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22 Companies |
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13 Products |
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13 Products |
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29 Mechanisms of Action |
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3 Trials |
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142 News |
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- | Trial primary completion date: IPCRR: International Pachyonychia Congenita Research Registry (clinicaltrials.gov) - Nov 21, 2018
P=N/A, N=2000, Recruiting, Sponsor: Pachyonychia Congenita Project
Trial primary completion date: Dec 2058 --> Dec 2030
- | Phase classification, Enrollment change: Studies of Heritable Disorders of Connective Tissue (clinicaltrials.gov) - Apr 6, 2018
P, N=929, Completed, Sponsor: National Institute on Aging (NIA)
Trial primary completion date: Dec 2058 --> Dec 2030 Phase classification: P=N/A --> P | N=1335 --> 929
- Zyclara (imiquimod) / Mochida, Viatris, Bausch Health
Enrollment change, Trial termination: The Effects of Aldara as an Adjunct to Laser Treatment (clinicaltrials.gov) - Mar 22, 2018
P2, N=7, Terminated, Sponsor: Henry Vasconez
Phase classification: P=N/A --> P | N=1335 --> 929 N=20 --> 7 | Completed --> Terminated; Failure to enroll enough participants
- | Tracleer (bosentan) / J&J, Roche
Trial completion: Treatment of Resistant Port-wine Stains With Bosentan and Pulsed Dye Laser: a Pilot Study (clinicaltrials.gov) - Feb 5, 2018
P2, N=4, Completed, Sponsor: Centre Hospitalier Universitaire de Nice
N=20 --> 7 | Completed --> Terminated; Failure to enroll enough participants Active, not recruiting --> Completed
- | Humira (adalimumab) / Eisai, AbbVie
Trial completion: Clinical Trial Using Humira in Netherton Syndrome (clinicaltrials.gov) - Oct 18, 2017
P2, N=11, Completed, Sponsor: Assistance Publique - Hôpitaux de Paris
Active, not recruiting --> Completed Active, not recruiting --> Completed
- ||| Trial completion, Trial primary completion date: ECP-015: Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia (clinicaltrials.gov) - Sep 5, 2017
P=N/A, N=150, Completed, Sponsor: Edimer Pharmaceuticals
Active, not recruiting --> Completed Active, not recruiting --> Completed | Trial primary completion date: Oct 2019 --> Dec 2016
- | Phase classification: Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa (clinicaltrials.gov) - Jun 23, 2017
P2, N=7, Completed, Sponsor: Vanderbilt University Medical Center
Active, not recruiting --> Completed | Trial primary completion date: Oct 2019 --> Dec 2016 Phase classification: P=N/A --> P2
- | Amevive (alefacept) / Astellas
Trial termination: Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - Jun 7, 2017
P=N/A, N=3, Terminated, Sponsor: Emory University
Phase classification: P=N/A --> P2 Suspended --> Terminated; drug company is no longer making the drug
- | Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Clinical: Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA (clinicaltrials.gov) - May 31, 2017
P2/3, N=36, Completed, Sponsor: Masonic Cancer Center, University of Minnesota
Suspended --> Terminated; drug company is no longer making the drug Active, not recruiting --> Completed
- Trial completion: GenTAC: National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions (clinicaltrials.gov) - Dec 20, 2016
P=N/A, N=3706, Completed, Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Active, not recruiting --> Completed Active, not recruiting --> Completed
- Phase classification: Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC) (clinicaltrials.gov) - Oct 7, 2016
P1b, N=15, Completed, Sponsor: TransDerm, Inc.
Active, not recruiting --> Completed Phase classification: P1/2 --> P1b
- Enrollment change, Trial primary completion date: IPCRR: International Pachyonychia Congenita Research Registry (clinicaltrials.gov) - Sep 16, 2016
P=N/A, N=1800, Recruiting, Sponsor: Pachyonychia Congenita Project
Phase classification: P1/2 --> P1b N=1500 --> 1800 | Trial primary completion date: Dec 2028 --> Dec 2058
- Trial termination: Treatment of Port Wine Stains Using Pulsed Dye Laser, Erbium Yag Laser and Topical Sirolimus (clinicaltrials.gov) - Aug 9, 2016
P=N/A, N=17, Terminated, Sponsor: Erasmus Medical Center
N=1500 --> 1800 | Trial primary completion date: Dec 2028 --> Dec 2058 Active, not recruiting --> Terminated; Lack of efficacy / adverse events
- Trial completion: EVACARNEY: Primary Pigmented Nodular Adrenocortical Disease (PPNAD) and the CARNEY Complex (CNC) (clinicaltrials.gov) - Jul 7, 2016
P=N/A, N=133, Completed, Sponsor: Assistance Publique - Hôpitaux de Paris
Active, not recruiting --> Terminated; Lack of efficacy / adverse events Active, not recruiting --> Completed
- Humira (adalimumab) / Eisai, AbbVie
Enrollment closed, Enrollment change: Clinical Trial Using Humira in Netherton Syndrome (clinicaltrials.gov) - Jun 29, 2016
P2, N=11, Active, not recruiting, Sponsor: Assistance Publique - Hôpitaux de Paris
Active, not recruiting --> Completed Recruiting --> Active, not recruiting | N=23 --> 11
- | Adempas (riociguat) / Bayer, Merck (MSD)
Trial termination, Trial primary completion date: RISE-IIP: Efficacy and Safety of Riociguat in Patients With Symptomatic Pulmonary Hypertension (PH) Associated With Idiopathic Interstitial Pneumonias (IIP) (clinicaltrials.gov) - May 20, 2016
P2, N=147, Terminated, Sponsor: Bayer
Recruiting --> Terminated | Trial primary completion date: Oct 2016 --> May 2016; Study terminated per recommendation of iDMC. On iDMC request, protocol amended to include 4-month safety follow-up for patients after withdrawal of riociguat.
