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EudraCT Number: 2019-002936-97

Sponsor Protocol Number: LTFU-ABO-101

Start Date:

Sponsor Name: Abeona Therapeutics Europe SL.

Full Title: A Long-term Follow-up Study of Patients with MPS IIIB from Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU)

Medical condition: MPS IIIB is a devastating lysosomal storage disease, caused by a N-α-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB appear normal at birth, but the disease is relentlessly progres...

Disease:	Version	SOC Term	Classification Code	Term	Level
	20.1	10010331 - Congenital, familial and genetic disorders	10056890	Mucopolysaccharidosis III	PT

Population Age: Children, Adolescents, Under 18

Gender: Male, Female

Trial protocol: DE (Prematurely Ended) FR (Ongoing)

Trial results: (No results available)

EudraCT Number: 2008-003801-15

Sponsor Protocol Number: 12011.202

Start Date:

Sponsor Name: PARIPharma GmbH

Full Title: A phase II, randomised, double-blind, placebo controlled, parallel group, dose-finding clinical trial to investigate the efficacy and safety of 10 and 20 mg/day aerosolised liposomal ciclosporin A ...

Medical condition: Bronchiolitis Obliterans Syndrome

Disease:	Version	SOC Term	Classification Code	Term	Level
	9.1		10049202	Bronchiolitis obliterans	LLT

Population Age: Adolescents, Under 18, Adults, Elderly

Gender: Male, Female

Trial protocol: BE (Completed) GB (Prematurely Ended)

Trial results: (No results available)

EudraCT Number: 2010-023502-12

Sponsor Protocol Number: UCL 08/0350

Start Date:

Sponsor Name: University College London

Full Title: A Proof of Principle, Double-Blind, Randomised Placebo-Controlled,Multi-centre Trial of pravaStatin to Ameliorate Early Onset Pre-eclampsia

Medical condition: Pre-eclampsia

Disease:	Version	SOC Term	Classification Code	Term	Level
			10036485	Pre-eclampsia	LLT
			10040444	Severe pre-eclampsia	LLT

Population Age: Adolescents, Under 18, Adults

Gender: Female

Trial protocol: GB (GB - no longer in EU/EEA)

Trial results: (No results available)

EudraCT Number: 2005-000928-17

Sponsor Protocol Number: A/201/05

Start Date:

Sponsor Name: POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI

Full Title: Hyperthermic intra-arterial chemotherapy with oxaliplatin in total hepatic vascular exclusion. A Phase II study

Medical condition: no resecable liver metastasis from colo-rectal and gastric cancer

Disease:	Version	SOC Term	Classification Code	Term	Level
	6.1		10055114		PT

Population Age: Adults, Elderly

Gender: Male, Female

Trial protocol: IT (Prematurely Ended)

Trial results: (No results available)

EudraCT Number: 2014-004641-27

Sponsor Protocol Number: UZLeuven

Start Date:

Sponsor Name: UZLeuven

Full Title: BOTOX in the treatment of urinary incontinence due to neurogenic detrusor overactivity in patients 3 months - 17 years

Medical condition: Urinary incontinence due to neurogenic detrusor overactivity

Disease:	Version	SOC Term	Classification Code	Term	Level
	18.0	10038359 - Renal and urinary disorders	10029279	Neurogenic bladder	PT

Population Age: Infants and toddlers, Children, Adolescents, Under 18

Gender: Male, Female

Trial protocol: BE (Prematurely Ended)

Trial results: (No results available)

EudraCT Number: 2014-000227-24

Sponsor Protocol Number: RG_13-320

Start Date:

Sponsor Name: University of Birmingham

Full Title: A Study of Thiotepa, Ifosphamide, Etoposide and Rituximab for the treatment of relapsed or refractory primary central nervous system lymphoma

Medical condition: Relapsed or refractory primary central neurvous system lymphoma.

Disease:	Version	SOC Term	Classification Code	Term	Level
	20.0	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)	10007953	Central nervous system lymphoma	PT

Population Age: Adolescents, Under 18, Adults, Elderly

Gender: Male, Female

Trial protocol: GB (GB - no longer in EU/EEA)

Trial results: (No results available)

EudraCT Number: 2017-002383-40

Sponsor Protocol Number: ICC_APL_Study_02

Start Date:

Sponsor Name: AIEOP- Associazione Italiana Ematologia Oncologia Pediatrica

Full Title: Treatment study for children and adolescents with Acute Promyelocytic Leukemia

Medical condition: acute promyelocytic leukemia (APL) in children and adolescents

Disease:

Version

SOC Term

Classification Code

Term

Level

Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Adolescents, Under 18

Gender: Male, Female

Trial protocol: IT (Ongoing)

Trial results: (No results available)

EudraCT Number: 2022-002954-25

Sponsor Protocol Number: ASND0036

Start Date:

Sponsor Name: Ascendis Pharma Growth Disorders A/S

Full Title: ApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in C...

Medical condition: Achondroplasia (ACH) in prepubertal children

Disease:	Version	SOC Term	Classification Code	Term	Level
	25.0	100000004850	10000452	Achondroplasia	LLT

Population Age: Children, Under 18

Gender: Male, Female

Trial protocol: IE (Ongoing) DK (Ongoing)

Trial results: (No results available)

EudraCT Number: 2021-005478-24

Sponsor Protocol Number: DYNE251-DMD-201

Start Date:

Sponsor Name: Dyne Therapeutics, Inc

Full Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participan...

Medical condition: Duchenne Muscular Dystrophy

Disease:	Version	SOC Term	Classification Code	Term	Level
	20.0	10010331 - Congenital, familial and genetic disorders	10013801	Duchenne muscular dystrophy	PT

Population Age: Children, Adolescents, Under 18

Gender: Male

Trial protocol: IE (Ongoing) BE (Ongoing) ES (Ongoing)

Trial results: (No results available)

EudraCT Number: 2017-002961-23

Sponsor Protocol Number: ODO-TE-B301

Start Date:

Sponsor Name: Odonate Therapeutics, Inc.

Full Title: A Multinational, Multicenter, Randomized, Phase 3 Study of Tesetaxel plus a Reduced Dose of Capecitabine versus Capecitabine Alone in Patients with HER2 Negative, Hormone Receptor Positive, Locally...

Medical condition: Patients with Breast Cancer

Disease:

Version

SOC Term

Classification Code

Term

Level

Population Age: Adults, Elderly

Gender: Male, Female

Trial protocol: CZ (Prematurely Ended) HU (Completed) ES (Prematurely Ended) GB (GB - no longer in EU/EEA) AT (Prematurely Ended) DE (Completed) BE (Prematurely Ended) FR (Prematurely Ended) PL (Prematurely Ended) IT (Prematurely Ended)

Trial results: (No results available)

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