{
  "api_version": "1",
  "request": {
    "url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Pelizaeus-Merzbacher+Disease",
    "query": {
      "condition": "Pelizaeus-Merzbacher Disease"
    },
    "page_size": 10
  },
  "pagination": {
    "page": 1,
    "page_size": 10,
    "total_count": 11,
    "total_pages": 2,
    "next_page_url": "https://clinicaltrialsfind.com/api/v1/search.json?condition=Pelizaeus-Merzbacher+Disease&page=2&page_size=10",
    "previous_page_url": null
  },
  "source": "remote",
  "last_synced_at": "2026-05-21T23:41:00.956Z",
  "attribution": "Data derived from public ClinicalTrials.gov records. The official record at https://clinicaltrials.gov/ remains the source of truth for current availability, contacts, and full study details.",
  "notes": [
    "This endpoint exposes only public summary fields — no participant contact emails, phone numbers, or scraped investigator contact details.",
    "For contact information and full protocol detail, follow each trial's `official_url` to the ClinicalTrials.gov record.",
    "This API is a navigational aid. It does not provide medical advice, eligibility determinations, or compensation guarantees."
  ],
  "trials": [
    {
      "nct_id": "NCT01391637",
      "title": "Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pelizaeus-Merzbacher Disease",
        "PMD"
      ],
      "interventions": [
        {
          "name": "HuCNS-SC transplant in the lead-in phase",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "StemCells, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "MALE",
        "summary": "Male only"
      },
      "enrollment_count": 4,
      "start_date": "2011-06",
      "completion_date": "2016-03",
      "has_results": false,
      "last_update_posted_date": "2016-05-13",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 1,
      "location_summary": "San Francisco, California",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01391637"
    },
    {
      "nct_id": "NCT03333200",
      "title": "Longitudinal Study of Neurodegenerative Disorders",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "MLD",
        "Krabbe Disease",
        "ALD",
        "MPS I",
        "MPS II",
        "MPS III",
        "Vanishing White Matter Disease",
        "GM3 Gangliosidosis",
        "PKAN",
        "Tay-Sachs Disease",
        "NP Deficiency",
        "Osteopetrosis",
        "Alpha-Mannosidosis",
        "Sandhoff Disease",
        "Niemann-Pick Diseases",
        "MPS IV",
        "Gaucher Disease",
        "GAN",
        "GM1 Gangliosidoses",
        "Morquio Disease",
        "S-Adenosylhomocysteine Hydrolase Deficiency",
        "Batten Disease",
        "Pelizaeus-Merzbacher Disease",
        "Leukodystrophy",
        "Lysosomal Storage Diseases",
        "Purine Nucleoside Phosphorylase Deficiency",
        "Multiple Sulfatase Deficiency Disease"
      ],
      "interventions": [
        {
          "name": "Palliative Care",
          "type": "OTHER"
        },
        {
          "name": "Hematopoetic Stem Cell Transplantation",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "OTHER",
        "BIOLOGICAL"
      ],
      "sponsor": "University of Pittsburgh",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 1500,
      "start_date": "2012-01-11",
      "completion_date": "2035-01",
      "has_results": false,
      "last_update_posted_date": "2026-02-09",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 1,
      "location_summary": "Pittsburgh, Pennsylvania",
      "locations": [
        {
          "city": "Pittsburgh",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03333200"
    },
    {
      "nct_id": "NCT03047369",
      "title": "The Myelin Disorders Biorepository Project",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leukodystrophy",
        "White Matter Disease",
        "Leukoencephalopathies",
        "4H Syndrome",
        "Adrenoleukodystrophy",
        "AMN",
        "ALD",
        "ALD Gene Mutation",
        "ALD (Adrenoleukodystrophy)",
        "X-linked Adrenoleukodystrophy",
        "X-ALD",
        "Adrenomyeloneuropathy",
        "Aicardi Goutieres Syndrome",
        "AGS",
        "Alexander Disease",
        "Alexanders Leukodystrophy",
        "AxD",
        "ADLD",
        "Canavan Disease",
        "CTX",
        "Cerebrotendinous Xanthomatoses",
        "Krabbe Disease",
        "GALC Deficiency",
        "Globoid Leukodystrophy",
        "TUBB4A-Related Leukodystrophy",
        "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum",
        "HBSL",
        "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity",
        "LBSL",
        "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)",
        "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation",
        "ALSP",
        "CSF1R Gene Mutation",
        "HCC - Hypomyelination and Congenital Cataract",
        "MLC1",
        "Megalencephalic Leukoencephalopathy With Subcortical Cysts",
        "MLD",
        "Metachromatic Leukodystrophy",
        "PMD",
        "Pelizaeus-Merzbacher Disease",
        "PLP1 Null Syndrome",
        "PLP1 Gene Duplication | Blood or Tissue | Mutations",
        "Pelizaeus Merzbacher Like Disease",
        "Peroxisomal Biogenesis Disorder",
        "Zellweger Syndrome",
        "Refsum Disease",
        "Salla Disease",
        "Sialic Storage Disease",
        "Sjögren",
        "Sjogren-Larsson Syndrome",
        "Van Der Knapp Disease",
        "Vanishing White Matter Disease",
        "Charcot-Marie-Tooth",
        "CMT",