- || Adempas (riociguat) / Bayer, Merck (MSD)
Trial primary completion date: RISE-IIP: Efficacy and Safety of Riociguat in Patients With Symptomatic Pulmonary Hypertension (PH) Associated With Idiopathic Interstitial Pneumonias (IIP) (clinicaltrials.gov) - May 5, 2016
P2, N=135, Recruiting, Sponsor: Bayer
On iDMC request, protocol amended to include 4-month safety follow-up for patients after withdrawal of riociguat. Trial primary completion date: Jan 2017 --> Oct 2016
- recombinant ectodysplasin A1 (APO200) / Onxeo, Pierre Fabre
Enrollment closed: ECP-002e: Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002 (clinicaltrials.gov) - Apr 7, 2016
P=N/A, N=10, Active, not recruiting, Sponsor: Edimer Pharmaceuticals
Trial primary completion date: Jan 2017 --> Oct 2016 Recruiting --> Active, not recruiting
- linsitinib (ASP7487) / VasaraGen
Trial completion, Enrollment change, Stroma: Linsitinib in Treating Patients With Gastrointestinal Stromal Tumors (clinicaltrials.gov) - Feb 11, 2016
P2, N=20, Completed, Sponsor: National Cancer Institute (NCI)
Recruiting --> Active, not recruiting Active, not recruiting --> Completed | N=40 --> 20
- | Trial completion, Trial primary completion date: Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC) (clinicaltrials.gov) - Feb 4, 2016
P1/2, N=15, Completed, Sponsor: TransDerm, Inc.
Active, not recruiting --> Completed | N=40 --> 20 Active, not recruiting --> Completed | Trial primary completion date: Oct 2015 --> Jul 2015
- Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Clinical: Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA (clinicaltrials.gov) - Jan 25, 2016
P2/3, N=33, Active, not recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
Active, not recruiting --> Completed | Trial primary completion date: Oct 2015 --> Jul 2015 Trial primary completion date: Dec 2015 --> Mar 2015
- Enrollment closed: Treatment of Port Wine Stains Using Pulsed Dye Laser, Erbium Yag Laser and Topical Sirolimus (clinicaltrials.gov) - Jan 21, 2016
P=N/A, N=20, Active, not recruiting, Sponsor: Erasmus Medical Center
Trial primary completion date: Dec 2015 --> Mar 2015 Recruiting --> Active, not recruiting
- recombinant ectodysplasin A1 (APO200) / Valerio Therap, Pierre Fabre
Trial completion, IO biomarker: Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (clinicaltrials.gov) - Jan 20, 2016
P2, N=6, Completed, Sponsor: Edimer Pharmaceuticals
Recruiting --> Active, not recruiting Active, not recruiting --> Completed
- Humira (adalimumab) / Eisai, AbbVie
Trial primary completion date: Clinical Trial Using Humira in Netherton Syndrome (clinicaltrials.gov) - Nov 16, 2015
P2, N=23, Recruiting, Sponsor: Assistance Publique - Hôpitaux de Paris
Active, not recruiting --> Completed Trial primary completion date: Jun 2016 --> Dec 2016
- Trial withdrawal: Pilot Study Evaluating the Efficiency and the Tolerance of the PDT in the Treatment of Epidermal Dysplasia for Patients Affected by Hereditary DEB (clinicaltrials.gov) - Oct 1, 2015
P=N/A, N=0, Withdrawn, Sponsor: Centre Hospitalier Universitaire de Nice
Trial primary completion date: Jun 2016 --> Dec 2016 Not yet recruiting --> Withdrawn
- Biomarker, Enrollment change, Trial primary completion date: Peripheral Blood Biomarkers in Idiopathic Interstitial Pneumonias (clinicaltrials.gov) - Sep 9, 2015
P=N/A, N=269, Terminated, Sponsor: Duke University
Not yet recruiting --> Withdrawn N=28 --> 269 | Trial primary completion date: Dec 2013 --> Feb 2013
- recombinant ectodysplasin A1 (APO200) / Valerio Therap, Pierre Fabre
Enrollment closed, IO biomarker: Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (clinicaltrials.gov) - Sep 8, 2015
P2, N=6, Active, not recruiting, Sponsor: Edimer Pharmaceuticals
N=28 --> 269 | Trial primary completion date: Dec 2013 --> Feb 2013 Recruiting --> Active, not recruiting
