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 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:3] 

Searched query = "Achondroplasia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04085523November 201930 September 2019A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With AchondroplasiaACcomplisH: 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 AchondroplasiaAchondroplasiaDrug: TransCon CNP;Drug: Placebo for TransCon CNPAscendis Pharma A/SNot recruiting2 Years10 YearsAll60Phase 2United States;Australia
2NCT03989947June 12, 201926 August 2019An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaA Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With AchondroplasiaAchondroplasiaDrug: Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111BioMarin PharmaceuticalRecruiting15 MonthsN/AAll70Phase 2United States
3NCT03583697May 23, 20183 June 2019A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With AchondroplasiaA 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 MonthsAchondroplasiaDrug: BMN 111;Drug: PlaceboBioMarin PharmaceuticalRecruitingN/A59 MonthsAll70Phase 2United States;Australia;United Kingdom
4NCT03424018December 12, 20174 November 2019An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaA Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With AchondroplasiaAchondroplasiaDrug: BMN 111BioMarin PharmaceuticalRecruiting6 YearsN/AAll110Phase 3United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom
5EUCTR2015-003836-11-GB20/01/201730 April 2019A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with AchondroplasiaA 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
110Phase 3United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03197766December 12, 201611 June 2019A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaA Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaAchondroplasiaDrug: BMN 111;Drug: PlaceboBioMarin PharmaceuticalNot recruiting5 Years18 YearsAll121Phase 3United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom
7NCT02724228January 26, 201611 November 2019A 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 AchondroplasiaAchondroplasiaDrug: BMN 111BioMarin PharmaceuticalNot recruiting7 YearsN/AAll30Phase 2United States;Australia;France;United Kingdom
8NCT02055157January 13, 201418 December 2018A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With AchondroplasiaA Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With AchondroplasiaAchondroplasiaDrug: BMN 111BioMarin PharmaceuticalNot recruiting5 Years14 YearsAll35Phase 2United States;Australia;France;United Kingdom
9NCT01435629November 22, 201216 December 2017A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With SomatropinOpen-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;AchondroplasiaDrug: somatropinNovo Nordisk A/SNot recruitingN/AN/AAll81N/AJapan
10NCT01590446February 201219 February 2015A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult VolunteersA 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 VolunteersAchondroplasiaDrug: BMN 111;Drug: Normal SalineBioMarin PharmaceuticalNot recruiting22 Years45 YearsMale74Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT01516229May 1, 199716 December 2017Special Survey for Long Term ApplicationSpecific Survey of Norditropin® in Achondroplasia: Survey for Long-term ApplicationGenetic Disorder;AchondroplasiaDrug: somatropinNovo Nordisk A/SNot recruiting1 Year15 YearsAll395N/AJapan

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