        "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency",
        "Allan-Herndon-Dudley Syndrome",
        "Cadasil",
        "Cockayne Syndrome",
        "Multiple Sulfatase Deficiency",
        "Gangliosidoses",
        "GM2 Gangliosidosis",
        "BPAN",
        "Labrune Syndrome",
        "LCC",
        "Mucopolysaccharidoses",
        "TBCK-Related Intellectual Disability Syndrome"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Children's Hospital of Philadelphia",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": null,
        "sex": "ALL",
        "summary": "Not listed"
      },
      "enrollment_count": 12000,
      "start_date": "2016-12-08",
      "completion_date": "2030-12-08",
      "has_results": false,
      "last_update_posted_date": "2025-10-23",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 23,
      "location_summary": "Los Angeles, California • Orange, California • Palo Alto, California + 18 more",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "Orange",
          "state": "California"
        },
        {
          "city": "Palo Alto",
          "state": "California"
        },
        {
          "city": "Sacramento",
          "state": "California"
        },
        {
          "city": "San Diego",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT03047369"
    },
    {
      "nct_id": "NCT06150716",
      "title": "Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Pelizaeus-Merzbacher Disease"
      ],
      "interventions": [
        {
          "name": "ION356",
          "type": "DRUG"
        }
      ],
      "intervention_types": [
        "DRUG"
      ],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "2 Years",
        "maximum_age": "17 Years",
        "sex": "MALE",
        "summary": "2 Years to 17 Years · Male only"
      },
      "enrollment_count": 24,
      "start_date": "2024-04-10",
      "completion_date": "2028-06",
      "has_results": false,
      "last_update_posted_date": "2025-12-12",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 3,
      "location_summary": "Atlanta, Georgia • Philadelphia, Pennsylvania • Salt Lake City, Utah",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        },
        {
          "city": "Salt Lake City",
          "state": "Utah"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT06150716"
    },
    {
      "nct_id": "NCT01005004",
      "title": "Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects",
      "overall_status": "COMPLETED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Pelizaeus-Merzbacher Disease"
      ],
      "interventions": [
        {
          "name": "HuCNS-SC cells",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "StemCells, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "5 Years",
        "sex": "MALE",
        "summary": "6 Months to 5 Years · Male only"
      },
      "enrollment_count": 4,
      "start_date": "2009-11",
      "completion_date": "2012-12",
      "has_results": false,
      "last_update_posted_date": "2015-01-15",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 1,
      "location_summary": "San Francisco, California",
      "locations": [
        {
          "city": "San Francisco",
          "state": "California"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01005004"
    },
    {
      "nct_id": "NCT01003912",
      "title": "Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases",
      "overall_status": "WITHDRAWN",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Lysosomal Storage Diseases",
        "Inborn Errors of Metabolism"
      ],
      "interventions": [
        {
          "name": "ALD-601",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Joanne Kurtzberg, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "18 Years",
        "maximum_age": null,
        "sex": "FEMALE",
        "summary": "18 Years and older · Female only"
      },
      "enrollment_count": 0,
      "start_date": "2009-10",
      "completion_date": "2017-03",
      "has_results": false,
      "last_update_posted_date": "2017-06-29",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 2,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT01003912"
    },
    {
      "nct_id": "NCT00654433",
      "title": "ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases",
      "overall_status": "TERMINATED",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE3"
      ],
      "conditions": [
        "Inherited Metabolic Diseases",
        "Lysosomal Storage Disorders",
        "Peroxisomal Storage Diseases",
        "Inborn Errors of Metabolism",
        "Mucopolysaccharidosis"
      ],
      "interventions": [
        {
          "name": "ALD-101",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Aldagen",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "16 Years",
        "sex": "ALL",
        "summary": "Up to 16 Years"
      },
      "enrollment_count": 40,
      "start_date": "2008-03",
      "completion_date": "2011-11",
      "has_results": false,
      "last_update_posted_date": "2014-07-08",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 3,
      "location_summary": "Los Angeles, California • New York, New York • Durham, North Carolina",
      "locations": [
        {
          "city": "Los Angeles",
          "state": "California"
        },
        {
          "city": "New York",
          "state": "New York"
        },
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT00654433"
    },
    {
      "nct_id": "NCT05659901",
      "title": "Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease",