- || Enrollment closed: ECP-015: Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia (clinicaltrials.gov) - Sep 8, 2015
P=N/A, N=150, Active, not recruiting, Sponsor: Edimer Pharmaceuticals
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting
- Enrollment change, Trial withdrawal: Effect of Laser Treatment on Capillary Malformations (clinicaltrials.gov) - Aug 26, 2015
P=N/A, N=0, Withdrawn, Sponsor: Children's Hospital Medical Center, Cincinnati
Recruiting --> Active, not recruiting N=100 --> 0 | Recruiting --> Withdrawn
- Biomarker, Trial completion: Biomarkers in Vascular Ehlers-Danlos Syndrome (clinicaltrials.gov) - Jul 21, 2015
P=N/A, N=211, Completed, Sponsor: Assistance Publique - Hôpitaux de Paris
N=100 --> 0 | Recruiting --> Withdrawn Recruiting --> Completed
- Humira (adalimumab) / Eisai, AbbVie
Trial primary completion date: Clinical Trial Using Humira in Netherton Syndrome (clinicaltrials.gov) - Jun 11, 2015
P2, N=23, Recruiting, Sponsor: Assistance Publique - Hôpitaux de Paris
Recruiting --> Completed Trial primary completion date: Jan 2016 --> Jun 2016
- | Enrollment closed, Trial primary completion date: Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC) (clinicaltrials.gov) - Jun 9, 2015
P1/2, N=15, Active, not recruiting, Sponsor: TransDerm, Inc.
Trial primary completion date: Jan 2016 --> Jun 2016 Recruiting --> Active, not recruiting | Trial primary completion date: Jan 2015 --> Oct 2015
- recombinant ectodysplasin A1 (APO200) / Valerio Therap, Pierre Fabre
Trial primary completion date, IO biomarker: Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (clinicaltrials.gov) - May 8, 2015
P2, N=6, Recruiting, Sponsor: Edimer Pharmaceuticals
Recruiting --> Active, not recruiting | Trial primary completion date: Jan 2015 --> Oct 2015 Trial primary completion date: Oct 2014 --> Dec 2015
- linsitinib (ASP7487) / VasaraGen
Trial primary completion date, Stroma: Linsitinib in Treating Patients With Gastrointestinal Stromal Tumors (clinicaltrials.gov) - May 1, 2015
P2, N=40, Active, not recruiting, Sponsor: National Cancer Institute (NCI)
Trial primary completion date: Oct 2014 --> Dec 2015 Trial primary completion date: Dec 2016 --> Dec 2015
- Trial primary completion date: EVACARNEY: Primary Pigmented Nodular Adrenocortical Disease (PPNAD) and the CARNEY Complex (CNC) (clinicaltrials.gov) - Apr 21, 2015
P=N/A, N=133, Active, not recruiting, Sponsor: Assistance Publique - Hôpitaux de Paris
Trial primary completion date: Dec 2016 --> Dec 2015 Trial primary completion date: Jan 2015 --> May 2015
- linsitinib (ASP7487) / VasaraGen
Enrollment change, Trial primary completion date, Stroma: Linsitinib in Treating Patients With Gastrointestinal Stromal Tumors (clinicaltrials.gov) - Apr 17, 2015
P2, N=40, Active, not recruiting, Sponsor: National Cancer Institute (NCI)
Trial primary completion date: Jan 2015 --> May 2015 N=20 --> 40 | Trial primary completion date: Dec 2013 --> Dec 2016
- ||| Trial completion, Trial primary completion date: Vascular Fundus Changes in Patients With High Probability of Chronic Cerebrospinal Venous Insufficiency (CCSVI) (clinicaltrials.gov) - Apr 3, 2015
P=N/A, N=60, Completed, Sponsor: Genetic Disease Investigators
N=20 --> 40 | Trial primary completion date: Dec 2013 --> Dec 2016 Active, not recruiting --> Completed | Trial primary completion date: Jul 2012 --> Apr 2015
- Trial completion, Trial primary completion date: Head Circumference Growth in Children With Ehlers-Danlos Syndrome Who Develop Dysautonomia Later in Life (clinicaltrials.gov) - Apr 3, 2015
P=N/A, N=21, Completed, Sponsor: Genetic Disease Investigators
Active, not recruiting --> Completed | Trial primary completion date: Jul 2012 --> Apr 2015 Recruiting --> Completed | Trial primary completion date: Oct 2011 --> Apr 2015
- Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Clinical: Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA (clinicaltrials.gov) - Mar 11, 2015
