Years | All | 121 | Phase 3 | United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom | |
7 | NCT02724228 | January 26, 2016 | 11 November 2019 | A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111 | BioMarin Pharmaceutical | Not recruiting | 7 Years | N/A | All | 30 | Phase 2 | United States;Australia;France;United Kingdom | |
8 | NCT02055157 | January 13, 2014 | 18 December 2018 | A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia | A Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With Achondroplasia | Achondroplasia | Drug: BMN 111 | BioMarin Pharmaceutical | Not recruiting | 5 Years | 14 Years | All | 35 | Phase 2 | United States;Australia;France;United Kingdom | |
9 | NCT01435629 | November 22, 2012 | 16 December 2017 | A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin | Open-label, Multicenter, Observational, Non-intervention Study to Retrospectively Evaluate the Efficacy of Norditropin® (Adult Height) in Patients With Achondroplasia/Hypochondroplasia Enrolled in the GH-1941 Study [Follow-up Survey] | Genetic Disorder;Achondroplasia | Drug: somatropin | Novo Nordisk A/S | Not recruiting | N/A | N/A | All | 81 | N/A | Japan | |
10 | NCT01590446 | February 2012 | 19 February 2015 | A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers | A Phase 1, Two-Part, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of BMN 111 Administered to Healthy Adult Volunteers | Achondroplasia | Drug: BMN 111;Drug: Normal Saline | BioMarin Pharmaceutical | Not recruiting | 22 Years | 45 Years | Male | 74 | Phase 1 | United States | |
No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
11 | NCT01516229 | May 1, 1997 | 16 December 2017 | Special Survey for Long Term Application | Specific Survey of Norditropin® in Achondroplasia: Survey for Long-term Application | Genetic Disorder;Achondroplasia | Drug: somatropin | Novo Nordisk A/S | Not recruiting | 1 Year | 15 Years | All | 395 | N/A | Japan |