      "overall_status": "RECRUITING",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Pelizaeus-Merzbacher Disease"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Ionis Pharmaceuticals, Inc.",
      "sponsor_class": "INDUSTRY",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "6 Months",
        "maximum_age": "17 Years",
        "sex": "MALE",
        "summary": "6 Months to 17 Years · Male only"
      },
      "enrollment_count": 32,
      "start_date": "2022-10-03",
      "completion_date": "2029-03",
      "has_results": false,
      "last_update_posted_date": "2026-04-17",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 2,
      "location_summary": "Atlanta, Georgia • Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Atlanta",
          "state": "Georgia"
        },
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT05659901"
    },
    {
      "nct_id": "NCT02699190",
      "title": "LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies",
      "overall_status": "COMPLETED",
      "study_type": "OBSERVATIONAL",
      "phases": [],
      "conditions": [
        "Leukodystrophy",
        "White Matter Disease",
        "4H Syndrome",
        "Adrenoleukodystrophy",
        "AMN",
        "ALD",
        "ALD (Adrenoleukodystrophy)",
        "X-linked Adrenoleukodystrophy",
        "X-ALD",
        "Adrenomyeloneuropathy",
        "Aicardi Goutieres Syndrome",
        "AGS",
        "Alexander Disease",
        "Alexanders Leukodystrophy",
        "AxD",
        "ADLD",
        "Canavan Disease",
        "CTX",
        "Cerebrotendinous Xanthomatoses",
        "Krabbe Disease",
        "GALC Deficiency",
        "Globoid Leukodystrophy",
        "TUBB4A-Related Leukodystrophy",
        "H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum",
        "HBSL",
        "HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity",
        "LBSL",
        "Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)",
        "Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation",
        "ALSP",
        "CSF1R Gene Mutation",
        "HCC - Hypomyelination and Congenital Cataract",
        "MLC1",
        "Megalencephalic Leukoencephalopathy With Subcortical Cysts 1",
        "MLD",
        "Metachromatic Leukodystrophy",
        "PMD",
        "Pelizaeus-Merzbacher Disease",
        "PLP1 Null Syndrome",
        "PLP1 Gene Duplication | Blood or Tissue | Mutations",
        "Pelizaeus-Merzbacher-Like Disease, 1",
        "Peroxisomal Biogenesis Disorder",
        "Zellweger Syndrome",
        "Refsum Disease",
        "Salla Disease",
        "Sialic Storage Disease",
        "Sjögren",
        "Sjogren-Larsson Syndrome",
        "Van Der Knapp Disease",
        "Vanishing White Matter Disease",
        "Charcot-Marie-Tooth",
        "CMT",
        "Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency",
        "Allan-Herndon-Dudley Syndrome",
        "Cadasil",
        "Cockayne Syndrome",
        "Multiple Sulfatase Deficiency",
        "Gangliosidoses",
        "GM2 Gangliosidosis",
        "BPAN",
        "Labrune Syndrome",
        "LCC",
        "Mucopolysaccharidoses",
        "TBCK-Related Intellectual Disability Syndrome"
      ],
      "interventions": [],
      "intervention_types": [],
      "sponsor": "Children's Hospital of Philadelphia",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": null,
        "maximum_age": "18 Years",
        "sex": "ALL",
        "summary": "Up to 18 Years"
      },
      "enrollment_count": 236,
      "start_date": "2017-01-06",
      "completion_date": "2024-10-31",
      "has_results": true,
      "last_update_posted_date": "2025-11-10",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 1,
      "location_summary": "Philadelphia, Pennsylvania",
      "locations": [
        {
          "city": "Philadelphia",
          "state": "Pennsylvania"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02699190"
    },
    {
      "nct_id": "NCT02254863",
      "title": "UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells",
      "overall_status": "RECRUITING",
      "study_type": "INTERVENTIONAL",
      "phases": [
        "PHASE1"
      ],
      "conditions": [
        "Adrenoleukodystrophy",
        "Batten Disease",
        "Mucopolysaccharidosis II",
        "Leukodystrophy, Globoid Cell",
        "Leukodystrophy, Metachromatic",
        "Neimann Pick Disease",
        "Pelizaeus-Merzbacher Disease",
        "Sandhoff Disease",
        "Tay-Sachs Disease",
        "Brain Diseases, Metabolic, Inborn",
        "Alpha-Mannosidosis",
        "Sanfilippo Mucopolysaccharidoses"
      ],
      "interventions": [
        {
          "name": "DUOC-01",
          "type": "BIOLOGICAL"
        }
      ],
      "intervention_types": [
        "BIOLOGICAL"
      ],
      "sponsor": "Joanne Kurtzberg, MD",
      "sponsor_class": "OTHER",
      "healthy_volunteers": false,
      "eligibility": {
        "minimum_age": "1 Week",
        "maximum_age": "22 Years",
        "sex": "ALL",
        "summary": "1 Week to 22 Years"
      },
      "enrollment_count": 40,
      "start_date": "2014-09",
      "completion_date": "2026-10",
      "has_results": false,
      "last_update_posted_date": "2025-09-08",
      "last_synced_at": "2026-05-21T23:41:00.956Z",
      "location_count": 1,
      "location_summary": "Durham, North Carolina",
      "locations": [
        {
          "city": "Durham",
          "state": "North Carolina"
        }
      ],
      "official_url": "https://clinicaltrials.gov/study/NCT02254863"
    }
  ]
}