DDrare: Database of Drug Development for Rare Diseases

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.

Drug = Active bmn 111: daily subcutaneous injection of 15 ug/kg of bmn 111; Bmn 111; Modified recombinant human c-type natriuretic peptide; Modified rhcnp; Normal saline; Somatropin; Transcon cnp; Vosoritide

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
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

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