Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

15.1%

67 terminated/withdrawn out of 445 trials

Success Rate

84.2%

-2.3% vs industry average

Late-Stage Pipeline

50%

224 trials in Phase 3/4

Results Transparency

84%

301 of 357 completed trials have results

Key Signals

2 recruiting301 with results51 terminated16 withdrawn

Enrollment Performance

Analytics

Phase 3
160(41.3%)
Phase 2
77(19.9%)
Phase 1
71(18.3%)
Phase 4
64(16.5%)
N/A
15(3.9%)
387Total
Phase 3(160)
Phase 2(77)
Phase 1(71)
Phase 4(64)
+1 more

Activity Timeline

Global Presence

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Clinical Trials (445)

Showing 20 of 445 trials
NCT01887938Phase 1Completed

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy

Role: lead

NCT03771898Phase 2Active Not Recruiting

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Role: lead

NCT04085172Phase 4Completed

A Study of TAK-503 in Children and Teenagers With Attention Deficit Hyperactivity Disorder (ADHD)

Role: lead

NCT04683003Phase 3Active Not Recruiting

A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Role: collaborator

NCT02455622Phase 4Completed

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Role: lead

NCT01303146Phase 2Completed

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Role: collaborator

NCT04002531Not ApplicableCompleted

A One Visit Follow Up of Adults With Fabry Disease Who Started Long-term Enzyme Replacement Therapy As Children

Role: collaborator

NCT01574274Phase 2Active Not Recruiting

SC-PEG Asparaginase vs. Oncaspar in Pediatric Acute Lymphoblastic Leukemia (ALL) and Lymphoblastic Lymphoma

Role: collaborator

NCT05556629Completed

A Survey to Assess Participants' and Physicians' Knowledge, Attitudes and Behavior When Using NATPARA

Role: lead

NCT01990040Active Not Recruiting

Registry for Participants With Short Bowel Syndrome

Role: lead

NCT03291223Recruiting

Gaucher Disease Outcome Survey (GOS)

Role: lead

NCT05067868Phase 4Recruiting

A Study of Replagal in Children and Adults With Fabry Disease in India

Role: lead

NCT01920737Phase 2Active Not Recruiting

A Novel "Pediatric-Inspired" Regimen With Reduced Myelosuppressive Drugs for Adults (Aged 18-60) With Newly Diagnosed Ph Negative Acute Lymphoblastic Leukemia

Role: collaborator

NCT02716233Phase 3Active Not Recruiting

A French Protocol for the Treatment of Acute Lymphoblastic Leukemia (ALL) in Children and Adolescents

Role: collaborator

NCT04497883Phase 1Completed

A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of a Single Oral Dose of Maribavir Administered in Healthy Japanese Participants Compared With Matched, Healthy, Non-Hispanic, Caucasian Participants and to Assess Dose-Proportionality of 3 Doses of Maribavir in Japanese Participants

Role: lead

NCT01071044Phase 4Completed

Efficacy and Safety of Lisdexamfetamine Dimesylate in Adults With Chronic Fatigue Syndrome

Role: collaborator

NCT04131556Phase 1Terminated

A Study Comparing the Pharmacokinetics and Palatability of Two Candidate Pediatric Powder-for-Oral-Suspension Formulations of Maribavir to the Current Maribavir Tablet Formulation Administered in Healthy Adult Participants

Role: lead

NCT04130191Completed

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II

Role: lead

NCT02141113Phase 4Completed

Efficacy and Safety Eval of Guanfacine Hydrochloride in Combination With Psychostimulants in Adults (18-65).

Role: collaborator

NCT02412787Phase 2Completed

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Role: lead