Key Insights

Highlights

Success Rate

80% trial completion

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 49/100

Termination Rate

12.0%

3 terminated out of 25 trials

Success Rate

80.0%

-6.5% vs benchmark

Late-Stage Pipeline

12%

3 trials in Phase 3/4

Results Transparency

50%

6 of 12 completed with results

Key Signals

6 with results80% success

Data Visualizations

Phase Distribution

17Total
Not Applicable (4)
P 1 (4)
P 2 (6)
P 3 (2)
P 4 (1)

Trial Status

Completed12
Recruiting5
Terminated3
Unknown3
Active Not Recruiting1
Withdrawn1

Trial Success Rate

80.0%

Benchmark: 86.5%

Based on 12 completed trials

Clinical Trials (25)

Showing 20 of 20 trials
NCT05003648Phase 4Active Not RecruitingPrimary

Treating Leg Symptoms in Women With X-linked Adrenoleukodystrophy

NCT02699190Completed

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

NCT03047369Recruiting

The Myelin Disorders Biorepository Project

NCT04925349RecruitingPrimary

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

NCT02254863Phase 1RecruitingPrimary

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

NCT05687474Completed

Baby Detect : Genomic Newborn Screening

NCT03789721Recruiting

Adrenoleukodystrophy National Registry Study

NCT03231878Phase 2CompletedPrimary

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

NCT04090268Not ApplicableRecruiting

Precision Exercise in Children With Malignant Hemopathies

NCT00007020Phase 3Completed

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

NCT03513328Phase 1Completed

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT01586455Phase 1Completed

Human Placental-Derived Stem Cell Transplantation

NCT04303416Phase 2Completed

Plasma Exchange With Albumin in AMN Patients

NCT02559830Phase 1Unknown

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

NCT00004418Phase 2TerminatedPrimary

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

NCT00383448Phase 2CompletedPrimary

HSCT for High Risk Inherited Inborn Errors

NCT02952482CompletedPrimary

Newborn Screening for Adrenoleukodystrophy

NCT02948062WithdrawnPrimary

Early Diagnosis Of Childhood Cerebral ALD

NCT00176904Phase 2CompletedPrimary

Stem Cell Transplant for Inborn Errors of Metabolism

NCT02961803Phase 2Completed

MD1003-AMN MD1003 in Adrenomyeloneuropathy

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