P2/3, N=34, Active, not recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
Recruiting --> Completed | Trial primary completion date: Oct 2011 --> Apr 2015 Recruiting --> Active, not recruiting
- Enrollment change, Trial termination: A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa (clinicaltrials.gov) - Mar 10, 2015
P4, N=3, Terminated, Sponsor: University Hospital, Basel, Switzerland
Recruiting --> Active, not recruiting N=18 --> 3 | Recruiting --> Terminated
- Trial completion, Trial primary completion date: Treatment of Nevus Flammeus With Alexandrite Laser (clinicaltrials.gov) - Feb 24, 2015
P=N/A, N=19, Completed, Sponsor: Bispebjerg Hospital
N=18 --> 3 | Recruiting --> Terminated Recruiting --> Completed | Trial primary completion date: Dec 2015 --> Dec 2014
- || Adempas (riociguat) / Bayer, Merck (MSD)
Trial primary completion date: RISE-IIP: Efficacy and Safety of Riociguat in Patients With Symptomatic Pulmonary Hypertension (PH) Associated With Idiopathic Interstitial Pneumonias (IIP) (clinicaltrials.gov) - Jan 30, 2015
P2, N=120, Recruiting, Sponsor: Bayer
Recruiting --> Completed | Trial primary completion date: Dec 2015 --> Dec 2014 Trial primary completion date: Mar 2016 --> Jan 2017
- Trial completion, IO biomarker: Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment (clinicaltrials.gov) - Jan 15, 2015
P=N/A, N=125, Completed, Sponsor: University Hospital Freiburg
Trial primary completion date: Mar 2016 --> Jan 2017 Recruiting --> Completed
- Tracleer (bosentan) / J&J, Roche
New P2 trial: Treatment of Resistant Port-wine Stains With Bosentan and Pulsed Dye Laser: a Pilot Study (clinicaltrials.gov) - Dec 17, 2014
P2, N=4, Active, not recruiting, Sponsor: Centre Hospitalier Universitaire de Nice
- Trial completion, Enrollment change, Trial primary completion date: Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa (clinicaltrials.gov) - Dec 10, 2014
P=N/A, N=7, Completed, Sponsor: Vanderbilt University
Recruiting --> Completed Recruiting --> Completed | N=20 --> 7 | Trial primary completion date: Mar 2014 --> Oct 2014
- Trial completion, Enrollment change: Port Wine Stains Treatment Matrix RF Study (clinicaltrials.gov) - Dec 5, 2014
P=N/A, N=10, Completed, Sponsor: Syneron Medical
Recruiting --> Completed | N=20 --> 7 | Trial primary completion date: Mar 2014 --> Oct 2014 Recruiting --> Completed | N=20 --> 10
- Trial primary completion date: A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa (clinicaltrials.gov) - Nov 3, 2014
P4, N=18, Recruiting, Sponsor: University Hospital, Basel, Switzerland
Recruiting --> Completed | N=20 --> 10 Trial primary completion date: Jun 2014 --> Dec 2014
- danazol oral / Generic mfg.
Enrollment change, Trial termination, Trial primary completion date: Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita (clinicaltrials.gov) - Sep 15, 2014
P1/2, N=5, Terminated, Sponsor: Children's Hospital Boston
Trial primary completion date: Jun 2014 --> Dec 2014 N=20 --> 5 | Recruiting --> Terminated | Trial primary completion date: Nov 2013 --> May 2014; Study was terminated due to under enrollment
- Clinical: Treatment of Port Wine Stains Using Pulsed Dye Laser, Erbium Yag Laser and Topical Sirolimus (clinicaltrials.gov) - Aug 12, 2014
P=N/A, N=20, Recruiting, Sponsor: Erasmus Medical Center
- lenalidomide / Generic mfg.
Enrollment change, Trial termination, Trial primary completion date: LENAKAP: Lenalidomide in Kaposi Disease Associated With HIV Infection (clinicaltrials.gov) - Aug 1, 2014
P2, N=12, Terminated, Sponsor: French National Agency for Research on AIDS and Viral Hepatitis
N=20 --> 5 | Recruiting --> Terminated | Trial primary completion date: Nov 2013 --> May 2014; Study was terminated due to under enrollment N=35 --> 12 | Active, not recruiting --> Terminated | Trial primary completion date: Oct 2013 --> Feb 2014