Clinical trials: Dwarfism
Achondroplasia
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
- Trial name
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 people
- Sponsor
- BioMarin Pharmaceutical
Growth Hormone Deficiency
Study to Assess Safety and Tolerability in Adults With Growth Hormone-Deficiency (GHD)
This study assesses the safety and tolerability of weekly TV-1106 compared to daily rhGH in adults with GHD who have previously been treated with rhGH.
- Trial name
- NCT02410356
- Testing
- TV-1106, dGH
- Where
- 50 sites in United States, Austria and 6 more
- Volunteers
- 34 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
The purpose of this clinical trial was to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of 100 µg TransCon CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period…
- Trial name
- ApproaCH (NCT05598320)
- Testing
- Navepegritide (TransCon CNP)
- Where
- 10 sites in United States, Australia and 5 more
- Volunteers
- 84 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
Growth Hormone Deficiency
Easypod United States User Trial
This study will help to determine whether an injection log on a recombinant human growth hormone (rhGH) delivery device improves subjects' adherence with injection schedules.
- Trial name
- NCT00689260
- Testing
- easypod
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- EMD Serono
Growth Hormone Deficiency
VRS-317 in Adult Subjects With Growth Hormone Deficiency
The purpose of this research study is to determine the safety and tolerability of up to five doses of VRS-317 in Adult Growth Hormone Deficient patients.
- Trial name
- NCT01359488
- Testing
- VRS-317
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Versartis Inc.
Idiopathic Short Stature
Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature
To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of…
- Trial name
- NCT00396097
- Testing
- Genotropin
- Where
- 43 sites in United States
- Volunteers
- 316 people
- Sponsor
- Pfizer
Growth Hormone Disorder, Adult Growth Hormone Deficiency
Cardiovascular Effects on Growth Hormone Therapy in Adults With Growth Hormone Deficiency
This trial is conducted in Europe. The aim of this trial is to investigate the effect of growth hormone on left ventricle morphology and function (systolic and diastolic).
- Trial name
- NCT01698944
- Testing
- somatropin
- Where
- Greece
- Volunteers
- 7 people
- Sponsor
- Novo Nordisk A/S
Achondroplasia
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.
- Trial name
- NCT05813314
- Testing
- BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Adult Growth Hormone Deficiency (AGHD)
A Study of GS3-007a for Oral Suspension in the Diagnosis of Adult Growth Hormone Deficiency (AGHD)
The GS3-007a Growth Hormone Stimulation Test (GHST) will be compared with the Insulin Tolerance Test (ITT) in an open-label, randomized, three-way crossover trial.
- Trial name
- NCT07259564
- Testing
- GS3-007a dry suspension, Insulin
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Safety, Tolerability
A Clinical Study of GenSci134 in Healthy Adults and Adult Growth Hormone Deficiency(AGHD)
To check how safe and well-tolerated a single subcutaneous injection of GenSc134 is in healthy male volunteers， a multiple-doses subcutaneous injection of GenSc134 is in healthy volunteers，a single subcutaneous injection of…
- Conditions
- Safety, Tolerability, Adult Growth Hormone Deficiency
- Trial name
- NCT07016802
- Testing
- GenSci134, Recombinant Human Growth Hormone Injection (Norditropin®)
- Where
- China
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Carcinoma, Non-Small-Cell Lung
Cilengitide and Cetuximab in Combination With Platinum-based Chemotherapy as First-line Treatment for Subjects With Advanced Non Small Cell Lung Cancer (NSCLC)
Primary objective of the study's Safety run-in: To determine the maximum tolerated dose (MTD) of cilengitide in combination with cetuximab, and platinum-based chemotherapy (cisplatin/vinorelbine or cisplatin/gemcitabine).
- Trial name
- CERTO (NCT00842712)
- Testing
- Cilengitide, Cetuximab, Cisplatin
- Where
- 44 sites in Belgium, Czechia and 6 more
- Volunteers
- 232 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Achondroplasia
A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
This is a multicenter, non-randomized, open-label, phase I/II study in children with ACH. This study will start with a dose escalation of ABSK061 in children with ACH to evaluate the safety, tolerability, PK, and efficacy.
- Trial name
- NCT07297875
- Testing
- ABSK061
- Where
- 7 sites in China
- Volunteers
- 110 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Achondroplasia
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly…
- Trial name
- COACH (NCT06433557)
- Testing
- Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections
- Where
- 3 sites in Denmark, Ireland and 1 more
- Volunteers
- 22 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
Genetic Disorder, Noonan Syndrome
Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome
This trial is conducted in Asia. The aim of the trial is to investigate the long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome.
- Trial name
- NCT01927861
- Testing
- somatropin
- Where
- 27 sites in Japan
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Achondroplasia, Hypochondroplasia
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.
- Trial name
- SKY-MOT3 (NCT07388966)
- Where
- 4 sites in Belgium, France and 1 more
- Volunteers
- 140 people
- Sponsor
- SYSNAV
Growth Hormone Deficiency
Cross Over Convenience And Preference Study Of New Mark VII Compared To Genotropin Pen In Pediatric And Adult Subjects
Convenience and preference for the new Mark VII pen compared to the current Genotropin pen will be assessed using a questionaire. it is expected that the new pen will be preferred or at least no different to the current pen.
- Trial name
- CHOOSE (NCT01112865)
- Testing
- Genotropin Pen, MARK VII pen
- Where
- 23 sites in Czechia, Germany and 5 more
- Volunteers
- 120 people
- Sponsor
- Pfizer
Idiopathic Short Stature
A Clinical Study of Long-Term Administration of Inpegsomatropin Injection in Participants With Idiopathic Short Stature Who Have Completed Phase Ⅲ Clinical Trials
This study is a multicenter, open-label, single-arm extension study to evaluate the safety and efficacy of long-term administration of Inpegsomatropin Injection in participants with Idiopathic Short Stature (ISS) who have…
- Trial name
- NCT07894341
- Testing
- Inpegsomatropin Injection
- Where
- 27 sites in China
- Volunteers
- 300 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Growth Hormone Deficiency
Dose Finding Study of GX-H9 in Paeditaric Patients With Growth Hormone Deficiency
This is a randomized, open-label, active controlled, Phase 2 study designed to assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of weekly and semi-monthly doses of GX-H9 in the treatment of…
- Trial name
- NCT03309891
- Testing
- GX-H9, Genotropin
- Where
- Ukraine
- Volunteers
- 56 people
- Sponsor
- Genexine, Inc.
Congenital Hypothyroidism
Use of Tirosint®-SOL or Tablet Formulations of Levothyroxine in Pediatric Patients With Congenital Hypothyroidism (CH)
This is a multi-center, prospective, parallel-group, open-label, randomized clinical study in one hundred and twenty-six (126) neonates and infants diagnosed with CH.
- Trial name
- NCT05228184
- Testing
- Tirosint®-SOL, Levothyroxine Sodium
- Where
- 10 sites in United States
- Volunteers
- 34 people
- Sponsor
- IBSA Institut Biochimique SA
Growth Hormone Deficiency
Safety, Pharmacokinetics and Pharmacodynamics Study of HL-032 in Healthy Male Volunteers
The purpose of this study is to evaluate the safety, pharmacokinetics and pharmacodynamics of HL-032 after oral administration in healthy male volunteers.
- Trial name
- HL-032 (NCT01440686)
- Testing
- Somatropin
- Where
- South Korea
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- HanAll BioPharma Co., Ltd.